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Purpose

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

Condition

Eligibility

Eligible Ages
Between 13 Years and 45 Years
Eligible Sex
Male
Accepts 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

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Double (Participant, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
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
  • Drug: Low dose of MRM-3379
    Oral capsule
Experimental
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
  • Drug: Middle Dose of MRM-3379
    Oral capsule
Experimental
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
  • Drug: High dose of MRM-3379
    Oral capsule
Placebo Comparator
Arm 4 :Placebo 		Participants 16-45 years of age randomized to this arm will receive Placebo
  • Drug: Placebo
    Capsules matched to study drug without the active pharmaceutical ingredient
Experimental
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
  • Drug: Low dose of MRM-3379 Open-Label
    Oral capsule

Recruiting Locations

UMass Memorial Health
Worcester, Massachusetts 01655
Contact:
Taylor Merk
774-455-4119
Taylor.merk@umassmed.edu

More Details

Status
Recruiting
Sponsor
Mirum Pharmaceuticals, Inc.

Study Contact

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.