Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity (NCT04966741) | Clinical Trial Compass
CompletedPhase 3
Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity
United States, Australia, Spain12 participantsStarted 2022-03-08
Plain-language 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).
Who can participate
Age range2 Years – 5 Years
SexALL
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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 \<6 years at the time of informed consent
✓. Obesity, defined as body mass index (BMI) ≥97th percentile for age and gender and body weight of at least 15 kilograms (kg) at the time of enrollment.
✓. Symptoms or behaviors of hyperphagia
✓. Parent or guardian of study participant is able to understand and comply with the requirements of the study (including QD injection regimen and all other study procedures) and is able to understand and sign the written consent/assent.
Exclusion criteria
✕. Glycated hemoglobin (HbA1c) \>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.
✕
What they're measuring
1
Percentage of Participants With Greater Than or Equal to (≥) 0.2 Reduction of BMI Z-Score From Baseline to Week 52