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Real-World Effects of MC4R Agonist Therapy in BBS and Severe Genetic Obesity

NCT07674290 · Status: RECRUITING · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 200

Last updated 2026-06-29

No results posted yet for this study

Summary

Bardet-Biedl syndrome (BBS) and other rare disorders associated with impairment of the melanocortin-4 receptor (MC4R) pathway are characterized by severe early-onset obesity, hyperphagia, and substantial morbidity. Setmelanotide, an MC4R agonist, is approved in Europe for selected genetic obesity disorders and reimbursed in Germany for eligible patients. This study aims to evaluate the effectiveness, safety, treatment persistence, metabolic outcomes, and patient-reported outcomes of Setmelanotide under real-world conditions. The registry is designed to allow future inclusion of additional MC4R agonists as they become approved and clinically available. The study will primarily be conducted at University Hospital Essen and will collect longitudinal routine clinical data from pediatric and adult patients receiving MC4R agonist therapy according to approved indications.

Conditions

  • Bardet Biedl Syndrome (BBS)
  • Bardet Biedl Syndrome
  • Bardet-Biedl Syndrome (BBS)
  • Alstrom Syndrome

Interventions

DRUG

Setmelanotide

Administration according to approved product labeling and treating physician discretion

Sponsors & Collaborators

  • Rhythm Pharmaceuticals, Inc.

    collaborator INDUSTRY

  • Tom Hühne

    lead OTHER

Principal Investigators

  • Metin Cetiner, PD Dr. med. · Universitätsmedizin Essen

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-01-01
Primary Completion
2030-12-31
Completion
2030-12-31

Countries

  • Germany

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07674290 on ClinicalTrials.gov