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

Phase 2RecruitingNCT07209462

Mirum Pharmaceuticals, Inc.60 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Fragile X Syndrome

Interventions

Low dose of MRM-3379DRUG

Oral capsule

Middle Dose of MRM-3379DRUG

Oral capsule

High dose of MRM-3379DRUG

Oral capsule

Placebo

Eligibility

Sex: MALEMin age: 13 YearsMax age: 45 Years

Medical Language ↔ Plain English

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

Locations (15)

Southwest Autism Research & Resource Center

Phoenix, Arizona, United States

RECRUITING

Outcomes

Primary Outcomes

Incidence and severity of treatment-emergent adverse events and Withdrawal due to adverse events and withdrawal due to adverse events

Time frame: Baseline to Week 12

Data from ClinicalTrials.gov

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