NCT04966741

Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity

A Phase 3 Multi-Center, 1-Year, Open-Label Study of Setmelanotide in Pediatric Patients Aged 2 to <6 Years of Age With Rare Genetic Causes of Obesity

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Rhythm Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 2 Years – 5 Years
• Healthy volunteers: Not accepted

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).

Detailed description

Pediatric participants aged 2 to \<6 years with obesity due to either biallelic variants of the POMC, PCSK1 or LEPR genes or BBS will be enrolled into this phase 3 open-label clinical trial at one of approximately 8 clinical centers in North America, Europe, or Australia. All participants will be assigned to receive setmelanotide via daily subcutaneous (SC) injection for 1 year.

Conditions

• Bardet-Biedl Syndrome
• POMC Deficiency Obesity
• PCSK1 Deficiency Obesity
• LEPR Deficiency Obesity

Interventions

Type	Name	Description
DRUG	Setmelanotide	SC injection once daily.

Timeline

• Start date: 2022-03-08
• Primary completion: 2023-09-18
• Completion: 2024-11-08
• First posted: 2021-07-19
• Last updated: 2024-11-27
• Results posted: 2024-07-10

Locations

6 sites across 4 countries: United States, Australia, Spain, United Kingdom

Regulatory

• FDA-regulated drug study