Phase 2
Completed N=18
Open-Label Study of Setmelanotide in Hypothalamic Obesity
Hypothalamic Obesity
Source: ClinicalTrials.gov NCT04725240 ↗
Enrolled (actual)
18
Serious AEs
5.6%
Results posted
Sep 2023
Primary outcomePrimary: Percentage of Participants With ≥ 5% Reduction in BMI From Baseline After 16 Weeks of Setmelanotide Treatment — 88.9 percentage of participants — p=<0.0001
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).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With ≥ 5% Reduction in BMI From Baseline After 16 Weeks of Setmelanotide Treatment |
88.9 | <0.0001 sig |
| SECONDARY Composite Percentage of Participants Aged ≥6 to <18 Years With ≥0.2 Reduction of BMI Z-Score or Participants Aged ≥18 Years With 5% Reduction of Body Weight From Baseline After 16 Weeks of Setmelanotide Treatment |
88.9 | <0.0001 sig |
| SECONDARY Percentage of Participants Aged ≥6 to <18 Years With ≥0.2 Reduction of BMI Z-Score From Baseline After 16 Weeks of Setmelanotide Treatment |
92.3 | <0.0001 sig |
| SECONDARY Percentage of Participants Aged ≥18 Years With ≥5% Reduction of Body Weight From Baseline After 16 Weeks of Setmelanotide Treatment |
80.0 | <0.0001 sig |
| SECONDARY Change From Baseline in Waist Circumference in Participants Aged ≥18 Years After 16 Weeks of Setmelanotide Treatment |
-10.275 | — |
| SECONDARY Change From Baseline in Daily Hunger Questionnaire Score in Participants Aged <12 Years After 16 Weeks of Setmelanotide Treatment |
-1.490 | — |
| SECONDARY Change From Baseline in Daily Hunger Questionnaire Scores in Participants Aged ≥12 Years After 16 Weeks of Setmelanotide Treatment |
-2.922; -3.122; -2.717 | — |
| SECONDARY Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged < 12 Years |
0; 0; 0; 2; 0; 0 | — |
| SECONDARY Number of Participants With Shift From Baseline in Global Hunger Questionnaire Score to Week 16 in Participants Aged ≥12 Years |
0; 1; 3; 0; 1; 0 | — |
| SECONDARY Number of Participants With Adverse Events (AEs) |
18 | — |
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 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 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.
Data sourced from ClinicalTrials.gov (NCT04725240). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.