Clinical Trial: Autologous Cryopreserved CD34+ Hematopoietic Cells Transduced With EFS-ADA Lentivirus for ADA SCID
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With EFS Lentiviral Vector Encoding for Human ADA Gene in Subjects With ADA Deficienc
Brief Summary: This is a prospective, non-randomized, single-cohort, longitudinal, single-center, clinical study designed to assess the efficacy and safety of a cryopreserved formulation of OTL-101 (autologous CD34+ hematopoietic stem/progenitor cells transduced ex vivo with EFS LV encoding for the human ADA gene) administered to ADA-SCID subjects between the ages of 30 days and 17 years of age, who are not eligible for an HLA-matched sibling/family donor and meeting the inclusion/exclusion criteria. The OTL-101 product will be infused after a minimal interval of at least 24 hours following the completion of reduced intensity conditioning. For subjects who have successfully received the OTL-101 product, PEG-ADA ERT will be discontinued at Day+30 (+/-3) after the transplant. After their discharge from hospital, the subjects will be seen at regular intervals to review their history, perform examinations and draw blood samples to assess immunity and safety.
Detailed Summary:
This is a prospective, non-randomized, single-cohort, longitudinal, single-center, clinical study designed to assess the efficacy and safety of a cryopreserved formulation of OTL-101 (autologous CD34+ hematopoietic stem cells transduced ex vivo with EFS LV encoding for the human ADA gene) administered to ADA-SCID subjects between the ages of 30 days and 17 years, who are not eligible for an HLA-matched sibling/family donor and meeting the inclusion/exclusion criteria.
The aim of this study is to assess the success of treatment for overall survival and event free survival.
Eligible subjects will be hospitalized to undergo the harvesting of autologous CD34+ cells. To enable the release of the cell product for infusion, the product must meet various quality control criteria for safety, identity, viability, purity and potency. If OTL-101 meets the acceptance criteria and is released, the subjects will be readmitted for conditioning prior to infusion of OTL-101.
For subjects who have successfully received the OTL-101 product, PEG-ADA ERT will be discontinued at Day+30 (+/-3) after the transplant. After their discharge from hospital, the subjects will be seen at regular intervals to review their history, perform examinations and draw blood samples at Months 1, 3, 6, 9, 12, 18, and 24. Any medically-indicated interventions will be determined at these visits. After Month 24 visit, the subjects will have completed the study and may enter a long term registry.
Sponsor: University of California, Los Angeles
Current Primary Outcome: Overall survival [ Time Frame: 12 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Event-free survival [ Time Frame: 24 months ]
Original Secondary Outcome: Same as current
Information By: University of California, Los Angeles
Dates:
Date Received: December 19, 2016
Date Started: December 2016
Date Completion: June 2020
Last Updated: January 4, 2017
Last Verified: January 2017