NCT03282656View on ClinicalTrials.gov

Gene Transfer for Sickle Cell Disease

PHASE1Active, not recruitingINTERVENTIONAL
Enrollment

10

Participants

Timeline

Start Date

February 13, 2018

Primary Completion Date

September 13, 2024

Study Completion Date

December 13, 2024

Conditions
Sickle Cell Disease
Interventions
BIOLOGICAL

single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a

single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a

Trial Locations (2)

90095

UCLA - Mattel Children's Hospital, Los Angeles

02115

Boston Children's Hospital, Boston

All Listed Sponsors
lead

David Williams

OTHER