Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With EFS Lentiviral Vector Encoding for the Human ADA Gene
|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: NCT01852071|
Recruitment Status : Completed
First Posted : May 13, 2013
Last Update Posted : April 13, 2020
|Condition or disease||Intervention/treatment||Phase|
|ADA-SCID||Genetic: Infusion of autologous EFS-ADA LV CD34+ (OTL-101) Drug: busulfan Drug: PEG-ADA ERT||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Autologous Transplantation of Bone Marrow CD34+ Stem/Progenitor Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID)|
|Actual Study Start Date :||August 2, 2013|
|Actual Primary Completion Date :||August 27, 2018|
|Actual Study Completion Date :||August 27, 2018|
Experimental: Gene Therapy
Infusion of autologous EFS-ADA Lentiviral (LV) CD34+ cells
Genetic: Infusion of autologous EFS-ADA LV CD34+ (OTL-101)
autologous EFS-ADA LV CD34+ cells (OTL-101) are infused intravenously
Other Name: OTL-101
Busulfan is used for non-myoablative conditioning
Drug: PEG-ADA ERT
PEG-ADA ERT is discontinued at Day +30 (-3/+15 days) after successful engraftment
- Assess safety by recording clinical toxicities. [ Time Frame: 2 years ]Safety will be assessed by recording clinical adverse events.
- Assess safety by determining absence or presence of exposure to replication-competent lentivirus (RCL) [ Time Frame: 2 years ]Replication-competent lentivirus exposure will be assessed by polymerase chain reaction (PCR) to VSV-G protein.
- Assess safety by evaluating the absence or development of monoclonal expansion or leukoproliferative complications from vector insertional effects [ Time Frame: 2 years ]Monoclonal expansion of blood cells by vector-mediated activity will be assessed by nrLAM-PCR
- Overall survival [ Time Frame: 1 year ]Overall survival will be assessed as the proportion of subjects alive.
- Event-free survival [ Time Frame: 1 year ]Event-free survival will be assessed by determining the numbers of subjects who remain alive with adequate immune reconstitution and do not need an allogeneic hematopoietic stem cell transplant or re-institution of enzyme replacement therapy.
- Overall survival [ Time Frame: 2 years ]Overall survival will be assessed as the proportion of subjects alive.
- Event-free survival [ Time Frame: 2 years ]Event-free survival will be assessed by determining the numbers of subjects who remain alive with adequate immune reconstitution and do not need an allogeneic hematopoietic stem cell transplant or re-institution of enzyme replacement therapy.
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): NCT01852071
|United States, California|
|Mattel Children's Hospital, UCLA|
|Los Angeles, California, United States, 90095|
|United States, Maryland|
|Mark O. Hatfield Clinical Research Center, NIH|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||Donald B Kohn, MD||University of California, Los Angeles|