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

Not Applicable

Recruiting

• Conditions: Fragile X Syndrome
• 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

• Registration Number: NCT07209462
• Lead Sponsor: Mirum Pharmaceuticals, Inc.

Brief 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

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-10
• Study First Submitted: 2025-10-02
• Study First Submitted QC: 2025-10-02
• Last Update Submitted: 2025-10-06
• Study First Posted: 2025-10-07
• Last Update Posted: 2025-10-08
• Study Start: 2025-12-01
• Primary Completion: 2027-10
• Study Completion: 2027-10-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Male
• Target Recruitment: 60

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Arm 1: Low Dose of MRM-3379	Low dose of MRM-3379	Participants 16-45 years of age randomized to this arm will receive the low dose of MRM-3379
Arm 2: Middle dose of MRM-3379	Middle Dose of MRM-3379	Participants 16-45 years of age randomized to this arm will receive the middle dose of MRM-3379
Arm 3: High dose of MRM-3379	High dose of MRM-3379	Participants 16-45 years of age randomized to this arm will receive the high dose of MRM-3379
Arm 4 :Placebo	Placebo	Participants 16-45 years of age randomized to this arm will receive Placebo
Arm 5: Low dose of MRM-3379 Open-Label	Low dose of MRM-3379 Open-Label	Participants 13 to \< 16 years of age will receive the low dose of MRM-3370 Open-Label

Primary Outcome Measures

Name	Time	Method
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 Locations

Locations (5)

California Clinical Trials Medical Group(CCTMG) managed by Parexel; 🇺🇸 Glendale, California, United States

Amnova Clinical Research, LLC; 🇺🇸 Irvine, California, United States

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States

Suburban Research Associates; 🇺🇸 Media, Pennsylvania, United States