Open-Label Study of Setmelanotide in Hypothalamic Obesity

NCT ID: NCT04725240

Last Updated: 2025-02-07

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 18 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-06-07
• Study Completion Date: 2022-06-28

Brief Summary

Open-label, single-arm study designed to evaluate the body weight response to setmelanotide administered subcutaneously (SC) daily in participants with hypothalamic obesity (HO).

Conditions

Hypothalamic Obesity

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Setmelanotide

Participants received setmelanotide once daily (QD) via SC injection for 16 weeks. All participants initiated treatment with setmelanotide (starting dose being age dependent) and dose escalated up to a maximum dose of 3.0 milligrams (mg) QD.

Group Type: EXPERIMENTAL

Setmelanotide

Intervention Type: DRUG

Setmelanotide for SC injection

Interventions

Setmelanotide

Setmelanotide for SC injection

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

Participants must meet all of the following criteria to be eligible for study participation:

• Participant has documented evidence of HO, including:

• Recent evidence of hypothalamic injury on magnetic resonance imaging (MRI); AND
• Diagnosis of craniopharyngioma or other non-malignant brain tumor affecting the hypothalamic region; AND
• Has undergone surgery, or chemotherapy, or radiation ≥6 months and ≤15 years before Screening.
• Participant has either unilateral hypothalamic lesions or bilateral hypothalamic lesions, as assessed by MRI.
• Obesity, documented by a body mass index (BMI) ≥35 kilograms/square meter (kg/m^2) for participants ≥18 years of age or BMI ≥95th percentile for age and gender for participants 6 to <18 years of age.
• Documented increase in BMI (change from pre-surgery baseline in BMI z-score ≥0.2 for participants <18 years of age or BMI >5% for participants >18 years of age) either during the first 6 months following surgery or within 1 year before surgery AND still present at Screening.
• More than 6 months after the end of post-tumor treatment, including chemotherapy, surgery, or radiation.
• Highly effective contraception throughout the study and for 90 days following the study.
• Ability to communicate well with the Investigator, understand and comply with the requirements of the study, and understand and sign the written informed consent, or, for participants aged <18 years, a parent/legal guardian that can sign.
• If receiving hormone replacement therapy, the dose of such therapy has remained stable for at least 2 months prior to Screening.

Exclusion Criteria

Participants meeting any of the following criteria are not eligible for study participation:

• Weight gain >5% in the previous 3 months.
• Weight loss ≥2% in the previous 3 months.
• Bariatric surgery or procedure within the last 6 months.
• Diagnosis of severe psychiatric disorders
• Glycated hemoglobin (HbA1c) >10.0% at Screening.
• Current, clinically significant pulmonary, cardiac, or oncologic disease considered severe enough to interfere with the study and/or confound the results.
• Glomerular filtration rate (GFR) <30mL/min/1.73m^2 during Screening.
• Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions.
• History or close family history (parents or siblings) of skin cancer or melanoma
• Participation in any clinical study with an investigational drug/device within 3 months or 5 half-lives, whichever is longer, prior to the first setmelanotide dose.
• Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide.
• Inability to comply with QD injection regimen.
• Pregnant and/or breastfeeding, or desiring to become pregnant during this trial.
• Cognitive impairment that, in the Investigator's opinion, precludes participation to the study and completions of study procedures or questionnaires.
• Participant is, in Investigator's opinion, otherwise not suitable to participate in the study.

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 40 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Rhythm Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

David Meeker, MD

Role: STUDY_CHAIR

Rhythm Pharmaceuticals, Inc.

Locations

Rady Children's Hospital

San Diego, California, United States

University of Florida

Gainesville, Florida, United States

Children's Minnesota

Saint Paul, Minnesota, United States

Vanderbilt University School of Medicine

Nashville, Tennessee, United States

Seattle Children's Research Institute

Seattle, Washington, United States

Countries

United States

References

Roth CL, Scimia C, Shoemaker AH, Gottschalk M, Miller J, Yuan G, Malhotra S, Abuzzahab MJ. Setmelanotide for the treatment of acquired hypothalamic obesity: a phase 2, open-label, multicentre trial. Lancet Diabetes Endocrinol. 2024 Jun;12(6):380-389. doi: 10.1016/S2213-8587(24)00087-1. Epub 2024 Apr 30.

Reference Type: BACKGROUND

PMID: 38697184 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2022-004107-32

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

RM-493-030

Identifier Type: -

Identifier Source: org_study_id