NCT01410825View on ClinicalTrials.gov

Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

5

Participants

Timeline

Start Date

July 31, 2011

Primary Completion Date

September 30, 2024

Study Completion Date

September 30, 2024

Conditions
Wiskott-Aldrich Syndrome
Interventions
BIOLOGICAL

Retrovirus-mediated gene transfer

Two procedures: 1) Bone marrow harvest from the patient's posterior iliac crests or collection of peripheral blood stem cells via apheresis procedure. 2) One time infusion of patient's transduced bone marrow cells.

Trial Locations (1)

02116

Children's Hospital Boston, Boston

All Listed Sponsors
lead

David Williams

OTHER

NCT01410825 - Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome | Biotech Hunter | Biotech Hunter