Ignite Creation Date:
2025-12-25 @ 12:43 AM
Ignite Modification Date:
2025-12-27 @ 11:19 AM
Study NCT ID:
NCT04988867
Status:
TERMINATED
Last Update Posted:
2024-09-24
First Post:
2021-07-23
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2024-09-19', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D015518', 'term': 'Rett Syndrome'}], 'ancestors': [{'id': 'D038901', 'term': 'X-Linked Intellectual Disability'}, {'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000656362', 'term': 'trofinetide'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medicalinformation@acadia-pharm.com', 'phone': '+1-858-261', 'title': 'Sr. Dir. Medical Information and Medical Communications', 'phoneExt': '2897', 'organization': 'ACADIA Pharmaceuticals Inc.'}, 'certainAgreement': {'otherDetails': "Investigator may publish the study results, relative to his/her own patients, only after review, comment and approval by the sponsor. No publication of confidential information shall be made without the sponsor's prior written consent. At least 60 days prior to submitting a manuscript or prior to any public presentation, a copy of the manuscript or presentation will be provided to the sponsor for review and comment. The sponsor has 60 days to review and comment.", 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': 'As a result of marketing approval of trofinetide on 10 March 2023, the study was terminated by the Sponsor with the intent of switching patients to commercially available product. The study was terminated on 31 May 2023.'}}, 'adverseEventsModule': {'timeFrame': 'Mean study drug exposure 434 days, corresponding to 1.2 years', 'eventGroups': [{'id': 'EG000', 'title': 'Trofinetide', 'description': 'Trofinetide oral solution was administered twice daily for up to approximately 26 months, orally or administered by gastrostomy (G) tube.', 'otherNumAtRisk': 15, 'deathsNumAtRisk': 15, 'otherNumAffected': 14, 'seriousNumAtRisk': 15, 'deathsNumAffected': 0, 'seriousNumAffected': 4}], 'otherEvents': [{'term': 'Iron deficiency anaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Rett syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 26, 'numAffected': 12}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 11, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Dysphagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Flatulence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Salivary hypersecretion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Toothache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 9, 'numAffected': 7}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 7, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 7, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Candida infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Croup infectious', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Oral candidiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Otitis media acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Pharyngitis streptococcal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Skin infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Arthropod bite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Weight decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Alanine aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Aspartate aminotransferase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Feeding disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Decreased appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Scoliosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Seizure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 9, 'numAffected': 5}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Epilepsy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Somnolence', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Altered state of consciousness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Irritability', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Dermatitis diaper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Dermatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Erythema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Excessive granulation tissue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}], 'seriousEvents': [{'term': 'Dysphagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Gastroenteritis sapovirus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Seizure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}, {'term': 'Altered state of consciousness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 24.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Safety and Tolerability of Treatment With Oral Trofinetide', 'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Trofinetide', 'description': 'Trofinetide oral solution was administered twice daily for up to approximately 26 months, orally or administered by gastrostomy (G) tube.'}], 'classes': [{'title': 'Patients with TEAEs', 'categories': [{'measurements': [{'value': '14', 'groupId': 'OG000'}]}]}, {'title': 'Patients with SAEs', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Patients with AEs leading to discontinuation', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Alanine transaminase ≥3 ULN', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Aspartate transaminase ≥3 ULN', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Potassium ≤3.0 mmol/L', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Leukocytes ≥15 × 10Ê9/L', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Neutrophils ≤1.5 × 10E9/L', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Hematocrit <0.3', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Hemoglobin <100 g/L', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Mean study drug exposure 434 days, corresponding to 1.2 years', 'description': 'Percentage of patients with treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), withdrawals due to AEs, potentially clinically important (PCI) changes in other safety assessments (laboratory values, vital signs, or ECGs, following protocol-defined PCI criteria)', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All patients enrolled and treated'}, {'type': 'PRIMARY', 'title': 'AUC0-12,ss (Area Under the Concentration-time Curve From Time 0 to 12 h at Steady State)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Trofinetide', 'description': 'Trofinetide oral solution was administered twice daily for up to approximately 26 months, orally or administered by gastrostomy (G) tube.'}], 'classes': [{'categories': [{'measurements': [{'value': '1015.7', 'spread': '135.9', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'PK samples were taken predose and at Weeks 2, 4, 8, and 12', 'description': 'Area under the concentration-time curve from time 0 to 12 h at steady state as obtained from population pharmacokinetic (PK) modelling', 'unitOfMeasure': 'μg x h/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Patients enrolled, treated, and with at least one quantifiable PK concentration'}, {'type': 'PRIMARY', 'title': 'Cmax,ss (Maximum Observed Drug Concentration at Steady State)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Trofinetide', 'description': 'Trofinetide oral solution was administered twice daily for up to approximately 26 months, orally or administered by gastrostomy (G) tube.'}], 'classes': [{'categories': [{'measurements': [{'value': '181.5', 'spread': '22.8', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'PK samples were taken predose and at Weeks 2, 4, 8, and 12', 'description': 'Maximum observed drug concentration at steady state as obtained from population pharmacokinetic (PK) modelling', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Patients enrolled, treated, and with at least one quantifiable PK concentration'}, {'type': 'PRIMARY', 'title': 'Cmin,ss (Minimum Observed Drug Concentration at Steady State of Oral Trofinetide)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Trofinetide', 'description': 'Trofinetide oral solution was administered twice daily for up to approximately 26 months, orally or administered by gastrostomy (G) tube.'}], 'classes': [{'categories': [{'measurements': [{'value': '19.6', 'spread': '6.3', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'PK samples were taken predose and at Weeks 2, 4, 8, and 12', 'description': 'Minimum observed drug concentration at steady state of oral trofinetide as obtained from population pharmacokinetic (PK) modeling', 'unitOfMeasure': 'μg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Patients enrolled, treated, and with at least one quantifiable PK concentration'}, {'type': 'PRIMARY', 'title': 'Tmax (Time of the Maximum Observed Drug Concentration at Steady State)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Trofinetide', 'description': 'Trofinetide oral solution was administered twice daily for up to approximately 26 months, orally or administered by gastrostomy (G) tube.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.8', 'spread': '0.2', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'PK samples were taken predose and at Weeks 2, 4, 8, and 12', 'description': 'Time of the maximum observed drug concentration at steady state as obtained from population pharmacokinetic (PK) modelling', 'unitOfMeasure': 'h', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Patients enrolled, treated, and with at least one quantifiable PK concentration'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Trofinetide', 'description': 'Trofinetide oral solution was administered twice daily for up to approximately 26 months, orally or administered by gastrostomy (G) tube.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}]}], 'dropWithdraws': [{'type': 'Study terminated by Sponsor', 'reasons': [{'groupId': 'FG000', 'numSubjects': '12'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Study drug noncompliance', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Trofinetide', 'description': 'Trofinetide oral solution was administered twice daily for up to approximately 26 months, orally or administered by gastrostomy (G) tube.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '3.1', 'spread': '0.8', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '15', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '0', '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': '1', 'groupId': 'BG000'}]}, {'title': 'White', 'measurements': [{'value': '13', 'groupId': 'BG000'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '15', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'All patients enrolled and treated'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2022-12-19', 'size': 810746, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2024-07-15T07:07', 'hasProtocol': True}, {'date': '2023-06-26', 'size': 682679, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2024-07-15T07:03', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 15}}, 'statusModule': {'whyStopped': 'As a result of marketing approval of trofinetide on 10 March 2023, the study was terminated by the Sponsor with the intent of switching patients to commercially available product.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2021-09-22', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-09', 'completionDateStruct': {'date': '2023-05-31', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-09-17', 'studyFirstSubmitDate': '2021-07-23', 'resultsFirstSubmitDate': '2024-08-19', 'studyFirstSubmitQcDate': '2021-08-03', 'lastUpdatePostDateStruct': {'date': '2024-09-24', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-09-17', 'studyFirstPostDateStruct': {'date': '2021-08-04', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-09-24', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-05-31', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety and Tolerability of Treatment With Oral Trofinetide', 'timeFrame': 'Mean study drug exposure 434 days, corresponding to 1.2 years', 'description': 'Percentage of patients with treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), withdrawals due to AEs, potentially clinically important (PCI) changes in other safety assessments (laboratory values, vital signs, or ECGs, following protocol-defined PCI criteria)'}, {'measure': 'AUC0-12,ss (Area Under the Concentration-time Curve From Time 0 to 12 h at Steady State)', 'timeFrame': 'PK samples were taken predose and at Weeks 2, 4, 8, and 12', 'description': 'Area under the concentration-time curve from time 0 to 12 h at steady state as obtained from population pharmacokinetic (PK) modelling'}, {'measure': 'Cmax,ss (Maximum Observed Drug Concentration at Steady State)', 'timeFrame': 'PK samples were taken predose and at Weeks 2, 4, 8, and 12', 'description': 'Maximum observed drug concentration at steady state as obtained from population pharmacokinetic (PK) modelling'}, {'measure': 'Cmin,ss (Minimum Observed Drug Concentration at Steady State of Oral Trofinetide)', 'timeFrame': 'PK samples were taken predose and at Weeks 2, 4, 8, and 12', 'description': 'Minimum observed drug concentration at steady state of oral trofinetide as obtained from population pharmacokinetic (PK) modeling'}, {'measure': 'Tmax (Time of the Maximum Observed Drug Concentration at Steady State)', 'timeFrame': 'PK samples were taken predose and at Weeks 2, 4, 8, and 12', 'description': 'Time of the maximum observed drug concentration at steady state as obtained from population pharmacokinetic (PK) modelling'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Rett Syndrome']}, 'referencesModule': {'references': [{'pmid': '40043705', 'type': 'DERIVED', 'citation': 'Percy AK, Ryther R, Marsh ED, Neul JL, Benke TA, Berry-Kravis EM, Feyma T, Lieberman DN, Ananth AL, Fu C, Buhrfiend C, Barrett A, Doshi D, Darwish M, An D, Bishop KM, Youakim JM. Results from the phase 2/3 DAFFODIL study of trofinetide in girls aged 2-4 years with Rett syndrome. Med. 2025 Jun 13;6(6):100608. doi: 10.1016/j.medj.2025.100608. Epub 2025 Mar 4.'}, {'pmid': '37460385', 'type': 'DERIVED', 'citation': 'Parent H, Ferranti A, Niswender C. Trofinetide: a pioneering treatment for Rett syndrome. Trends Pharmacol Sci. 2023 Oct;44(10):740-741. doi: 10.1016/j.tips.2023.06.008. Epub 2023 Jul 16.'}]}, 'descriptionModule': {'briefSummary': 'To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls with Rett syndrome'}, 'eligibilityModule': {'sex': 'FEMALE', 'stdAges': ['CHILD'], 'maximumAge': '5 Years', 'minimumAge': '2 Years', 'genderBased': True, 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Female subject\n\n 1. 2 to 4 years of age and body weight ≥9 kg and \\<20 kg at Screening OR\n 2. 5 years of age and body weight ≥9 kg and \\<12 kg at Screening\n* Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube\n* The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments\n* Has classic/typical Rett syndrome (RTT) or possible RTT according to the Rett Syndrome Diagnostic Criteria\n* Has a documented disease-causing mutation in the MECP2 gene\n* Has a stable pattern of seizures, or has had no seizures, within 8 weeks prior to Screening\n* Subject and caregiver(s) must reside at a location to which study drug can be delivered and have been at their present residence for at least 4 weeks prior to Screening\n\nExclusion Criteria:\n\n* Has been treated with insulin within 12 weeks of Baseline\n* Has current clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, Type 1 diabetes mellitus, or uncontrolled Type 2 diabetes mellitus), renal, hepatic, respiratory or gastrointestinal disease (such as celiac disease or inflammatory bowel disease) or has major surgery planned during the study\n* Has a history of, or current, cerebrovascular disease or brain trauma\n* Has significant, uncorrected visual or uncorrected hearing impairment\n* Has a history of, or current, malignancy\n* Has any of the following:\n\n 1. QTcF interval of \\>450 ms at Screening or Baseline\n 2. History of a risk factor for torsades de pointes (e.g., heart failure or family history of long QT syndrome)\n 3. History of clinically significant QT prolongation that is deemed to put the subject at increased risk of clinically significant QT prolongation\n 4. Other clinically significant finding on ECG at Screening or Baseline\n\nAdditional inclusion/exclusion criteria apply. Patients will be evaluated at baseline to ensure that all criteria for study participation are met. Patients may be excluded from the study based on these assessments (and specifically, if it is determined that their baseline health and condition do not meet all prespecified entry criteria)."}, 'identificationModule': {'nctId': 'NCT04988867', 'acronym': 'DAFFODIL™', 'briefTitle': 'An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome', 'organization': {'class': 'INDUSTRY', 'fullName': 'ACADIA Pharmaceuticals Inc.'}, 'officialTitle': 'An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome', 'orgStudyIdInfo': {'id': 'ACP-2566-009'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Drug - trofinetide', 'description': 'Oral dose of trofinetide', 'interventionNames': ['Drug: Trofinetide']}], 'interventions': [{'name': 'Trofinetide', 'type': 'DRUG', 'description': "Trofinetide solution of 10-30 mL based on subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)", 'armGroupLabels': ['Drug - trofinetide']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35233', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': 'University of Alabama at Birmingham', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Rush University Medical Center', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Boston Children's Hospital/Harvard Medical School", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '55101', 'city': 'Saint Paul', 'state': 'Minnesota', 'country': 'United States', 'facility': "Gillette Children's Hospital", 'geoPoint': {'lat': 44.94441, 'lon': -93.09327}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '37232', 'city': 'Nashville', 'state': 'Tennessee', 'country': 'United States', 'facility': 'Vanderbilt University Medical Center', 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'ACADIA Pharmaceuticals Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}