EMANATE: A Study of Setmelanotide in Patients With Specific Gene Variants in the MC4R Pathway

Completed · Phase 3 · Has a placebo group

Conditions studied: Obesity, Genetic Obesity

In brief

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.

Key facts

Study ID

NCT05093634

Run by

Rhythm Pharmaceuticals, Inc.

People needed

296

Starts

2021-12-10

Expected to finish

2026-06-30

Last updated by the study team

2026-08-06

Who can join

Age: 6 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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
• Key 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

Where it is running

• HonorHealth Bariatric Center — Scottsdale, Arizona, United States
• Children's Hospital Los Angeles — Los Angeles, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• Yale Center for Clinical Investigation — New Haven, Connecticut, United States
• Advent Health — Altamonte Springs, Florida, United States
• InQuest Medical Research — Suwanee, Georgia, United States
• UMASS Memorial Medical Center — Worcester, Massachusetts, United States
• University of Michigan — Ann Arbor, Michigan, United States
• Metro Detroit Endocrinology Center — Dearborn, Michigan, United States
• Mayo Clinic — Rochester, Minnesota, United States
• St. Luke's Hospital — Chesterfield, Missouri, United States
• Washington University in St. Louis — St Louis, Missouri, United States
• University of Buffalo — Buffalo, New York, United States
• Metropolitan Hospital Center — New York, New York, United States
• Mount Sinai Hospital — New York, New York, United States
• Columbia University — New York, New York, United States
• Ten's Medical Center - Pediatric Endocrinology Clinic — Staten Island, New York, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Oregon Health & Science University — Portland, Oregon, United States
• Geisinger Clinic — Danville, Pennsylvania, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• UPMC Montefiore — Pittsburgh, Pennsylvania, United States
• Vanderbilt University School of Medicine — Nashville, Tennessee, United States
• Texas Tech — Amarillo, Texas, United States
• Phoenix Children's Hospital — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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