Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03300453 | Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome | PHASE1 | COMPLETED | 4 | — | — | Sep 17, 2013 | Nov 27, 2019 | Dec 2, 2019 | 1 | France |
Multiple measurements will be aggregated to derive the number of participants with Abnormal Laboratory Values and/or Adverse Events that are related to Treatment.
| Arm | Type | Description |
|---|---|---|
| rAAV2/5-hNAGLU | EXPERIMENTAL | Each patient will receive 960 µL of vector suspension. The vector suspension will be deposited simultaneously at 16 sites, each deposit containing 2.4x 1011 vg (4x1012 vg in total). |
| Name | Type | Description |
|---|---|---|
| rAAV2/5-hNAGLU | DRUG | one-time brain intraparenchymal gene therapy dose |
Inclusion criteria: * Age: 18 months up to 60 months (5th birthday); * Onset of clinical manifestations related to mucopolysaccharidosis type IIIB (MPSIIIB); * NAGLU activity in peripheral blood cell and/or cultured fibroblast extracts of less than 10% of controls; * Patient affiliated to, or cover...