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
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Rhythm Pharmaceuticals, Inc.
- Enrollment
- 205
- Locations
- 27
- Primary Endpoint
- Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Study Overview
Brief Summary
This was a long-term extension trial to study the safety and tolerability of continued setmelanotide treatment in participants who had completed a previous clinical trial on treatment with setmelanotide for obesity associated with genetic defects upstream of the Melanocortin-4 (MC4) receptor in the leptin-melanocortin pathway.
Detailed Description
The primary objectives of this extension trial were to explore the long-term safety and tolerability of setmelanotide for up to 7 years or until drug was otherwise available through authorized use. Participants entered this protocol immediately upon completion of their index protocol such that dosing of setmelanotide continued without gaps in therapy.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 2 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Participants aged 2 or older (or aged >2 years as per local regulations) who had completed participation in a previous setmelanotide trial and demonstrated adequate safety and meaningful clinical benefit (efficacy)
- •Participant and/or parent or guardian was 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
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 milliliters per minute (mL/min).
- •History or close family history of melanoma or participant history of oculocutaneous albinism
- •Other protocol defined Inclusion/Exclusion criteria may apply.
Arms & Interventions
Setmelanotide
Participants received setmelanotide at the same starting dose as received in the index trials as a Subcutaneous (SC) injection once daily for up to 5.6 years in this extension trial.
Intervention: Setmelanotide (Drug)
Outcomes
Primary Outcomes
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time Frame: From first dose up to 5.6 years
An adverse event (AE) was any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention. A TEAE was defined as any AE that began or worsened in intensity on or after the date of the first administration of study drug.
Secondary Outcomes
No secondary outcomes reported