Ignite Creation Date:
2025-12-25 @ 2:28 AM
Ignite Modification Date:
2025-12-26 @ 1:02 AM
Study NCT ID:
NCT05093634
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-10-15
First Post:
2021-09-30
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
EMANATE: A Study of Setmelanotide in Patients With Specific Gene Variants in the MC4R Pathway
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009765', 'term': 'Obesity'}, {'id': 'D006963', 'term': 'Hyperphagia'}], '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'}, {'id': 'D012817', 'term': 'Signs and Symptoms, Digestive'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C579663', 'term': 'setmelanotide'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 400}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2021-12-10', 'type': 'ACTUAL'}, 'statusVerifiedDate': '2025-10', 'completionDateStruct': {'date': '2025-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-10-14', 'studyFirstSubmitDate': '2021-09-30', 'studyFirstSubmitQcDate': '2021-10-13', 'lastUpdatePostDateStruct': {'date': '2025-10-15', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2021-10-26', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Difference in mean change in body weight in patients treated with setmelanotide compared to placebo, assessed as percent change in BMI', 'timeFrame': 'Baseline to 52 weeks'}], 'secondaryOutcomes': [{'measure': 'The proportion of patients who achieve at least 5% reduction in BMI in patients treated with setmelanotide compared to placebo', 'timeFrame': 'Baseline to 52 weeks'}, {'measure': 'The difference in mean change in body weight in adult patients treated with setmelanotide compared to placebo, assessed as percent change in baseline body weight', 'timeFrame': 'Baseline to 52 weeks'}, {'measure': 'The difference in mean percent change in the weekly average most hunger score in patients treated with setmelanotide compared to placebo', 'timeFrame': 'Baseline to 52 weeks'}, {'measure': 'The proportion of patients who achieve at least 10% reduction in BMI in patients treated with setmelanotide compared to placebo', 'timeFrame': 'Baseline to 52 weeks'}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Melanocortin-4 Receptor Pathway', 'Genetic Obesity', 'Hunger', 'Hyperphagia', 'POMC', 'PCSK1', 'LEPR', 'SH2B1', 'NCOA1 (SRC1)'], 'conditions': ['Obesity', 'Genetic Obesity']}, 'descriptionModule': {'briefSummary': 'The protocol describes a randomized, double-blind, placebo-controlled trial with independent sub-studies of setmelanotide in patients with obesity and at least one of the specific gene variants in the Melanocortin-4 Receptor pathway:\n\n* POMC or PCSK1 (Sub-study 035a)\n* LEPR (Sub-study 035b)\n* SRC1 (Sub-study 035c)\n* SH2B1 (Sub-study 035d)\n\nThe objectives and endpoints are identical for these sub-studies.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patients must have a pre-identified:\n\n * Heterozygous genetic variant in the POMC gene or PCSK1 gene\n * Heterozygous genetic variant in the LEPR gene\n * Homozygous, heterozygous, or compound heterozygous variant in the NCOA1 (SRC1)\n * Homozygous, heterozygous, or compound heterozygous variant in SH2B1 gene, or chromosomal 16p11.2 deletion encompassing the SH2B1 gene\n* Between 6 and 65 years of age at the time of provision of informed consent/assent\n* Obesity, defined as BMI ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th percentile for age and gender for patients 6 up to 17 years of age\n* Patient and/or parent or guardian is able to understand and comply with the requirements of the study and is able to understand and sign the written informed consent/assent\n* Patient and/or parent or guardian reports that patient experienced childhood obesity, defined as the patient and/or parent or guardian reporting that the patient was significantly overweight during childhood\n* Agree to use a highly effective form of contraception throughout the study and for 90 days following the study\n* Reported history of lifestyle intervention of diet and exercise\n* Reported history of hyperphagia\n\nKey Exclusion Criteria:\n\n* Weight loss of 2% or greater in the previous 3 months\n* Recent history of bariatric surgery\n* Significant psychiatric disorder(s)\n* Suicidal ideation, attempt or behavior\n* Clinically significant pulmonary, cardiac, endocrine/metabolic, hepatic or oncologic disease\n* Glycated hemoglobin (HbA1C) \\>10% at Screening\n* History of significant liver disease or severe kidney disease\n* History or close family history (parents or siblings) of melanoma, or patient history of oculocutaneous albinism\n* Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)\n* Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing\n* Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide\n* Significant hypersensitivity to any excipient in the study drug\n* If female, pregnant or breastfeeding'}, 'identificationModule': {'nctId': 'NCT05093634', 'briefTitle': 'EMANATE: A Study of Setmelanotide in Patients With Specific Gene Variants in the MC4R Pathway', 'organization': {'class': 'INDUSTRY', 'fullName': 'Rhythm Pharmaceuticals, Inc.'}, 'officialTitle': 'A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial: Multiple Independent Sub-studies of Setmelanotide in Patients With POMC/PCSK1, LEPR, NCOA1(SRC1), or SH2B1 Gene Variants in the Melanocortin-4 Receptor Pathway', 'orgStudyIdInfo': {'id': 'RM-493-035'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'POMC or PCSK1 variant', 'description': '1:1 Randomization', 'interventionNames': ['Drug: Setmelanotide', 'Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'LEPR variant', 'description': '1:1 Randomization', 'interventionNames': ['Drug: Setmelanotide', 'Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'NCOA1 (SRC1) variant', 'description': '1:1 Randomization', 'interventionNames': ['Drug: Setmelanotide', 'Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'SH2B1 variant', 'description': '1:1 Randomization', 'interventionNames': ['Drug: Setmelanotide', 'Drug: Placebo']}], 'interventions': [{'name': 'Setmelanotide', 'type': 'DRUG', 'description': 'Subcutaneous Injection', 'armGroupLabels': ['LEPR variant', 'NCOA1 (SRC1) variant', 'POMC or PCSK1 variant', 'SH2B1 variant']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Subcutaneous Injection', 'armGroupLabels': ['LEPR variant', 'NCOA1 (SRC1) variant', 'POMC or PCSK1 variant', 'SH2B1 variant']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85016', 'city': 'Phoenix', 'state': 'Arizona', 'country': 'United States', 'facility': "Phoenix Children's Hospital", 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '85258', 'city': 'Scottsdale', 'state': 'Arizona', 'country': 'United States', 'facility': 'HonorHealth Bariatric Center', 'geoPoint': {'lat': 33.50921, 'lon': -111.89903}}, {'zip': '90027', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital Los Angeles", 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '06519', 'city': 'New Haven', 'state': 'Connecticut', 'country': 'United States', 'facility': 'Yale Center for Clinical Investigation', 'geoPoint': {'lat': 41.30815, 'lon': -72.92816}}, {'zip': '32701', 'city': 'Altamonte Springs', 'state': 'Florida', 'country': 'United States', 'facility': 'Advent Health', 'geoPoint': {'lat': 28.66111, 'lon': -81.36562}}, {'zip': '30024', 'city': 'Suwanee', 'state': 'Georgia', 'country': 'United States', 'facility': 'InQuest Medical Research', 'geoPoint': {'lat': 34.05149, 'lon': -84.0713}}, {'zip': '01655', 'city': 'Worcester', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'UMASS Memorial Medical Center', 'geoPoint': {'lat': 42.26259, 'lon': 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'University of Buffalo', 'geoPoint': {'lat': 42.88645, 'lon': -78.87837}}, {'zip': '10029', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Metropolitan Hospital Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '10029', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Mount Sinai Hospital', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '10306', 'city': 'Staten Island', 'state': 'New York', 'country': 'United States', 'facility': "Ten's Medical Center - Pediatric Endocrinology Clinic", 'geoPoint': {'lat': 40.56233, 'lon': -74.13986}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': 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Armand Trousseau', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '33604', 'city': 'Pessac', 'country': 'France', 'facility': 'Centre Hospitalier Universitaire (CHU) de Bordeaux', 'geoPoint': {'lat': 44.80565, 'lon': -0.6324}}, {'zip': '97400', 'city': 'Saint-Denis', 'country': 'France', 'facility': 'Hôpital Félix Guyon Bellepierre', 'geoPoint': {'lat': 48.93564, 'lon': 2.35387}}, {'zip': '10117', 'city': 'Berlin', 'country': 'Germany', 'facility': 'Charite - Universitatsmedizin Berlin', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'zip': '60596', 'city': 'Frankfurt', 'country': 'Germany', 'facility': 'Endokrinologikum Frankfurt', 'geoPoint': {'lat': 49.68333, 'lon': 10.53333}}, {'zip': '04103', 'city': 'Leipzig', 'country': 'Germany', 'facility': 'Universitaetsklinikum Leipzig', 'geoPoint': {'lat': 51.33962, 'lon': 12.37129}}, {'zip': '89075', 'city': 'Ulm', 'country': 'Germany', 'facility': 'Universitaetsklinikum Ulm', 'geoPoint': {'lat': 48.39841, 'lon': 9.99155}}, 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'Hospital Fundación Jimenez Díaz', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': '29010', 'city': 'Málaga', 'country': 'Spain', 'facility': 'Hospital Universitario Virgen de la Victoria de Málaga', 'geoPoint': {'lat': 36.72016, 'lon': -4.42034}}, {'zip': '46014', 'city': 'Valencia', 'country': 'Spain', 'facility': 'Hospital General de Valencia', 'geoPoint': {'lat': 39.47391, 'lon': -0.37966}}, {'zip': 'BS2 8AE', 'city': 'Bristol', 'country': 'United Kingdom', 'facility': 'Bristol Royal Hospital for Children', 'geoPoint': {'lat': 51.45523, 'lon': -2.59665}}, {'zip': 'CB2 0QQ', 'city': 'Cambridge', 'country': 'United Kingdom', 'facility': 'University of Cambridge', 'geoPoint': {'lat': 52.2, 'lon': 0.11667}}, {'zip': 'L9 7AL', 'city': 'Liverpool', 'country': 'United Kingdom', 'facility': 'Aintree University Hospital', 'geoPoint': {'lat': 53.41058, 'lon': -2.97794}}, {'zip': 'NW1 2PG', 'city': 'London', 'country': 'United Kingdom', 'facility': 'London Medical - The London Diabetes Centre', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'WC1E6JF', 'city': 'London', 'country': 'United Kingdom', 'facility': 'University College London Hospitals NHS Foundation Trust', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'David Meeker, MD', 'role': 'STUDY_CHAIR', 'affiliation': 'Rhythm Pharmaceuticals, Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Rhythm Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}