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Melanocortin 4 Receptor (MC4R) Agonist

Setmelanotide 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
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights

Summary

This trial is testing setmelanotide, a drug that may help with weight loss, in children aged 2-6 with obesity due to certain genetic conditions. The trial will last for one year to evaluate the safety and effectiveness of the drug.

Eligible Conditions
  • Bardet-Biedl Syndrome
  • Obesity
  • Leprosy Deficiency Obesity

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Mean percent change in BMI
Proportion of patients demonstrating >0.2 decrease from baseline in body weight
Secondary outcome measures
Mean absolute change from baseline in BMI Z-score
Mean change in Ages & Stages Questionnaires (ASQ®-3)
Mean change in bone age
+1 more
Other outcome measures
Change in metabolic parameters, HBA1c mmol/mol
Change in metabolic parameters, fasting glucose mg/dL
Change in pharmacokinetics (PK) concentration values in AUC
+2 more

Side effects data

From 2022 Phase 4 trial • 77 Patients • NCT05046132
52%
Injection site pain
26%
Injection site erythema
17%
Injection site swelling
4%
Dizziness
4%
Abdominal pain
4%
Dry throat
4%
Arthralgia
4%
Medical device site dermatitis
4%
Injection site pruritus
4%
Palpitations
4%
Injection site induration
4%
Pain in extremity
100%
80%
60%
40%
20%
0%
Study treatment Arm
Group 2: Moxifloxacin 400mg
Group 1: Setmelanotide 3 mg
Group 1: Setmelanotide 5 mg
Group 1: Setmelanotide 2 mg
Group 3: Moxifloxacin 400mg
Group 1: Setmelanotide 7 mg
Group 2: Placebo
Group 3: Placebo

Trial Design

1Treatment groups
Experimental Treatment
Group I: SetmelanotideExperimental Treatment1 Intervention
Investigational product: Setmelanotide,10 mg/mL in a sterile solution for Subcutaneous (SC) injection
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Setmelanotide
2017
Completed Phase 4
~650

Find a Location

Who is running the clinical trial?

Rhythm Pharmaceuticals, Inc.Lead Sponsor
27 Previous Clinical Trials
10,319 Total Patients Enrolled
4 Trials studying Bardet-Biedl Syndrome
1,072 Patients Enrolled for Bardet-Biedl Syndrome
Murry Stewart, BM/DMStudy ChairRhythm Pharmaceuticals, Inc.
Chief Medical OfficerStudy ChairRhythm Pharmaceuticals, Inc.
124 Previous Clinical Trials
21,744 Total Patients Enrolled

Media Library

Setmelanotide (Melanocortin 4 Receptor (MC4R) Agonist) Clinical Trial Eligibility Overview. Trial Name: NCT04966741 — Phase 3
Bardet-Biedl Syndrome Research Study Groups: Setmelanotide
Bardet-Biedl Syndrome Clinical Trial 2023: Setmelanotide Highlights & Side Effects. Trial Name: NCT04966741 — Phase 3
Setmelanotide (Melanocortin 4 Receptor (MC4R) Agonist) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04966741 — Phase 3
~4 spots leftby Jul 2025