EMANATE: A Study of Setmelanotide in Patients With Specific Gene Variants in the MC4R Pathway
Purpose
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: - POMC or PCSK1 (Sub-study 035a) - LEPR (Sub-study 035b) - SRC1 (Sub-study 035c) - SH2B1 (Sub-study 035d) The objectives and endpoints are identical for these sub-studies.
Conditions
- Obesity
- Genetic Obesity
Eligibility
- Eligible Ages
- Between 6 Years and 65 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Patients must have a pre-identified: - Heterozygous genetic variant in the POMC gene or PCSK1 gene - Heterozygous genetic variant in the LEPR gene - Homozygous, heterozygous, or compound heterozygous variant in the NCOA1 (SRC1) - Homozygous, heterozygous, or compound heterozygous variant in SH2B1 gene, or chromosomal 16p11.2 deletion encompassing the SH2B1 gene - Between 6 and 65 years of age at the time of provision of informed consent/assent - 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 - 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 - 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 - Agree to use a highly effective form of contraception throughout the study and for 90 days following the study - Reported history of lifestyle intervention of diet and exercise - Reported history of hyperphagia
Exclusion Criteria
- Weight loss of 2% or greater in the previous 3 months - Recent history of bariatric surgery - Significant psychiatric disorder(s) - Suicidal ideation, attempt or behavior - Clinically significant pulmonary, cardiac, endocrine/metabolic, hepatic or oncologic disease - Glycated hemoglobin (HbA1C) >10% at Screening - History of significant liver disease or severe kidney disease - History or close family history (parents or siblings) of melanoma, or patient history of oculocutaneous albinism - Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion) - Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing - Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide - Significant hypersensitivity to any excipient in the study drug - If female, pregnant or breastfeeding
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Care Provider, Investigator)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental POMC or PCSK1 variant |
1:1 Randomization |
|
|
Experimental LEPR variant |
1:1 Randomization |
|
|
Experimental NCOA1 (SRC1) variant |
1:1 Randomization |
|
|
Experimental SH2B1 variant |
1:1 Randomization |
|
Recruiting Locations
Vanderbilt University School of Medicine
Nashville, Tennessee 37232
Nashville, Tennessee 37232
More Details
- Status
- Recruiting
- Sponsor
- Rhythm Pharmaceuticals, Inc.