Ignite Creation Date:
2025-12-25 @ 2:12 AM
Ignite Modification Date:
2026-03-07 @ 2:28 PM
Study NCT ID:
NCT03287960
Status:
COMPLETED
Last Update Posted:
2023-05-23
First Post:
2017-09-14
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Setmelanotide for the Treatment of Leptin Receptor (LEPR) Deficiency Obesity
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['United States'], 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2023-05-06', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D009765', 'term': 'Obesity'}], 'ancestors': [{'id': 'D050177', 'term': 'Overweight'}, {'id': 'D044343', 'term': 'Overnutrition'}, {'id': 'D009748', 'term': 'Nutrition Disorders'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D001835', 'term': 'Body Weight'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C579663', 'term': 'setmelanotide'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@rhythmtx.com', 'phone': '857-264-4280', 'title': 'Rhythm Clinical Trials', 'organization': 'Rhythm Pharmaceuticals, Inc.'}, 'certainAgreement': {'otherDetails': 'All information regarding setmelanotide supplied by Rhythm to the investigator is privileged and confidential information. The investigator agrees to use this information to accomplish the study and will not use it for other purposes without consent from Rhythm. The information obtained from the clinical study will be used towards the development of setmelanotide and may be disclosed to regulatory authority(ies), other investigators, corporate partners, or consultants as required.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': 'Based on meetings with the Federal Drug Administration (FDA), the structure of the endpoints was slightly different in the statistical analysis plan (SAP) and the protocol.\n\nIn addition, there was no planned comparison to a randomized placebo group given the rarity of monogenic LEPR deficiency obesity.'}}, 'adverseEventsModule': {'timeFrame': 'From first dose up to ~30 days after last dose of study drug (Up to ~ 1 year)', 'description': 'SAS population. Participants in pivotal and supplemental cohort were included. Adverse events were analyzed for participants in placebo \\& setmelanotide groups combined as one group per planned analysis.', 'eventGroups': [{'id': 'EG000', 'title': 'Setmelanotide', 'description': 'Participants received titrated doses of setmelanotide once daily, by SC injection during titration period for 2 - 12 weeks. Thereafter, participants continued setmelanotide at their specific therapeutic dose for an additional 10 weeks during the open label treatment period. Participants who achieved at least a 5 kg weight loss (or at least 5% weight loss if baseline body weight was \\<100 kg) at the end of the open label treatment period, continued into the 8-week double-blind withdrawal period and received 4 weeks setmelanotide and 4 weeks placebo. Following the withdrawal period, participants entered open label treatment period and received setmelanotide to complete approximately 52 weeks (\\~1 year) of treatment at a therapeutic dose.', 'otherNumAtRisk': 15, 'deathsNumAtRisk': 15, 'otherNumAffected': 15, 'seriousNumAtRisk': 15, 'deathsNumAffected': 1, 'seriousNumAffected': 3}], 'otherEvents': [{'term': 'Flushing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Melanocytic naevus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Eye naevus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Injection site erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 32, 'numAffected': 11}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Injection site induration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 16, 'numAffected': 7}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Injection site pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 14, 'numAffected': 7}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Injection site oedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 13, 'numAffected': 6}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Injection site pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 13, 'numAffected': 8}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 9, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Injection site bruising', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 8, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 7, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Influenza like illness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Injection site haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Injection site hypersensitivity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Malaise', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Injection site atrophy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Injection site reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Injection site urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Medical device pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Medical device site erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Oedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Xerosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 7, 'numAffected': 4}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Depression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 4, 'numAffected': 2}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Depressed mood', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Suicidal ideation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Affect lability', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Drug abuse', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Fear of injection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Illusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Dysmenorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 5, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Spontaneous penile erection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Ejaculation disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Metrorrhagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Amenorrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Ovarian cyst', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Vaginal haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Arthropod bite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Foot fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Joint Injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Ligament sprain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Blood creatine phosphokinase abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Blood follicle stimulating hormone increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Blood lactate dehydrogenase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Blood luteinising hormone increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Blood urea decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Blood uric acid increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Heart rate increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Insulin tolerance test abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Neutrophil count increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Weight increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Cardiac flutter', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Asthma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Anemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Eosinophilia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Iron deficiency anemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 18, 'numAffected': 5}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 5, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Sleep paralysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Presyncope', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Sciatica', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Syncope', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Migraine', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Tic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Ear pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Tinnitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Vertigo', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 16, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 13, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 13, 'numAffected': 7}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Gingival discolouration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Abdominal discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Abdominal distension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Haemorrhoids', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Cholestasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Hepatocellular injury', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Haematuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Proteinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Renal colic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Renal failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Skin hyperpigmentation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 20, 'numAffected': 11}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Hyperhidrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Skin striae', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Urticaria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Hair growth rate abnormal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Hyperkeratosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Lentigo', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Lipodystrophy acquired', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Melanocytic naevus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Pain of skin', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Pigmentation disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Pruritus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Rash', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 7, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Musculoskeletal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 4, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Muscle spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Musculoskeletal chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Neck pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Torticollis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Hypothyroidism', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Endocrine disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Hypogonadism', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Endocrine disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Decreased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Dyslipidaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Folate deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Glucose tolerance impaired', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Gout', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Vitamin A deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Lower respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 6, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 6, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Gastrointestinal infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Arthritis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Fungal infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Injection site abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Otitis externa', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}], 'seriousEvents': [{'term': 'Cholecystitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Road Traffic Accident', 'notes': 'Leading to event', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA'}, {'term': 'Suicidal Ideation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}, {'term': 'Gastric Banding Reversal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 22.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Percentage of Participants Who Reached ≥10% Weight Loss Threshold After 1 Year (Pivotal Cohort)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Setmelanotide (Entire Study)', 'description': 'Participants received setmelanotide once daily by SC injection for approximately 52 weeks (\\~ 1 year).'}], 'classes': [{'categories': [{'measurements': [{'value': '45.5', 'groupId': 'OG000', 'lowerLimit': '19.96', 'upperLimit': '72.88'}]}]}], 'analyses': [{'pValue': '0.0001', 'groupIds': ['OG000'], 'statisticalMethod': 'Clopper-Pearson method', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Two-sided confidence interval obtained using Clopper-Pearson method and one-sided p-value obtained from exact binomial test, testing that at least 5% of participants in the population of interest will achieve 10% weight loss.'}], 'paramType': 'NUMBER', 'timeFrame': 'Week 52', 'description': 'The percentage of participants who met the ≥10% weight loss threshold (responders) after approximately Week 52 (\\~1 year) of treatment were analyzed.', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '90% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The full analysis set (FAS) consisted of participants who received any of the study drug injections and at least one baseline assessment (included those who did and did not demonstrate ≥5 kg weight loss or 5% of body weight if weight is \\<100 kg at baseline over the initial12-week open label treatment period and proceeded into the double blind, placebo-controlled withdrawal period). Participants in pivotal cohort were included in the analysis.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Who Reached ≥10% Weight Loss Threshold After 1 Year (Pivotal + Supplemental Cohort)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Setmelanotide (Entire Study)', 'description': 'Participants received setmelanotide once daily by SC injection for approximately 52 weeks (\\~ 1 year).'}], 'classes': [{'categories': [{'measurements': [{'value': '53.3', 'groupId': 'OG000', 'lowerLimit': '30.00', 'upperLimit': '75.63'}]}]}], 'analyses': [{'pValue': '<0.0001', 'groupIds': ['OG000'], 'statisticalMethod': 'Clopper-Pearson method', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Two-sided confidence interval (CI) obtained using Clopper-Pearson method and one-sided p-value obtained from exact binomial test, testing that at least 5% of patients in the population of interest would achieve 10% weight loss.'}], 'paramType': 'NUMBER', 'timeFrame': 'Week 52', 'description': 'The percentage of participants who met the ≥10% weight loss threshold (responders) after approximately Week 52 (\\~1 year) of treatment were analyzed.', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '90% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS Population. Participants in pivotal and supplemental cohort were included in the analysis.'}, {'type': 'SECONDARY', 'title': 'Mean Percent Change From Baseline in Body Weight', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Setmelanotide (Entire Study)', 'description': 'Participants received setmelanotide once daily by SC injection for approximately 52 weeks (\\~ 1 year).'}], 'classes': [{'categories': [{'measurements': [{'value': '-12.34', 'spread': '7.534', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '<.0001', 'groupIds': ['OG000'], 'paramType': 'Least Squares Mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '90', 'paramValue': '-12.37', 'ciLowerLimit': '-15.08', 'ciUpperLimit': '-9.66', 'statisticalMethod': 'ANOVA', 'nonInferiorityType': 'OTHER', 'statisticalComment': 'Longitudinal mixed ANOVA', 'nonInferiorityComment': 'Model based summary statistics from longitudinal mixed analysis of variance (ANOVA) model with fixed effect for visit, baseline body weight and random effect for participant, one sided p-value from model.'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 52', 'description': 'The mean percent change from baseline in body weight at 52 weeks was analyzed.', 'unitOfMeasure': 'Percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Designated Use Set (DUS) consisted of participants who received any of the study drug injections, demonstrated ≥5 kg weight loss \\[or 5% of body weight if weight was \\<100 kg at baseline\\] during the initial 12-week open label treatment period, and proceeded into the double-blind, placebo-controlled withdrawal period. Participants in pivotal and supplemental cohort were included in the analysis.'}, {'type': 'SECONDARY', 'title': "Mean Percent Change From Baseline in Hunger Score ('Most Hunger')", 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Setmelanotide (Entire Study)', 'description': 'Participants received setmelanotide once daily by SC injection for approximately 52 weeks (\\~ 1 year).'}], 'classes': [{'categories': [{'measurements': [{'value': '-42.7', 'spread': '27.49', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '<.0001', 'groupIds': ['OG000'], 'paramType': 'Least Squares Mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '90', 'paramValue': '-42.69', 'ciLowerLimit': '-56.35', 'ciUpperLimit': '-29.02', 'statisticalMethod': 'ANOVA', 'nonInferiorityType': 'OTHER', 'statisticalComment': 'Longitudinal mixed analysis of variance (ANOVA) model', 'nonInferiorityComment': 'Model based summary statistics from longitudinal mixed analysis of variance model with fixed effect for week, baseline daily hunger score and random effect for participant, one sided p-value from model.'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 52', 'description': 'The mean percent change in hunger scores for participants ≥12 years of age with leptin receptor (LEPR) deficiency obesity in treatment with setmelanotide was evaluated. Hunger score ranged from 0 - 10 on a Likert-type scale; 0 = not hungry at all and 10 = hungriest possible. On the Daily Hunger Questionnaire, each of the 3 items (average hunger, most/worst hunger, and morning hunger) was scored separately and averaged on a weekly basis.', 'unitOfMeasure': 'Percent Change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'DUS Population. Participants in pivotal and supplemental cohort were included in the analysis.'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants Achieving at Least 25% Improvement in Daily Hunger From Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Setmelanotide (Entire Study)', 'description': 'Participants received setmelanotide once daily by SC injection for approximately 52 weeks (\\~ 1 year).'}], 'classes': [{'categories': [{'measurements': [{'value': '71.4', 'groupId': 'OG000', 'lowerLimit': '46.00', 'upperLimit': '89.60'}]}]}], 'analyses': [{'pValue': '<.0001', 'groupIds': ['OG000'], 'statisticalMethod': 'Clopper-Pearson method', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'Two-sided CI obtained using Clopper-Pearson method and one-sided p-value obtained from exact binomial test, testing that ≥5 % of participants in the population of interest would achieve ≥25 % improvement in daily hunger score.'}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline and Week 52', 'description': 'The percentage of participants (≥12 years of age) achieving a ≥25% improvement from baseline in hunger score at Week 52 (i.e., after treatment with setmelanotide for 52 weeks at the therapeutic dose) was assessed. Hunger ranged from 0 - 10 on a Likert-type scale; 0 = not hungry at all and 10 = hungriest possible. On the Daily Hunger Questionnaire, each of the 3 items (average hunger, most/worst hunger, and morning hunger) was scored separately and averaged on a weekly basis.', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '90% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'FAS population with available data at specified time point. Participants in pivotal and supplemental cohort were included in the analysis.'}, {'type': 'SECONDARY', 'title': 'Absolute Change From Baseline in Waist Circumference', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Setmelanotide (Entire Study)', 'description': 'Participants received setmelanotide once daily by SC injection for approximately 52 weeks (\\~ 1 year).'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '131.58', 'spread': '24.047', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-9.80', 'spread': '6.095', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '0.0031', 'groupIds': ['OG000'], 'paramType': 'Least Squares Mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '90', 'paramValue': '-8.9', 'ciLowerLimit': '-14.10', 'ciUpperLimit': '-3.61', 'statisticalMethod': 'ANOVA', 'nonInferiorityType': 'OTHER', 'statisticalComment': 'Longitudinal mixed ANOVA', 'nonInferiorityComment': 'Model based summary statistics from longitudinal mixed analysis of variance model with fixed effect for visit, baseline waist circumference and random effect for participant, one sided p-value from model.'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 52', 'description': 'Waist circumference (cm) was measured according to the National Heart, Lung, and Blood Institute (NHLBI) criteria. Waist circumference was measured when participants were fasting at approximately the same time at each visit. The absolute change from baseline in waist circumference was assessed.', 'unitOfMeasure': 'Centimeter', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'DUS population with available data at specified time point. Participants in pivotal and supplemental cohort were included in the analysis.'}, {'type': 'SECONDARY', 'title': 'Absolute Change in Body Weight (Reversal of Weight Loss) During Double-Blind Placebo-Controlled Withdrawal Period', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Setmelanotide (Double-Blind)', 'description': 'Participants received setmelanotide once daily by SC injection for 4 weeks and placebo matched to setmelanotide for 4 weeks during the double-blind placebo controlled withdrawal period.'}], 'classes': [{'title': 'Change at Week 4: Setmelanotide', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1.2', 'spread': '3.04', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 4: Placebo', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.9', 'spread': '2.82', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 8 of withdrawal period', 'description': 'A comparison of weight change was evaluated during the 8 week placebo-controlled withdrawal period for each participant, during which each participant received 4 weeks of placebo and 4 weeks active therapy in a blinded fashion.', 'unitOfMeasure': 'Kilogram', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'DUS population with available data at specified time point. Participants in pivotal and supplemental cohort were included in the analysis.'}, {'type': 'SECONDARY', 'title': 'Absolute Daily Hunger Reduction Score During the Double-Blind Placebo-Controlled Withdrawal Period', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Setmelanotide (Double-Blind)', 'description': 'Participants received setmelanotide once daily by SC injection for 4 weeks and placebo matched to setmelanotide for 4 weeks during the double-blind placebo controlled withdrawal period.'}], 'classes': [{'title': 'Setmelanotide', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '3.4', 'spread': '1.63', 'groupId': 'OG000'}]}]}, {'title': 'Placebo', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.3', 'spread': '2.13', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Week 8 of withdrawal period', 'description': 'The absolute score in daily hunger reduction during the double-blind placebo-controlled withdrawal period (≥12 Years of Age) was assessed. Hunger ranged from 0 - 10 on a Likert-type scale; 0 = not hungry at all and 10 = hungriest possible. On the Daily Hunger Questionnaire, each of the 3 items (average hunger, most/worst hunger, and morning hunger) was scored separately and averaged on a weekly basis. The weekly average hunger score of the daily worst (most) hunger score in 24 hours is the hunger score used to assess this study endpoint. Lower scores represent lower hunger, higher scores represent greater hunger.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'DUS population with available data at specified time point. Participants in pivotal and supplemental cohort were included in the analysis.'}, {'type': 'SECONDARY', 'title': 'Mean Percent Change From Baseline in Body Mass Index', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Setmelanotide (Entire Study)', 'description': 'Participants received setmelanotide once daily by SC injection for approximately 52 weeks (\\~ 1 year).'}], 'classes': [{'categories': [{'measurements': [{'value': '-14.24', 'spread': '9.096', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '<.0001', 'groupIds': ['OG000'], 'paramType': 'Least Squares Mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '90', 'paramValue': '-14.0', 'ciLowerLimit': '-16.80', 'ciUpperLimit': '-11.20', 'statisticalMethod': 'ANOVA', 'nonInferiorityType': 'OTHER', 'statisticalComment': 'Longitudinal mixed ANOVA model', 'nonInferiorityComment': 'Model based summary statistics from longitudinal mixed analysis of variance model with fixed effect for week, baseline BMI and random effect for participant, one sided p-value from model.'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 52', 'description': 'The mean percent change from baseline in body mass index (BMI) was assessed.', 'unitOfMeasure': 'Percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'DUS population with available data at specified time point. Participants in pivotal and supplemental cohort were included in the analysis.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Glucose Parameters', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Setmelanotide (Entire Study)', 'description': 'Participants received setmelanotide once daily by SC injection for approximately 52 weeks (\\~ 1 year).'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.586', 'spread': '2.3740', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 52', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.465', 'spread': '1.4350', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 52', 'description': 'Glucose parameters included glucose, glycated hemoglobin (HbA1c) and Oral glucose tolerance test (OGTT). Data is planned to be reported only for change from baseline in glucose levels.', 'unitOfMeasure': 'millimole per liter', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The Safety Analysis Set (SAS) was defined as all participants who received any study drug injections at least one post-dose safety assessment. Participants with available data were analyzed. Participants in pivotal and supplemental cohort were included in the analysis.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Setmelanotide', 'description': 'Participants received titrated doses of setmelanotide once daily, by subcutaneous (SC) injection during titration period for 2 - 12 weeks. Thereafter, participants continued setmelanotide at their specific therapeutic dose for an additional 10 weeks during the open label treatment period. Participants who achieved at least a 5 kg weight loss (or at least 5% weight loss if baseline body weight was \\<100 kg) at the end of the open label treatment period, continued into the 8-week double-blind withdrawal period and received 4 weeks setmelanotide and 4 weeks placebo. Following the withdrawal period, participants entered open label treatment period and received setmelanotide to complete approximately 52 weeks (\\~1 year) of treatment at a therapeutic dose.'}], 'periods': [{'title': 'Titration Period (2 to 12 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}, {'title': 'Open-Label Period (10 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}, {'title': 'Double-Blind Withdrawal Period (8 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}, {'title': 'Open-Label Period (32 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '13'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Serious Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'Participants were enrolled into the pivotal cohort (11 participants) or supplemental cohort (4 participants).\n\nPivotal cohort: Set of participants under study constituted a collection of detailed clinical case reports with a comprehensive baseline and past medical history assessment and complete clinical efficacy, safety and laboratory evaluations conducted for each participant.\n\nSupplemental cohort: Any additional participants enrolled were included in supplemental cohort.', 'preAssignmentDetails': 'At screening, a blood sample was obtained for genotyping for mechanisms considered to be possibly related to the safety or efficacy response to the study medication (e.g., other obesity related genes). Complete physical, relevant bloodwork and other standard assessments were performed.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Setmelanotide', 'description': 'Participants received titrated doses of setmelanotide once daily, by SC injection during titration period for 2 - 12 weeks. Thereafter, participants continued setmelanotide at their specific therapeutic dose for an additional 10 weeks during the open label treatment period. Participants who achieved at least a 5 kg weight loss (or at least 5% weight loss if baseline body weight was \\<100 kg) at the end of the open label treatment period, continued into the 8-week double-blind withdrawal period and received 4 weeks setmelanotide and 4 weeks placebo. Following the withdrawal period, participants entered open label treatment period and received setmelanotide to complete approximately 52 weeks (\\~1 year) of treatment at a therapeutic dose.'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '10', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '21.67', 'spread': '8.52', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '9', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '13', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '12', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Netherlands', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}]}]}, {'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'France', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}, {'title': 'Germany', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}, {'title': 'Canada', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2018-12-10', 'size': 16117230, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2023-04-05T08:13', 'hasProtocol': True}, {'date': '2019-04-16', 'size': 1787032, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2023-04-05T08:15', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE', 'maskingDescription': 'During the 8 week double-blind placebo-controlled withdrawal period, participants received 4 weeks of daily setmelanotide and 4 weeks of daily placebo. Participants, investigators, and sites remained blinded as to when placebo treatment was administered.'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Open-label with an 8 week Double-Blind Placebo-Controlled Withdrawal Period'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 15}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2018-01-30', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-05', 'completionDateStruct': {'date': '2020-09-25', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-05-18', 'studyFirstSubmitDate': '2017-09-14', 'resultsFirstSubmitDate': '2023-04-11', 'studyFirstSubmitQcDate': '2017-09-15', 'lastUpdatePostDateStruct': {'date': '2023-05-23', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-05-18', 'studyFirstPostDateStruct': {'date': '2017-09-19', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2023-05-23', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2020-09-25', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percentage of Participants Who Reached ≥10% Weight Loss Threshold After 1 Year (Pivotal Cohort)', 'timeFrame': 'Week 52', 'description': 'The percentage of participants who met the ≥10% weight loss threshold (responders) after approximately Week 52 (\\~1 year) of treatment were analyzed.'}], 'secondaryOutcomes': [{'measure': 'Percentage of Participants Who Reached ≥10% Weight Loss Threshold After 1 Year (Pivotal + Supplemental Cohort)', 'timeFrame': 'Week 52', 'description': 'The percentage of participants who met the ≥10% weight loss threshold (responders) after approximately Week 52 (\\~1 year) of treatment were analyzed.'}, {'measure': 'Mean Percent Change From Baseline in Body Weight', 'timeFrame': 'Baseline and Week 52', 'description': 'The mean percent change from baseline in body weight at 52 weeks was analyzed.'}, {'measure': "Mean Percent Change From Baseline in Hunger Score ('Most Hunger')", 'timeFrame': 'Baseline and Week 52', 'description': 'The mean percent change in hunger scores for participants ≥12 years of age with leptin receptor (LEPR) deficiency obesity in treatment with setmelanotide was evaluated. Hunger score ranged from 0 - 10 on a Likert-type scale; 0 = not hungry at all and 10 = hungriest possible. On the Daily Hunger Questionnaire, each of the 3 items (average hunger, most/worst hunger, and morning hunger) was scored separately and averaged on a weekly basis.'}, {'measure': 'Percentage of Participants Achieving at Least 25% Improvement in Daily Hunger From Baseline', 'timeFrame': 'Baseline and Week 52', 'description': 'The percentage of participants (≥12 years of age) achieving a ≥25% improvement from baseline in hunger score at Week 52 (i.e., after treatment with setmelanotide for 52 weeks at the therapeutic dose) was assessed. Hunger ranged from 0 - 10 on a Likert-type scale; 0 = not hungry at all and 10 = hungriest possible. On the Daily Hunger Questionnaire, each of the 3 items (average hunger, most/worst hunger, and morning hunger) was scored separately and averaged on a weekly basis.'}, {'measure': 'Absolute Change From Baseline in Waist Circumference', 'timeFrame': 'Baseline and Week 52', 'description': 'Waist circumference (cm) was measured according to the National Heart, Lung, and Blood Institute (NHLBI) criteria. Waist circumference was measured when participants were fasting at approximately the same time at each visit. The absolute change from baseline in waist circumference was assessed.'}, {'measure': 'Absolute Change in Body Weight (Reversal of Weight Loss) During Double-Blind Placebo-Controlled Withdrawal Period', 'timeFrame': 'Baseline and Week 8 of withdrawal period', 'description': 'A comparison of weight change was evaluated during the 8 week placebo-controlled withdrawal period for each participant, during which each participant received 4 weeks of placebo and 4 weeks active therapy in a blinded fashion.'}, {'measure': 'Absolute Daily Hunger Reduction Score During the Double-Blind Placebo-Controlled Withdrawal Period', 'timeFrame': 'Week 8 of withdrawal period', 'description': 'The absolute score in daily hunger reduction during the double-blind placebo-controlled withdrawal period (≥12 Years of Age) was assessed. Hunger ranged from 0 - 10 on a Likert-type scale; 0 = not hungry at all and 10 = hungriest possible. On the Daily Hunger Questionnaire, each of the 3 items (average hunger, most/worst hunger, and morning hunger) was scored separately and averaged on a weekly basis. The weekly average hunger score of the daily worst (most) hunger score in 24 hours is the hunger score used to assess this study endpoint. Lower scores represent lower hunger, higher scores represent greater hunger.'}, {'measure': 'Mean Percent Change From Baseline in Body Mass Index', 'timeFrame': 'Baseline and Week 52', 'description': 'The mean percent change from baseline in body mass index (BMI) was assessed.'}, {'measure': 'Change From Baseline in Glucose Parameters', 'timeFrame': 'Baseline and Week 52', 'description': 'Glucose parameters included glucose, glycated hemoglobin (HbA1c) and Oral glucose tolerance test (OGTT). Data is planned to be reported only for change from baseline in glucose levels.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['LEPR deficiency obesity', 'setmelanotide', 'RM-493', 'Leptin receptor', 'Obesity', 'Melanocortin 4 receptor'], 'conditions': ['Leptin Receptor Deficiency Obesity']}, 'referencesModule': {'references': [{'pmid': '33137293', 'type': 'DERIVED', 'citation': 'Clement K, van den Akker E, Argente J, Bahm A, Chung WK, Connors H, De Waele K, Farooqi IS, Gonneau-Lejeune J, Gordon G, Kohlsdorf K, Poitou C, Puder L, Swain J, Stewart M, Yuan G, Wabitsch M, Kuhnen P; Setmelanotide POMC and LEPR Phase 3 Trial Investigators. Efficacy and safety of setmelanotide, an MC4R agonist, in individuals with severe obesity due to LEPR or POMC deficiency: single-arm, open-label, multicentre, phase 3 trials. Lancet Diabetes Endocrinol. 2020 Dec;8(12):960-970. doi: 10.1016/S2213-8587(20)30364-8. Epub 2020 Oct 30.'}]}, 'descriptionModule': {'briefSummary': 'To demonstrate statistically significant and clinically meaningful effects of setmelanotide on percent body weight change in participants with LEPR deficiency obesity due to rare bi-allelic or loss-of function mutations at the end of 1 year of treatment.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Bi-allelic, homozygous or compound heterozygous (a different gene mutation on each allele) genetic status for the LEPR gene, with the loss-of-function (LOF) variant for each allele conferring a severe obesity phenotype.\n2. Age 6 years and above. 6+: Germany, Netherlands, UK. 12+: France\n3. If adult age ≥18 years, obesity with body mass index (BMI) ≥ 30 kilograms per meters squared (kg/m\\^2); if child or adolescent (\\< 18 years of age), obesity with weight \\> 97th percentile for age on growth chart assessment.\n4. Study participant and/or parent or guardian is able to communicate well with the investigator, to understand and comply with the requirements of the study, and is able to understand and sign the written informed consent/assent, after being informed about the study.\n5. Female participants of child-bearing potential must agree to use contraception as outlined in the protocol. Female participants of non-childbearing potential, defined as surgically sterile (status post hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) post-menopausal for at least 12 months (and confirmed with a screening FSH level in the post-menopausal lab range), or failure to have progressed to Tanner Stage V and/or failure to have achieved menarche, do not require contraception during the study.\n6. Male participants with female partners of childbearing potential must agree to a double barrier method if they become sexually active during the study. Male participants must not donate sperm during and for 90 days following their participation in the study.\n\nExclusion Criteria:\n\n1. Recent intensive (within 2 months) diet and/or exercise regimen with or without the use of weight loss agents including herbal medications, that has resulted in weight loss or weight stabilization.\n2. Prior gastric bypass surgery resulting in \\>10% weight loss durably maintained from the baseline pre-operative weight with no evidence of weight regain.\n3. Diagnosis of schizophrenia, bipolar disorder, personality disorder or other Diagnostic and Statistical Manual of Mental Disorders (DSM-III) disorders that the investigator believes will interfere significantly with study compliance.\n4. A Patient Health Questionnaire-9 (PHQ-9) score of ≥ 15.\n5. Any suicidal ideation of type 4 or 5 on the Columbia Suicide Severity Rating Scale (C-SSRS). Any lifetime history of a suicide attempt, or any suicidal behavior in the last month.\n6. Current, severe stable restrictive or obstructive lung disease arising because of extreme obesity, evidence of significant heart failure (New York Heart Association \\[NYHA\\] Class 3 or greater), or oncologic disease, if these were severe enough to interfere with the study and/or would confound the results.\n7. History of significant liver disease or liver injury, or current liver assessment for a cause of abnormal liver tests \\[as indicated by abnormal liver function tests, alanine transaminase (ALT), aspartate transaminase (AST), alkaline phosphatase, or serum bilirubin (\\> 2.0 x upper limit of normal (ULN) for any of these tests)\\] for an etiology other than non-alcoholic fatty liver disease (NAFLD).\n8. History or presence of impaired renal function as indicated by clinically significant abnormal creatinine, blood urea nitrogen (BUN), or urinary constituents (e.g., albuminuria) or moderate to severe renal dysfunction as defined by the Cockcroft Gault equation \\< 30 milliliter/minute (mL/min).\n9. History or close family history (parents or siblings) of skin cancer or melanoma, or participant history of ocular-cutaneous albinism.\n10. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions, determined as part of a screening comprehensive skin evaluation performed by a qualified dermatologist.\n11. Volunteer is, in the opinion of the study investigator, not suitable to participate in the study.\n12. Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing.\n13. Significant hypersensitivity to study drug.\n14. Inability to comply with every day (QD) injection regimen.\n15. Participants who have been placed in an institution through an official or court order, as well as those who are dependent on the sponsor, Investigator or study site.'}, 'identificationModule': {'nctId': 'NCT03287960', 'briefTitle': 'Setmelanotide for the Treatment of Leptin Receptor (LEPR) Deficiency Obesity', 'organization': {'class': 'INDUSTRY', 'fullName': 'Rhythm Pharmaceuticals, Inc.'}, 'officialTitle': 'An Open Label, 1-Year Trial, Including a Double-Blind Placebo-Controlled Withdrawal Period, of Setmelanotide (RM-493), a Melanocortin 4 Receptor (MC4R) Agonist, in Leptin Receptor (LEPR) Deficiency Obesity Due to Bi-Allelic Loss-of-Function LEPR Genetic Mutation', 'orgStudyIdInfo': {'id': 'RM-493-015'}, 'secondaryIdInfos': [{'id': '2017-002005-36', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Setmelanotide', 'description': 'Participants received titrated doses of setmelanotide once daily, by SC injection during titration period for 2 - 12 weeks. Thereafter, participants continued setmelanotide at their specific therapeutic dose for an additional 10 weeks during the open label treatment period. Participants who achieved at least a 5 kg weight loss (or at least 5% weight loss if baseline body weight was \\<100 kg) at the end of the open label treatment period, continued into the 8-week double-blind withdrawal period and received 4 weeks setmelanotide and 4 weeks placebo. Following the withdrawal period, participants entered open label treatment period and received setmelanotide to complete approximately 52 weeks (\\~1 year) of treatment at a therapeutic dose.', 'interventionNames': ['Drug: Setmelanotide', 'Drug: Placebo']}], 'interventions': [{'name': 'Setmelanotide', 'type': 'DRUG', 'otherNames': ['RM-493'], 'description': 'Once daily subcutaneous injection', 'armGroupLabels': ['Setmelanotide']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Once daily subcutaneous injection', 'armGroupLabels': ['Setmelanotide']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'L3P 7P3', 'city': 'Markham', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Markham Stouffville Hospital', 'geoPoint': {'lat': 43.86682, 'lon': -79.2663}}, {'zip': '75013', 'city': 'Paris', 'country': 'France', 'facility': 'Institute of Cardiometabolism and Nutrition / Pitié-Salpêtrière Hospital', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'city': 'Ulm', 'country': 'Germany', 'facility': 'University of Ulm', 'geoPoint': {'lat': 48.39841, 'lon': 9.99155}}, {'city': 'Rotterdam', 'country': 'Netherlands', 'facility': 'Erasmus Medical Center', 'geoPoint': {'lat': 51.9225, 'lon': 4.47917}}, {'zip': '97405', 'city': 'Saint-Denis', 'country': 'Reunion', 'facility': 'Hôpital Félix GUYON', 'geoPoint': {'lat': -20.88231, 'lon': 55.4504}}, {'zip': 'CB2 0QQ', 'city': 'Cambridge', 'country': 'United Kingdom', 'facility': 'University of Cambridge Metabolic Research Laboratories', 'geoPoint': {'lat': 52.2, 'lon': 0.11667}}], 'overallOfficials': [{'name': 'David Meeker, MD', 'role': 'STUDY_CHAIR', 'affiliation': 'Rhythm Pharmaceuticals, Inc.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Rhythm Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}