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

NCT ID: NCT05093634

Last Updated: 2025-10-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

400 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-12-10

Study Completion Date

2025-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Obesity Genetic Obesity

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

POMC or PCSK1 variant

1:1 Randomization

Group Type EXPERIMENTAL

Setmelanotide

Intervention Type DRUG

Subcutaneous Injection

Placebo

Intervention Type DRUG

Subcutaneous Injection

LEPR variant

1:1 Randomization

Group Type EXPERIMENTAL

Setmelanotide

Intervention Type DRUG

Subcutaneous Injection

Placebo

Intervention Type DRUG

Subcutaneous Injection

NCOA1 (SRC1) variant

1:1 Randomization

Group Type EXPERIMENTAL

Setmelanotide

Intervention Type DRUG

Subcutaneous Injection

Placebo

Intervention Type DRUG

Subcutaneous Injection

SH2B1 variant

1:1 Randomization

Group Type EXPERIMENTAL

Setmelanotide

Intervention Type DRUG

Subcutaneous Injection

Placebo

Intervention Type DRUG

Subcutaneous Injection

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Setmelanotide

Subcutaneous Injection

Intervention Type DRUG

Placebo

Subcutaneous Injection

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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
Minimum Eligible Age

6 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Rhythm Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

David Meeker, MD

Role: STUDY_CHAIR

Rhythm Pharmaceuticals, Inc.

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status

HonorHealth Bariatric Center

Scottsdale, Arizona, United States

Site Status

Children's Hospital Los Angeles

Los Angeles, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

Yale Center for Clinical Investigation

New Haven, Connecticut, United States

Site Status

Advent Health

Altamonte Springs, Florida, United States

Site Status

InQuest Medical Research

Suwanee, Georgia, United States

Site Status

UMASS Memorial Medical Center

Worcester, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Metro Detroit Endocrinology Center

Dearborn, Michigan, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

St. Luke's Hospital

Chesterfield, Missouri, United States

Site Status

Washington University in St. Louis

St Louis, Missouri, United States

Site Status

University of Buffalo

Buffalo, New York, United States

Site Status

Metropolitan Hospital Center

New York, New York, United States

Site Status

Mount Sinai Hospital

New York, New York, United States

Site Status

Columbia University

New York, New York, United States

Site Status

Ten's Medical Center - Pediatric Endocrinology Clinic

Staten Island, New York, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Oregon Health & Science University

Portland, Oregon, United States

Site Status

Geisinger Clinic

Danville, Pennsylvania, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

UPMC Montefiore

Pittsburgh, Pennsylvania, United States

Site Status

Vanderbilt University School of Medicine

Nashville, Tennessee, United States

Site Status

Texas Tech

Amarillo, Texas, United States

Site Status

Texas Children's Hospital

Houston, Texas, United States

Site Status

Biopharma Informatic

Houston, Texas, United States

Site Status

Rio Grande Valley Endocrine Center

McAllen, Texas, United States

Site Status

Endocrine Associates of Dallas and Plano

Plano, Texas, United States

Site Status

MultiCare Health System Institute for Research and Innovation

Tacoma, Washington, United States

Site Status

West Virginia University

Morgantown, West Virginia, United States

Site Status

Marshfield Clinic Research Foundation, a Division of Marshfield Clinic, Inc

Marshfield, Wisconsin, United States

Site Status

University of Alberta - Alberta Diabetes Institute (ADI)

Edmonton, Alberta, Canada

Site Status

GH Est Hopital Femme Mere Enfant

Lyon, , France

Site Status

Hu Pitie Salpetriere Aphp

Paris, , France

Site Status

Hopital Armand Trousseau

Paris, , France

Site Status

Centre Hospitalier Universitaire (CHU) de Bordeaux

Pessac, , France

Site Status

Hôpital Félix Guyon Bellepierre

Saint-Denis, , France

Site Status

Charite - Universitatsmedizin Berlin

Berlin, , Germany

Site Status

Endokrinologikum Frankfurt

Frankfurt, , Germany

Site Status

Universitaetsklinikum Leipzig

Leipzig, , Germany

Site Status

Universitaetsklinikum Ulm

Ulm, , Germany

Site Status

University of Patras School of Medicine

Rio, , Greece

Site Status

Hasharon Hospital

Petah Tikva, , Israel

Site Status

Chaim Sheba MC, Safra Children's Hospital

Ramat Gan, , Israel

Site Status

Erasmus Medisch Centrum

Rotterdam, , Netherlands

Site Status

University of Puerto Rico

San Juan, , Puerto Rico

Site Status

Hospital Sant Joan de Deu, Barcelona

Barcelona, , Spain

Site Status

Hospital Fundación Jimenez Díaz

Madrid, , Spain

Site Status

Hospital Universitario Virgen de la Victoria de Málaga

Málaga, , Spain

Site Status

Hospital General de Valencia

Valencia, , Spain

Site Status

Bristol Royal Hospital for Children

Bristol, , United Kingdom

Site Status

University of Cambridge

Cambridge, , United Kingdom

Site Status

Aintree University Hospital

Liverpool, , United Kingdom

Site Status

London Medical - The London Diabetes Centre

London, , United Kingdom

Site Status

University College London Hospitals NHS Foundation Trust

London, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Canada France Germany Greece Israel Netherlands Puerto Rico Spain United Kingdom

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

RM-493-035

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Open-Label Extension Study of Setmelanotide
NCT06596135 ENROLLING_BY_INVITATION PHASE3
Long Term Extension Trial of Setmelanotide
NCT03651765 COMPLETED PHASE2/PHASE3
Study of NGM395 in Adult Participants
NCT04187339 COMPLETED PHASE1