Klinische Studien im Bereich innovativer Zelltherapien werden in enger Zusammenarbeit mit der Klinik für Hämatologie durchgeführt. Weiters pflegt unsere Transplantationsimmunologie eine enge Zusammenarbeit mit verschiedenen Kliniken der MedUni Wien, aber auch anderen Universitätskliniken in Österreich.
This is a first-in-human study to evaluate the feasibility, safety and preliminary antitumor efficacy of autologous T cells genetically engineered with a CD19-specific chimeric antigen receptor (CAR) and manufactured with a new process. CAR-T cells will be investigated in combination with ibrutinib in chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) and as single agent in diffuse large B-cell lymphoma (DLBCL) and in adult acute lymphoblastic leukemia (ALL).
The purpose of this study is to determine the clinical benefit and characterize the safety profile of tabelecleucel for the treatment of Epstein-Barr virus-associated post-transplant lymphoproliferative disease (EBV+ PTLD) in the setting of (1) solid organ transplant (SOT) after failure of rituximab and rituximab plus chemotherapy or (2) allogeneic hematopoietic cell transplant (HCT) after failure of rituximab. This is a multicenter, open-label, phase 3 study to assess the efficacy and safety of tabelecleucel for the treatment of EBV+ PTLD in the setting of SOT after failure of rituximab and rituximab plus chemotherapy (SOT cohort) or HCT after failure of rituximab (HCT cohort).
ALLELE Study; ATA129-EBV-302; ATARA
The purpose of this study is to assess the efficacy and safety of tabelecleucel in participants with Epstein-Barr virus (EBV) associated diseases. This is a multicenter, multicohort, open label, single-arm, Phase 2 study to assess the efficacy and safety of tabelecleucel for the treatment of EBV-associated diseases in participants who are newly diagnosed or relapsed/refractory to prior treatment. Newly diagnosed or relapsed/refractory participants will be enrolled in one of the following cohorts:
- EBV+ lymphoproliferative disease (LPD) in the setting of primary immunodeficiency (PID) (PID LPD)
- EBV+ LPD in the setting of acquired (non-congenital) immunodeficiency (AID) (AID LPD)
- EBV+ posttransplant lymphoproliferative disorder involving the central nervous system (CNS PTLD)
- EBV+ PTLD where standard first line therapy (rituximab or chemotherapy) is not appropriate, including CD20 negative disease
- EBV+ sarcomas, including leiomyosarcoma (LMS)
- Chronic active EBV (CAEBV) and EBV+ hemophagocytic lymphohistiocytosis (HLH) (CAEBV/HLH cohort)
Das Ziel der APN401-104 Studie ist es, die empfohlene Phase 2 Dosis (RP2D) von APN401 zu ermitteln, sowie die Sicherheit und Veträglichkeit von APN401 bei Patienten mit fortgeschrittenen soliden Tumoren, bei denen etablierte Standardtherapien nicht mehr wirksam sind, weiter zu evaluieren.
APN401 ist eine Suspension autologer mononukleärer Zellen des peripheren Blutes (PBMCs), die transient mit einer siRNA zur Reduzierung des Proteins Cbl-b transfiziert wurden. Dies ist eine offene, multizentrische Phase Ib Dosis-Eskalationsstudie. Das Ziel dieser Studie ist es, die Sicherheit und Verträglichkeit von APN401 weiter zu untersuchen und die RP2D zu bestimmen. Darüber hinaus sollen erste vorläufige Daten zur klinischen Wirksamkeit der Behandlung mit APN401 gesammelt werden.
(PBMC-basierende Leukozyten- Immuntherapie in einer nationalen Dosisfindung (Studie) für RP2D, eine offene multizentrische Studie)
This is a global Phase 2, open-label, single-arm, multicohort, multicenter study to evaluate efficacy and safety of JCAR017 in adult subjects with r/r FL or MZL. The study will be conducted in compliance with the International Council on Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory requirements.
This study is divided into three periods: Pretreatment, which consists of screening assessments, leukapheresis and the Pretreatment evaluation; Treatment, which starts with the administration of lymphodepleting (LD) chemotherapy and continues through JCAR017 administration at Day 1 with follow-up through Day 29; Posttreatment, which includes follow-up assessments for disease status and safety for 2 years.
This is a pivotal Phase II randomised, multi-centre, open-label study to evaluate the efficacy and safety of MB-CART2019.1 compared to standard of care therapy in participants with relapsed/refractory diffuse large B-cell lymphoma, who are not eligible for high-dose chemotherapy and autologous stem cell transplantation.
This study should determine superiority of MB-CART2019.1 treatment compared to SoC therapy with R-GemOx (rituximab, gemcitabine and oxaliplatin) with respect to progression-free survival in second-line therapy in participants with R-R DLBCL, who are non-eligible for high-dose chemotherapy and autologous stem cell transplantation. MB-CART2019.1 is designed to effectively target malignant B cells in patients suffering from late stage haematological B-cell malignancies. MB-CART2019.1 consists of autologous cluster of differentiation CD20/CD19 chimeric antigen receptor (CAR) transduced CD4/CD8 enriched T cells, derived from a leukapheresis and processed by using the CliniMACS Prodigy®. Patients who are suitable for this study will be randomized 1:1 to either MB-CART2019.1 or SoC. Both treatment arms are unblinded. MB-CART2019.1 arm: Single infusion of fresh formulation of 2.5 × 10^6 CAR-transduced autologous T cells. IMP is only to be administered after a lymphodepleting chemotherapy with fludarabine and cyclophosphamide. For MB-CART2019.1 production, patients will undergo a leukapheresis. SoC arm: R-GemOx (8 cycles of 14 days each) or (10% of SoC arm) BR (Bendamustine/Rituximab) + polatuzumab vedotin (6 cycles of 21 days each).The duration of the active part of the study for each individual participant from screening to the end of the 1-year follow-up after infusion of MB-CART2019.1 cells (experimental arm) or the start of SoC therapy (comparator arm) will be approximately 55 weeks. The LTFU in Year 2 after infusion of MB-CART2019.1 cells or the start of treatment in the comparator arm will not be part of the active part of the clinical study and will be reported separately.
(R-R DLBCL) (MB-CART2019.1; Miltenyi Biotec)
A Phase 3 Randomized Study Comparing Bortezomib, Lenalidomide and Dexamethasone (VRd) followed by Ciltacabtagene Autoleucel, a CAR-T Therapy Directed Against BCMA vs. Bortezomib, Lenalidomide, and Dexamethasone (VRd) followed by lenalidomide and Dexamethasone (Rd) Therapy in Participants with Newly Diagn. Multiple Myeloma for Whom HSC Transplant is Not Planned as Initial Therapy.
JNJ-68284528 (ciltacabtagene autoleucel [cilta-cel]) is an autologous CAR-T therapy that targets B-cell maturation antigen (BCMA), a molecule expressed on the surface of mature B lymphocytes and malignant plasma cells. Results from the Phase 1b and Phase 2 portions of Study 68284528MMY2001 in participants with relapsed/refractory multiple myeloma (MM) indicate that cilta-cel has significant anti-myeloma
activity and a safety profile consistent with the known mechanism of action of CAR-T therapies.
All eligible participants who complete the initial six 21-day cycles of VRd therapy will then be randomized to 1 of 2 treatment arms.
Following randomization, participants in Arm A will first receive two 21-day cycles of VRd followed by Rd maintenance (28-day cycles) until confirmed progressive disease (PD), death, intolerable toxicity, withdrawal of consent, or end of study. Following randomization, participants in Arm B will undergo apheresis to acquire mononuclear cells (MNCs). Cilta-cel will be generated from the participant’s T-cells selected from the apheresis product.
After apheresis these participants will receive 2 cycles of VRd as bridging therapy, at the same dose and regimen as was administered during the initial 6 cycles (including dose adjustments if applicable). After cilta-cel production and product release, participants will receive a conditioning regimen of cyclophosphamide and fludarabine. After the infusion of cilta-cel, participants will move into a treatmentfree observation phase until confirmed PD or the start of subsequent anti-myeloma therapy. Cilta-cel will be administered 5 to 7 days after the start of the conditioning regimen. Participants will have intensive monitoring for safety, pharmacokinetics, biomarkers, and efficacy during the first 112 days after cilta-cel
administration (post-infusion follow-up phase). After the post-infusion follow-up phase is complete (Day 112), participants will move into the post-treatment follow-up phase. During the post-treatment follow-up phase, participants will continue to be monitored for efficacy until confirmed PD, death, withdrawal of consent, or final PFS analysis. After confirmed PD, participants will be followed for subsequent anti-myeloma therapies, PFS on next-line therapy (PFS2), delayed AEs (including SPMs), and survival, every 16 weeks until the end of the study. All participants who receive cilta-cel will continue to be monitored for long-term safety under a separate study (68284528MMY4002) for up to 15 years after cilta-cel administration.