Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID) Clinical Trial
Official title:
Gene Transfer for Adenosine Deaminase-severe Combined Immunodeficiency (ADA-SCID) Using an Improved Self-inactivating Lentiviral Vector (TYF-ADA)
Gene transfer for ADA-SCID using an improved lentiviral vector (TYF-ADA)
Status | Recruiting |
Enrollment | 10 |
Est. completion date | December 31, 2021 |
Est. primary completion date | December 31, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Month and older |
Eligibility |
Inclusion Criteria: - Diagnosis of classical ADA-SCID based on: - A proven defective adenosine deaminase (ADA) gene as defined by direct sequencing of patient DNA. - T-cell immune deficiency defined as one or more of the following: CD3+ autologous T cells < 300/ul, or less than 50% of normal value for in vitro mitogen stimulation, or absent proliferation in vitro to antigens. - With severe infections, including but not limited to: pneumonitis; protracted diarrhea requiring total parenteral nutrition; infection with herpes viruses or adenovirus or fungus; disseminated BCG infection. - No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia of children. - No prior allogeneic stem cell transplantation. - Life expectancy = 2 months. - Negative for HIV infection. - Written, informed consent obtained prior to any study-specific procedures. Exclusion Criteria: - None |
Country | Name | City | State |
---|---|---|---|
China | Shenzhen Geno-immune Medical Institute | Shenzhen | Guangdong |
Lead Sponsor | Collaborator |
---|---|
Shenzhen Geno-Immune Medical Institute |
China,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Overall survival up to a year | Patient will be monitored for overall health condition, including immune cell assessments, blood biochemistry and metabolitic activities, metabolic detoxification, gene-modified cell percentage and vector copy number (VCN) in the blood, and continued follow-up for 15 years. | 15 years | |
Secondary | 1. Success of immune reconstitution | Immunological and metabolic values including all leukocyte counts (ALC), T, B and NK cell counts (CD3, CD4, CD8, CD19, CD56), T cell TREC levels, T cell repertoire diversity, PHA proliferation rate, immunoglobulins and dATP levels will be measured. | 12 month | |
Secondary | 2. Change of infection status | Immune recovery associated with reduction of infection episodes and frequencies, including viral, fungal and bacterial infections will be documented. | 12 month |