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Melanocortin 4 Receptor (MC4R) Agonist
Setmelanotide for Genetic Obesity
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
Pivotal Trial
No Placebo-Only Group
Summary
This trial tests setmelanotide, an injection, in young children with genetic causes of obesity. The medication helps control hunger and energy use, aiming to manage their weight more effectively. Setmelanotide has been approved for treating obesity caused by specific genetic deficiencies and is being developed for other rare genetic disorders associated with obesity.
Who is the study for?
This trial is for children aged 2 to <6 with severe obesity linked to specific genetic conditions: POMC, PCSK1, LEPR deficiencies or Bardet-Biedl Syndrome. They must be significantly overweight and have a history of intense hunger. Children can't join if they've had certain skin lesions, poor liver/kidney function, high HbA1c levels, or previous setmelanotide treatment.
What is being tested?
The study tests the effectiveness and safety of setmelanotide over one year in young kids with rare genetic obesity disorders. It's an open-label phase 3 trial where all participants receive the drug via daily injections.
What are the potential side effects?
Potential side effects are not detailed here but may include reactions related to the injection site, general discomforts like headaches or nausea, and possibly others as observed in earlier phases or related treatments.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 year
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Mean Percent Change From Baseline in BMI
Percentage of Participants With Greater Than or Equal to (≥) 0.2 Reduction of BMI Z-Score From Baseline to Week 52
Secondary study objectives
Change From Baseline in Body Weight
Mean Absolute Change From Baseline in BMI Z-score
Mean Change From Baseline in Bone Age
+4 moreOther study objectives
Change in metabolic parameters, HBA1c mmol/mol
Change in metabolic parameters, fasting glucose mg/dL
Change in pharmacokinetics (PK) concentration values in AUC
+2 moreSide effects data
From 2022 Phase 4 trial • 77 Patients • NCT0504613246%
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%
Injection site pruritus
11%
Fatigue
7%
Abdominal pain
7%
Constipation
4%
Pancreatitis acute
4%
Hepatitis acute
4%
Asthenia
4%
Chills
4%
Injection site induration
4%
Medical device site dermatitis
4%
Aphthous ulcer
4%
Change of bowel habit
4%
Chapped lips
4%
Diarrhoea
4%
Gingival discolouration
4%
Hyperaesthesia teeth
4%
Pruritus
4%
Rash
4%
Cough
4%
Dry throat
4%
Dyspnoea
4%
Rhinorrhoea
4%
Muscle spasms
4%
Pain in extremity
4%
Palpitations
4%
Skin abrasion
4%
Blood creatine phosphokinase increased
4%
Abnormal dreams
100%
80%
60%
40%
20%
0%
Study treatment Arm
Group 1: Setmelanotide 2 mg
Group 1: Setmelanotide 3 mg
Group 2: Moxifloxacin 400mg
Group 1: Setmelanotide 5 mg
Group 1: Setmelanotide 7 mg
Group 2: Placebo
Group 3: Placebo
Group 3: Moxifloxacin 400mg
Awards & Highlights
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
2Treatment groups
Experimental Treatment
Group I: Setmelanotide: PPL GroupExperimental Treatment1 Intervention
Participants with POMC)/PCSK1/LEPR biallelic mutations collectively referred to as PPL received setmelanotide at a dose of 0.5 milligrams (mg) per day (QD) via SC injection for 52 weeks. The dose was escalated by increments of 0.5 mg every 2 weeks, if tolerated, at the dose escalation visits (Weeks 2, 4, and 6) to a maximum dose of 0.5 to 2.0 mg QD with the maximum dose based on body weight. Following the last dose in this study, participants who were considered likely to benefit from continued setmelanotide treatment and who had completed this trial could be eligible to enter an open-label long-term extension (LTE) trial with setmelanotide.
Group II: Setmelanotide: BBS GroupExperimental Treatment1 Intervention
Participants with BBS received setmelanotide at a dose of 0.5 mg QD via SC injection for 52 weeks. The dose was escalated by increments of 0.5 mg every 2 weeks, if tolerated, at the dose escalation visits (Weeks 2, 4, and 6) to a maximum dose of 0.5 to 2.0 mg QD with the maximum dose based on body weight. Following the last dose in this study, participants who were considered likely to benefit from continued setmelanotide treatment and who had completed this trial could be eligible to enter an open-label long-term extension (LTE) trial with setmelanotide.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Setmelanotide
2017
Completed Phase 4
~800
Find a Location
Who is running the clinical trial?
Rhythm Pharmaceuticals, Inc.Lead Sponsor
28 Previous Clinical Trials
10,363 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.
126 Previous Clinical Trials
21,979 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You are very overweight for your age and gender, and weigh at least 15 kilograms at the time of joining the study.You have a strong allergic reaction to any ingredient in the study drug.The doctor has noticed that you have had episodes of excessive eating at any point in your life.You have excessive eating or a strong urge to eat more than usual.You or someone in your family has had melanoma (a type of skin cancer), or you have a history of oculocutaneous albinism (a condition that affects skin, hair, and eye color).You have been part of a previous research study that involved using setmelanotide, or you have taken setmelanotide before.You have a medical condition that affects your body weight and it is not under control.You have had a serious liver disease in the past.
Research Study Groups:
This trial has the following groups:- Group 1: Setmelanotide: PPL Group
- Group 2: Setmelanotide: BBS Group
Awards:
This trial has 2 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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