NCT05194124

Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Participants With Specific Gene Defects in the MC4R Pathway

A Phase 3, Randomized, Double-Blind Trial of Two Formulations of Setmelanotide (Daily and Weekly) With a Crossover to Open-Label Once Weekly Setmelanotide in Patients With Specific Gene Defects in the Melanocortin-4 Receptor Pathway Who Are Currently on a Stable Dose of the Once Daily Formulation

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 19 (actual)
• Sponsor: Rhythm Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

A trial to compare the weekly and daily formulations of setmelanotide in participants with genetic defects in the melanocortin-4 receptor pathway.

Detailed description

This study is designed to compare the safety, pharmacokinetics, and efficacy of weekly and daily formulations of setmelanotide in participants with obesity associated with biallelic or heterozygous POMC (pro-opiomelanocortin), PCSK1 (proprotein convertase subtilisin/kexin Type 1), LEPR (leptin receptor) genetic variants, and participants with Bardet-Biedl Syndrome (BBS).

Conditions

• Bardet-Biedl Syndrome
• POMC Deficiency

Interventions

Type	Name	Description
DRUG	Setmelanotide 2 mg	Administered as SC injection
DRUG	Setmelanotide 2.5 mg	Administered as SC injection
DRUG	Setmelanotide 3 mg	Administered as SC injection
DRUG	Setmelanotide 20 mg	Administered as SC injection
DRUG	Setmelanotide 25 mg	Administered as SC injection
DRUG	Setmelanotide 30 mg	Administered as SC injection

Timeline

• Start date: 2021-12-21
• Primary completion: 2023-10-19
• Completion: 2023-10-19
• First posted: 2022-01-18
• Last updated: 2024-11-26
• Results posted: 2024-11-26

Locations

7 sites across 6 countries: United States, Canada, Germany, Netherlands, Puerto Rico, United Kingdom

Regulatory

• FDA-regulated drug study