Autologous Cryopreserved CD34+ Hematopoietic Cells Transduced With EFS-ADA Lentivirus for ADA SCID
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02999984|
Recruitment Status : Active, not recruiting
First Posted : December 21, 2016
Last Update Posted : February 5, 2018
|Condition or disease||Intervention/treatment||Phase|
|Severe Combined Immunodeficiency Due to ADA Deficiency||Biological: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells||Phase 1 Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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 Deficiency Severe Combined Immunodeficiency|
|Actual Study Start Date :||December 2016|
|Estimated Primary Completion Date :||October 2019|
|Estimated Study Completion Date :||June 2020|
Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells
Biological: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells
Infusion of OTL-101 after reduced intensity conditioning
Other Name: OTL-101
- Overall survival [ Time Frame: 12 months ]The proportion of subjects alive at the Month 12 visit.
- Event-free survival [ Time Frame: 24 months ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02999984
|United States, California|
|University of California, Los Angeles|
|Los Angeles, California, United States, 90095|
|Study Director:||Donald B. Kohn, MD||University of California, Los Angeles|