Phase 2
N=18
Open-Label Study of Setmelanotide in Hypothalamic Obesity
Hypothalamic Obesity
Bottom Line
View on ClinicalTrials.gov: NCT04725240 ↗Enrolled (actual)
18
Serious AEs
5.6%
Results posted
Sep 2023
Primary outcome: Primary: Percentage of Participants With ≥ 5% Reduction in BMI From Baseline After 16 Weeks of Setmelanotide Treatment — 88.9 percentage of participants — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Setmelanotide (Drug)
- Age
- Pediatric, Adult · 6+ yrs
- Sex
- All
- Sponsor
- Rhythm Pharmaceuticals, Inc.
- Primary completion
- Jun 2022
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 | — |
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 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.