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NCT07209462From ClinicalTrials.govRecruiting

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

  • Fragile X Syndrome

At a glance

Phase
Phase 2
Study type
Interventional
Sponsor
Mirum Pharmaceuticals, Inc.
Enrolment target
60
Started
22 November 2025
Main results due
October 2027
Study sites
18
Registry updated
29 September 2026

Can you take part?

  • Ages 13 years to 45 years.
  • Men only.
  • You need the condition being studied — healthy volunteers are not accepted.

These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.

Read the full eligibility criteria
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

What this study is about

In the sponsor’s own words, from the registry.

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

Study sites(18)

  • Southwest Autism Research & Resource Center

    Phoenix, Arizona, United States

    Recruiting

  • Amnova Clinical Research, LLC

    Irvine, California, United States

    Recruiting

  • Children's Hospital of Orange County - Pediatric Subspecialty

    Orange, California, United States

    Recruiting

  • Uc Davis Mind Institute

    Sacramento, California, United States

    Recruiting

  • Children's Hospital Colorado

    Aurora, Colorado, United States

    Recruiting

  • Neurology

    Miami, Florida, United States

    Recruiting

  • Emory University - The Emory Clinic - Clifton Road

    Atlanta, Georgia, United States

    Recruiting

  • Rush University Medical Center-Clinical Operation

    Chicago, Illinois, United States

    Recruiting

  • Kennedy Krieger Institute, John Hopkins School of Medicine

    Baltimore, Maryland, United States

    Recruiting

  • Boston Children's Hospital

    Boston, Massachusetts, United States

    Recruiting

  • UMass Memorial Health

    Worcester, Massachusetts, United States

    Recruiting

  • University of Minnesota Department of Medicine

    Minneapolis, Minnesota, United States

    Recruiting

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, United States

    Recruiting

  • Suburban Research Associates

    Media, Pennsylvania, United States

    Recruiting

  • Intermountain Healthcare - Park City Specialty Clinic

    Salt Lake City, Utah, United States

    Not yet recruiting

  • Parc Tauli Hospital Universitari

    Barcelona, Spain

    Recruiting

  • Sant Joan De Deu Barcelona Hospital

    Barcelona, Spain

    Recruiting

  • Hospital Regional Universitario de Málaga (H. Civil)

    Málaga, Spain

    Recruiting

Showing 18 of 18 sites. 17 of the 18 sites on this study are recruiting right now — a site can stop enrolling while the study as a whole is still open.

Source: ClinicalTrials.gov record NCT07209462. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.

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