Hairy Cell Leukemia

Researchers are investigating a new combination of three drugsazacitidine, venetoclax, and tagraxofuspto treat patients with Acute Myeloid Leukemia AML who have leftover leukemia cells that cannot be seen with the naked eye. This Phase 12 clinical trial aims to assess the safety and how well this drug combination controls residual AML and prevents the disease from coming back. The study builds on FDA approvals of venetoclax and azacitidine together for AML and tagraxofusp alone for another leukemia type, but this combination is not yet FDA-approved for AML treatment. The study involves two groups of participants. In Phase 1, up to 12 people receive escalating doses of tagraxofusp combined with fixed doses of azacitidine and venetoclax to find the safest and best dose. Treatment cycles last 28 days, with azacitidine given daily for seven days, tagraxofusp infused on days 4 to 6, and venetoclax taken on days 1 and 14. In Phase 2, 19 participants receive the recommended dose of tagraxofusp plus azacitidine and venetoclax with the same schedule. Bone marrow biopsies and aspirations occur regularly during treatment to monitor response. After treatment, participants are followed for up to two years. Participants will have regular visits including blood tests, imaging scans like CT, MRI, or PET, heart function tests, and bone marrow examinations. Researchers will monitor for side effects, measure disease remission, and check for minimal residual disease to evaluate treatment impact. The study expects to last about four years with around 31 participants. Outcomes such as remission duration, survival, relapse rates, and safety events will be assessed during treatment and follow-up.

This research aims to understand how well routine childhood vaccinations protect pediatric patients under 21 years old who have been treated for B-cell Acute Lymphoblastic Leukemia or Lymphoma B-ALLLy, especially those who have received the immunotherapy drug blinatumomab. The study compares patients treated with blinatumomab to those who have not, to see if immunotherapy affects the lasting protection from vaccines. It also seeks to establish clear vaccination guidelines for these patients, since chemotherapy and immunotherapy may weaken their immune defenses. Participants are observed in two groups those who have received blinatumomab and those who have not. The study evaluates whether patients maintain protective antibody levels at least six months after completing blinatumomab therapy. For patients with low immunity, the study will assess if revaccination restores protective antibody levels within six months. The study does not involve new treatments but monitors immune responses over time to routine vaccinations. During the study, participants will have blood tests to measure their antibody levels against common vaccine-preventable diseases. Researchers will track who maintains immunity and who responds to revaccination. The primary measurement is the proportion of patients with protective antibody levels six months post-treatment. The study will continue to monitor participants immune status and response to boosters, helping to inform future vaccination protocols. Participation may last through follow-up visits over several months after therapy completion.

Researchers are studying the use of unlicensed cryopreserved cord blood units CBUs for transplantation in both children and adults with blood cancers and other related disorders. This observational study involves patients with hematologic malignancies and various inherited and acquired disorders affecting the blood and immune system. The main goal is to monitor how well neutrophil recovery occurs after transplantation using these unlicensed CBUs in multiple institutions. Participants receive unlicensed cryopreserved CBUs as part of their transplant treatment. The study includes patients of any age receiving these CBUs for approved indications. The protocol focuses on the access and distribution of these unlicensed units rather than a specific treatment intervention. The study gathers data from recipients who receive these CBUs, tracking outcomes after transplantation. Participants are monitored for neutrophil recovery at 60 and 100 days after transplant, defined by a neutrophil count of at least 500mm3. Researchers also collect information on infection transmission, infusion reactions, survival rates at one year, and incidence of acute and chronic graft versus host disease. Platelet recovery is also evaluated. Safety and efficacy outcomes are followed over time to better understand the effects of unlicensed CBUs in this patient population.

Researchers are evaluating whether telehealth music therapy can be a practical treatment for cognitive difficulties in adults who have survived blood cancers such as lymphoma, leukemia, or myeloma. The study also examines if music therapy and music education can help improve cognitive function as well as symptoms like anxiety, depression, and fatigue in this population. This pilot trial is exploring these effects in hematologic cancer survivors who experience cancer-related cognitive dysfunction. Participants will be assigned to one of three groups the experimental music therapy MT group, the therapist-attention music education TAME control group, or a wait-list control WLC group receiving usual care. Those in the MT and TAME groups will receive 12 weekly 60-minute sessions, with homework assignments between sessions to reinforce skills and concepts. The WLC group completes assessments during a 24-week wait period and can later choose to receive either the MT or TAME intervention. During the study, participants will complete assessments to measure the feasibility of telehealth music therapy, defined by completing at least 9 of the 12 sessions. Researchers will monitor cognitive function, mood symptoms, and fatigue. Participants must be able to complete study assessments independently and consent in English. The total study duration includes the intervention period and follow-up assessments to evaluate outcomes related to cancer-related cognitive dysfunction.

Researchers are studying the safety, tolerability, and preliminary effects of CG009301 for injection in adults with relapsed or refractory blood cancers, including acute myeloid leukemia AML, high-risk myelodysplastic syndromes HR-MDS, and acute lymphoblastic leukemia ALL. This Phase 1, open-label trial aims to find the best dose and dosing schedule for CG009301 and to better understand its safety profile in participants who have limited treatment options. Participants will receive CG009301 through an intravenous infusion diluted in sodium chloride daily for 7 days per 28-day cycle. The study has two phases a dose-escalation phase for various relapsed or refractory blood cancers, followed by a dose-expansion phase focusing on AML, HR-MDS, and ALL. Treatment continues until the disease progresses. During the trial, participants will undergo regular assessments including safety monitoring, laboratory tests, and evaluations of drug levels in the body. Researchers will measure outcomes such as the recommended dose for expansion, duration of treatment cycles, adverse events, and clinical responses over periods up to 20 months or longer. Participants are expected to comply with scheduled visits and tests throughout the study duration.

Researchers are evaluating IM-1021, an antibody-drug conjugate, in a Phase 1 study involving participants with advanced B-cell lymphomas and solid tumors. This first-in-human, open-label study aims to assess the safety, tolerability, pharmacokinetics, and preliminary anti-tumor activity of IM-1021. The study includes a dose escalation phase to find safe and tolerable doses and an expansion phase to further evaluate these doses in specific cancer types. The study has two parts Part A focuses on escalating doses of IM-1021 given intravenously to determine safety and recommended dosing schedules, including the possibility of alternative dosing. Part B involves expanding participant groups to further test safety and early effectiveness of IM-1021 at doses chosen from Part A. Participants receive the study drug intravenously on an intermittent basis throughout these phases. Participants will undergo multiple assessments including monitoring for treatment-related adverse events, pharmacokinetic blood tests, and evaluations of anti-tumor effects from week 6 until disease progression or study discontinuation. Safety and tolerability will be tracked from the first dose until about 37 days after the last dose. The study duration spans from screening, treatment, and follow-up with data collection continuing up to the study end in 2029.

Researchers are studying JV-213, a new type of autologous CAR T-cell therapy targeting CD79b, in adults with relapsed or refractory B-cell lymphomas. The main goal is to find the highest dose of JV-213 that patients can tolerate safely. This Phase 1 trial also aims to evaluate the therapys safety, tolerability, and the best dose to use in future studies. Participants receive JV-213 through an intravenous infusion after their own immune cells are collected via leukapheresis. The study has two parts the first tests increasing doses of JV-213 in small groups to find the maximum tolerated dose, while the second part treats more patients at this recommended dose. Each participants treatment depends on the dose level assigned during the trial. Throughout the study, participants are closely monitored for side effects using standard criteria for adverse events. Researchers also assess tumor response and symptom relief to measure effectiveness. Blood and tumor samples are collected to study how the therapy works in the body and to identify biomarkers linked to treatment response or side effects. The study continues until December 2028, with approximately one year of follow-up after treatment to assess safety and outcomes.

Researchers are evaluating the safety and early effectiveness of TT-01488, an oral tablet that reversibly inhibits BTK, in adults with B-cell malignancies who have not responded to or tolerated previous treatments. This open-label Phase I study aims to find a suitable dose for further testing and to assess how the drug behaves in the body. The study is sponsored by TransThera Sciences Nanjing, Inc. The trial has two parts a dose escalation phase where TT-01488 tablets are given once daily in 28-day cycles at increasing doses to find the recommended dose for expansion followed by a dose expansion phase where this dose is further tested for safety and early effectiveness. The dose escalation uses a modified 33 design starting at 50 mg daily, with the goal of identifying a recommended Phase II dose based on safety, drug levels, and response. Participants will take daily tablets and undergo assessments including monitoring for dose-limiting toxicities within 28 days of the first dose, along with safety and response evaluations over three years. Researchers will track adverse events, drug concentration in the blood, and measures like disease control and survival. The study is non-randomized and open-label, and participant involvement may last up to three years depending on outcomes and follow-up needs.

Researchers are evaluating the safety and effectiveness of a new fourth-generation gene-modified T cell therapy called 4SCAR19, which targets CD19-positive B cell malignancies. This phase III trial involves multiple clinical centers and focuses on patients with relapsed or refractory B cell cancers that have not responded to standard treatments. The study also aims to develop a standardized method for producing these modified cells using lentiviral vectors. Participants will undergo a process where their peripheral blood mononuclear cells are collected through apheresis, and their T cells are activated and genetically modified with the 4SCAR19 gene. Before receiving the modified T cells, participants will get a conditioning chemotherapy regimen of cyclophosphamide and fludarabine to prepare their immune system. The modified 4SCAR19 T cells will then be infused, and participants will be closely monitored for treatment responses and safety. Throughout the study, participants will have regular assessments to track the presence of CAR T cells and observe clinical responses. Safety will be evaluated over 24 weeks using standard criteria for adverse events, while anti-tumor activity will be followed for up to one year. The trial requires ongoing monitoring after treatment to understand the therapys effects and safety profile over time.

Researchers are evaluating revumenib, a drug also known as SNDX-5613, in people with leukemia linked to increased expression of HOX genes. This Phase II study aims to assess how well revumenib works in controlling such leukemias and to understand its effects on measurable residual disease, cytogenetic remission, event-free survival, duration of response, and overall survival. The study also explores molecular markers that might predict response or resistance to treatment. Participants who qualify will take revumenib orally twice daily, about 12 hours apart, during each 28-day treatment cycle. The study focuses on relapsed or refractory acute leukemia of various lineages with specific genetic alterations associated with HOX gene upregulation. The treatment continues in cycles, with ongoing monitoring to assess effectiveness and safety. During the study, participants will be closely monitored for safety and adverse events, with an average follow-up of about one year. Researchers will conduct assessments including blood tests and other evaluations to track response and side effects. Participants or their guardians must provide informed consent, and the study collects data on molecular and cellular markers to understand treatment response better over the course of participation.