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

Last updated: April 20, 2026
Sponsor: Mirum Pharmaceuticals, Inc.
Overall Status: Active - Recruiting

Phase

2

Condition

Fragile X Syndrome

Treatment

Middle Dose of MRM-3379

Low dose of MRM-3379

High dose of MRM-3379

Clinical Study ID

NCT07209462
ENV-202
  • Ages 13-45
  • Male

Study 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 Criteria

Inclusion

Inclusion Criteria:

  • Willing and able to provide signed informed consent/assent. Where local regulationspermit inclusion of participants deemed unable to provide informed consent, alegally authorized representative must provide informed consent on the participant'sbehalf, 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 andreliably with the participant

  • Able to swallow tablets or capsules

Exclusion

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

Study Design

Total Participants: 60
Treatment Group(s): 5
Primary Treatment: Middle Dose of MRM-3379
Phase: 2
Study Start date:
November 22, 2025
Estimated Completion Date:
October 31, 2027

Connect with a study center

  • Southwest Autism Research & Resource Center

    Phoenix, Arizona 85006
    United States

    Active - Recruiting

  • Southwest Autism Research & Resource Center

    Phoenix 5308655, Arizona 5551752 85006
    United States

    Site Not Available

  • Amnova Clinical Research, LLC

    Irvine, California 92604
    United States

    Active - Recruiting

  • Children's Hospital of Orange County - Pediatric Subspecialty

    Orange, California 92868
    United States

    Site Not Available

  • Uc Davis Mind Institute

    Sacramento, California 95817
    United States

    Site Not Available

  • California Clinical Trials Medical Group(CCTMG) managed by Parexel

    Glendale 5352423, California 5332921 91206
    United States

    Site Not Available

  • Amnova Clinical Research, LLC

    Irvine 5359777, California 5332921 92604
    United States

    Site Not Available

  • Children's Hospital of Orange County - Pediatric Subspecialty

    Orange 5379513, California 5332921 92868
    United States

    Site Not Available

  • Uc Davis Mind Institute

    Sacramento 5389489, California 5332921 95817
    United States

    Site Not Available

  • Children's Hospital Colorado

    Aurora, Colorado 80045
    United States

    Site Not Available

  • Children's Hospital Colorado

    Colorado Springs, Colorado 80045
    United States

    Site Not Available

  • Children's Hospital Colorado

    Colorado Springs 5417598, Colorado 5417618 80045
    United States

    Site Not Available

  • Neurology

    Miami, Florida 33136
    United States

    Site Not Available

  • Neurology

    Miami 4164138, Florida 4155751 33136
    United States

    Site Not Available

  • Emory University - The Emory Clinic - Clifton Road

    Atlanta, Georgia 30322
    United States

    Site Not Available

  • Emory University - The Emory Clinic - Clifton Road

    Atlanta 4180439, Georgia 4197000 30322
    United States

    Site Not Available

  • Rush University Medical Center-Clinical Operation

    Chicago, Illinois 60612
    United States

    Active - Recruiting

  • Rush University Medical Center-Clinical Operation

    Chicago 4887398, Illinois 4896861 60612
    United States

    Site Not Available

  • Kennedy Krieger Institute, John Hopkins School of Medicine

    Baltimore, Maryland 21205
    United States

    Site Not Available

  • Kennedy Krieger Institute, John Hopkins School of Medicine

    Baltimore 4347778, Maryland 4361885 21205
    United States

    Site Not Available

  • Boston Children's Hospital

    Boston, Massachusetts 02115
    United States

    Site Not Available

  • UMass Memorial Health

    Worcester, Massachusetts 01655
    United States

    Active - Recruiting

  • Boston Children's Hospital

    Boston 4930956, Massachusetts 6254926 02115
    United States

    Site Not Available

  • UMass Memorial Health

    Worcester 4956184, Massachusetts 6254926 01655
    United States

    Site Not Available

  • University of Minnesota Department of Medicine

    Minneapolis, Minnesota 55414
    United States

    Site Not Available

  • University of Minnesota Department of Medicine

    Minneapolis 5037649, Minnesota 5037779 55414
    United States

    Site Not Available

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 42229
    United States

    Site Not Available

  • Cincinnati Children's Hospital Medical Center

    Cincinnati 4508722, Ohio 5165418 42229
    United States

    Site Not Available

  • Suburban Research Associates

    Media, Pennsylvania 19063
    United States

    Active - Recruiting

  • Suburban Research Associates

    Media 4559575, Pennsylvania 6254927 19063
    United States

    Site Not Available

  • Intermountain Healthcare - Park City Specialty Clinic

    Salt Lake City, Utah 84108
    United States

    Site Not Available

  • Intermountain Healthcare - Park City Specialty Clinic

    Salt Lake City 5780993, Utah 5549030 84108
    United States

    Site Not Available

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