Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)

Recruiting now · Phase 2 · Has a placebo group

Conditions studied: Fragile X Syndrome

In brief

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

Key facts

Study ID

NCT07209462

Run by

Mirum Pharmaceuticals, Inc.

People needed

60

Starts

2025-11-22

Expected to finish

2027-10-01

Last updated by the study team

2026-08-07

Who can join

Age: 13 and older, up to 45. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• History of or current medical condition other than FXS and related issues that would place the participant at higher risk from study participation

Where it is running

• Southwest Autism Research & Resource Center — Phoenix, Arizona, United States (enrolling)
• Amnova Clinical Research, LLC — Irvine, California, United States (enrolling)
• Children's Hospital of Orange County - Pediatric Subspecialty — Orange, California, United States (enrolling)
• Uc Davis Mind Institute — Sacramento, California, United States (enrolling)
• Children's Hospital Colorado — Aurora, Colorado, United States (enrolling)
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (enrolling)
• Suburban Research Associates — Media, Pennsylvania, United States (enrolling)
• Emory University - The Emory Clinic - Clifton Road — Atlanta, Georgia, United States (enrolling)
• Rush University Medical Center-Clinical Operation — Chicago, Illinois, United States (enrolling)
• Kennedy Krieger Institute, John Hopkins School of Medicine — Baltimore, Maryland, United States (enrolling)
• Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
• UMass Memorial Health — Worcester, Massachusetts, United States (enrolling)
• University of Minnesota Department of Medicine — Minneapolis, Minnesota, United States (enrolling)
• Neurology — Miami, Florida, United States
• Intermountain Healthcare - Park City Specialty Clinic — Salt Lake City, Utah, United States

Full record on ClinicalTrials.gov

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