Trial Profile

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)

Status: Active, no longer recruiting

Phase of Trial: Phase III

Latest Information Update: 02 Feb 2024

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At a glance

Drugs OTL-103 (Primary) ; Busulfan; Fludarabine; Lenograstim; Plerixafor; Rituximab
Indications Wiskott-Aldrich syndrome
Focus Pharmacodynamics; Registrational; Therapeutic Use
Sponsors Orchard Therapeutics

02 Feb 2023 Planned End Date changed from 1 Jan 2024 to 1 Sep 2027.
02 Feb 2023 Planned primary completion date changed from 1 Jul 2023 to 1 Sep 2025.
27 Sep 2022 Status changed from recruiting to active, no longer recruiting.

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