Evidence map›Paper›PMID 41703984›Full record

Trial reportObesity (Silver Spring, Md.)2026

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

Jesús Argente, Andrea M Haqq, Jan Luca Schorfheide, Nicolas Touchot, Caroline Huber, Urs Wiedemann, Jeremy Pomeroy, Phase 3 BBS Trial Investigators

Erratum issued Registry-linked trialAbstract readClinical Trial, Phase IIIComparative StudyMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Obesity (Silver Spring, Md.), 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to trial NCT03746522 (A Phase 3 Trial of Setmelanotide), which is not on this map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT03746522 phase3completednot on this map

A Phase 3 Trial of Setmelanotide (RM-493), a Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Patients With Moderate to Severe Obesity

TypeinterventionalSponsorRhythm Pharmaceuticals, Inc.Ran2018 to 2021Enrolled52ConditionsBardet Biedl Syndrome (BBS), Alström Syndrome (AS)ArmsSetmelanotide, Placebo
3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Trial
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

8 authors.

Jesús ArgenteDepartment of Pediatrics and Pediatric Endocrinology, University Hospital Niño Jesús, Research Institute La Princesa, Madrid, Spain.
Andrea M HaqqDivision of Pediatric Endocrinology, University of Alberta, Edmonton, Alberta, Canada.
Jan Luca Schorfheidestradoo Gmbh, Munich, Germany.
Nicolas TouchotRhythm Pharmaceuticals, Inc., Boston, Massachusetts, USA.
Caroline HuberRhythm Pharmaceuticals, Inc., Boston, Massachusetts, USA.
Urs Wiedemannstradoo Gmbh, Munich, Germany.
Jeremy PomeroyMarshfield Clinic Research Institute, Marshfield, Wisconsin, USA.
Phase 3 BBS Trial Investigators

Funding

Bardet Biedl Syndrome Foundation and Family AssociationRhythm Pharmaceuticals, Inc.
6 · The paper itself

Abstract

objectiveThis 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).

methodsPatients 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.

resultsA significantly greater proportion of patients treated with setmelanotide met the primary endpoint compared with control patients (58.6% vs. 6.9%; p < 0.001). In pediatric patients, 71.4% achieved the primary endpoint vs. 10.7% of controls (p < 0.001); in adults, corresponding numbers were 46.7% vs. 3.3% (p = 0.001). Secondary outcomes demonstrated consistent benefits of setmelanotide treatment. Sensitivity analysis using inverse probability of treatment weighting confirmed these findings.

conclusionsThis indirect comparison provides additional strong evidence that setmelanotide significantly improves weight outcomes in patients with BBS. These findings further support its clinical benefit over 52 weeks in managing obesity associated with BBS.

trial registrationClinicalTrials.gov identifier: NCT03746522.

Indexed as

alpha-MSHBardet-Biedl SyndromeObesityAdolescentAdultBody Mass IndexChildFemaleHumansMaleRegistriesTreatment OutcomeWeight LossYoung Adultalpha-MSHsetmelanotideBardet‐Biedl syndromeindirect treatment comparisonssetmelanotideweight outcomes

Identifiers

PMID41703984
PMCPMC12933228

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.