drugset / Trial / NCT04174105
Gene Transfer Study in Patients With Late Onset Pompe Disease
Non-randomizedSequentialOpen-labelTreatment
Summary
This is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD).
Timeline
- Start
- 2020-10-27
- Primary completion
- 2035-02-28
- Completion
- 2035-02-28
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | zocaglusagene nuzaparvovec | Unknown | 3e+13 vg/kg | Intravenous |
| Subject | zocaglusagene nuzaparvovec | Unknown | 6e+13 vg/kg | Intravenous |
| Subject | zocaglusagene nuzaparvovec | Unknown | 1e+14 vg/kg | Intravenous |