Ireland
--:--IST
Latest
Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide
Clinical Trials in the USA / NCT07209462
Recruiting Phase 2

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

NCT07209462 · tracked via the Priya Life Science USA tracker
Sponsor
Mirum Pharmaceuticals, Inc
Phase
Phase 2
Started
2025-11-22
Last updated
2026-09-29

Condition(s) studied

Fragile X Syndrome

Investigational drug(s) / intervention(s)

Low dose of MRM-3379Middle Dose of MRM-3379High dose of MRM-3379PlaceboLow dose of MRM-3379 Open-Label

Low dose of MRM-3379: Oral capsule

Middle Dose of MRM-3379: Oral capsule

High dose of MRM-3379: Oral capsule

Placebo: Capsules matched to study drug without the active pharmaceutical ingredient

Low dose of MRM-3379 Open-Label: Oral capsule

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

Sex
MALE
Min age
13 Years
Max age
45 Years
Healthy volunteers
No
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

Primary outcome measure(s)

  • Incidence and severity of treatment-emergent adverse events and Withdrawal due to adverse events and withdrawal due to adverse events — Baseline to Week 12

Trial sites (18)

FacilityCityRegionStatus
Southwest Autism Research & Resource Center Phoenix Arizona Recruiting
Amnova Clinical Research, LLC Irvine California Recruiting
Children's Hospital of Orange County - Pediatric Subspecialty Orange California Recruiting
Uc Davis Mind Institute Sacramento California Recruiting
Children's Hospital Colorado Aurora Colorado Recruiting
Neurology Miami Florida Recruiting
Emory University - The Emory Clinic - Clifton Road Atlanta Georgia Recruiting
Rush University Medical Center-Clinical Operation Chicago Illinois Recruiting
Kennedy Krieger Institute, John Hopkins School of Medicine Baltimore Maryland Recruiting
Boston Children's Hospital Boston Massachusetts Recruiting
UMass Memorial Health Worcester Massachusetts Recruiting
University of Minnesota Department of Medicine Minneapolis Minnesota Recruiting
Cincinnati Children's Hospital Medical Center Cincinnati Ohio Recruiting
Suburban Research Associates Media Pennsylvania Recruiting
Intermountain Healthcare - Park City Specialty Clinic Salt Lake City Utah 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

More Mirum Pharmaceuticals, Inc. trials in the USA

The REPLACE Registry for Cholbam® (Cholic Acid) Active, not recruiting
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis Active, not recruiting
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC) Active, not recruiting
A Trial Evaluating Brelovitug vs Delayed Treatment for the Treatment of Chronic Hepatitis Delta Infection (AZURE-4) Active, not recruiting
A Trial Evaluating BJT-778 vs Delayed Treatment for the Treatment of Chronic Hepatitis Delta Infection Active, not recruiting

Other trials for the same condition

Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS Active, not recruiting
An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07209462 on ClinicalTrials.gov ↗ ← All trials in the USA