Long Term Extension Trial of Setmelanotide

Study Overview
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Brief Summary
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This is a long-term extension trial to study the safety and tolerability of continued setmelanotide treatment in patients who have completed a previous clinical trial on treatment with setmelanotide for obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway.

Detailed Description
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The primary objectives of this extension trial are to explore the long-term safety and tolerability of setmelanotide for up to 5 years or until drug is otherwise available through authorized use. Patients can enter this protocol immediately upon completion of their index protocol such that dosing of setmelanotide continues without gaps in therapy.

Official Title
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Long Term Extension Trial of Setmelanotide (RM-493) for Patients Who Have Completed a Trial of Setmelanotide for the Treatment of Obesity Associated With Genetic Defects Upstream of the MC4 Receptor in the Leptin-melanocortin Pathway

Conditions
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Obesity Associated With Defects in Leptin-melanocortin Pathway

Intervention / Treatment
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* Drug: Setmelanotide


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Patients aged 2 or older (or aged >2 years as per local regulations) who have completed participation in a previous setmelanotide trial and demonstrated adequate safety and meaningful clinical benefit (efficacy) Patient and/or parent or guardian is able to communicate with the investigator, understand and sign the written informed consent/assent, and comply with the trial requirements Agree to use a highly effective form of contraception throughout the trial Key Exclusion Criteria: Pregnant and/or breastfeeding women Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion) Current, clinically significant disease Documented diagnosis of schizophrenia, bipolar disorder, personality disorder, major depressive disorder, or other psychiatric disorder(s) Suicidal ideation, attempt or behavior History of significant liver disease Moderate to severe renal dysfunction as defined by a glomerular filtration rate (GFR) <30 mL/min. History or close family history of melanoma or patient history of oculocutaneous albinism Other protocol defined Inclusion/Exclusion criteria may apply.

Ages Eligible for Study
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Minimum Age: 2 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Open label treatment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Setmelanotide<br>Once daily subcutaneous injection | Drug: Setmelanotide<br>* Once daily subcutaneous injection<br>* Other names: RM-493;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability of setmelanotide | Frequency and severity of adverse events (AEs) as well as changes in physical examinations, electrocardiograms (ECGs), vital signs (including resting BP and HR), laboratory evaluations, and injection site reactions. | 5 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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RM-493, setmelanotide, obesity, leptin-melanocortin, melanocortin 4 receptor