NCT05353647View on ClinicalTrials.gov

A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)

PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

25

Participants

Timeline

Start Date

July 12, 2022

Primary Completion Date

July 31, 2027

Study Completion Date

July 31, 2027

Conditions
Sickle Cell Disease
Interventions
BIOLOGICAL

Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a

A single infusion of autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a

Trial Locations (9)

30322

Children's Healthcare of Atlanta/Emory University, Atlanta

53226

Medical College of Wisconsin, Milwaukee

60611

Lurie Children's Hospital of Chicago, Chicago

90027

Children's Hospital of Los Angeles, Los Angeles

90095

UCLA Medical Center, Los Angeles

94609

UCSF Benioff Children's Hospital Oakland, Oakland

95817

UC Davis Medical Center, Sacramento

02115

Boston Children's Hospital, Boston

Dana-Farber Cancer Institute/Brigham and Women's Hospital, Boston

Sponsors
All Listed Sponsors
collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

collaborator

California Institute for Regenerative Medicine (CIRM)

OTHER

collaborator

Genetix Biotherapeutics Inc.

INDUSTRY

collaborator

Blood and Marrow Transplant Clinical Trials Network

NETWORK

lead

David Williams

OTHER

NCT05353647 - A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001) | Biotech Hunter | Biotech Hunter