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Melanocortin-4 Receptor Agonist

Setmelanotide 2mg daily for Bardet-Biedl Syndrome

Phase 3
Waitlist Available
Research Sponsored by Rhythm Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 27 weeks
Awards & highlights

Study Summary

This trial is testing a new medication for people with a genetic defect that prevents the body from processing a hormone called melanocortin-4.

Eligible Conditions
  • Bardet-Biedl Syndrome
  • Pro-opiomelanocortin Deficiency

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~27 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 27 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Area under the plasma concentration-time curve over the dosing interval (AUC0-tau)
Maximum plasma concentration (Cmax)
Time to maximum plasma concentration (Tmax)
+1 more
Secondary outcome measures
Number of adverse events and serious adverse events

Trial Design

6Treatment groups
Experimental Treatment
Placebo Group
Group I: Setmelanotide subcutaneous injection Weekly 30 mgExperimental Treatment1 Intervention
Patients on 3mg setmelanotide daily will be randomized 1:1 to receive setmelanotide either QD or QW during the double blind period, and all will be assigned to this arm in the open label period.
Group II: Setmelanotide subcutaneous injection Weekly 20 mgExperimental Treatment1 Intervention
Patients on 2mg setmelanotide daily will be randomized 1:1 to receive setmelanotide either QD or QW during the double blind period, and all will be assigned to this arm in the open label period.
Group III: Setmelanotide subcutaneous injection Daily 3 mgExperimental Treatment1 Intervention
Patients on 3mg setmelanotide daily will be randomized 1:1 to receive setmelanotide either QD or QW during the double blind period.
Group IV: Setmelanotide subcutaneous injection Daily 2 mgExperimental Treatment1 Intervention
Patients on 2mg setmelanotide daily will be randomized 1:1 to receive setmelanotide either QD or QW during the double blind period.
Group V: Placebo subcutaneous injection DailyPlacebo Group1 Intervention
Patients will be randomized 1:1 to receive either QD or QW placebo during the double blind period (and setmelanotide in the other formulation).
Group VI: Placebo subcutaneous injection WeeklyPlacebo Group1 Intervention
Patients will be randomized 1:1 to receive either QD or QW placebo during the double blind period (and setmelanotide in the other formulation).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Setmelanotide 2mg daily
2021
Completed Phase 3
~20
Setmelanotide 20mg weekly
2021
Completed Phase 3
~20
Setmelanotide 3mg daily
2021
Completed Phase 3
~20
Setmelanotide 30mg weekly
2021
Completed Phase 3
~20

Find a Location

Who is running the clinical trial?

Rhythm Pharmaceuticals, Inc.Lead Sponsor
25 Previous Clinical Trials
9,283 Total Patients Enrolled
3 Trials studying Bardet-Biedl Syndrome
64 Patients Enrolled for Bardet-Biedl Syndrome
David Meeker, MDStudy ChairRhythm Pharmaceuticals, Inc.
13 Previous Clinical Trials
1,545 Total Patients Enrolled
2 Trials studying Bardet-Biedl Syndrome
64 Patients Enrolled for Bardet-Biedl Syndrome
Linda Shapiro, MD, PhDStudy ChairRhythm Pharmaceuticals, Inc.
2 Previous Clinical Trials
95 Total Patients Enrolled

Frequently Asked Questions

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~6 spots leftby Mar 2025