Open-Label Study of Setmelanotide in Hypothalamic Obesity

Completed · Phase 2

Conditions studied: Hypothalamic Obesity

In brief

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

Key facts

Study ID

NCT04725240

Run by

Rhythm Pharmaceuticals, Inc.

People needed

18

Starts

2021-06-07

Expected to finish

2022-06-28

Last updated by the study team

2025-02-07

Who can join

Age: 6 and older, up to 40. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• 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

Full record on ClinicalTrials.gov

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