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Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome

Trial Profile

Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 28 May 2018

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At a glance

  • Drugs WASp gene therapy (Primary)
  • Indications Wiskott-Aldrich syndrome
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Sponsors Genethon
  • Most Recent Events

    • 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
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