Table 1.
Ongoing CAR-T and CAR-NK cell trials in Germany (listed on EU (clinicaltrialsregister.eu) and/or US Clinical Trials Register (clinicaltrials.gov)).
| EudraCT (NCT) |
Study title (acronym) | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Status | Antigen | Disease | Phase | Start | No. Patients | Age | Vector | IMP | Sponsor | |
| CD19-CAR-T cell products | ||||||||||
| 2018-000973-57(o) (NCT03630159) | Phase Ib study of tisagenlecleucel in combination with pembrolizumab in r/r diffuse large B-cell lymphoma (DLBCL) patients (PORTIA) | |||||||||
| O | CD19 | DLBCL | 1 | >18 | LV | Tisagenlecleucel | Novartis | |||
| 2017-002116-14 (NCT03876769) | A phase II trial of tisagenlecleucel in first-line high-risk (HR) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (B-ALL) who are minimal residual disease (MRD) positive at the end of consolidation (EOC) therapy (CASSIOPEIA) | |||||||||
| O | CD19 | B-ALL | 2 | 2019 | 9 | <18–64 | LV | Tisagenlecleucel | Novartis | |
| 2017-004385-94 (NCT03568461) | A phase II, single arm, multicenter open-label trial to determine the efficacy and safety of tisagenlecleucel (CTL019) in adult patients with refractory or relapsed follicular lymphoma (ELARA) | |||||||||
| O | CD19 | FL | 2 | 2019 | 12 | >18 | LV | Tisagenlecleucel | Novartis | |
| 2013-003205-25 (NCT02435849) | A phase II, single arm, multicenter trial to determine the efficacy and safety of CTL019 in pediatric patients with relapsed and refractory B-cell acute lymphoblastic leukemia (ELIANA) | |||||||||
| O | CD19 | B-ALL | 2 | 2016 | 6 | <18–64 | LV | Tisagenlecleucel | Novarts | |
| 2014-003060-20 (NCT02445248) | A phase II, single arm, multicenter trial to determine the efficacy and safety of CTL019 in adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) (JULIET) | |||||||||
| O | CD19 | DLBCL | 2 | 2016 | 8 | >18 | LV | Tisagenlecleucel | Novartis | |
| 2016-002966-29 (NCT03570892) | Tisagenlecleucel versus standard of care in adult patients with relapsed or refractory aggressive B-cell non-Hodgkin lymphoma: A randomized, open label, phase III trial (BELINDA) | |||||||||
| O | CD19 | B-NHL | 3 | 2019 | 37 | >18 | LV | Tisagenlecleucel | Novartis | |
| 2016-001991-31 (NCT03123939) | Phase IIIb study for relapsed/refractory pediatric/young adult acute lymphoblastic leukemia patients to be treated with CTL019 | |||||||||
| C | CD19 | B-ALL | 3 | 2017 | 5 | <18–64 | LV | Tisagenlecleucel | Novartis | |
| 2015-005007-86 (NCT02348216) | A phase I/II multicenter study evaluating the safety and efficacy of KTE-C19 in subjects with refractory aggressive Non-Hodgkin lymphoma (NHL) (ZUMA-1) | |||||||||
| O | CD19 | DLBCL, PMBCL, TFL | 1/2 | 2017 | 60 | 18–64 | RV | Axicabtagene ciloleucel | Kite | |
| 2017-002261-22 (NCT03391466) | A phase III, randomized, open-label study evaluating the efficacy of Axicabtagene Ciloleucel versus standard of care therapy in subjects with relapsed/refractory diffuse large B-cell lymphoma (ZUMA-7) | |||||||||
| O | CD19 | DLBCL | 3 | 2018 | 48 | >18 | RV | Axicabtagene ciloleucel | Kite | |
| 2015-005010-30 (NCT02625480) | A phase I/II multicenter study evaluating the safety and efficacy of KTE-X19 in pediatric and adolescent subjects with relapsed/refractory B-precursor acute lymphoblastic leukemia or relapsed/refractory B-cell non-Hodgkin lymphoma (ZUMA-4) | |||||||||
| O | CD19 | B-ALL, B-NHL | 1/2 | 2020 | 10 | <18–64 | RV | Brexucabtagene autoleucel | Kite | |
| 2018-001923-38 (NCT03624036) | Phase I/II multicenter study evaluating the safety and efficacy of KTE-X19 in adult subjects with relapsed/refractory chronic lymphocytic leukemia (ZUMA-8) | |||||||||
| T | CD19 | B-CLL | 1/2 | 2019 | 15 | >18 | RV | Brexucabtagene autoleucel | Kite | |
| 2015-005009-35 (NCT02614066) | A phase I/II multicenter study evaluating the safety and efficacy of KTE-X19 in adult subjects with relapsed/refractory B-precursor acute lymphoblastic leukemia (r/r ALL) (ZUMA-3) | |||||||||
| O | CD19 | B-ALL | 1/2 | 2018 | 5 | 18–64 | RV | Brexucabtagene autoleucel | Kite | |
| 2015-005008-27 (NCT02601313) | A phase II multicenter study evaluating the efficacy of KTE-X19 in subjects with relapsed/refractory mantle cell lymphoma (r/r MCL) (ZUMA-2) | |||||||||
| O | CD19 | MCL | 2 | 2018 | 20 | >18 | RV | Brexucabtagene autoleucel | Kite | |
| 2018-001246-34 (NCT03743246) | A phase I/II, open-label, single arm, multicohort, multicenter trial to evaluate the safety and efficacy of JCAR017 in pediatric subjects with relapsed/refractory B-ALL and B-NHL (TRANSCEND PEDALL) | |||||||||
| O | CD19 | B-ALL, B-NHL | 1/2 | 2019 | 10 | <18 | LV | Lisocabtagene maraleucel | Celgene | |
| 2019-004081-18(o) (NCT04245839) | A phase II, open-label, single-arm, multicohort, multicenter trial to evaluate the efficacy and safety of JCAR017 in adult subjects with relapsed or refractory indolent B-cell non-Hodgkin lymphoma (NHL) (TRANSCEND FL) | |||||||||
| O | CD19 | FL | 2 | >18 | LV | Lisocabtagene maraleucel | Celgene | |||
| 2017-000106-38 (NCT03484702) | A phase II, single-arm, multi-cohort, multicenter trial to determine the efficacy and safety of JCAR017 in adult subjects with aggressive B-cell non-Hodgkin lymphoma (TRANSCEND WORLD) | |||||||||
| O | CD19 | B-NHL | 2 | 2018 | 16 | >18 | LV | Lisocabtagene maraleucel | Celgene | |
| 2018-000929-32(o) (NCT03575351) | A global randomized multicenter phase III trial of JCAR017 compared to standard of care in adult subjects with high-risk, second-line, transplant-eligible relapsed or refractory aggressive B-cell non-Hodgkin lymphomas (TRANSFORM). | |||||||||
| O | CD19 | B-NHL | 3 | >18 | LV | Lisocabtagene maraleucel | Celgene | |||
| 2017-002848-32 (NCT03853616) | A phase I/II safety, dose finding and feasibility trial of MB-CART19.1 in patients with relapsed or refractory CD19 positive B-cell malignancies. | |||||||||
| O | CD19 | B-ALL, B-NHL, B-CLL | 1/2 | 2018 | 48 | <18, >18 | MB-CART19.1 | Miltenyi | ||
| 2018-003916-38 (NCT04035434) | A phase I/II dose escalation and cohort expansion study of the safety and efficacy of allogeneic CRISPR-Cas9-engineered T cells (CTX110) in subjects with relapsed or refractory B-cell malignancies | |||||||||
| O | CD19 | B-NHL | 1/2 | 2019 | 16 | >18 | CTX110 | CRISPR | ||
| 2016-004808-60 (NCT03676504) | Treatment of patients with relapsed or refractory CD19+ lymphoid disease with T-lymphocytes transduced by RV-SFG.CD19.CD28.4-1BBzeta retroviral vector—a unicenter Phase I /II clinical trial | |||||||||
| O | CD19 | B-ALL, B-NHL | 1/2 | 2018 | 48 | <18–64 | RV | CD19.CAR T cells | UK HD | |
| 2007-007612-29 (NCT01195480) | Immunotherapy with CD19ζ chimeric antigen receptor gene-modified EBV-specific CTLs after stem cell transplant in children with high-risk acute lymphoblastic leukemia (CD19TPALL) | |||||||||
| T(*) | CD19 | B-ALL | 1 | 2013 | 12 | <18 | RV | CD19 transduced EBV-CTL | UCL | |
| CD19/CD20 -Dual-CAR-T cell products | ||||||||||
| 2018-001253-27(o) (NCT03870945) | A phase I/II safety, dose finding and feasibility trial of MB-CART2019.1 in patients with relapsed or resistant CD20 and CD19 positive B-NHL | |||||||||
| O | CD19, CD20 | B-NHL | 1/2 | >18 | LV | MB-CART2019.1 | Miltenyi | |||
| CD20 -CAR-T cell products | ||||||||||
| 2017-000120-10(o) (NCT03893019) | Multicenter phase I trial of MB-CART20.1 for the treatment of patients with metastatic melanoma | |||||||||
| O | CD20 | Melanoma | 1 | >18 | LV | MB-CART20.1 | Miltenyi | |||
| 2017-000121-12(o) (NCT03664635) | A phase I/II safety, dose finding and feasibility trial of MB-CART20.1 in patients with relapsed or resistant CD20-positive B-NHL | |||||||||
| O | CD20 | B-NHL | 1/2 | >18 | LV | MB-CART20.1 | Miltenyi | |||
| CD123-CAR-T cell products | ||||||||||
| 2019-001339-30(o) (NCT04230265) | Multicenter, open-label, adaptive design phase I trial with genetically modified T cells carrying universal chimeric antigen receptors (UniCAR02-T) in combination with CD123 Target Module (TM123) for the treatment of patients with hematologic and lymphatic malignancies positive for CD123 | |||||||||
| O | CD123 | AML, B-ALL, BPDCN | 1 | >18 | LV | UniCAR02-T + TM123 | Cellex | |||
| BCMA-CAR-T cell products | ||||||||||
| 2018-000264-28 (NCT03601078) | A phase II, multi-cohort, open-label, multicenter study to evaluate the efficacy, and safety of bb2121 in subjects with relapsed and refractory multiple myeloma and in subjects with clinical high-risk multiple myeloma (KarMMa-2) | |||||||||
| O | BCMA | MM | 2 | 2019 | 10 | >18 | LV | Idecabtagene vicleucel | Celgene | |
| 2017-002245-29 (NCT03361748) | A phase 2, multicenter study to determine the efficacy and safety of bb2121 in subjects with relapsed and refractory multiple myeloma (KarMMa) | |||||||||
| O | BCMA | MM | 2 | 2018 | 15 | >18 | LV | Idecabtagene vicleucel | Celgene | |
| 2018-001023-38 (NCT03651128) | A phase III, multicenter, randomized, open-label study to compare the efficacy and safety of BB2121 versus standard regimens in subjects with relapsed and refractory multiple myeloma (RRMM) (KarMMa-3) | |||||||||
| O | BCMA | MM | 3 | 2019 | 40 | >18 | LV | Idecabtagene vicleucel | Celgene | |
| 2018-004124-10(o) (NCT04133636) | A phase 2, multicohort open-label study of JNJ-68284528, a chimeric antigen receptor T cell (CAR-T) therapy directed against BCMA in subjects with multiple myeloma (CARTITUDE-2) | |||||||||
| O | BCMA | MM | 2 | >18 | LV | JNJ-68284528 | Janssen | |||
| SLAMF7-CAR-T cell product | ||||||||||
| 2019-001264-30 (NCT04499339) | A phase I/IIa clinical trial to assess feasibility, safety and antitumor activity of autologous SLAMF7 CAR-T cells in multiple myeloma | |||||||||
| O | SLAMF7 | MM | 1/2 | 2020 | 12 | >18 | SB | SLAMF7 CAR-T | UK Würzburg | |
| CLDN6-CAR-T cell product | ||||||||||
| 2019-004323-20 (NCT04503278) | Phase I/IIa, first-in-human, open-label, dose-escalation trial with expansion cohorts to evaluate safety, and preliminary efficacy of CLDN6-CAR-T with or without CLDN6 RNA-LPX in patients with CLDN6-positive relapsed or refractory advanced solid tumors | |||||||||
| O | Claudin-6 | Solid tumor | 1/2 | 2020 | 18 | >18 | RV | CLDN6-CAR-T | BioNTech | |
| HER2-CAR-NK-cell product | ||||||||||
| 2016-000225-39(o) (NCT03383978) | Multicenter, open label, phase I study of intracranial injection of NK-92/5.28.z cells in patients with recurrent HER2-positive glioblastoma (CAR2BRAIN) | |||||||||
| O | Her2 | Glioblastoma | 1 | >18 | LV | NK-92/5.28.z cells | KGU | |||
| Long-term follow-up studies | ||||||||||
| 2017-001465-24 (NCT03435796) | Long-term follow-up protocol for subjects treated with gene-modified T cells. | |||||||||
| O | 2018 | 55 | Celgene | |||||||
| 2014-001673-14 (NCT02445222) | Long-term follow-up of patients exposed to lentiviral-based CAR T cell therapy (PAVO) | |||||||||
| O | 2016 | 55 | Novartis | |||||||
Trials are subdivided depending on the target antigen, investigational medicinal product (IMP), phase and start date of study (in chronological order). In addition, the study title, study acronym, status of the study in Germany, disease for which the drug is tested, intended number of trial participants in Germany, age of participants, vector which was used to transduce cells, and the sponsor of the study is listed.
Status: O ongoing, T terminated, C completed. Vector: LV lentiviral vector, RV retroviral vector, SB sleeping beauty. Disease: ALL acute lymphoblastic leukemia, BPDCN blastic plasmacytoid dendritic cell neoplasm, CLL chronic lymphocytic leukemia, DLBCL diffuse large B-cell lymphoma, FL follicular lymphoma, IMP investigational medicinal product, MCL mantel cell lymphoma, MM multiple myeloma, NHL Non-Hodgkin Lymphoma, PMBCL primary mediastinal B-cell lymphoma, TFL transformed follicular lymphoma, r/r relapsed/refractory. Sponsor: Celgene Celgene Corporation, Cellex Cellex Patient Treatment GmbH, KGU Johann Wolfgang Goethe University Hospital, Kite Kite Pharma, Inc., Miltenyi Miltenyi Biomedicine GmbH and Miltenyi Biotec B.V. & Co. KG Novartis, Novartis Pharma.
(o)Complete data set derived from clinicaltrials.gov.
(*)Indicated data derived from clinicaltirals.gov.