Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Low dose of MRM-3379, Middle Dose of MRM-3379, High dose of MRM-3379, Placebo.
- Who it may be relevant to
- Registry conditions: Fragile X Syndrome. Basic parameters: 13 years — 45 years · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of MRM-3379 in Male Participants With Fragile X Syndrome
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
Interventions
- Drug Low dose of MRM-3379
Oral capsule - Drug Middle Dose of MRM-3379
Oral capsule - Drug High dose of MRM-3379
Oral capsule - Drug Placebo
Capsules matched to study drug without the active pharmaceutical ingredient - Drug Low dose of MRM-3379 Open-Label
Oral capsule
Primary outcome measures
- 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]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Double blind
- Primary purpose
- Treatment
Study locations
United States · 15 centers
- Southwest Autism Research & Resource Center — Phoenix
- Amnova Clinical Research, LLC — Irvine
- Children's Hospital of Orange County - Pediatric Subspecialty — Orange
- Uc Davis Mind Institute — Sacramento
- Children's Hospital Colorado — Aurora
- Neurology — Miami
- Emory University - The Emory Clinic - Clifton Road — Atlanta
- Rush University Medical Center-Clinical Operation — Chicago
- … and 7 more centers
Identifiers
NCT: NCT07209462 · ENV-202