Fragile X Syndrome
Conditions
Brief summary
To evaluate the safety and tolerability of MRM-3379 in male participants with FXS on the bases of the following endpoints: -Incidence and severity of TEAEs; - Withdrawal due to AEs.
Detailed description
To evaluate the preliminary efficacy of MRM-3379 in male participants with FXS on the basis of the following endpoints: - Key secondary endpoint: Change from baseline on the NIH-TCB Crystallized Cognition Composite (CCC): Composed from the Picture Vocabulary Test (PVT) and Oral Reading Recognition Test (ORRT) change sensitive scores; - Other secondary endpoint: Clinician Global Impression-Change (CGI-C) score, To describe the PK properties of MRM-3379 on the basis of the following endpoint: Steady-state MRM-3379 and metabolite plasma concentrations
Interventions
DRUGPlacebo for MRM-3379
DRUGMRM-3379
Sponsors
Mirum Pharmaceuticals Inc.
Eligibility
Sex/Gender
Male
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To evaluate the safety and tolerability of MRM-3379 in male participants with FXS on the bases of the following endpoints: -Incidence and severity of TEAEs; - Withdrawal due to AEs. | — |
Secondary
| Measure | Time frame |
|---|---|
| To evaluate the preliminary efficacy of MRM-3379 in male participants with FXS on the basis of the following endpoints: - Key secondary endpoint: Change from baseline on the NIH-TCB Crystallized Cognition Composite (CCC): Composed from the Picture Vocabulary Test (PVT) and Oral Reading Recognition Test (ORRT) change sensitive scores; - Other secondary endpoint: Clinician Global Impression-Change (CGI-C) score, To describe the PK properties of MRM-3379 on the basis of the following endpoint: Steady-state MRM-3379 and metabolite plasma concentrations | — |
Outcome results
None listed