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Gene-Targeted obesity drug shows promise in phase 3 trial

NCT ID NCT05093634

Disease control Sponsor: Rhythm Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Aug 07, 2026 · Updated 3 times

Summary

This phase 3 trial is testing a drug called setmelanotide in people with obesity linked to certain gene variants (POMC, PCSK1, LEPR, NCOA1, or SH2B1). About 296 participants, aged 6 to 65, will receive either the drug or a placebo to see if it helps reduce body weight. The study aims to find a new treatment option for these rare genetic forms of obesity.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Setmelanotide (a drug injected under the skin)
What this could lead to
If successful, this could provide a targeted treatment for people with obesity caused by certain gene variants, helping them lose weight.
What could go wrong
This is still an early-stage trial, and results may not show significant weight loss. Side effects from the drug are possible, and it may not work for everyone with these gene variants.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

296 people

The number who actually took part.

Started

Dec 2021

Finished

Jun 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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 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 * If female, pregnant or breastfeeding

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Conditions

The condition(s) this trial relates to.

Hyperphagia inherited obesity Obesity obesity disorder obesity due to leptin receptor gene deficiency obesity due to pro-opiomelanocortin deficiency obesity due to prohormone convertase I deficiency severe early-onset obesity-insulin resistance syndrome due to SH2B1 deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Genetic obesity Obesity

Contacts and locations

Locations

  • Advent Health

    Altamonte Springs, Florida, 32701, United States

  • Aintree University Hospital

    Liverpool, L9 7AL, United Kingdom

  • Biopharma Informatic

    Houston, Texas, 77084, United States

  • Bristol Royal Hospital for Children

    Bristol, BS2 8AE, United Kingdom

  • Centre Hospitalier Universitaire (CHU) de Bordeaux

    Pessac, 33604, France

  • Chaim Sheba MC, Safra Children's Hospital

    Ramat Gan, 52621, Israel

  • Charite - Universitatsmedizin Berlin

    Berlin, 10117, Germany

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Columbia University

    New York, New York, 10032, United States

  • Endocrine Associates of Dallas and Plano

    Plano, Texas, 75093, United States

  • Endokrinologikum Frankfurt

    Frankfurt, 60596, Germany

  • Erasmus Medisch Centrum

    Rotterdam, 3015 GD, Netherlands

  • GH Est Hopital Femme Mere Enfant

    Lyon, 69677, France

  • Geisinger Clinic

    Danville, Pennsylvania, 17822, United States

  • Hasharon Hospital

    Petah Tikva, 49372, Israel

  • HonorHealth Bariatric Center

    Scottsdale, Arizona, 85258, United States

  • Hopital Armand Trousseau

    Paris, 75771, France

  • Hospital Fundación Jimenez Díaz

    Madrid, 28040, Spain

  • Hospital General de Valencia

    Valencia, 46014, Spain

  • Hospital Sant Joan de Deu, Barcelona

    Barcelona, 08950, Spain

  • Hospital Universitario Virgen de la Victoria de Málaga

    Málaga, 29010, Spain

  • Hu Pitie Salpetriere Aphp

    Paris, 75651, France

  • Hôpital Félix Guyon Bellepierre

    Saint-Denis, 97400, France

  • InQuest Medical Research

    Suwanee, Georgia, 30024, United States

  • London Medical - The London Diabetes Centre

    London, NW1 2PG, United Kingdom

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

    Marshfield, Wisconsin, 54449, United States

  • Mayo Clinic

    Rochester, Minnesota, 55902, United States

  • Metro Detroit Endocrinology Center

    Dearborn, Michigan, 48126-2619, United States

  • Metropolitan Hospital Center

    New York, New York, 10029, United States

  • Mount Sinai Hospital

    New York, New York, 10029, United States

  • MultiCare Health System Institute for Research and Innovation

    Tacoma, Washington, 98405, United States

  • Oregon Health & Science University

    Portland, Oregon, 97239, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • Rio Grande Valley Endocrine Center

    McAllen, Texas, 78503, United States

  • St. Luke's Hospital

    Chesterfield, Missouri, 63017, United States

  • Ten's Medical Center - Pediatric Endocrinology Clinic

    Staten Island, New York, 10306, United States

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • Texas Tech

    Amarillo, Texas, 79106, United States

  • UMASS Memorial Medical Center

    Worcester, Massachusetts, 01655, United States

  • UPMC Montefiore

    Pittsburgh, Pennsylvania, 15213, United States

  • Universitaetsklinikum Leipzig

    Leipzig, 04103, Germany

  • Universitaetsklinikum Ulm

    Ulm, 89075, Germany

  • University College London Hospitals NHS Foundation Trust

    London, WC1E6JF, United Kingdom

  • University of Alberta - Alberta Diabetes Institute (ADI)

    Edmonton, Alberta, T6G 2E1, Canada

  • University of Buffalo

    Buffalo, New York, 14260, United States

  • University of Cambridge

    Cambridge, CB2 0QQ, United Kingdom

  • University of Michigan

    Ann Arbor, Michigan, 48105, United States

  • University of Patras School of Medicine

    Rio, 26504, Greece

  • University of Puerto Rico

    San Juan, 00925-253, Puerto Rico

  • Vanderbilt University School of Medicine

    Nashville, Tennessee, 37232, United States

  • Washington University in St. Louis

    St Louis, Missouri, 63130, United States

  • West Virginia University

    Morgantown, West Virginia, 26506, United States

  • Yale Center for Clinical Investigation

    New Haven, Connecticut, 06519, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.