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RM-493-035: Setmelanotide in Patients with Gene Defects in the MC4R Pathway
This study is now recruiting.
The purpose of this study is to find out if an investigational drug called setmelanotide (RM-493) can help control body weight in people with certain genetic modifications in their genes that play a key role in the regulation of body weight. Males and females, age 6 to 65, with obesity and certain genetic variants (MC4R pathway) may be eligible to participate. If you are confirmed eligible for the study, then participation will last up to 64 Weeks. Participants will complete Screening (Up to 8 Weeks), Double-Blind Treatment (Up to 52 Weeks), and Follow-Up Period (4 Weeks). Participants will complete research procedures including physical exams, blood draws, ECGs, and questionnaires. Participant will also need to self-inject the study drug once daily for the duration of their participation.
Who Do I Contact?
If you are interested in participating in the study or want to learn more please contact our study team at wadekl [at] chop.edu or 267-398-5761.
Eligibility & Criteria
IRB #:
22-019798
Official Title:
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study: 5 Independent Sub-studies of Setmelanotide in Patients with POMC, PCSK1, LEPR, SRC1, SH2B1, and PCSK1 N221D Gene Defects in the Melanocortin-4 Receptor Pathway
Study Phase:
Phase III
Eligible Age Range:
6 - 65 Years
Gender:
All
Visit Criteria
There are three (3) parts to this trial: Screening Period, Double-Blind Treatment Period, and Follow-Up Period.
Screening Period (Up to 8 Weeks): During the initial Screening Period we will see if you are eligible to participate. You will complete 1 in-person study visit, which will take place up to 8 Weeks (56 days) before your first dose of study drug.
Double-Blind Treatment Period (Up to 52 Weeks): If you are found to be eligible you will enter the double-blind treatment period which will last for up to 52 weeks. During this period, you will have up to 6 in-person visits, up to 9 virtual visits and 1 phone call. You will be randomly assigned (like the flip of a coin or drawing lots) to one of two groups: the study drug group or placebo group. You will have an equal chance of receiving the study drug or the placebo.
Follow-Up Period (4 Weeks): You will have an end-of-study visit completed over the phone approximately 4 weeks after the double-blind treatment period ends. The study doctor will also check whether the study drug works for you. If so, you may have an option to enroll in a separate extension study to continue receiving the study drug. Your study doctor will let you know if this option is available to you. If it is not, your participation in the study will end.
Leader
