Fragile X Syndrome
Conditions
Keywords
Fragile X Syndrome, FXS, FMR, Phosphodiesterase 4, PDE4
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
Interventions
DRUGLow dose of MRM-3379
Oral capsule
DRUGMiddle Dose of MRM-3379
Oral capsule
DRUGHigh dose of MRM-3379
Oral capsule
DRUGPlacebo
Capsules matched to study drug without the active pharmaceutical ingredient
DRUGLow dose of MRM-3379 Open-Label
Oral capsule
Sponsors
Mirum Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
MALE
Age
13 Years to 45 Years
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence and severity of treatment-emergent adverse events and Withdrawal due to adverse events and withdrawal due to adverse events | Baseline to Week 12 |
Countries
United States
Outcome results
None listed