drugset / Trial / NCT04411654
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
NaSingle-groupOpen-labelTreatment
Summary
J3Z-MC-OJAB is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose LY3884961 (formerly PR001) in infants diagnosed with Type 2 Gaucher disease (GD2). For each patient, the study will be approximately 5 years in duration. During the first 12 months after dosing, patients will be evaluated for the effects of LY3884961 on safety, tolerability, immunogenicity, biomarkers, and efficacy. Patients will be followed up for an additional 4 years to monitor safety and changes on selected biomarkers and clinical outcomes.
Timeline
- Start
- 2021-06-29
- Primary completion
- 2028-05
- Completion
- 2028-05
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | LY3884961 | Gene therapy (AAV / viral vector) | — | Other |
| Background | Methylprednisolone | Other / unclassified | — | Intravenous |
| Background | Prednisone | Other / unclassified | — | Oral |
| Background | Sirolimus | Small molecule | — | — |