NCT00598481View on ClinicalTrials.gov

ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID

PHASE2CompletedINTERVENTIONAL
Enrollment

12

Participants

Timeline

Start Date

October 2, 2002

Primary Completion Date

July 10, 2011

Study Completion Date

June 19, 2019

Conditions
Immunologic Deficiency Syndromes
Interventions
GENETIC

Gene Therapy

Infusion of autologous CD34+ cells transduced with retroviral vector encoding ADA after non-myeloablative conditioning with busulfan

DRUG

Busulfan

Busulfan is used for non-myeloablative conditioning

Trial Locations (2)

20132

Ospedale San Raffaele, Milan

Unknown

Investigational Site, Jerusalem

All Listed Sponsors
lead

Fondazione Telethon

OTHER

NCT00598481 - ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID | Biotech Hunter | Biotech Hunter