Long Term Extension Trial of Setmelanotide

Rhythm Pharmaceuticals

Status and phase: Completed; Phase 3; Phase 2

Conditions: Obesity Associated With Defects in Leptin-melanocortin Pathway

Treatments: Drug: Setmelanotide

Study type: Interventional

Funder types: Industry

Identifiers

NCT03651765

RM-493-022

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 205 patients

Sex: All

Ages: 2+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

1. 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)
2. 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
3. Agree to use a highly effective form of contraception throughout the trial

Key Exclusion Criteria:

1. Pregnant and/or breastfeeding women
2. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)
3. Current, clinically significant disease
4. Documented diagnosis of schizophrenia, bipolar disorder, personality disorder, major depressive disorder, or other psychiatric disorder(s)
5. Suicidal ideation, attempt or behavior
6. History of significant liver disease
7. Moderate to severe renal dysfunction as defined by a glomerular filtration rate (GFR) <30 mL/min.
8. History or close family history of melanoma or patient history of oculocutaneous albinism

Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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205 participants in 1 patient group

Setmelanotide

Experimental group

Description:

Once daily subcutaneous injection

Treatment:

Drug: Setmelanotide