(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted...
Advanced Systemic MastocytosisAggressive Systemic Mastocytosis2 moreThis is an open-label, single arm, Phase 2 study evaluating the efficacy and safety of avapritinib (BLU-285) in patients with advanced systemic mastocytosis (AdvSM), including patients with aggressive SM (ASM), SM with associated hematologic neoplasm (SM-AHN), and mast cell leukemia (MCL)
Len/Dex/DLI in Relapsed Multiple Myeloma After Allogeneic Stem Cell Transplant
Relapsed Hematologic MalignancyMultiple MyelomaMultiple Myeloma (MM) is a morbid disease which can only be cured with an allogeneic hematopoietic stem cell transplant (HSCT). Approximately 50% of allotransplanted patients will relapse, with a median survival of 5 years. Better approaches to improve disease control at relapse, while decreasing toxicity, are urgently needed. Relapse after allogeneic transplant is a failure of the graft versus MM effect (GvMM). DLIs can be used to control disease following relapse, but the optimal dose, schedule of administration and drug association remain elusive, while the immunosuppression found in MM patients can compromise their effect. One reason for immunotherapy failure relates to the immunological environment: as much as myeloma cells depend on their microenvironment to survive and proliferate, the immunotherapeutic effect of allogeneic HSCT depends on both systemic and local immunological status to be efficacious. Immunomodulatory drugs such as Lenalidomide (Len) have been tried in various settings after allogeneic transplantation with the aim to reverse immunosuppression and stimulate the GvMM, but if and how Len influences a GvMM and thereby promotes an immunotherapeutic success remained uncharacterized. Therefore, a deeper understanding of the immunological environment in MM patients is needed in order to establish and / or restore a potent GvMM effect. This study proposes the powerful combination of the two following goals, one clinical and one biological : Clinical: The investigators propose a two-step treatment using first Len in association with Dexamethasone (Dex), followed by Donor Leukocytes Infusions (DLIs) to offer an optimal disease control strategy in relapsed patients. The cytoreductive and immunomodulatory effects of Len is expected to induce a permissive immunological environment for the immunotherapeutic activity of DLIs to develop, while the association with Dex will lessen the risk of graft-versus-host disease (GVHD). This treatment combination has the potential to further improve depth of myeloma response, delay myeloma progression and improve patient survival. Biological: In an attempt to gain knowledge on how the GvMM behaves in MM patients post-relapse after having received a combined treatment of Len/Dex/DLIs, the investigators propose to characterize the immune environment of their bone marrow (BM) using both minimal residual disease (MRD) assessement by flow cytometry and an unbiased analysis of the transcriptome at various time points.
Cytomegalovirus (CMV) Reactivation in Allogeneic HSCT Recipient
Cytomegalovirus InfectionsHaematological Malignancy2 moreThis study consists of two parts: 1) Part 1, a retrospective part on 250 consecutive patients following allogeneic haematopoietic stem cell transplant (allo-HSCT) at the Royal Melbourne Hospital from 2012 to 2017, inclusive, and 2) Part 2, a prospective part on 120 allo-HSCT patients from 4 sites in Australia: the Royal Melbourne Hospital, Peter MacCallum Cancer Centre, Austin Hospital, and Westmead Hospital. In Part 1, medical records of allo-HSCT recipients will be evaluated to determine the incidence and clinical outcomes of CMV viremia post HSCT, including both the direct (CMV disease) and indirect (such as invasive fungal infection, other viral infections, bacterial infection) effects on clinical outcomes. In Part 2, allo-HSCT participants at risk of CMV disease will be assessed to determine the association of host CMV-specific immunity with clinical management and outcomes over one year post allo-HSCT. The overall aims of the study are to establish if CMV infection in allo-HSCT patients are associated with poor clinical outcomes; and whether measurement of immunological functions could provide an early indicator to identify patients at risk and appropriate timing for initiation of CMV treatment.
Durvalumab and Tremelimumab for Pediatric Malignancies
Pediatric CancerSolid Tumor Pediatric1 moreThe purpose of the study is to determine the recommended dose of durvalumab and tremelimumab (immunotherapy drugs) in pediatric patients with advanced solid and hematological cancers and expand in a second phase to test the efficacy of these drugs once this dose is determined.
Serial Measurements of Molecular and Architectural Responses to Therapy (SMMART) PRIME Trial
Accelerated Phase Chronic Myelogenous LeukemiaBCR-ABL1 Positive55 moreThis phase Ib trial determines if samples from a patient's cancer can be tested to find combinations of drugs that provide clinical benefit for the kind of cancer the patient has. This study is also being done to understand why cancer drugs can stop working and how different cancers in different people respond to different types of therapy.
Expanded Access of Omidubicel, for Allogeneic Transplantation in Patients With Hematological Malignancies...
Hematological MalignanciesOmidubicel is an investigational therapy for patients with high-risk hematologic malignancies.
A Study to Evaluate the Tolerability, Safety, Pharmacokinetics of HEC89736 in Patients With Relapsed...
Relapsed or Refractory B-cell Hematologic MalignanciesEvaluate the safety, pharmacokinetics and efficacy of HEC89736 in patients with Relapsed or Refractory B-cell Hematologic Malignancies
A Study of Teclistamab in Participants With Relapsed or Refractory Multiple Myeloma
Hematological MalignanciesThe purpose of this study is to evaluate the efficacy of teclistamab at the recommended Phase 2 dose (RP2D).
Tildrakizumab for Prevention of Acute Graft-Versus-Host Disease
Hematologic MalignanciesThis is a phase 2 open-label trial designed to evaluate the efficacy of tildrakizumab in improving graft-versus-host disease (GVHD)-free relapse-free survival after myeloablative allogeneic hematopoietic cell transplantation (alloHCT) for hematologic malignancy.
Study of 3D189 in Patients With Hematologic Malignancies
Acute LeukemiaMultiple Myeloma2 moreTo assess the safety, immunogenicity and preliminary efficacy of 3D189 in patients with hematological malignancies.