drugset / Trial / NCT04105166
Gene Therapy for Pyruvate Kinase Deficiency (PKD)
NaSequentialOpen-labelTreatment
Summary
This is an open-label Phase I trial to evaluate the safety of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency (PKD).
Timeline
- Start
- 2020-07-06
- Primary completion
- 2025-06-09
- Completion
- 2025-06-09
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | RP-L301 | Unknown | — | Intravenous |