NCT06239116

A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment

A Study of RM-718 Weekly Formulation in Healthy Subjects With Obesity and in Patients With Obesity Due to MC4R Impairment

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 150 (estimated)
• Sponsor: Rhythm Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 12 Years – 65 Years
• Healthy volunteers: Accepted

Summary

The purpose of this study is to evaluate the safety, tolerability, and PK of RM-718 in healthy subjects with obesity and in patients with MC4R Pathway Impairment

Detailed description

This is a first-in-human and first-in-patient, 4-part study that includes the evaluation of safety, tolerability, and PK of: single ascending doses (SAD) of RM-718 weekly (RM-718) in healthy subjects 18 to 55 years of age with obesity (Part A), multiple ascending doses (MAD) of RM-718 in healthy subjects 18 to 55 years of age with obesity (Part B), MAD of RM-718 in patients 12 to 65 years of age with HO (Part C), and MAD of RM-718 in patients with PWS (Part D). Cohorts in Parts A and B are double-blind, placebo-controlled, and randomized 2:1 (4 subjects receive RM-718, 2 subjects receive placebo). Part C evaluates open-label dose escalation in patients 12 to 65 years of age with HO. Part D evaluates open-label dose escalation in patients 12 to 65 years of age. Study participants will receive: 1 weekly dose of either RM-718 or placebo in Part A, 4 weekly doses of either RM-718 or placebo in Part B,16 weekly doses of open-label RM-718 in Part C, and 26 weekly doses of RM-718 in Part D. Study drug (RM-718 or placebo) doses are administered weekly via subcutaneous injection.

Conditions

• Hypothalamic Obesity
• Prader-Willi Syndrome
• PWS

Interventions

Type	Name	Description
DRUG	Part A: RM-718 or placebo (matched to specific RM-718 dose cohort)	Single ascending dose of RM-718 or placebo (matched to specific RM-718 Part A dose cohort)
DRUG	Part B: RM-718 or placebo (matched to specific RM-718 dose cohort)	Multiple ascending doses of RM-718 or placebo (matched to specific RM-718 Part B dose cohorts)
DRUG	Part C: RM-718	Multiple ascending doses of RM-718
DRUG	Part D: RM-718	Multiple ascending doses of RM-718

Timeline

• Start date: 2024-03-05
• Primary completion: 2027-11-01
• Completion: 2028-11-01
• First posted: 2024-02-02
• Last updated: 2025-12-22

Locations

7 sites across 1 country: United States

Regulatory

• FDA-regulated drug study