Trial Profile

A PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME

Status: Completed

Phase of Trial: Phase I/II

Latest Information Update: 09 Apr 2025

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At a glance

Drugs OTL-103 (Primary) ; Antithymocyte globulin; Busulfan; Fludarabine; Lenograstim; Plerixafor; Rituximab
Indications Wiskott-Aldrich syndrome
Focus Adverse reactions; First in man; Pharmacodynamics; Registrational; Therapeutic Use
Acronyms TIGET-WAS
Sponsors GlaxoSmithKline; GSK; Orchard Therapeutics

Most Recent Events

29 Jan 2024 Status changed from active, no longer recruiting to completed.
02 Feb 2023 Planned End Date changed from 11 Sep 2025 to 11 Sep 2030.
10 Dec 2019 Updated results presented at the 61st Annual Meeting and Exposition of the American Society of Hematology

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