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Clinical Studies

The ultimate goal of our research is its translation into therapies that improve patients' lives. Several of our scientific discoveries have been turned into drugs that are now being tested in patients with various severe diseases at clinical trials. These include the following studies:

01 | Prevention & therapy of organ transplant complications with regulatory T cells

 

Therapy of graft-versus-host-disease (GvHD) with regulatory T cells

Allogeneic stem-cell transplantation is a curative treatment option for patients with malignant and some non-malignant hematopoietic diseases.

Having donor T cells in the transplant is vital, as they strengthen immune function after transplantation and mediate antitumor effects (graft-versus-leukemia/lymphoma effect, GvL). On the other hand though, donor T cells can also attack the recipient’s organs soon after bone marrow transplantation, or even chronically over a long period thereafter, thus triggering potentially lethal donor-versus-host disease (acute [a]GvHD and chronic [c]GvHD).

In animal studies, Professor Matthias Edinger’s team demonstrated that treatment with donor-derived immune-suppressive regulatory T cells (Tregs) can block aGvHD and cGvHD, without blocking the GvL activity of conventional donor T cells. This strategy is now clinically translated into therapeutic concepts to treat acute and chronic GvHD using donor-derived regulatory T cells that are manufactured within the LIT’s Jose Carreras GMP facility.

 

Therapy for acute graft-versus-host-disease (aGvHD) with regulatory T cells

In a clinical phase I/II study, Professor Edinger’s team is exploring whether the transfer of large numbers of donor-derived regulatory T cells can ameliorate severe acute GvHD in patients

  • Title of the study | Therapy of Steroid-Refractory Gastrointestinal Graft-Versus-Host Disease with Regulatory Donor T Cells (Treg Therapy)
  • Eudra CT number | 2012-002685-12
  • Current status of the study | recruiting
  • Sponsor | University Hospital of Regensburg, represented by Professor Matthias Edinger
  • Study design | national, multicenter, single-arm, non-controlled, phase I/II
  • Head of the clinical trial | Professor Matthias Edinger
  • Participating trial centers | University Hospital of Erlangen, University Hospital of Mainz, University Hospital of Regensburg, University Hospital of Würzburg
  • Investigated product | in-vitro expanded regulatory T cells
  • Pharmaceutical manufacturer | LIT José Carreras Center for Somatic Cell Therapy, central manufacturer for all participating trial centers

The clinical study is funded by the Free State of Bavaria as part of the Bavarian Immunotherapy Network (BayImmuNet).

 

Therapy for chronic graft-versus-host-disease (cGvHD) with regulatory T cells

This clinical trial will investigate whether regulatory T cells from the donor can be used to treat severe, treatment-refractory chronic GvHD

  • Title of the study | Therapy of Steroid-Refractory Chronic Graft-Versus-Host Disease With Regulatory Donor T Cells
  • Eudra CT number | 2016-003947-1
  • Current status of the study | recruiting
  • Sponsor | University Hospital of Regensburg, represented by Professor Matthias Edinger
  • Study design | national, monocentric, single-arm, non-controlled, phase I/II
  • Head of the clinical trial | Professor Matthias Edinger
  • Participating trial centers | University Hospital of Regensburg
  • Investigated product | in-vitro expanded regulatory T cells
  • Pharmaceutical manufacturer |LIT José Carreras Center for Somatic Cell Therapy

This clinical study is funded by the European Union as part of the Horizon 2020 research program. It is embedded in the TREGeneration project, led by Professor João Forjaz de Lacerda from the Institute of Molecular Medicine in Lisbon, Portugal. The aim of the consortium is to conduct a total of five clinical trials with regulatory T cells in chronic GvHD. For more information please contact:

 

Professor Matthias Edinger
Director of JCC & Head of Research Group | Immunoregulation

Email: matthias.edinger@ukr.de

02 | Regulatory T cells in kidney transplantation

Organ transplantation is sometimes the only life-saving treatment for chronic kidney failure. However, lifelong immune suppression is often needed to prevent the rejection of the transplant by the recipient’s immune system. This necessary drug-based immunosuppression causes serious side effects and is often insufficient to rescue the transplant.

A clinical study launched in 2019 at the Medical University of Vienna, under the direction of Professor Thomas Wekerle, aims to help the recipient’s immune system tolerate the transplanted kidney so that it no longer treats the transplanted organ as ‘foreign’. Drug-based immunosuppression can then be stopped. In this study, tolerance to the transplanted organ shall be achieved through combined treatment with regulatory T cells from the recipient and bone marrow cells from the kidney donor. In the long term, the study should contribute to transplant patients being able to manage without immunosuppression. The LIT’s Jose Carreras GMP facility is acting as the pharmaceutical manufacturer of the regulatory T cells in this study.

  • Title of the study | A Prospective Controlled Trial Of In-Vitro Expanded Recipient Regulatory T-cell Therapy And Tocilizumab Together With Donor Bone Marrow Infusion In HLA-Mismatched Living Donor Kidney Transplant Recipients (Trex)
  • Eudra-CT-Nummer | 2018-003142-16
  • Current status of the study | recruiting
  • Sponsor | Medical University of Vienna, represented by Professor Thomas Wekerle
  • Pharmaceutical manufacturer | LIT José Carreras Center for Somatic Cell Therapy

The clinical study is funded by the German federal state (BmBF). For more information please visit: http://www.meduniwien.ac.at/transplantation-immunology/wwtf-project.html or contact:

 

Professor Matthias Edinger
Director of JCC & Head of Research Group | Immunoregulation

Email: matthias.edinger@ukr.de

03 | GD2-IL18 CAR T-cell Therapy

Phase I clinical trial to determine safety, dose-finding, and feasibility of adoptive T-cell therapy with GD2-IL18 CAR T cells in patients with relapsed or refractory GD2-positive solid tumors.

The treatment concept is based on the ground-breaking work of both Professor Hinrich Abken at the LIT, and of his collaborators, Professor Claudia Rössig (UMC Münster, Germany) and Professor Axel Schambach (UMC Hannover, Germany). T cells that are genetically equipped to express chimeric antigen receptors (CAR T cells) can selectively recognize tumor cells and are potent therapeutics against leukemias and lymphomas. However, they fail to be effective against solid cancers. One reason for this failure is the lack of proinflammatory cytokines such as IL18 within the tumor microenvironment; these are required for appropriate immune cell activity.

In this clinical trial CAR T cells that recognize the tumor antigen GD2 are genetically engineered to produce IL18 themselves once they recognize tumor cells. This novel principle of cellular immunotherapy has been successfully tested in vivo and is now being clinically explored in treatment-refractory patients with GD2 positive tumors at the UMC Münster. GD2-IL18 CAR T-cell production is conducted at the UMC Erlangen by Professor Andreas Mackensen.

  • Title of the study: A Phase I Safety, Dose Finding And Feasibility Trial Of GD2-IL18 CART In Patients With Relapsed Or Refractory GD2-Positive Solid Cancers
  • Eudra CT number | TBC
  • Current status of the study | recruitment scheduled to start in Q1/2024
  • Sponsor | University Hospital of Münster
  • Study design | national, monocentric, single-arm, non-controlled, phase I
  • Head of the clinical trial | Professor Claudia Rössig
  • Clinical trial centers|University Hospital of Münster, University Hospital of Erlangen
  • Investigated product | GD2-IL18 engineered CAR T cells
  • Pharmaceutical manufacturer |University Hospital of Erlangen

The clinical study is funded by the federal state of Germany (BmBF). For more information please contact:

 

Professor Hinrich Abken
Head of Research Division | Genetic Immunotherapy

Email: hinrich.abken@ukr.de

04 | SIK3 inhibitor OMX-0407

This is a single-arm, open-label, multicenter, phase I clinical trial assessing the safety and tolerability of OMX-0407 as a monotherapy in patients with previously treated, unresectable solid tumors.

OMX-0407, the first-in-class oral inhibitor of SIK3 (salt-inducible kinase 3), has been developed by iOmx Therapeutics AG, a spin-off business which was co-founded in 2016 by Professor Philipp Beckhove, Scientific Director at the LIT.  SIK3—a member of the SIK family—was identified by Professor Beckhove’s team at the LIT as a novel immune-protective kinase. Inhibition of SIK3 with OMX-0407 triggers a novel immune-evasion biological process by sensitizing tumors to immune-derived ligands from the TNF superfamily—and driving tumor reduction through NF-κB activation. In preclinical studies OMX-0407 showed strong monotherapy effects in reshaping the immune compartment and accelerating tumor cell death.

The study is a first-in-human, dose-escalation trial of OMX-0407 monotherapy in patients with previously-treated unresectable solid tumors. The phase I trial is designed to characterize the safety, tolerability, and pharmacodynamic activity of OMX-0407. The dose escalation scheme is established on the 3+3 design to determine the maximum tolerated dose (MTD).

For more information please visit: https://iomx.com or contact:

 

Professor Philipp Beckhove
Scientific Director LIT & Head of Research Division | Interventional Immunology

Email: beckhove@rcii.de