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NCT07209462PHASE2Recruiting

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

Mirum Pharmaceuticals, Inc.

Start Date

11/22/2025

Completion Date

10/1/2027

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

Age Range: 13 years to 45 years

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

Interventions

DRUG

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

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Conditions

Fragile X Syndrome

Locations

Southwest Autism Research & Resource Center

Phoenix, Arizona 85006

United States

Amnova Clinical Research, LLC

Irvine, California 92604

United States

Children's Hospital of Orange County - Pediatric Subspecialty

Orange, California 92868

United States

Uc Davis Mind Institute

Sacramento, California 95817

United States

Children's Hospital Colorado

Aurora, Colorado 80045

United States

Neurology

Miami, Florida 33136

United States

Emory University - The Emory Clinic - Clifton Road

Atlanta, Georgia 30322

United States

Rush University Medical Center-Clinical Operation

Chicago, Illinois 60612

United States

Kennedy Krieger Institute, John Hopkins School of Medicine

Baltimore, Maryland 21205

United States

Boston Children's Hospital

Boston, Massachusetts 02115

United States

UMass Memorial Health

Worcester, Massachusetts 01655

United States

University of Minnesota Department of Medicine

Minneapolis, Minnesota 55414

United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio 42229

United States

Suburban Research Associates

Media, Pennsylvania 19063

United States

Intermountain Healthcare - Park City Specialty Clinic

Salt Lake City, Utah 84108

United States