Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
NCT ID: NCT02333760
Last Updated: 2021-06-03
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
10 participants
INTERVENTIONAL
2014-09-30
2032-10-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
OTHER
NONE
Interventions
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Autologous CD34+ cells transduced with WASP lentiviral vector
Follow up of ex vivo gene therapy transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WASP gene
Eligibility Criteria
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Inclusion Criteria
* Parents, guardians or patient signed informed consent, guardians or patient signed informed consent
Exclusion Criteria
MALE
No
Sponsors
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Genethon
OTHER
Responsible Party
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Locations
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Hopital Necker - Enfants Malades
Paris, , France
UCL Institute of Child Health
London, , United Kingdom
Countries
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References
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Magnani A, Semeraro M, Adam F, Booth C, Dupre L, Morris EC, Gabrion A, Roudaut C, Borgel D, Toubert A, Clave E, Abdo C, Gorochov G, Petermann R, Guiot M, Miyara M, Moshous D, Magrin E, Denis A, Suarez F, Lagresle C, Roche AM, Everett J, Trinquand A, Guisset M, Bayford JX, Hacein-Bey-Abina S, Kauskot A, Elfeky R, Rivat C, Abbas S, Gaspar HB, Macintyre E, Picard C, Bushman FD, Galy A, Fischer A, Six E, Thrasher AJ, Cavazzana M. Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome. Nat Med. 2022 Jan;28(1):71-80. doi: 10.1038/s41591-021-01641-x. Epub 2022 Jan 24.
Other Identifiers
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GNT-WAS-03
Identifier Type: -
Identifier Source: org_study_id
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