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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

Study 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
46%
Nausea
39%
Injection site pain
29%
Vomiting
25%
Injection site erythema
21%
Ephelides
21%
Decreased appetite
18%
Skin hyperpigmentation
14%
Injection site swelling
14%
Headache
14%
Erection increased
11%
Fatigue
11%
Injection site pruritus
7%
Abdominal pain
7%
Constipation
4%
Hepatitis acute
4%
Asthenia
4%
Chills
4%
Medical device site dermatitis
4%
Aphthous ulcer
4%
Change of bowel habit
4%
Gingival discolouration
4%
Pruritus
4%
Cough
4%
Dyspnoea
4%
Rhinorrhoea
4%
Pain in extremity
4%
Blood creatine phosphokinase increased
4%
Pancreatitis acute
4%
Injection site induration
4%
Chapped lips
4%
Hyperaesthesia teeth
4%
Diarrhoea
4%
Rash
4%
Dry throat
4%
Muscle spasms
4%
Skin abrasion
4%
Palpitations
4%
Abnormal dreams
100%
80%
60%
40%
20%
0%
Study treatment Arm
Group 1: Setmelanotide 2 mg
Group 1: Setmelanotide 3 mg
Group 1: Setmelanotide 7 mg
Group 1: Setmelanotide 5 mg
Group 2: Placebo
Group 2: Moxifloxacin 400mg
Group 3: Moxifloxacin 400mg
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
26 Previous Clinical Trials
9,319 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.
123 Previous Clinical Trials
21,682 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does the age limit for this research exclude adults over 30 years old?

"This research is open to children that have not yet turned 5 years old."

Answered by AI

To join this experiment, what are the requirements that a person must meet?

"This trial is looking for 12 young patients, aged 2-5, who have already been diagnosed with bardet-biedl syndrome. Candidates must also match the following criteria: POMC, PCSK1, or LEPR deficiency (as confirmed by genetic testing which meets American College of Medical Genetics and Genomics criteria), or BBS as defined by both the Beales Criteria, 1999 and genetic confirmation of homozygous or compound heterozygous loss-off-function mutation in BBS genes. In addition, eligible participants must be obese (BMI ≥97th percentile for age and gender AND body weight of"

Answered by AI

How does Setmelanotide compare to other treatments?

"Currently, 4 clinical trials are underway to study the efficacy of setmelanotide. Of these 4, 3 are in Phase 3. Setmelanotide trials are being conducted in 52 different locations, with many taking place in Marshfield, Wisconsin."

Answered by AI

Have there been similar trials to this one before?

"Since 2018, Setmelanotide has undergone various clinical trials. The first study, involving 150 patients, was completed in 2018 and sponsored by Rhythm Pharmaceuticals, Inc. After the first study, Setmelanotide received Phase 2 & 3 drug approval. Currently, there are 4 ongoing trials in 31 cities and 9 different countries."

Answered by AI

How can interested patients sign up for this research?

"As of right now, this particular trial is not looking for any more participants. However, this may change in the future as the study was posted on February 16th, 2022 and was edited as recently as October 21st, 2022. If you are looking for other trials to participate in, there are 996 trials actively recruiting participants with bardet-biedl syndrome and 4 trials for Setmelanotide that are currently searching for patients."

Answered by AI

What are the conditions that Setmelanotide is known to help alleviate?

"Setmelanotide can be used to manage chronic weight, obesity, and even obesity caused by a deficiency in pro-opiomelanocortin."

Answered by AI

Who else is applying?

What state do they live in?
Nebraska
How old are they?
< 18
What site did they apply to?
Columbia University Medical Center, Division of Pediatric Endocrinology, Diabetes and Metabolism
What portion of applicants met pre-screening criteria?
Met criteria

How responsive is this trial?

Typically responds via
Email
Recent research and studies
clinicaltrials.govStudy
Setmelanotide in Pediatric Patients With Rare Genetic Diseases of Obesity
2022. "Setmelanotide in Pediatric Patients With Rare Genetic Diseases of Obesity". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04966741.
~4 spots leftby May 2025
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