A Phase 3, Randomized, Double-Blind Trial of Two Formulations of Setmelanotide (Daily and Weekly) With a Crossover to Open-Label Once Weekly Setmelanotide in Patients With Specific Gene Defects in the Melanocortin-4 Receptor Pathway Who Are Currently on a Stable Dose of the Once Daily Formulation
Who is this study for? Patients with genetic defects in the melanocortin-4 receptor pathway
What treatments are being studied? Setmelanotide
Status: Completed
Location: See all (7) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
A trial to compare the weekly and daily formulations of setmelanotide in participants with genetic defects in the melanocortin-4 receptor pathway.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Healthy Volunteers: f
View:
• Biallelic or heterozygous POMC/PCSK1 or LEPR (PPL) genetic variants or Bardet-Biedl syndrome (BBS), for which they are being treated with QD setmelanotide.
• 6 years or older at screening.
• Taking the setmelanotide QD formulation for at least 6 months in the RM-493-022 (NCT03013543) study with acceptable safety and tolerability, and dose level.
• Participant and/or parent or guardian is able to communicate well with the Investigator, to understand and comply with the requirements of the study and is able to understand and sign the written informed consent/assent.
• Use of a highly effective form of contraception throughout the study and for 90 days following the study.
Locations
United States
Arizona
Honor Health Research Institute
Scottsdale
Wisconsin
Marshfield Clinic Research Institute
Marshfield
Other Locations
Canada
Alberta Health Services
Edmonton
Germany
Charité - Universitätsmedizin Berlin, Campus Virchow-Klinikum
Berlin
Netherlands
Erasmus MC
Rotterdam
Puerto Rico
UPR Medical Sciences Campus
Rio Piedras
United Kingdom
Addenbrooke's Hospital
Cambridge
Time Frame
Start Date: 2021-12-21
Completion Date: 2023-10-19
Participants
Target number of participants: 19
Treatments
Experimental: Run-in Period: Setmelanotide 2 mg QD
Participants received subcutaneous (SC) injection of 2 mg setmelanotide once daily (QD) for 1 week in the run-in period.
Experimental: Run-in Period: Setmelanotide 2.5 mg QD
Participants received SC injection of 2.5 mg setmelanotide QD for 1 week in the run-in period.
Experimental: Run-in Period: Setmelanotide 3 mg QD
Participants received SC injection of 3 mg setmelanotide QD for 1 week in the run-in period.
Experimental: DB Period: Setmelanotide 20 mg QW
Participants who received 2 mg setmelanotide QD in the run-in period, received SC injection of 20 mg setmelanotide once weekly (QW) and placebo matched to setmelanotide QD for 13 weeks in the DB period.
Experimental: DB Period: Setmelanotide 25 mg QW
Participants who received 2.5 mg setmelanotide QD in the run-in period, received SC injection of 25 mg setmelanotide QW and placebo matched to setmelanotide QD for 13 weeks in the DB period.
Experimental: DB Period: Setmelanotide 3 mg QD
Participants who received 3 mg setmelanotide QD in the run-in period, received SC injection of 3 mg setmelanotide QD and placebo matched to setmelanotide QW for 13 weeks in the DB period.
Experimental: DB Period: Setmelanotide 30 mg QW
Participants who received 3 mg setmelanotide QD in the run-in period, received SC injection of 30 mg setmelanotide QW and placebo matched to setmelanotide QD for 13 weeks in the DB period.
Experimental: OL Period: Setmelanotide 20 mg QW
Participants who received 2 mg setmelanotide QD in the run-in period and 20 mg setmelanotide QW in the DB period, received SC injection of 20 mg setmelanotide QW for 13 weeks in the OL period.
Experimental: OL Period: Setmelanotide 25 mg QW
Participants who received 2.5 mg setmelanotide QD in the run-in period and 25 mg setmelanotide QW in the DB period, received SC injection of 25 mg setmelanotide QW for 13 weeks in the OL period.
Experimental: OL Period: Setmelanotide 30 mg QW
Participants who received 3 setmelanotide QD in the run-in period and 3 mg setmelanotide QD or 30 mg setmelanotide QW in the DB period, received SC injection of 30 mg setmelanotide QW for 13 weeks in the OL period.
Related Therapeutic Areas
Sponsors
Leads: Rhythm Pharmaceuticals, Inc.