Gene Therapy for Wiskott-Aldrich Syndrome (WAS)

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

6

Participants

Timeline

Start Date

March 31, 2011

Primary Completion Date

November 13, 2019

Study Completion Date

November 13, 2019

Conditions
Wiskott-Aldrich Syndrome
Interventions
GENETIC

Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene

transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WAS gene

Trial Locations (2)

WC1N 1EH

Great Ormond Street Hospital, London

Royal Free Hospital, London

Sponsors

Lead Sponsor

All Listed Sponsors
collaborator

Great Ormond Street Hospital for Children NHS Foundation Trust

OTHER

collaborator

Institute of Child Health

OTHER

lead

Genethon

OTHER

NCT01347242 - Gene Therapy for Wiskott-Aldrich Syndrome (WAS) | Biotech Hunter | Biotech Hunter