A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome

NCTID	NCT03837483 (View at clinicaltrials.gov)
Description	This is an open-label, single arm study to evaluate the cryopreserved formulation of OTL-103 Gene Therapy. OTL-103 consists of autologous CD34+ hematopoietic stem cells in which the gene encoding for the Wiskott-Aldrich Syndrome is introduced by means of a third generation lentiviral vector. (Show More)
Indication	Wiskott-Aldrich Syndrome
Compound Name	Etuvetidigene autotemcel (formerly OTL-103)
Sponsor	Fondazione Telethon
Funder Type	Other
Status	Active not recruiting
Enrollment Count	10

Target Gene/Variant	WAS
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	LV
Editor Type
Dose 1	Transduced CD34+ cells (4.4 - 14.5 x 10^6 cells/kg)
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States,Italy

Has US IND	True
Recent Updates	Telethon intends to seek regulatory approval in Europe