Setmelanotide in Bardet-Biedl Syndrome: A 52-Week Comparison of Phase 3 Trial Participants With a Matched Registry Cohort.

ABSTRACT:

Objective

This analysis aimed to assess the efficacy of setmelanotide over 52 weeks in patients with Bardet-Biedl syndrome (BBS) compared with an external natural history cohort from the international Clinical Registry Investigating BBS (CRIBBS).

Methods

Patients with BBS ≥ 6 years of age (n = 29) treated with setmelanotide for 52 weeks in a phase 3 trial (NCT03746522) and a propensity-score matched external control cohort (n = 58) from CRIBBS were included. Responder rate at week 52 was defined as ≥ 0.3-point decrease in BMI z-score (patients ≤ 18 years) or ≥ 10% body weight reduction (adults). Secondary outcomes included changes in BMI, BMI z-score, and body weight.

Results

A significantly greater proportion of patients treated with setmelanotide met the primary end

SUBMITTER: Argente J 

PROVIDER: S-EPMC12933228 | biostudies-literature | 2026 Mar

REPOSITORIES: biostudies-literature