Wiskott-Aldrich Syndrome
Conditions
Brief summary
Evaluation of the clinical efficacy: • annualized rate of severe infections from 6 to 18 months after gene therapy (GT) compared with 1 year prior to GT; • annualized rate of moderate and severe bleeding episodes up to 1 year after GT compared with 1 year prior to GT.
Detailed description
• Evaluation of the overall survival at 12, 24 and 36 months • Evaluation of the safety of treatment: 1) safety and tolerability as measured by adverse event (AE) reporting; 2) absence of malignancy or abnormal clonal proliferation (ACP) development due to insertional oncogenesis; 3) absence of replication-competent lentivirus (RCL). • Evaluation of the engraftment at 6 months • Evaluation of biological correlates of efficacy at 12 months, 2 years and 3 years
Interventions
Sponsors
Fondazione Telethon Ets
Eligibility
Sex/Gender
Male
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Evaluation of the clinical efficacy: • annualized rate of severe infections from 6 to 18 months after gene therapy (GT) compared with 1 year prior to GT; • annualized rate of moderate and severe bleeding episodes up to 1 year after GT compared with 1 year prior to GT. | — |
Secondary
| Measure | Time frame |
|---|---|
| • Evaluation of the overall survival at 12, 24 and 36 months • Evaluation of the safety of treatment: 1) safety and tolerability as measured by adverse event (AE) reporting; 2) absence of malignancy or abnormal clonal proliferation (ACP) development due to insertional oncogenesis; 3) absence of replication-competent lentivirus (RCL). • Evaluation of the engraftment at 6 months • Evaluation of biological correlates of efficacy at 12 months, 2 years and 3 years | — |
Countries
Italy
Outcome results
None listed