Duchenne Muscular Dystrophy
Conditions
Brief summary
Phase 1: The frequency of the incidence and severity of all adverse events – AEs, SAEs and AESI., Phase 1: Mean changes from the baseline (V0a) recorded in the functional assessments adjusted to the stage of the disease: for ambulatory patients: 6MWT, timed functions of NSAA: supine to rise and 10-meter walk/run, for non-ambulatory patients: PUL 2.0, at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7)., Phase 1: Mean changes from the baseline (V0a) in the EMG assessment of MUP duration of the selected muscles of upper and lower extremity in both ambulatory and non-ambulatory patients, at month 3 (V5), 6 (V6), and 12 (V7)., Phase 2: The frequency of the incidence and severity of all adverse events – AEs, SAEs and AESI., Phase 2: Mean changes from the baseline (V0a) recorded in the functional assessments adjusted to the stage of the disease: a. for ambulatory patients: 6MWT, timed functions of NSAA: supine to rise and 10-meter walk/run b. for non-ambulatory patients: PUL 2.0 at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7)., Phase 2: Mean changes from the baseline in the EMG assessment of MUP duration of the selected muscles of upper and lower extremity in both ambulatory and non-ambulatory patients at month 3, 6 and 12 .
Detailed description
Phase 2: Mean changes from the baseline (V0a) recorded by functional assessments (adjusted to the stage of the disease): for ambulatory patients: PUL 2.0, assessment of grip strength by dynamometer; for non-ambulatory patients: assessment of grip strength by dynamometer. At month 1 (V4), 3 (V5), 6 (V6), and 12 (V7)., Phase 2: Mean changes from the baseline (V0a) in QoL assessed by PODCI at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7)., Phase 2: Evaluation of Overall Treatment Effect at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7).
Interventions
DRUGDT-DEC01
Sponsors
Dystrogen Therapeutics Technology Polska Sp. z o.o.
Eligibility
Sex/Gender
Male
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Phase 1: The frequency of the incidence and severity of all adverse events – AEs, SAEs and AESI., Phase 1: Mean changes from the baseline (V0a) recorded in the functional assessments adjusted to the stage of the disease: for ambulatory patients: 6MWT, timed functions of NSAA: supine to rise and 10-meter walk/run, for non-ambulatory patients: PUL 2.0, at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7)., Phase 1: Mean changes from the baseline (V0a) in the EMG assessment of MUP duration of the selected muscles of upper and lower extremity in both ambulatory and non-ambulatory patients, at month 3 (V5), 6 (V6), and 12 (V7)., Phase 2: The frequency of the incidence and severity of all adverse events – AEs, SAEs and AESI., Phase 2: Mean changes from the baseline (V0a) recorded in the functional assessments adjusted to the stage of the disease: a. for ambulatory patients: 6MWT, timed functions of NSAA: supine to rise and 10-meter walk/run b. for non-ambulatory patients: PUL 2.0 at month 1 (V4), 3 | — |
Secondary
| Measure | Time frame |
|---|---|
| Phase 2: Mean changes from the baseline (V0a) recorded by functional assessments (adjusted to the stage of the disease): for ambulatory patients: PUL 2.0, assessment of grip strength by dynamometer; for non-ambulatory patients: assessment of grip strength by dynamometer. At month 1 (V4), 3 (V5), 6 (V6), and 12 (V7)., Phase 2: Mean changes from the baseline (V0a) in QoL assessed by PODCI at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7)., Phase 2: Evaluation of Overall Treatment Effect at month 1 (V4), 3 (V5), 6 (V6), and 12 (V7). | — |
Countries
Poland
Outcome results
None listed