NCT04774536View on ClinicalTrials.gov

Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

9

Participants

Timeline

Start Date

September 18, 2024

Primary Completion Date

March 1, 2027

Study Completion Date

March 1, 2029

Conditions
Sickle Cell Disease
Interventions
DRUG

CRISPR_SCD001

CRISPR\_SCD001 is administered by IV infusion following myeloablative conditioning with busulfan.

Trial Locations (2)

90095

RECRUITING

University of California, Los Angeles, Los Angeles

94609

RECRUITING

UCSF Benioff Children's Hospital, Oakland

All Listed Sponsors
collaborator

University of California, Los Angeles

OTHER

collaborator

University of California, Berkeley

OTHER

lead

Mark Walters, MD

OTHER