Drugs / EFS-ADA lentiviral vector
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT02022696 | Dec 2013 | severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency | National Human Genome Research Institute (NHGRI) | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT05432310 | Jan 2023 → Nov 2027 expected | severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency | University of California, Los Angeles | Recruiting | No outcome recorded |
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | EFS-ADA lentiviral vector | “The EFS-ADA lentiviral vector with the human ADA complementary DNA (cDNA) will be used to transduce autologous CD34+ cells from Granulocyte Colony Stimulating Factor...” NCT05432310 ↗1“use of the EFS-ADA lentiviral vector to introduce the human adenosine deaminase (ADA) gene into the hematopoietic progenitors” NCT02022696 ↗ |
| Action | Restore | “During the follow-up phase, the investigators aim to determine whether the cells could engraft and produce mature cells that contain and express the corrected ADA gene in the...” NCT05432310 ↗ |
| Route | Intravenous | “re-infused into the patient through a peripheral vein” NCT02022696 ↗ |
| Target | ADA | “The EFS-ADA lentiviral vector with the human ADA complementary DNA (cDNA) will be used to transduce autologous CD34+ cells from Granulocyte Colony Stimulating Factor...” NCT05432310 ↗ |