Table 1.
Current Gene Therapy Trials using Suicide genes, Immunostimulation, and RNAi for Hematologic Malignancies
| Gene Therapy Application | Trial Identifier | Title | Phase | Conditions | Intervention | Expected Completion |
|---|---|---|---|---|---|---|
| RNAi | NCT01961063 | Gene Therapy After Frontline Chemotherapy in Treating Patients With AIDS-Related Non-Hodgkin Lymphoma | 1 | AIDS-related Diffuse Large Cell Lymphoma|AIDS-related Diffuse Mixed Cell Lymphoma|AIDS-related Diffuse Small Cleaved Cell Lymphoma|AIDS-related Immunoblastic Large Cell Lymphoma|AIDS-related Lymphoblastic Lymphoma|AIDS- related Small Noncleaved Cell Lymphoma|HIV Infection | Lentivirus vector rHIV7-shI-TAR- CCR5RZ-transduced hematopoietic progenitor cells | Jun 2031 |
| NCT02378922 | Autologous Transplantation and Stem Cell Based-Gene Therapy With LVsh5/C46 (CAL-1), a Dual Anti-HIV Lentiviral Vector, for the Treatment of HIV-Associated Lymphoma | 1 | AIDS-Related Hodgkin Lymphoma; AIDS-Related Non-Hodgkin Lymphoma | Gene-modified hematopoietic stem cells with low CCR5 via RNAi and inhibititory for HIV-1 fusion via C46 antiviral peptide | n/a | |
| NCT02493829 | B7.1/IL-2 Leukaemia Cell Vaccine for Non-Transplant AML RFUSIN2-AML2 (NTX) | 1 | AML | AML Cell Vaccine | Sep 2021 | |
| Suicide Gene Therapy for GVHD | NCT01875237 | A Phase 1/2 Trial Evaluating Treatment of Emergent Graft Versus Host Disease (GvHD) With AP1903 After Planned Donor Infusions (DLIs) of T-cells Genetically Modified With the iCasp9 Suicide Gene in Patients With Hematologic Malignancies | 1|2 | Leukemia; Myeloma; Myeloproliferative Diseases | Donor Lymphocyte Infusion (DLI)|Drug: AP1903 | n/a |
| NCT00710892 | CASPALLO: A Phase I Study Evaluating the Use of Allodepleted T Cells Transduced With Inducible Caspase 9 Suicide Gene After Haploidentical Stem Cell Transplantation | 1 | ALL; NHL; MDS; CML | Alloreactive T cell depletion with RFT5- dgA; iCasp9: AP1903 | Jul 2026 | |
| NCT01744223 | A Phase 1/2 Dose Escalation Study Evaluating Safety and Feasibility of BPX-501 T Cells After Partially Mismatched, Related, T Cell-Depleted HSCT (Hematopoietic Stem Cell Transplant) | 1|2 | AML; ALL; Lymphoma | iCasp9: BPX-501 cells and AP1903 | Dec 2018 | |
| NCT02487459 | A Phase I/II Safety Study of Planned BPX-501 T Cell Infusion After Partially Mismatched, Related, TCR αβ+T Cell Depleted HSCT in Adults With Advanced Hematologic Malignances at High Risk for Relapse | 1|2 | High-risk ALL in 1st or subsequent CR; High-risk AML in 1st or subsequent CR; CML refractory to at least 2 TKIs or resistant mutations or progression to blast phase CML; Intermediate/high risk MDS; HL or NHL:, relapsed or refractory; other high-risk hematologic malignancies otherwise eligible for stem cell transplantation without an HLA matched donor or in need of a fast transplant | iCasp9: BPX-501 cells and AP1903 | ||
| Immune modulation | NCT01773395 | Randomized Placebo-controlled Phase II Trial of Irradiated, Adenovirus Vector Transferred GM-CSF Secreting Autologous Leukemia Cell Vaccination (GVAX) Versus Placebo Vaccination in Patients With Advanced MDS/AML After Allogeneic Hematopoietic Stem Cell Transplantation | 2 | AML; MDS-RAEB not in remission | Adenovirus Vector Transferred GM-CSF Secreting Autologous Leukemia Cell Vaccination (GVAX) | Jan 2033 |
| Transplant related infection | NCT02276820 | Most Closely Human Leukocyte Antigen (HLA)-Matched Adenovirus-specific T Lymphocytes (Viralym-A) | 1 | Adenovirus Infection | Viralym-A | Dec 2017 |
| NCT02108522 | Multivirus-specific T Cells for the Treatment of Virus Infections After Stem Cell Transplant | 1 | Infection | Multivirus Specific T cells | Mar 2018 |