NCT03287960 study context · version 1

Rare disease · Phase 3

Setmelanotide: LEPR-deficiency pivotal cohort

What was tested?

Setmelanotide in a genetically defined obesity cohort, including response-selected blinded withdrawal. ↗

Registered primary question: The proportion of the pivotal cohort reaching at least 10% weight loss after approximately one year. ↗

What the reviewed report found

Journal year 2020 · Article summary reviewed 2026-09-20

Five of eleven participants achieved at least 10% weight loss at approximately one year. The hunger-score analysis involved a selected subset of seven participants aged at least 12 years who had first met the weight-loss threshold.^↗

Keep in context: The registry’s total enrollment is 15; the selected paper reports the eleven-person pivotal LEPR cohort. The primary one-year result is not a parallel placebo comparison, and the hunger analysis selects initial responders. ↗↗

Participants, design, limitations and sources

Registry snapshot

Registry enrollment: 15 · reported actual
Registry status: Completed
Allocation and masking: Na · Open label
Design: Single group
Eligible ages: 6 Years; maximum not specified
Registry results: Posted in this snapshot

Registry source ↗ · Updated 2023-05-23; retrieved 2026-09-20. Registry enrollment is not necessarily the analyzed sample in a publication. Posted registry results and published article results are separate.

LEPR-deficiency obesity trial

Design in the report: A sponsor-funded, single-arm, open-label phase 3 trial included a blinded placebo-withdrawal sequence for participants who first met a weight-loss threshold.^↗
Participants in the report: Eleven participants aged at least six years with LEPR-deficiency obesity were included in the full analysis and safety sets.^↗
Limitations: The primary one-year endpoint lacked a parallel control group. The small cohort and response-selected withdrawal and hunger analyses limit interpretation and do not establish outcomes in other causes of obesity.^↗

This reading covers the article abstract and record metadata. Read its preserved Human Evidence Review →

How the study identity and sources were checked

Study context checked 2026-09-20. Existing article summaries keep their original review dates. This is a selected study reading, not a complete literature or full-text appraisal.

Registry

Setmelanotide for the Treatment of Leptin Receptor (LEPR) Deficiency Obesity ↗
Protocol identifier: RM-493-015. Sponsor: Rhythm Pharmaceuticals, Inc..
Retrieved 2026-09-20; SHA-256 05a6cee47787dfaf3e04ba4098eee7ff97a2538bf4a2cbe2ccc8c5ebcd6978f0.

PMID 33137293

Efficacy and safety of setmelanotide, an MC4R agonist, in individuals with severe obesity due to LEPR or POMC deficiency: single-arm, open-label, multicentre, phase 3 trials ↗

Exact trial identity is declared in PubMed’s ClinicalTrials.gov databank field. The registry independently labels this reference DERIVED. These source labels do not establish that every result in a paper applies to this trial.

Identity metadata retrieved 2026-09-20. EFetch XML; locate MedlineCitation/PMID 33137293. SHA-256 04d73cd4656ccf5ac7e3429077a068278bbe91f299a004c2a5a3b9cdad044c9c.

Scientific summary uses the original 2026-09-20 source snapshot: SHA-256 04d73cd4656ccf5ac7e3429077a068278bbe91f299a004c2a5a3b9cdad044c9c.