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

Plain-language summary

This Phase 1/2 open follow-up study monitors patients who were enrolled in the hematopoietic stem cell gene therapy trial for Wiskott-Aldrich Syndrome and treated with autologous CD34+ cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector. Its purpose is long-term safety follow-up. Participants are patients from the initial Phase 1/2 WAS studies conducted in France and the United Kingdom (GTG002.07 and GTG003.08) who, or whose parents or guardians, have provided informed consent.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Active, not recruiting

Sponsor

Genethon

Intervention

Autologous CD34+ cells transduced with WASP lentiviral vector

Start date

9/1/2014

Estimated completion

10/1/2032

Countries

France, United Kingdom

Related genes

Locations

• Paris, France
• London, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* Patients enrolled in the initial phase I/II WAS conducted in France and United Kingdom (GTG002.07 and GTG003.08).
* Parents, guardians or patient signed informed consent, guardians or patient signed informed consent

Exclusion Criteria:

• Parents, guardians, patients unwilling to return for the follow up study period.