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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
Symptoms or behaviors of hyperphagia at any time during the patient's life, as determined by the Investigator at screening
Symptoms or behaviors of hyperphagia
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

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
The doctor has noticed that you have had episodes of excessive eating at any point in your life.
Select...
You have excessive eating or a strong urge to eat more than usual.

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 2020 Phase 3 trial • 15 Patients • NCT03287960
73%
Injection site erythema
73%
Skin hyperpigmentation
53%
Injection site pruritus
53%
Nausea
47%
Injection site induration
47%
Injection site pain
47%
Diarrhea
40%
Injection site oedema
33%
Injection site bruising
33%
Headache
27%
Back pain
27%
Asthenia
27%
Insomnia
27%
Spontaneous penile erection
27%
Dizziness
27%
Abdominal pain upper
27%
Nasopharyngitis
27%
Arthralgia
20%
Influenza like illness
20%
Injection site hypersensitivity
20%
Anxiety
20%
Anemia
20%
Muscle spasms
13%
Melanocytic naevus
13%
Fatigue
13%
Injection site haematoma
13%
Malaise
13%
Depression
13%
Depressed mood
13%
Constipation
13%
Vomiting
13%
Erythema
13%
Hyperhidrosis
13%
Hypothyroidism
13%
Gastrointestinal infection
13%
Pharyngitis
13%
Rhinitis
7%
Pain of skin
7%
Rash
7%
Tinnitus
7%
Pigmentation disorder
7%
Migraine
7%
Ear pain
7%
Pruritus
7%
Tic
7%
Blood uric acid increased
7%
Fear of injection
7%
Road Traffic Accident
7%
Suicidal Ideation
7%
Gastric Banding Reversal
7%
Flushing
7%
Eye naevus
7%
Chest pain
7%
Injection site atrophy
7%
Injection site reaction
7%
Injection site urticaria
7%
Medical device pain
7%
Medical device site erythema
7%
Oedema
7%
Pyrexia
7%
Xerosis
7%
Suicidal ideation
7%
Affect lability
7%
Drug abuse
7%
Illusion
7%
Dysmenorrhoea
7%
Ejaculation disorder
7%
Metrorrhagia
7%
Amenorrhoea
7%
Ovarian cyst
7%
Vaginal haemorrhage
7%
Arthropod bite
7%
Foot fracture
7%
Joint Injury
7%
Ligament sprain
7%
Alanine aminotransferase increased
7%
Blood creatine phosphokinase abnormal
7%
Blood follicle stimulating hormone increased
7%
Blood lactate dehydrogenase increased
7%
Blood luteinising hormone increased
7%
Blood urea decreased
7%
Heart rate increased
7%
Insulin tolerance test abnormal
7%
Neutrophil count increased
7%
Weight increased
7%
Cardiac flutter
7%
Oropharyngeal pain
7%
Asthma
7%
Eosinophilia
7%
Iron deficiency anemia
7%
Sleep paralysis
7%
Presyncope
7%
Sciatica
7%
Syncope
7%
Vertigo
7%
Abdominal pain
7%
Gingival discolouration
7%
Abdominal discomfort
7%
Abdominal distension
7%
Dyspepsia
7%
Gastrooesophageal reflux disease
7%
Haemorrhoids
7%
Cholestasis
7%
Hepatocellular injury
7%
Haematuria
7%
Proteinuria
7%
Renal colic
7%
Renal failure
7%
Skin striae
7%
Urticaria
7%
Hair growth rate abnormal
7%
Hyperkeratosis
7%
Lentigo
7%
Lipodystrophy acquired
7%
Pain in extremity
7%
Torticollis
7%
Hypogonadism
7%
Decreased appetite
7%
Dyslipidaemia
7%
Folate deficiency
7%
Glucose tolerance impaired
7%
Gout
7%
Vitamin A deficiency
7%
Lower respiratory tract infection
7%
Influenza
7%
Arthritis viral
7%
Bronchitis
7%
Fungal infection
7%
Gastroenteritis
7%
Injection site abscess
7%
Otitis externa
7%
Cholecystitis
7%
Musculoskeletal pain
7%
Musculoskeletal chest pain
7%
Neck pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Setmelanotide

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
25 Previous Clinical Trials
9,291 Total Patients Enrolled
3 Trials studying Bardet-Biedl Syndrome
72 Patients Enrolled for Bardet-Biedl Syndrome
Murry Stewart, BM/DMStudy ChairRhythm Pharmaceuticals, Inc.
Chief Medical OfficerStudy ChairRhythm Pharmaceuticals, Inc.
122 Previous Clinical Trials
21,557 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
~4 spots leftby Mar 2025