A Clinical Study to Enable Process Validation of Commercial Grade OTL-101

Severe Combined Immunodeficiency Due to ADA Deficiency Clinical Trial

Official title: A Single Arm, Open Label Clinical Study to Enable Process Validation of Commercial Grade Ex Vivo Hematopoietic Stem Cell Gene Therapy (OTL-101) in Subjects With Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID)

Status Withdrawn

Clinical Trial Summary

The purpose of the current study is to treat at least 3 ADA-SCID patients with OTL-101 prepared by the commercial manufacturing process.

Clinical Trial Description

The safety and efficacy of OTL-101 for the treatment of patients with ADA- SCID have been established in previous clinical trials. The purpose of the current study is to treat at least 3 ADA-SCID patients with OTL-101 prepared by the commercial manufacturing process in order to facilitate collection of data necessary for final manufacturing.

Assessments will focus on monitoring safety and engraftment, through the evaluation of parameters describing immunological recovery, ADA enzyme activity and persistence of gene marking (VCN) at 6 months and 12 months. After completion of 12 months of follow-up on the current study protocol, subjects will be enrolled in an observational long-term follow-up study, in order to monitor the long-term safety of treatment with OTL-101. ;

Related Conditions & MeSH terms

• Immunologic Deficiency Syndromes
• Severe Combined Immunodeficiency
• Severe Combined Immunodeficiency Due to ADA Deficiency

• NCT number: NCT04140539
• Study type: Interventional
• Source: University of California, Los Angeles
• Status: Withdrawn
• Phase: Phase 2/Phase 3
• Start date: October 15, 2019
• Completion date: August 30, 2021