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Active Not RecruitingNCT02333760

Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome

Long Term Safety Follow up of Patients Enrolled in the Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome (GTG002-07 and GTG003-08).

Status
Active Not Recruiting
Phase
Phase 1 / Phase 2
Study type
Interventional
Enrollment
10 (estimated)
Sponsor
Genethon · Academic / Other
Sex
Male
Age
Healthy volunteers
Not accepted

Summary

An open follow up study of patients enrolled in the Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome and treated with autologous CD34+ cells transduced with the w1.6\_hWASP\_WPRE (VSVg) lentiviral vector.

Conditions

Interventions

TypeNameDescription
GENETICAutologous CD34+ cells transduced with WASP lentiviral vectorFollow up of ex vivo gene therapy transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WASP gene

Timeline

Start date
2014-09-01
Primary completion
2032-10-01
Completion
2032-10-01
First posted
2015-01-07
Last updated
2021-06-03

Locations

2 sites across 2 countries: France, United Kingdom

Source: ClinicalTrials.gov record NCT02333760. Inclusion in this directory is not an endorsement.