NCT03311503View on ClinicalTrials.gov

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 with Low Dose Targeted Busulfan Conditioning

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

12

Participants

Timeline

Start Date

February 26, 2018

Primary Completion Date

October 1, 2026

Study Completion Date

October 1, 2028

Conditions
Severe Combined Immunodeficiency, X LinkedGene Therapy
Interventions
BIOLOGICAL

autologous CD34+ cell transduced with G2SCID vector

single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) lentiviral vector G2SCID

Trial Locations (4)

30322

RECRUITING

Emory University/Childrens Healthcare of Atlanta, Atlanta

45229

RECRUITING

Cincinnati Children's Hospital Medical Center, Cincinnati

90095

RECRUITING

Mattel Children's Hospital - UCLA, Los Angeles

02115

RECRUITING

Boston Childrens Hospital, Boston

All Listed Sponsors
lead

David Williams

OTHER