drugset / Trial / NCT03223194
Gene Transfer Clinical Study in Crigler-Najjar Syndrome
RandomizedSequentialOpen-labelTreatment
Summary
This is a Phase 1/2, multinational, open-label, ascending-dose, delayed-treatment concurrent control clinical study to evaluate the safety and preliminary efficacy of AT342 in subjects with Crigler-Najjar aged ≥1 year. Subjects will receive a single dose of AT342 and will be followed for safety and efficacy for 5 years.
Timeline
- Start
- 2017-09-08
- Primary completion
- 2021-02-11
- Completion
- 2021-02-11
Outcome
Outcome not reported
Stopped (Business): “Sponsor Decision”
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | VOLRUBIGENE RALAPARVOVEC | Gene therapy (AAV / viral vector) | 1.5e+12 vg/kg | Intravenous |
| Subject | VOLRUBIGENE RALAPARVOVEC | Gene therapy (AAV / viral vector) | 6e+12 vg/kg | Intravenous |
| Subject | VOLRUBIGENE RALAPARVOVEC | Gene therapy (AAV / viral vector) | 1.5e+13 vg/kg | Intravenous |