Status:
COMPLETED
Open-Label Study of Setmelanotide in Hypothalamic Obesity
Lead Sponsor:
Rhythm Pharmaceuticals, Inc.
Conditions:
Hypothalamic Obesity
Eligibility:
All Genders
6-40 years
Phase:
PHASE2
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).
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
June 7 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 28 2022
Estimated Enrollment :
18 Patients enrolled
Trial Details
Trial ID
NCT04725240
Start Date
June 7 2021
End Date
June 28 2022
Last Update
February 7 2025
Active Locations (5)
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1
Rady Children's Hospital
San Diego, California, United States, 92123
2
University of Florida
Gainesville, Florida, United States, 32610-0296
3
Children's Minnesota
Saint Paul, Minnesota, United States, 55102
4
Vanderbilt University School of Medicine
Nashville, Tennessee, United States, 37232