Essential Thrombocythemia

Researchers are studying the use of 3'-deoxy-3'-[18F] fluorothymidine (FLT) positron emission tomography (PET) imaging in patients with cancer. This phase I trial aims to evaluate how well FLT PET imaging measures tumor growth and the activity of the DNA synthetic pathway in various cancers, including solid tumors and blood cancers. The study also seeks to determine how effective this imaging method is at detecting lesions and assessing response to treatment. Participants receive up to four FLT PET imaging procedures. During each procedure, a small amount of the FLT tracer compound is injected into the vein, followed by PET scan data collection for two hours to measure tumor growth. Blood samples may be taken during the scans, and urine samples collected afterward to analyze breakdown products of the tracer. Throughout the study, patients undergo assessments including PET or CT PET scans to measure tracer uptake and retention in tumors and normal organs. Researchers also evaluate changes in key enzymes related to DNA synthesis before and after therapy. These evaluations help monitor tumor activity and treatment response. The total time participants spend in the scanner during imaging is up to two hours per session, with a focus on capturing detailed tumor growth information.

Researchers are studying the use of unlicensed cryopreserved cord blood units (CBUs) for transplantation in both pediatric and adult patients with various blood-related cancers and other disorders affecting the blood-forming system. This observational study aims to evaluate outcomes such as the recovery of a certain level of white blood cells after transplantation, as well as the incidence of infections, infusion reactions, survival rates, and graft-versus-host disease over time. The study involves patients receiving unlicensed CBUs at multiple U.S. transplant centers. These CBUs are used for patients with hematologic malignancies and other blood disorders. The protocol collects data on patients who receive these unlicensed transplant units, without administering a new treatment but observing the outcomes after transplantation. Participants will be monitored for neutrophil recovery at 60 and 100 days post-transplant, along with assessments of infection transmission, infusion reactions, survival one year after transplant, and occurrences of acute and chronic graft-versus-host disease. Platelet engraftment levels will also be tracked. The study includes patients of any age and follows them through the transplantation and recovery process to gather information on these key outcomes.

Researchers are evaluating IM-1021, an antibody-drug conjugate, in participants with advanced cancers including B-cell lymphomas and solid tumors. This Phase 1 open-label study aims to assess the safety, tolerability, pharmacokinetics, and early anti-tumor effects of IM-1021. The study includes a dose escalation phase to find safe doses and schedules, followed by an expansion phase to further assess these doses in specific cancer types. IM-1021 is given intravenously on a 21-day cycle, starting at 2 mg/kg, with alternative dosing schedules possible. The study has two parts: Part A focuses on escalating doses to evaluate safety and determine recommended doses, while Part B expands treatment in groups with specific cancer types to further evaluate safety and preliminary activity. Participants will undergo regular safety assessments including monitoring for treatment-related side effects from the first dose through 37 days after the last dose. Researchers will also measure drug levels in the body and evaluate anti-tumor activity starting at week 6 until disease progression or study discontinuation. The total study duration varies per participant. Safety, tolerability, and pharmacokinetic data will guide future development of IM-1021.

Researchers are evaluating cytokine induced memory-like natural killer (CIML NK) cells combined with IL-2 in adults aged 18 and older with Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), and Myeloproliferative Neoplasms (MPN) who have relapsed after haploidentical or HLA matched stem cell transplantation. The study also includes pediatric patients aged 12 and older with AML, MDS, and Juvenile Myelomonocytic Leukemia (JMML) who relapse after stem cell transplantation. This is a Phase I clinical trial focused on testing the safety and determining the appropriate dose of these investigational CIML NK cells, which have not yet been approved by the FDA for relapsed disease treatment. The treatment involves intravenous infusion of CIML NK cells on day 0. Prior to this, patients receive chemotherapy with fludarabine administered once daily for three doses starting on day -5, and cyclophosphamide given on days -5 and -4. This regimen is designed to prepare the body for CIML NK cell infusion. Both adult and pediatric patients undergo this treatment schedule. Participants will be closely monitored for safety over 6 weeks, with additional evaluations including objective response rate at 28 days, and assessments for leukemia-free survival and overall survival at 100 days and one year. Researchers will also track the incidence and severity of acute and chronic graft-versus-host disease over time. The study involves various tests such as bone marrow examinations, blood tests, and pregnancy tests when applicable. Participants must meet eligibility criteria and provide informed consent to join this study, which continues until the end date in December 2026.

Researchers are evaluating drug combinations to prevent graft-versus-host disease (GVHD) in people who have received stem cell transplants from unrelated donors with different blood types. This platform protocol focuses on safety and effectiveness of post-transplant cyclophosphamide (PTCy) based GVHD prevention after mismatched unrelated donor hematopoietic cell transplants in patients with malignant blood diseases. The study compares new drug combinations to a standard treatment. Participants receive one of the drug combinations after transplant, including investigational arms named ACCEL-001 and ACCEL-002, or the shared comparator control group. Conditioning regimens vary and may include combinations of drugs such as busulfan, fludarabine, melphalan, cyclophosphamide, and total body irradiation before transplant. The donor stem cell graft infusion occurs on Day 0, followed by specific post-transplant medications like cyclophosphamide, tacrolimus, mycophenolate mofetil, abatacept, and ruxolitinib, with supportive care for infection prevention and other complications. During the study, participants have regular doctor visits for check-ups and routine tests, complete surveys on physical and emotional health, and provide blood and stool samples. Researchers monitor outcomes including graft-versus-host disease-free, relapse-free survival one year after transplant, infection rates, survival, graft failure, and immune recovery. Safety is closely tracked, including monitoring for cytokine release syndrome and infections. The study lasts for at least one year post-transplant, with detailed data collection on treatment response and side effects.

Researchers are evaluating the safety, tolerability, pharmacokinetics, and preliminary effectiveness of BL-M24D1 in patients with relapsed or refractory multiple myeloma and other blood cancers. This open, multicenter, non-randomized Phase I clinical trial aims to gather early data on how this investigational drug behaves and affects patients who have limited treatment options after standard therapies have failed or are unavailable. The study has two parts: a dose escalation phase (Phase Ia) and an expansion cohort phase (Phase Ib). Participants receive BL-M24D1 through intravenous infusion over a 2-week cycle. If clinical benefit is observed, patients may continue receiving additional cycles until disease progression, intolerable side effects, or other reasons lead to stopping treatment. During the trial, participants undergo regular assessments including monitoring for dose-limiting toxicities within 28 days after the first dose and evaluation of the maximum tolerated dose. Over approximately 24 months, researchers will track treatment-emergent side effects, pharmacokinetic measures like drug concentration and clearance, immune responses to the drug, and clinical responses such as objective response rate and duration of response. The study includes safety follow-up and long-term monitoring.

Researchers are evaluating the safety and effectiveness of bomedemstat (MK-3543) compared to the best available therapy (BAT) in adults with essential thrombocythemia (ET) who have not responded well to or cannot tolerate hydroxyurea. The study aims to determine if bomedemstat provides a better lasting clinical and blood response than current treatments. This is a phase 3, randomized, open-label trial sponsored by Merck Sharp & Dohme LLC. Participants will be randomly assigned to receive either bomedemstat or one of several approved therapies including anagrelide, busulfan, interferon alfa (or pegylated forms), or ruxolitinib. Bomedemstat will start at 50 mg daily, with dose adjustments to safely lower platelet counts. Each participant will be treated daily for up to 52 weeks, with an option to continue an extended treatment phase up to 156 weeks. Those initially on BAT who stop responding may switch to bomedemstat during the extension. During the study, participants will have regular assessments of blood counts, symptoms, and side effects. Researchers will measure the durable clinicohematologic response over about 52 weeks as the main outcome. Other outcomes include symptom changes, event rates like thrombosis or bleeding, disease progression, and safety up to 180 weeks. The study includes monitoring of fatigue and quality of life using patient questionnaires to understand treatment impact over time.

Researchers are evaluating GLB-001, an oral drug, in patients with relapsed, refractory, or intolerant myeloid malignancies including polycythemia vera, essential thrombocythemia, myelofibrosis, various myelodysplastic syndromes, and acute myeloid leukemia. This phase 1, open-label study aims to assess the safety, tolerability, how the drug moves through and affects the body, and early signs of efficacy. The study includes three parts: dose escalation, dose exploration, and dose expansion to find the best dose for future studies. The study involves giving GLB-001 orally to participants in three phases. Phase 1a uses a dose-escalation design to evaluate safety and tolerability in patients with polycythemia vera or essential thrombocythemia. Phase 1b also uses dose escalation to study safety in patients with myelofibrosis, myelodysplastic syndromes, and acute myeloid leukemia. Phase 1c expands dosing to further assess tolerability, efficacy, and to select the recommended dose for phase 2 trials. Dose levels and treatment schedules are adjusted based on participant response and safety. Participants will undergo assessments to monitor dose-limiting toxicities within 28 days of the first dose and longer-term safety up to three years. Researchers will evaluate drug levels in the body and clinical responses to treatment over periods ranging from weeks to years depending on the disease type and study phase. Major organ functions, blood tests, adverse events, and response to treatment will be closely monitored throughout the study. Participants must agree to follow the visit schedule and study requirements during their involvement.

Researchers are comparing two different approaches to prevent chronic graft-versus-host disease (GVHD) in people with blood cancers who are receiving an allogeneic hematopoietic stem cell transplant (allo-HCT). The study aims to find out which prevention method is better at avoiding chronic GVHD up to one year after transplant. This is a Phase 2 randomized study sponsored by Memorial Sloan Kettering Cancer Center. Participants will be randomly assigned to one of two treatment groups. One group will receive an intermediate dose of post-transplant cyclophosphamide (PTCY), tacrolimus, mycophenolate mofetil (MMF), and ruxolitinib taken twice daily. The other group will receive the standard approach with a full dose of PTCY, tacrolimus, and MMF. Tacrolimus is started on day 5, with tapering schedules based on standard care or starting within two weeks of ruxolitinib. MMF is given from day 5 to day 35. During the study, participants will be closely monitored for chronic GVHD-free survival up to one year after transplant. Researchers will also track infections of grade 2 to 4 during that time. The study involves regular clinical assessments, laboratory tests, and safety monitoring. Participation will last at least one year after the stem cell transplant, allowing comprehensive evaluation of treatment effects and side effects over time.

Researchers are studying the effects of multiple doses of venetoclax on how the body processes ethinyl estradiol and levonorgestrel in female participants who have various types of blood cancers, specifically different subtypes of non-Hodgkin's lymphoma. This Phase 1 trial aims to understand the pharmacokinetics of these drugs when given together to help guide treatment decisions. Participants receiving clinical benefit without disease progression may continue venetoclax treatment in a separate extension study. The study involves administering ethinyl estradiol/levonorgestrel tablets on the first day of the first and third periods, while venetoclax tablets are given starting on the first day of the second period and then daily thereafter. This design allows researchers to evaluate the interaction between the drugs over approximately 59 days after the initial dose. The trial does not include masking or placebo controls. During the study, participants undergo monitoring to measure key drug parameters such as the time to reach maximum concentration (Tmax), maximum concentration (Cmax), half-life (t1/2), and overall drug exposure (AUC) for both venetoclax and ethinyl estradiol/levonorgestrel. These assessments help understand how the drugs behave in the body. Safety and disease status are also monitored, and participants may continue treatment in an extension phase if benefiting from the therapy. The total study duration includes follow-up up to about 59 days after the initial dose.