Setmelanotide in Pediatric Patients With Rare Genetic Diseases of Obesity
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|ClinicalTrials.gov Identifier: NCT04966741|
Recruitment Status : Active, not recruiting
First Posted : July 19, 2021
Last Update Posted : December 15, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Bardet-Biedl Syndrome POMC Deficiency Obesity PCSK1 Deficiency Obesity LEPR Deficiency Obesity||Drug: Setmelanotide||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 3 Multi-Center, One-Year, Open-Label Study of Setmelanotide in Pediatric Patients Aged 2 to <6 Years of Age With Rare Genetic Causes of Obesity|
|Actual Study Start Date :||February 16, 2022|
|Estimated Primary Completion Date :||September 20, 2023|
|Estimated Study Completion Date :||September 20, 2023|
Investigational product: Setmelanotide,10 mg/mL in a sterile solution for Subcutaneous (SC) injection
All patients will begin treatment at a dose of 0.5 mg of setmelanotide per day. Patients will then increase their dose by 0.5 mg increments, every 2 weeks, until reaching their target maximum dose (not to exceed 2mg daily). The target maximum dose of setmelanotide used in this study will be based on the weight bands.
Other Name: IMCIVREE
- Proportion of patients demonstrating >0.2 decrease from baseline in body weight [ Time Frame: Baseline to Week 52 ]To evaluate the effect of setmelanotide on weight-related parameters by using measurement of decrease in body weight
- Mean percent change in BMI [ Time Frame: Baseline to Week 52 ]To evaluate the effect of setmelanotide on weight-related parameters by using the Body Mass Index measurement
- Mean absolute change from baseline in BMI Z-score [ Time Frame: Baseline to Week 52 ]Mean absolute change from baseline in BMI Z-score by age and gender
- Mean change in percent of the 95th percentile of BMI [ Time Frame: Baseline to Week 52 ]Mean change in percent of the 95th percentile of BMI by age and gender
- Mean change in bone age [ Time Frame: Baseline to Week 52 ]Mean change in bone age
- Mean change in Ages & Stages Questionnaires (ASQ®-3) [ Time Frame: Baseline to Week 52 ]Mean change in Ages & Stages Questionnaires score (ASQ®-3)
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|Ages Eligible for Study:||2 Years to 5 Years (Child)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
Key Inclusion Criteria:
Patients must have obesity due to either:
- POMC, PCSK1, or LEPR deficiency, confirmed by genetic testing demonstrating biallelic variants that are interpreted as pathogenic, likely pathogenic, or of undetermined significance (VUS) by the American College of Medical Genetics and Genomics criteria (ACMG), or
- BBS confirmed clinical and genetic diagnosis
- Age between 2 to <6 years at the time of informed consent
- Obesity, defined as BMI ≥97th percentile for age and gender AND body weight of at least 15 kg at the time of enrollment.
- Symptoms or behaviors of hyperphagia
- Parent or guardian of study participant is able to understand and comply with the requirements of the study (including once daily [QD] injection regimen and all other study procedures) and is able to understand and sign the written consent/assent.
Key Exclusion Criteria
- HbA1c >9.0% at screening
- History of significant liver disease
- Glomerular filtration rate (GFR) <60 mL/min/1.73 m2
- History or close family history of melanoma, or patient history of oculocutaneous albinism.
- Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)
- Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing.
- Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide.
- Significant hypersensitivity to any excipient in the study drug.
- Inadequate hepatic function
- Any other uncontrolled endocrine, metabolic or medical condition(s) known to impact body weight
Other protocol defined Inclusion/Exclusion criteria may apply.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04966741
|United States, Colorado|
|Children's Hospital Colorado|
|Aurora, Colorado, United States, 80045|
|United States, New York|
|Columbia University Medical Center, Division of Pediatric Endocrinology, Diabetes and Metabolism|
|New York, New York, United States, 10032|
|United States, Wisconsin|
|Marshfield Clinic Research Foundation|
|Marshfield, Wisconsin, United States, 54449|
|Sydney Children's Hospital|
|Randwick, Australia, NSW 2031|
|Hospital Infantil Niño Jesus|
|Madrid, Spain, 28009|
|Addenbrooke's Hospital, Wellcome Trust-MRC Institute of Metabolic Science|
|Cambridge, United Kingdom, CB2 0QQ|
|Study Chair:||David Meeker, MD||Rhythm Pharmaceuticals, Inc.|
|Responsible Party:||Rhythm Pharmaceuticals, Inc.|
|Other Study ID Numbers:||
|First Posted:||July 19, 2021 Key Record Dates|
|Last Update Posted:||December 15, 2022|
|Last Verified:||December 2022|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Bardet Biedl Syndrome
Central Nervous System Diseases
Nervous System Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn