Gene-Targeted obesity drug shows promise in phase 3 trial

NCT ID NCT05093634

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

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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

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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