Gene Therapy for Severe Combined Immunodeficiency (SCID)
Trial Summary
What is the purpose of this trial?
The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blood (mPB) of ADA-deficient SCID infants and children following human ADA gene transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
Will I have to stop taking my current medications?
The trial protocol does not specify whether you need to stop taking your current medications. However, it mentions that participants likely to require treatment with drugs not permitted by the study protocol are excluded, suggesting some medications might not be allowed.
What data supports the effectiveness of the treatment EFS-ADA Lentiviral Vector, OTL-101 for Severe Combined Immunodeficiency (SCID)?
Gene therapy has shown to be effective in treating severe combined immunodeficiencies, including those caused by adenosine deaminase deficiency, by providing sustained correction of T-cell deficiencies. Improved safety and effectiveness have been achieved with newer lentiviral vectors, which are more potent and safer than earlier versions.12345
Is gene therapy for SCID generally safe in humans?
Gene therapy for SCID has shown a good safety profile in humans, with most adverse events being mild to moderate and not related to the therapy itself. However, there have been rare cases of severe complications, such as leukemia-like conditions, in some patients with X-linked SCID, which have been addressed by improving the therapy's design.26789
How is the treatment EFS-ADA Lentiviral Vector (OTL-101) unique for SCID?
The EFS-ADA Lentiviral Vector (OTL-101) is unique because it uses a self-inactivating lentiviral vector to deliver a corrected gene into the patient's own stem cells, which reduces the risk of causing cancer compared to older methods. This approach aims to provide a safer and more effective long-term correction of the immune deficiency in SCID patients.125910
Research Team
Satiro De Oliveira, MD
Principal Investigator
Assistant Professor
Eligibility Criteria
This trial is for infants and children with ADA-SCID, a genetic disorder that affects the immune system. Participants must be at least 30 days old, unable to undergo bone marrow transplant from a family donor, and able to follow study procedures. Pregnant individuals or those with certain organ dysfunctions or previous gene therapy are excluded.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Conditioning
Participants receive pharmacokinetically-adjusted busulfan reduced intensity conditioning prior to re-infusion of their gene-modified cells
Treatment
Autologous CD34+ cells transduced with EFS-ADA lentiviral vector are infused into participants
Follow-up
Participants are monitored for safety and effectiveness after treatment, including immune reconstitution and cessation of PEG-ADA ERT
Long-term follow-up
Participants may enroll in a long-term follow-up study to reach a total of 15 years post-gene therapy
Treatment Details
Interventions
- EFS-ADA Lentiviral Vector
EFS-ADA Lentiviral Vector is already approved in United States, European Union for the following indications:
- Adenosine Deaminase Severe Combined Immunodeficiency (ADA SCID)
- Adenosine Deaminase Severe Combined Immunodeficiency (ADA SCID)
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of California, Los Angeles
Lead Sponsor