Autosomal Dominant RYR1-Related Myopathy
Conditions
Brief summary
Change from Day 1 in the 1-MSST measurements after approximately 28 days of dosing between active treatment and placebo.
Detailed description
1. Change from Day 1 in the 6 MNWT, TUG, 4 SCT, and for weight-scaled muscle strength as measured by QMA and MMT after approximately 28 days of dosing between active treatment and placebo, 2. Change from Day 1 in the PROMIS-F, PROMIS PF and IPAQ questionnaires after approximately 28 days of dosing between active treatment and placebo, 3. Incidence of AEs, serious adverse events (SAEs), treatment-emergent adverse events (TEAEs) and adverse events of special interest (AESIs) throughout the trial, 4. Change from Day 1 in safety assessments (vital signs, physical examinations, laboratory safety tests, electrocardiograms [ECGs], and Columbia Suicide Severity Rating Scale [C‑SSRS])
Interventions
DRUGSurlorian placebo
DRUGSurlorian
Sponsors
Rycarma Therapeutics Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from Day 1 in the 1-MSST measurements after approximately 28 days of dosing between active treatment and placebo. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Change from Day 1 in the 6 MNWT, TUG, 4 SCT, and for weight-scaled muscle strength as measured by QMA and MMT after approximately 28 days of dosing between active treatment and placebo, 2. Change from Day 1 in the PROMIS-F, PROMIS PF and IPAQ questionnaires after approximately 28 days of dosing between active treatment and placebo, 3. Incidence of AEs, serious adverse events (SAEs), treatment-emergent adverse events (TEAEs) and adverse events of special interest (AESIs) throughout the trial, 4. Change from Day 1 in safety assessments (vital signs, physical examinations, laboratory safety tests, electrocardiograms [ECGs], and Columbia Suicide Severity Rating Scale [C‑SSRS]) | — |
Outcome results
None listed