Not currently recruiting at UC Davis

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

a study on Fragile X Syndrome

Summary

Eligibility
for males ages 13-45 (full criteria)
Location
at Sacramento, California and other locations
Dates
study started
study ends around
Principal Investigator
by Randi Hagerman
Headshot of Randi Hagerman
Randi Hagerman

Description

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

Official Title

Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of MRM-3379 in Male Participants With Fragile X Syndrome

Keywords

Fragile X Syndrome, FXS, FMR, Phosphodiesterase 4, PDE4, Low dose of MRM-3379, Middle Dose of MRM-3379, High dose of MRM-3379, Low dose of MRM-3379 Open-Label

Eligibility

You can join if…

Open to males ages 13-45

  • 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 CAN'T join if...

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

Locations

  • Uc Davis Mind Institute not yet accepting patients
    Sacramento California 95817 United States
  • Children's Hospital of Orange County - Pediatric Subspecialty not yet accepting patients
    Orange California 92868 United States
  • Amnova Clinical Research, LLC accepting new patients
    Irvine California 92604 United States
  • Southwest Autism Research & Resource Center accepting new patients
    Phoenix Arizona 85006 United States

Lead Scientist at UC Davis

  • Randi Hagerman
    Professor, MED: Pediatrics, School of Medicine. Authored (or co-authored) 637 research publications

Details

Status
accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
(estimated)
Sponsor
Mirum Pharmaceuticals, Inc.
ID
NCT07209462
Phase
Phase 2 Fragile X Syndrome Research Study
Study Type
Interventional
Participants
Expecting 60 study participants
Last Updated