Phase 3
Completed N=12
Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity
Bardet-Biedl Syndrome · POMC Deficiency Obesity · PCSK1 Deficiency Obesity · LEPR Deficiency Obesity
Source: ClinicalTrials.gov NCT04966741 ↗
Enrolled (actual)
12
Serious AEs
0.0%
Results posted
Jul 2024
Primary outcomePrimary: Percentage of Participants With Greater Than or Equal to (≥) 0.2 Reduction of BMI Z-Score From Baseline to Week 52 — 85.7; 80.0 percentage of participants
◆ Published Evidence
Established
43citations · ~43 / year
Setmelanotide in patients aged 2-5 years with rare MC4R pathway-associated obesity (VENTURE): a 1 year, open-label, multicenter, phase 3 trial.
Summary
This is a phase 3 open-label, clinical study to evaluate the efficacy, safety and tolerability of setmelanotide over 1 year of treatment, in pediatric participants aged 2 to <6 years with obesity due to either biallelic variants of the pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) genes or Bardet-Biedl Syndrome (BBS).
Linked Publications
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Setmelanotide in patients aged 2-5 years with rare MC4R pathway-associated obesity (VENTURE): a 1 year, open-label, multicenter, phase 3 trial.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Greater Than or Equal to (≥) 0.2 Reduction of BMI Z-Score From Baseline to Week 52 |
85.7; 80.0 | — |
| PRIMARY Mean Percent Change From Baseline in BMI |
-25.597; -9.719 | — |
| SECONDARY Mean Absolute Change From Baseline in BMI Z-score |
-5.185; -1.331 | — |
| SECONDARY Mean Change From Baseline in Percent of the 95th Percentile of BMI |
-47.595; -14.462 | — |
| SECONDARY Mean Change From Baseline in Bone Age |
1.020; 0.700 | — |
| SECONDARY Number of Participants With Shift From Baseline in Ages & Stages Questionnaires, Third Edition (ASQ-3) |
5; 2; 0; 0; 0; 0 | — |
| SECONDARY Change From Baseline in Body Weight |
-7.139; -0.327 | — |
| SECONDARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) |
7; 5 | — |
| SECONDARY Number of Participants With TEAEs Graded by Severity |
2; 5; 5; 0; 0; 0 | — |
Eligibility Criteria
Key Inclusion Criteria
- Participants must have obesity due to either:
- POMC, PCSK1, or LEPR deficiency, confirmed by genetic testing demonstrating biallelic variants that are interpreted as pathogenic, likely pathogenic, or of undetermined significance (VUS) by the American College of Medical Genetics and Genomics criteria (ACMG), or
- BBS confirmed clinical and genetic diagnosis
- Age between 2 to 9.0% at screening
- History of significant liver disease
- Glomerular filtration rate (GFR) <60 milliliter per minute per 1.73 meter square (mL/min/1.73 m^2)
- History or close family history of melanoma, or participant history of oculocutaneous albinism.
- Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)
- Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing.
- Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide.
- Significant hypersensitivity to any excipient in the study drug.
- Inadequate hepatic function
- Any other uncontrolled endocrine, metabolic or medical condition(s) known to impact body weight
Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT04966741) and the linked publication. 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.