Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of MRM-3379 in Male Participants With Fragile X Syndrome
Status: Recruiting
Location: See all (15) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
This study is a multicenter, double-blind, randomized, placebo-controlled study to assess the safety and tolerability of 3 doses of MRM-3379 in male participants with Fragile X Syndrome ages 16 to 45 (inclusive). In addition, a parallel cohort of participants ages 13 to \<16 will receive open-label MRM-3379. All participants will participate for 12 weeks of treatment. The study is also intended as a proof-of-concept investigation to evaluate whether MRM-3379 can improve FXS symptoms
Eligibility
Participation Requirements
Sex: Male
Minimum Age: 13
Maximum Age: 45
Healthy Volunteers: f
View:
• Willing and able to provide signed informed consent/assent. Where local regulations permit inclusion of participants deemed unable to provide informed consent, a legally authorized representative must provide informed consent on the participant's behalf, and the participant must provide assent if applicable.
• Male, 13-45 years of age (inclusive)
• Weight ≥30 kg and BMI between 18 and 36 kg/m2 (inclusive) at the screening visit
• Diagnosis of FXS with a molecular genetic ≥200 CGG repetitions .
• Able to perform the PVT and ORRT of the NIH-TCB
• Have a consistent caregiver(s) who is willing and able to be present regularly and reliably with the participant
• Able to swallow tablets or capsules
Locations
United States
Arizona
Southwest Autism Research & Resource Center
RECRUITING
Phoenix
California
Amnova Clinical Research, LLC
RECRUITING
Irvine
Children's Hospital of Orange County - Pediatric Subspecialty
RECRUITING
Orange
Uc Davis Mind Institute
RECRUITING
Sacramento
Colorado
Children's Hospital Colorado
RECRUITING
Aurora
Florida
Neurology
RECRUITING
Miami
Georgia
Emory University - The Emory Clinic - Clifton Road
RECRUITING
Atlanta
Illinois
Rush University Medical Center-Clinical Operation
RECRUITING
Chicago
Massachusetts
Boston Children's Hospital
RECRUITING
Boston
UMass Memorial Health
RECRUITING
Worcester
Maryland
Kennedy Krieger Institute, John Hopkins School of Medicine
RECRUITING
Baltimore
Minnesota
University of Minnesota Department of Medicine
RECRUITING
Minneapolis
Ohio
Cincinnati Children's Hospital Medical Center
RECRUITING
Cincinnati
Pennsylvania
Suburban Research Associates
RECRUITING
Media
Utah
Intermountain Healthcare - Park City Specialty Clinic
NOT_YET_RECRUITING
Salt Lake City
Time Frame
Start Date:2025-11-22
Estimated Completion Date:2027-10
Participants
Target number of participants:60
Treatments
Experimental: Arm 1: Low Dose of MRM-3379
Participants 16-45 years of age randomized to this arm will receive the low dose of MRM-3379
Experimental: Arm 2: Middle dose of MRM-3379
Participants 16-45 years of age randomized to this arm will receive the middle dose of MRM-3379
Experimental: Arm 3: High dose of MRM-3379
Participants 16-45 years of age randomized to this arm will receive the high dose of MRM-3379
Placebo_comparator: Arm 4 :Placebo
Participants 16-45 years of age randomized to this arm will receive Placebo
Experimental: Arm 5: Low dose of MRM-3379 Open-Label
Participants 13 to \< 16 years of age will receive the low dose of MRM-3370 Open-Label