Clinical Research Directory

Oral Tetrahydrouridine-Decitabine in Patients With Advanced Myeloproliferative Neoplasms

This is a phase II, multicenter, open-label study evaluating the feasibility, safety, and efficacy of oral tetrahydrouridine-decitabine (THU-decitabine) in patients with advanced myeloproliferative neoplasms (MPN).

AlloHCT With PegINFa for Myelofibrosis

This is a single site, open-label, dose de-escalation, Phase 1 study of pegylated interferon alfa-2a administered after alloHCT in subjects with primary or secondary myelofibrosis. Part 1 of the study will assess the rate of dose-limiting toxicities (DLTs) during the DLT evaluation period and identify the Recommended Phase 2 Dose (RP2D). Once the RP2D is identified, 6 additional patients will be enrolled in the expansion cohort.

Feasibility Trial of Extracorporeal Iron Purification in Patients With Myelodysplastic Syndrome or Myelofibrosis

In transfusion-dependent myelodysplastic syndromes patients, regular blood transfusions lead to iron overload, which can cause organ damage, hormonal imbalances, and increased infection risk, ultimately impacting patient survival. Standard oral iron chelation therapies can be intolerable for some patients due to adverse effects. The MEX-CD1 device (class III) could potentially offer an alternative for these patients by reducing serum iron levels through a novel, extracorporeal approach. MEXIRON clinical investigation focuses on the use of MEX-CD1, a medical device designed for extracorporeal chelation therapy to reduce iron overload in patients suffering from transfusion-dependent myelodysplastic syndromes (MDS) and myelofibrosis. MEXIRON aims to evaluate the device's use feasibility, safety, and effectiveness in reducing iron levels. Transfusions needs, patient experience and quality of life are also assessed. Each enrolled patients will undergo three low-volume continuous veno-venous haemodialysis cycles within one week. Following the three- haemodialysis cycles, patients will be monitored through on-site follow-up visits at 7 days, 28 days, and 90 days post-treatment to assess long-term effects.

Darzalex Faspro (Daratumumab and Hyaluronidase-fihj) Before Standard Desensitization and Allogeneic Bone Marrow or Peripheral Blood Stem Cell Transplantation in Adult Patients at High-risk for Primary Graft Failure Secondary to Donor Specific Antibodies

This research is being done to investigate the safety and effectiveness of Darzalex Faspro (daratumumab and hyaluronidase-fihj) (a monoclonal antibody that targets plasma cells that make antibodies) and whether it can lower donor specific antibodies (DSA) levels to low enough levels to permit patients to proceed with allogeneic peripheral blood transplant (alloBMT). Those being asked to participate have high DSA levels that puts those being asked to participate at high risk of rejecting the available donor's blood stem cells and making those being asked to participate ineligible to receive a stem cell transplant.

A Study of Oral Nuvisertib (TP-3654) in Patients With Myelofibrosis

This study is a Phase 1/2, multicenter, dose-escalation, open-label trial to assess safety, tolerability, pharmacokinetics and pharmacodynamics of nuvisertib (TP-3654) in patients with intermediate or high-risk primary or secondary MF.

A Study to Compare Elritercept to Placebo in Adults With Myelofibrosis and Anemia Who Are Taking Ruxolitinib

The main aim of this study is to find out how well elritercept works to improve anemia in participants with myelofibrosis (MF) who are taking ruxolitinib when compared to placebo. Other aims are to learn how elritercept improves anemia compared to placebo; to learn if elritercept reduces tiredness, improves symptoms related to MF, and helps participants do physical activities more easily. The study also aims to find out how elritercept affects the bone marrow, the spleen, and whether participants develop antibodies to the study drug. The study will also check how safe elritercept is compared to placebo, and if elritercept stays safe over a long period of time. Participants will receive study treatment for at least 9 months (36 weeks). After this period, participants who received placebo will have the option to switch to elritercept.

Tolerability of Ropeginterferon Alfa-2b Add-on to Ongoing Ruxolitinib Therapy in Myelofibrosis

The purpose of this clinical trial is to learn if the study drug ropeginterferon alfa- 2b added to, standard of care, ruxolitinib is safe and effective in treating patients with Myelofibrosis.

Study of Selinexor in Combination With Ruxolitinib in Myelofibrosis

This is a global, multicenter, 2-part study to evaluate the efficacy and safety of selinexor plus ruxolitinib in JAK inhibitor (JAKi) treatment-naïve myelofibrosis (MF) participants. The study will be conducted in two phases: Phase 1 (open-label) and Phase 3 (double-blind). Phase 1 (enrollment completed) was an open-label evaluation of the safety and recommended Phase 2 dose (RP2D) of selinexor in combination with ruxolitinib and included a dose escalation using a standard 3+3 design (Phase 1a) and a dose expansion part (Phase 1b). Phase 3 (ongoing), double-blind, placebo-controlled part of the study comparing the efficacy and safety of combination therapy of selinexor + ruxolitinib with combination of placebo + ruxolitinib.

Mutant CALR-peptide Based Vaccine in Patients With Mutated CALR Myeloproliferative Neoplasm

The primary objective of this study is to assess the safety and tolerability of administrating mutated-CALR peptide Vaccine to patients with MPN. The researchers plan to enroll 10 patients over a 12 month period. Maximum length of participation in 80 weeks. Patients will be asked to complete questionnaires, bone marrow biopsies, research lab collection, and standard of care lab draw. This research will be taking place only at The Mount Sinai Hospital, specifically at the Ruttenberg Treatment Center.

CMV-MVA Triplex Vaccination of Stem Cell Donors in Preventing CMV Viremia in Participants With Allogeneic Transplant

This phase II trial studies how well multi-peptide CMV-modified vaccinia Ankara (CMV-MVA Triplex) vaccination of stem cell donors works in preventing cytomegalovirus (CMV) viremia in participants with blood cancer undergoing donor stem cell transplant. Giving a vaccine to the donors may boost the recipient's immunity to this virus and reduce the chance of CMV disease after transplant.

Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia

This phase 1b/2a open-label study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of DISC-0974 as well as categorize the effects on hematologic response in participants with myelofibrosis or myelodysplastic syndrome and anemia.

Hu8F4 in Treating Patients With Advanced Hematologic Malignancies

This phase I trial studies the side effects and best dose of anti-PR1/HLA-A2 monoclonal antibody Hu8F4 (Hu8F4) in treating patients with malignancies related to the blood (hematologic). Monoclonal antibodies, such as Hu8F4, may interfere with the ability of cancer cells to grow and spread.

Comparing Momelotinib and Ruxolitinib in People With Untreated Myelofibrosis and Low Blood Cell Counts

The purpose of this study is to compare momelotinib and ruxolitinib as treatments for myelofibrosis with low blood cell counts. Both drugs are approved by the FDA to treat myelofibrosis. The study asks which drug does a better job at shrinking the spleen.

High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly

To learn if giving ruxolitinib and busulfan before a stem cell transplant can help to reduce spleen size and help the transplant to succeed.

RVU120 in Patients With Intermediate or High-risk, Primary or Secondary Myelofibrosis

The objective of this clinical trial is to evaluate the efficacy (how well the drug works), safety, pharmacokinetics (PK), and pharmacodynamics (PD) of the study drug, RVU120, in treating adult patients with intermediate or high-risk, primary or secondary myelofibrosis. RVU120 will be given as a single agent or in combination with ruxolitinib.

Haplo-HSCT for Myelofibrosis

Myelofibrosis (MF) is a myeloproliferative neoplasm causing bone marrow failure and high risk of leukemia transformation. JAK2 inhibitors improve symptoms but do not cure MF. Allogeneic stem cell transplantation (allo-HSCT) is the only potential cure, though limited donor availability restricts access. Haploidentical transplantation shows promise but associated with higher graft failure and treatment related mortality. We recently developed a novel regimen of haplo-SCT for MF. This study aims to investigate this novel protocol in a prospective trial to improve MF outcomes.

A Telehealth Advance Care Planning Intervention

The objective of this project is to conduct a pilot randomized trial to assess the preliminary efficacy of a telehealth-delivered Serious Illness Care Program on healthcare communication, patient anxiety and distress, as well as completion of advance directives (specifically MOLST and healthcare proxy forms) for older patients with acute myeloid leukemia, myelodysplastic syndrome, and similar myeloid malignancies.

Peri-graft Cytokine Profile: Influence of Hematologic Disease and Donor Type

Cytokine profiles in the early post-transplant phase may influence the Development of early post-transplant inflammatory or immunological complications. This study propose to analyze these cytokine profiles based on the disease (myelofibrosis or other hematologic malignancies) and on the donor type (haploidentical, genotypically identical, or phenotypically identical). Patients with myelofibrosis have an inflammatory cytokine profile prior to transplantation. Patients who undergo haploidentical allogeneic hematopoietic stem cell transplantation (HSCT) most often experience a post-transplant cytokine release syndrome, and the question is whether these two conditions-myelofibrosis and haploidentical transplantation-increase the probability of an early cytokine release, potentially putting these patients at greater risk for acute GVHD than patients who undergo transplantation under other conditions or who have other diseases. In the prospective Phase 2 "FIBRAPLO" protocol (ClinicalTrials.gov ID NCT04728490), 28 patients with myelofibrosis received an allogeneic hematopoietic stem cell transplantation from a haploidentical donor, and serum samples were collected at Day-7 before transplantation, Day 0 and Day+7 after transplantation to analyse cytokine profiles around the time of allogeneic HSCT. The purpose of the present study is to match these FIBRAPLO patients (patients with myelofibrosis who received an allograft from a haploidentical donor) with patients without myelofibrosis who received a haploidentical transplant, in order to determine whether these profiles are specific to this population. The aim is to compare these profiles to other scenarios: patients with or without myelofibrosis who received transplants from 10/10 or geno-identical donors. This study propose to prospectively collect blood samples at time points (D-7, D0, and D7) from control patients : * receiving a haplo-identical allogeneic transplant for another condition (acute leukemia, myelodysplastic syndrome), * a geno-identical or 10/10 matched unrelated allogeneic transplant, for myelofibrosis or for other condition (acute leukemia, myelodysplastic syndrome)

A Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of Imetelstat in Combination With Ruxolitinib in Participants With Myelofibrosis

The purpose of the study is to identify the recommended Part 2 dose (R2PD) of imetelstat sodium in combination with ruxolitinib in participants with myelofibrosis (MF) in Part 1, and to evaluate the safety and preliminary clinical activity of the R2PD of imetelstat sodium in combination with ruxolitinib in participants with MF in Part 2.

HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation With Reduced Dose Post Transplantation Cyclophosphamide GvHD Prophylaxis

The goal of this clinical trial is to determine the effectiveness of Reduced Dose Post-Transplant Cyclophosphamide (PTCy) in patients with hematologic malignancies after receiving an HLA-Mismatched Unrelated Donor (MMUD) . The main question\[s\] it aims to answer are: * Does a reduced dose of PTCy reduce the occurrence of infections in the first 100 days after transplant? * Does a reduced dose of PTCy maintain the same level of protection against Graft Versus Host Disease (GvHD) as the standard dose of PTCy?

QT Interval/Corrected QT Interval （QT/QTc） Clinical Study to Evaluate the Cardiac Safety of TQ05105 Tablets in Healthy Participants

Primary Objective: To evaluate the effect of single-dose TQ05105 on QTcF interval in healthy Chinese participants. Secondary Objectives: To evaluate the effects of single-dose TQ05105 on other cardiac parameters (heart rate, QT, RR, PR and QRS intervals) in healthy Chinese participants; To verify assay sensitivity by evaluating the effect of moxifloxacin on QTcF interval; To evaluate the safety and tolerability of single-dose TQ05105 in healthy Chinese participants; To characterize the pharmacokinetic (PK) characteristics of TQ05105 and its metabolite (TQ12550) after single administration of TQ05105 tablets in healthy Chinese participants.

Study to Evaluate Safety, Pharmacokinetic and Pharmacodynamic Dose Escalation and Expansion Study of PXS-5505 in Patients With Primary, Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis

This study will be an open-label phase 1/2a study to evaluate the safety and tolerability of PXS-5505 in patients with primary, postpolycythemia vera (PV) or post-essential thrombocythemia (ET) myelofibrosis.

Quantitative MRI for Myelofibrosis

This study is for the development and validation of functional magnetic resonance imaging (MRI) parameters as biomarkers for analyzing extent of disease and quantifying response to treatment in patients with myelofibrosis.

Revumenib in Patients With Myelofibrosis

This is a Phase Ib/II 2 study investigating the safety and efficacy of revumenib in two cohorts of participants with myelofibrosis. COHORT-1 will investigate the safety of revumenib as monotherapy in participants with myelofibrosis previously treated with a JAK inhibitor. Following confirmation of safety in COHORT-1, the study will proceed with enrollment in COHORT-2, which will evaluate the efficacy and safety of revumenib in combination with a JAK inhibitor.