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Prof Adrian Thrasher


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Be Part of Research - Trial Details - Gene therapy for Wiskott-Aldrich Syndrome (WAS)
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Gene therapy for Wiskott-Aldrich Syndrome (WAS)

Not Recruiting

Open to: Male

Age: Adult

London

Medical Conditions

Wiskott-Aldrich Syndrome

This information is provided directly by researchers and we recognise that it isn't always easy to understand. We are working with researchers to improve the accessibility of this information.


Not provided at time of registration

Start dates may differ between countries and research sites. The research team are responsible for keeping the information up-to-date.  

The recruitment start and end dates are as follows:

23 Feb 2010 31 Dec 2013

Publications

2017 Results article in https://www.ncbi.nlm.nih.gov/pubmed/28716862 results

Interventional

Intervention Type : Drug
Intervention Description : Ex vivo gene therapy using patient's autologous CD34+ cells transduced with a lentiviral vector containing the human WASP gene.

Patients undergo either a bone marrow harvest or a leukapheresis. They then receive a conditioning myeloablative regimen while CD34+ cells are selected in their bone marrow and transduced with the lentiviral vector (3 days). Patients then receive their transduced CD34+ cells (as in autologous bone marrow transplantation).

There are no real doses, simply quantity of CD34+ cells transduced will depend on the amount of bone marrow harvest and quality of transduction. This is part of the parameters that are being assessed in the trial.

Duration of the study follow-up is 2 years.




You can take part if:



You may not be able to take part if:


1. Patient with HLA-genotypically identical bone marrow2. Patient with 10/10 antigen HLA-matched unrelated donor or cord blood3. Contraindication to leukapheresis3.1. Anaemia (Hb < 8g/dl)3.2. Cardiovascular instability3.3. Severe coagulopathy3.3.1. Contraindication to bone marrow harvest3.3.2. Contraindication to administration of conditioning medication3. Human immunodeficiency virus (HIV) positive patient


Below are the locations for where you can take part in the trial. Please note that not all sites may be open.

  • Institute of Child Health
    London
    WC1N 1EH

This information has not yet been provided by the study team. You'll have an opportunity to discuss any risks and benefits that may be associated with this study prior to consenting to taking part.

Prof Adrian Thrasher



The study is sponsored by Genethon (France) and funded by Genethon (France).



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What will you do next?
Read full details for Trial ID: ISRCTN46087965

Or CPMS 7314

Last updated 16 March 2018

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