Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity

Korábbi kezelés

• 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.

Más betegség

• History of significant liver disease
• History or close family history of melanoma, or participant history of oculocutaneous albinism.
• Significant hypersensitivity to any excipient in the study drug.
• Any other uncontrolled endocrine, metabolic or medical condition(s) known to impact body weight
  • Other protocol defined Inclusion/Exclusion criteria may apply.

Laborértékek

• Glycated hemoglobin (HbA1c) >9.0% at screening
• Glomerular filtration rate (GFR) <60 milliliter per minute per 1.73 meter square (mL/min/1.73 m\^2)

Egyéb

• Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)
• Inadequate hepatic function

• 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.