open-label study of setmelanotide in hypothalamic obesity

Conditions: Hypothalamic Obesity

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 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 \ 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.