NCT03837483

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

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 Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Fondazione Telethon · Academic / Other
• Sex: All
• Age: 65 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Wiskott-Aldrich Syndrome

Interventions

Type	Name	Description
GENETIC	OTL-103	Autologous hematopoietic stem cells collected from mobilized peripheral blood transduced ex vivo with a lentiviral vector encoding the WAS cDNA

Timeline

• Start date: 2019-01-21
• Primary completion: 2024-03-18
• Completion: 2027-09-01
• First posted: 2019-02-12
• Last updated: 2025-09-29

Locations

2 sites across 2 countries: United States, Italy

Regulatory

• FDA-regulated drug study