Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome
Phase of Trial: Phase I/II
Latest Information Update: 15 Apr 2016
At a glance
- Drugs WASp gene therapy (Primary)
- Indications Wiskott-Aldrich syndrome
- Focus Adverse reactions; Therapeutic Use
- Sponsors Genethon
- 19 Nov 2014 The primary endpoints have been changed from safety, immunological repose, engraftment outcomes, clinical outcomes to improvement in eczema, infection, bleeding, autoimmune disorders and reduction in number of hospitalization as per ClinicalTrials.gov.
- 19 Nov 2014 Planned primary completion date changed from 1 Dec 2013 to 1 Dec 2017, according to ClinicalTrials.gov record.
- 21 Apr 2014 New trial record