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Clinical Trials in Spain / NCT07209462
Recruiting Phase 2

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

NCT07209462 · tracked via the Priya Life Science Spain 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-3379 →Middle Dose of MRM-3379High dose of MRM-3379 →PlaceboLow 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)

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 Spain

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
Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND) Active, not recruiting
Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU) 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
Study to Evaluate Switching to Brelovitug for the Treatment of CHD in Participants Receiving Bulevirtide Recruiting

Other trials for the same condition

Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome 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 Spain