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Clinical trial

A Single Arm, Open-label Clinical Trial of Hematopoietic Stem Cell Gene Therapy With Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Encoding WAS cDNA in Subjects With Wiskott-Aldrich Syndrome (WAS)

NCT03837483 is registered in ClinicalTrials.gov with Fondazione Telethon as lead sponsor.

NCT NCT03837483
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Registry record

As filed in ClinicalTrials.gov.

Title
A Single Arm, Open-label Clinical Trial of Hematopoietic Stem Cell Gene Therapy With Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Encoding WAS cDNA in Subjects With Wiskott-Aldrich Syndrome (WAS)
Lead sponsor
Fondazione Telethon
Phase
Phase 3
Overall status
Active, not recruiting
Start date
January 21, 2019
Completion date
September 30, 2027
Enrollment
10 (actual)
Sites
2

Conditions

As the registry lists them; a condition with its own page is linked.

wiskott-aldrich syndrome