Duchenne muscular dystrophy (DMD)
Conditions
Brief summary
CORE PHASE: Area under the concentration-time curve from dosing (time 0) to time t at steady state (AUC0-T,ss) after at least 7 days of dosing (at Week 1 and at 6 months) CORE PHASE: Maximum plasma concentration at steady state (Cmax,ss) after at least 7 days of dosing (at Week 1 and at 6 months) CORE PHASE: Elimination half life (t1/2) assessed after at least 7 days of dosing dosing (at Week 1 and at 6 months), EXTENSION PHASE: • Type, incidence, and severity of TEAEs and SAEs from baseline up to Week 144 • Proportion of subjects experiencing TEAEs from baseline to Week 144
Detailed description
CORE PHASE: • Type, incidence, and severity of treatment-emergent adverse events (TEAEs) and SAEs from baseline to Week 48 • Proportion of subjects experiencing TEAEs from baseline to Week 48 • Change from baseline vital signs and clinical laboratory tests to each postbaseline visit up to Week 48 • Change from baseline electrocardiogram (ECG) to each postbaseline visit up to Week 48, CORE PHASE: • Change in physical function as per the Bayley III Gross Motor scale from baseline to Week 48 for subjects aged ≥2 to <3.5 years of age • Change in physical function as per the North Star Ambulatory Assessment (NSAA) total score from baseline to Week 48 for subjects aged ≥3.5 years of age, CORE PHASE: • Change in Paediatric Outcomes Data Collection Instrument (PODCI) from baseline to Week 48 for subjects aged ≥4 years of age in Cohort 1 only, EXTENSION PHASE: • Type, incidence, and severity of TEAEs and SAEs from baseline up to Week 144 • Proportion of patients experiencing TEAEs from baseline to Week 144 • Change from baseline vital signs and clinical laboratory tests to each postbaseline visit up to Week 144 • Change from baseline ECG to each postbaseline up to Week 144
Interventions
DRUGITF2357
Sponsors
Italfarmaco S.p.A.
Eligibility
Sex/Gender
Male
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| CORE PHASE: Area under the concentration-time curve from dosing (time 0) to time t at steady state (AUC0-T,ss) after at least 7 days of dosing (at Week 1 and at 6 months) CORE PHASE: Maximum plasma concentration at steady state (Cmax,ss) after at least 7 days of dosing (at Week 1 and at 6 months) CORE PHASE: Elimination half life (t1/2) assessed after at least 7 days of dosing dosing (at Week 1 and at 6 months), EXTENSION PHASE: • Type, incidence, and severity of TEAEs and SAEs from baseline up to Week 144 • Proportion of subjects experiencing TEAEs from baseline to Week 144 | — |
Secondary
| Measure | Time frame |
|---|---|
| CORE PHASE: • Type, incidence, and severity of treatment-emergent adverse events (TEAEs) and SAEs from baseline to Week 48 • Proportion of subjects experiencing TEAEs from baseline to Week 48 • Change from baseline vital signs and clinical laboratory tests to each postbaseline visit up to Week 48 • Change from baseline electrocardiogram (ECG) to each postbaseline visit up to Week 48, CORE PHASE: • Change in physical function as per the Bayley III Gross Motor scale from baseline to Week 48 for subjects aged ≥2 to <3.5 years of age • Change in physical function as per the North Star Ambulatory Assessment (NSAA) total score from baseline to Week 48 for subjects aged ≥3.5 years of age, CORE PHASE: • Change in Paediatric Outcomes Data Collection Instrument (PODCI) from baseline to Week 48 for subjects aged ≥4 years of age in Cohort 1 only, EXTENSION PHASE: • Type, incidence, and severity of TEAEs and SAEs from baseline up to Week 144 • Proportion of patients experiencing TEAEs from baselin | — |
Countries
Belgium, Italy, Netherlands
Outcome results
None listed