Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)
ConditionFragile X Syndrome
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexMale
Age13-45
SponsorMirum Pharmaceuticals, Inc.
About this trial
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 criteria
Qualifiers
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 .
Disqualifiers
None
Trial design
Treatments tested in this trial
- Low dose of MRM-3379
- Middle Dose of MRM-3379
- High dose of MRM-3379
- Placebo
- Low dose of MRM-3379 Open-Label
Treatment groups
60 Participants
are divided into 5 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov