Spinal Muscular Atrophy
Conditions
Brief summary
Number and proportion of patients reporting treatment-emergent serious adverse events (SAEs) by Medical Dictionary for Regulatory Activities (MedDRA) System Organ Class (SOC) and Preferred Term (PT) for the entire duration of the study (i.e., up to 5 years), Number and proportion of patients reporting treatment-emergent adverse events of special interest (AESIs) by AESI category and MedDRA SOC and PT within each AESI category for the entire duration of the study (i.e., up to 5 years)
Detailed description
The number and proportion of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist, The number and proportion of participants demonstrating maintenance of each developmental milestone, Change from Baseline in the Hammersmith Functional Motor Scale – Expanded (HFMSE) total score, Change from Baseline in the Revised Upper Limb Module (RULM) total score, Number and proportion of patients with potentially clinically significant vital sign findings, summarized individually for each vital sign parameter, Number and proportion of patients with potentially clinically significant laboratory values, summarized individually for each laboratory parameter
Interventions
DRUGOAV101B
Sponsors
Novartis Pharma AG
Eligibility
Sex/Gender
All
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number and proportion of patients reporting treatment-emergent serious adverse events (SAEs) by Medical Dictionary for Regulatory Activities (MedDRA) System Organ Class (SOC) and Preferred Term (PT) for the entire duration of the study (i.e., up to 5 years), Number and proportion of patients reporting treatment-emergent adverse events of special interest (AESIs) by AESI category and MedDRA SOC and PT within each AESI category for the entire duration of the study (i.e., up to 5 years) | — |
Secondary
| Measure | Time frame |
|---|---|
| The number and proportion of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist, The number and proportion of participants demonstrating maintenance of each developmental milestone, Change from Baseline in the Hammersmith Functional Motor Scale – Expanded (HFMSE) total score, Change from Baseline in the Revised Upper Limb Module (RULM) total score, Number and proportion of patients with potentially clinically significant vital sign findings, summarized individually for each vital sign parameter, Number and proportion of patients with potentially clinically significant laboratory values, summarized individually for each laboratory parameter | — |
Countries
Belgium, Denmark, France, Italy, Netherlands, Spain
Outcome results
None listed