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

NCT ID: NCT07209462

Last Updated: 2025-11-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

60 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-12-31

Study Completion Date

2027-10-31

Brief Summary

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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

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Conditions

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Fragile X Syndrome

Keywords

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Fragile X Syndrome FXS FMR Phosphodiesterase 4 PDE4

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Arm 1: Low Dose of MRM-3379

Participants 16-45 years of age randomized to this arm will receive the low dose of MRM-3379

Group Type EXPERIMENTAL

Low dose of MRM-3379

Intervention Type DRUG

Oral capsule

Arm 2: Middle dose of MRM-3379

Participants 16-45 years of age randomized to this arm will receive the middle dose of MRM-3379

Group Type EXPERIMENTAL

Middle Dose of MRM-3379

Intervention Type DRUG

Oral capsule

Arm 3: High dose of MRM-3379

Participants 16-45 years of age randomized to this arm will receive the high dose of MRM-3379

Group Type EXPERIMENTAL

High dose of MRM-3379

Intervention Type DRUG

Oral capsule

Arm 4 :Placebo

Participants 16-45 years of age randomized to this arm will receive Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Capsules matched to study drug without the active pharmaceutical ingredient

Arm 5: Low dose of MRM-3379 Open-Label

Participants 13 to \< 16 years of age will receive the low dose of MRM-3370 Open-Label

Group Type EXPERIMENTAL

Low dose of MRM-3379 Open-Label

Intervention Type DRUG

Oral capsule

Interventions

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Low dose of MRM-3379

Oral capsule

Intervention Type DRUG

Middle Dose of MRM-3379

Oral capsule

Intervention Type DRUG

High dose of MRM-3379

Oral capsule

Intervention Type DRUG

Placebo

Capsules matched to study drug without the active pharmaceutical ingredient

Intervention Type DRUG

Low dose of MRM-3379 Open-Label

Oral capsule

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

13 Years

Maximum Eligible Age

45 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Mirum Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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California Clinical Trials Medical Group(CCTMG) managed by Parexel

Glendale, California, United States

Site Status RECRUITING

Amnova Clinical Research, LLC

Irvine, California, United States

Site Status RECRUITING

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status NOT_YET_RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status NOT_YET_RECRUITING

Suburban Research Associates

Media, Pennsylvania, United States

Site Status RECRUITING

Countries

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United States

Facility Contacts

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Diana Devitskaya

Role: primary

Maggie Wang

Role: primary

Role: backup

Michelle Coughlin

Role: primary

Elizabeth Bank

Role: primary

Alex Hollis

Role: primary

Other Identifiers

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ENV-202

Identifier Type: -

Identifier Source: org_study_id