A Trial of Setmelanotide in Acquired Hypothalamic Obesity

NCT ID: NCT05774756

Last Updated: 2025-07-02

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

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

120 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-04-26

Study Completion Date

2027-04-16

Brief Summary

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The goal of this trial is to learn how well Setmelanotide works to improve weight reduction, hunger, and quality of life in patients 4 years of age and older with acquired Hypothalamic Obesity (HO). To determine how well setmelanotide works and how safe it is, patients with HO will take a daily injection of either setmelanotide or placebo and complete trial assessments for 52 weeks on a therapeutic regimen.

A separate sub-study in patients with congenital HO is detailed under NCT06760546.

Detailed Description

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Conditions

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Hypothalamic Obesity

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Setemelanotide

Randomized 2:1 (Setmelanotide: Placebo)

Group Type EXPERIMENTAL

Setmelanotide

Intervention Type DRUG

Solution for daily subcutaneous injection

Placebo

Randomized 2:1 (Setmelanotide: Placebo)

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo matched to setmelanotide for daily subcutaneous injection

Interventions

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Setmelanotide

Solution for daily subcutaneous injection

Intervention Type DRUG

Placebo

Placebo matched to setmelanotide for daily subcutaneous injection

Intervention Type DRUG

Other Intervention Names

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RM-493 Imcivree

Eligibility Criteria

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Inclusion Criteria

1. Documented evidence of acquired hypothalamic obesity (HO)
2. Age 4 years and older
3. Weight gain associated with the hypothalamic injury and a BMI of ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th percentile for age and sex for patients 4 to \<18 years of age
4. Agree to use a highly effective form of contraception throughout the study and for 90 days after the study

Exclusion Criteria

1. Diagnosis of Prader-Willi syndrome (PWS) or Rapid-onset obesity with hypoventilation, hypothalamic, autonomic dysregulation, neuroendocrine tumor syndrome (ROHHADNET)
2. Weight loss \>2% in the previous 3 months for patients aged ≥18 years or \>2% reduction in BMI for patients aged 4 to \<18 years
3. Bariatric surgery or procedure within last 2 years
4. Diagnosis of severe psychiatric disorders; any suicidal ideation, attempt or behavior
5. Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease
6. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)
7. History or close family history of skin cancer or melanoma
8. Participation in any clinical trial with an investigational drug/device within 3 months prior to the first trial dose
9. Previously enrolled in a clinical trial involving setmelanotide or any previous exposure to setmelanotide
10. Inability to comply with once daily (QD) injection regimen
11. If female, pregnant and/or breastfeeding
12. Patients with obesity attributable to other genetic or syndromic conditions (eg, PPL \[POMC, PCSK1, LEPR, collectively\], BBS) prior to the hypothalamic injury.
13. If receiving hormone replacement therapy, dose has remained stable for at least 2 months before Screening
Minimum Eligible Age

4 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Rhythm Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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David Meeker, MD

Role: STUDY_CHAIR

Rhythm Pharmaceuticals, Inc.

Locations

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UAN Pediatric Endocrinology

Birmingham, Alabama, United States

Site Status

Rady Children's Hospital

San Diego, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

Ann and Robert H. Lurie Children's Hospital

Chicago, Illinois, United States

Site Status

University of Iowa Stead Family Department of Pediatrics

Iowa City, Iowa, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status

Children's Minnesota

Saint Paul, Minnesota, United States

Site Status

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Site Status

Columbia University Irving Medical Center

New York, New York, United States

Site Status

Ohio State Wexner Medical Center

Columbus, Ohio, United States

Site Status

Lynn Health Science Institute

Oklahoma City, Oklahoma, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Vanderbilt University School of Medicine

Nashville, Tennessee, United States

Site Status

Seattle Children's Hospital, Research and Foundation - Center for Integrative Brain Research

Seattle, Washington, United States

Site Status

Hospital for Sick Children

Toronto, Ontario, Canada

Site Status

Universitaetsklinikum Hamburg-Eppendorf (UKE) - Ambulanzzentrum des UKE GmbH

Hamburg, , Germany

Site Status

Medicover Neuroendokrinologie

München, , Germany

Site Status

University Children's Hospital, Klinikum Oldenburg

Oldenburg, , Germany

Site Status

Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin

Ulm, , Germany

Site Status

Nagoya City University Hospital

Nagoya, Aichi-ken, Japan

Site Status

Nagano Children's Hospital

Azumino, Nagano, Japan

Site Status

Toranomon Hospital

Minato, Tokyo, Japan

Site Status

Prinses Maxima Center for Pediatric Oncology

Utrecht, , Netherlands

Site Status

Birmingham Women and Children's Hospital NHS Trust

Birmingham, , United Kingdom

Site Status

Hull University Teaching Hospital

Hull, , United Kingdom

Site Status

UCL Great Ormond Street Institute of Child Health

London, , United Kingdom

Site Status

Countries

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United States Canada Germany Japan Netherlands United Kingdom

Other Identifiers

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RM-493-040

Identifier Type: -

Identifier Source: org_study_id

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