Gene Therapy for Wiskott-Aldrich Syndrome

This is phase I/II protocol to evaluate the safety and efficacy of WAS gene transfer into hematopoietic stem/progenitor cells for the treatment of Wiskott Aldrich Syndrome.

Lead sponsor: Fondazione Telethon•1 locations•View source on ClinicalTrials.gov

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Trial snapshot

StatusCOMPLETED

PhasePHASE1/PHASE2

Enrollment8

Start dateApr 2010

Last updatedApr 4, 2025

Condition

Wiskott-Aldrich Syndrome (WAS)

Locations shown

Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)

Milan

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: April 4, 2025Data synced to Clareo: May 25, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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