Myotonic Dystrophy Type 1
Conditions
Brief summary
5×STS time, change from baseline at Week 49
Detailed description
Change from baseline at Week 49 in: vHOT; middle finger QMT total CGI-C 10-MWRT velocity (m/s) PGI-C DM1-ACTIVC total score MDHI total PGI-S CGI-S 9-HPT MDHI subscale, Maximum observed drug concentration (Cmax) Time to maximum concentration (tmax) Area under the concentration-time curve (AUC) from hour 0 to the last measurable concentration (AUCtlast) AUC extrapolated to infinity (AUC∞) Apparent terminal elimination rate constant (λZ) Apparent terminal elimination half-life (t½) Plasma clearance (CL) Volume of distribution at the terminal phase (Vz), if appropriate Volume of distribution at steady state (Vss), if appropriate, Incidence of anti-drug antibodies (ADAs), * Treatment-emergent adverse events (TEAEs) including: - Treatment-emergent serious adverse events (SAEs) - TEAEs considered related to study drug - TEAEs leading to discontinuation from study drug and discontinuation from the study * Other clinically significant safety and tolerability observations including: - Vital sign findings - 12-lead electrocardiogram (ECG) findings -Clinical laboratory values *C-SSRS results
Interventions
Sponsors
Eligibility
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 5×STS time, change from baseline at Week 49 | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline at Week 49 in: vHOT; middle finger QMT total CGI-C 10-MWRT velocity (m/s) PGI-C DM1-ACTIVC total score MDHI total PGI-S CGI-S 9-HPT MDHI subscale, Maximum observed drug concentration (Cmax) Time to maximum concentration (tmax) Area under the concentration-time curve (AUC) from hour 0 to the last measurable concentration (AUCtlast) AUC extrapolated to infinity (AUC∞) Apparent terminal elimination rate constant (λZ) Apparent terminal elimination half-life (t½) Plasma clearance (CL) Volume of distribution at the terminal phase (Vz), if appropriate Volume of distribution at steady state (Vss), if appropriate, Incidence of anti-drug antibodies (ADAs), * Treatment-emergent adverse events (TEAEs) including: - Treatment-emergent serious adverse events (SAEs) - TEAEs considered related to study drug - TEAEs leading to discontinuation from study drug and discontinuation from the study * Other clinically significant safety and tolerability observations including: - Vital si | — |