Trials / Completed
CompletedNCT01347242
Gene Therapy for Wiskott-Aldrich Syndrome (WAS)
Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome
- Status
- Completed
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 6 (actual)
- Sponsor
- Genethon · Academic / Other
- Sex
- Male
- Age
- —
- Healthy volunteers
- Not accepted
Summary
This is a phase I/II study to evaluate the safety and efficacy of Hematopoietic Stem Cell genetherapy for the Wiskott-Aldrich Syndrome.
Detailed description
This clinical trial is an ex vivo gene therapy trial. The investigational product corresponds to autologous CD34+ cells transduced with a lentiviral vector harboring the human WASP gene.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| 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 |
Timeline
- Start date
- 2011-03-01
- Primary completion
- 2019-11-13
- Completion
- 2019-11-13
- First posted
- 2011-05-04
- Last updated
- 2021-06-03
Locations
2 sites across 1 country: United Kingdom
Source: ClinicalTrials.gov record NCT01347242. Inclusion in this directory is not an endorsement.