drugset / Trial / NCT04774536
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
Phase 1/2
Recruiting
9 enrolled
Mark Walters, MD
University of California, Berkeley · collabUniversity of California, Los Angeles · collab
NaSingle-groupOpen-labelTreatment
Summary
This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will evaluate the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR\_SCD001 Drug Product).
Timeline
- Start
- 2025-08-22
- Primary completion
- 2030-03-01
- Completion
- 2030-03-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | CRISPR_SCD001 | CRISPR / gene editing | 3 cells/kg | Intravenous |
| Subject | CRISPR_SCD001 | CRISPR / gene editing | 20 cells/kg | Intravenous |