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
Mirum Pharmaceuticals, Inc.
60 participants
Nov 22, 2025
INTERVENTIONAL
Conditions
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
Inclusion Criteria7
- 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 Criteria1
- History of or current medical condition other than FXS and related issues that would place the participant at higher risk from study participation
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Interventions
Oral capsule
Oral capsule
Oral capsule
Capsules matched to study drug without the active pharmaceutical ingredient
Oral capsule
Locations(15)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT07209462