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Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)
Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of MRM-3379 in Male Participants With Fragile X Syndrome
Who can join
Ages 13 to 45 · Men
Full eligibility criteria
Inclusion Criteria: * 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 Exclusion Criteria: History of or current medical condition other than FXS and related issues that would place the participant at higher risk from study participation
About the study
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
What is being tested
- Low dose of MRM-3379 (drug)
- Middle Dose of MRM-3379 (drug)
- High dose of MRM-3379 (drug)
- Placebo (drug)
- Low dose of MRM-3379 Open-Label (drug)
Sponsor: Mirum Pharmaceuticals, Inc. · Participants: 60 · Started: Nov 22, 2025
Contact the study team
Official record on ClinicalTrials.gov — NCT07209462
Locations in the U.S.
Conditions
From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.