Krabbe Disease
Conditions
Brief summary
Incidence and severity of adverse events and serious adverse events that are attributed to FBX-101, HSCT incident of engraftment
Detailed description
Improvement of probability to achieve independent sitting compared to untreated patients or those receiving HSCT only at one year and two years post gene therapy, Improvement of gross motor function, as measured by Peabody Developmental Motor Scale 2nd Edition (PDMS-2), above a functional age equivalent of 12 months, compared to untreated patients or those receiving HSCT only, at 2 years post gene therapy
Interventions
DRUGFBX-101
Sponsors
Forge Biologics Inc.
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence and severity of adverse events and serious adverse events that are attributed to FBX-101, HSCT incident of engraftment | — |
Secondary
| Measure | Time frame |
|---|---|
| Improvement of probability to achieve independent sitting compared to untreated patients or those receiving HSCT only at one year and two years post gene therapy, Improvement of gross motor function, as measured by Peabody Developmental Motor Scale 2nd Edition (PDMS-2), above a functional age equivalent of 12 months, compared to untreated patients or those receiving HSCT only, at 2 years post gene therapy | — |
Countries
Spain
Outcome results
None listed