NCT03837483View on ClinicalTrials.gov

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

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

10

Participants

Timeline

Start Date

January 21, 2019

Primary Completion Date

March 18, 2024

Study Completion Date

September 30, 2027

Conditions
Wiskott-Aldrich Syndrome
Interventions
GENETIC

OTL-103

Autologous hematopoietic stem cells collected from mobilized peripheral blood transduced ex vivo with a lentiviral vector encoding the WAS cDNA

Trial Locations (2)

20132

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

30329

Children's Healthcare of Atlanta, Inc, Atlanta

All Listed Sponsors
collaborator

Ospedale San Raffaele

OTHER

lead

Fondazione Telethon

OTHER