Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of MRM-3379 in Male Participants With Fragile X Syndrome
Overview
- Phase
- Phase 2
- Status
- Recruiting
- Sponsor
- Mirum Pharmaceuticals, Inc.
- Enrollment
- 60
- Locations
- 16
- Primary Endpoint
- Incidence and severity of treatment-emergent adverse events and Withdrawal due to adverse events and withdrawal due to adverse events
Overview
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
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Double (Participant, Investigator)
Eligibility Criteria
- Ages
- 13 Years to 45 Years (Child, Adult)
- 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
Arms & Interventions
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
Intervention: Low dose of MRM-3379 (Drug)
Arm 4 :Placebo
Participants 16-45 years of age randomized to this arm will receive Placebo
Intervention: Placebo (Drug)
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
Intervention: Low dose of MRM-3379 Open-Label (Drug)
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
Intervention: Middle Dose of MRM-3379 (Drug)
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
Intervention: High dose of MRM-3379 (Drug)
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
Secondary Outcomes
No secondary outcomes reported