Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)
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
- Fragile X Syndrome
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
| Arm | Description | Assigned 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 |
|
|
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 |
|
|
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 |
|
|
Placebo Comparator Arm 4 :Placebo |
Participants 16-45 years of age randomized to this arm will receive Placebo |
|
|
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 |
|
Recruiting Locations
Southwest Autism Research & Resource Center
Phoenix, Arizona 85006
Phoenix, Arizona 85006
Amnova Clinical Research, LLC
Irvine, California 92604
Irvine, California 92604
Uc Davis Mind Institute
Sacramento, California 95817
Sacramento, California 95817
Children's Hospital Colorado
Aurora, Colorado 80045
Aurora, Colorado 80045
Emory University - The Emory Clinic - Clifton Road
Atlanta, Georgia 30322
Atlanta, Georgia 30322
Rush University Medical Center-Clinical Operation
Chicago, Illinois 60612
Chicago, Illinois 60612
Kennedy Krieger Institute, John Hopkins School of Medicine
Baltimore, Maryland 21205
Baltimore, Maryland 21205
Boston Children's Hospital
Boston, Massachusetts 02115
Boston, Massachusetts 02115
UMass Memorial Health
Worcester, Massachusetts 01655
Worcester, Massachusetts 01655
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 42229
Cincinnati, Ohio 42229
Suburban Research Associates
Media, Pennsylvania 19063
Media, Pennsylvania 19063
More Details
- Status
- Recruiting
- Sponsor
- Mirum Pharmaceuticals, Inc.