Neutropenia

The GENESIS clinical study aims to map HLA genetic variation in the Greek population and evaluate possible correlations with selected underlying diseases. It is a multicenter, prospective, non-interventional clinical study targeting 12,000 subjects over an anticipated duration of 36 months, with the goal of creating a pilot HLA map for medical research and possible clinical applications. Each subject will complete one visit at a participating site and provide demographic information, including date of birth, gender, race, ancestry, height, and weight, as well as information about smoking or vaping, alcohol consumption, arterial blood pressure, diagnosed diseases, and current treatments. Recent clinical laboratory results from up to 12 months before sample collection may also be collected when available, including blood count, metabolic, liver enzyme, and biochemical parameters. Two buccal swabs will be collected from each subject for DNA extraction and HLA genotyping analysis. Selected DNA samples will also undergo low-pass whole genome sequencing to further investigate associations between the HLA region and autoimmune diseases. After the analysis is completed, an individualized ancestry report will be securely available to study subjects if they elect to access it.

Researchers are evaluating the safety and effectiveness of emapalumab for treating prolonged severe cytopenia in participants with large B-cell lymphoma LBCL who have received CAR T-cell therapy CART. This pilot study aims to assess how well emapalumab works to improve blood cell counts and to monitor its safety and tolerability. Additionally, the study explores biomarkers that may indicate response or resistance to the treatment. Participants will be assigned to one of two dose levels of emapalumab, given by infusion. Up to two planned doses are being studied initially, with about 16 to 32 participants enrolled in total. The dosing approach involves evaluating improvement in blood test results among early participants to decide whether to continue enrollment at a given dose or to increase the dose. Treatment cycles and dose adjustments are based on the participants responses. Throughout the study, participants will undergo tests such as blood counts and bone marrow biopsies before and after each treatment cycle. Researchers will monitor safety by tracking adverse events and other health measures for about one year after treatment. Participants may also have biomarkers assessed to understand the treatments effects better. The study lasts until the primary completion date, with ongoing evaluations to assess safety and efficacy.

Researchers are evaluating the effectiveness and safety of using Trilaciclib to prevent chemotherapy-related myelosuppression in patients with Ewings sarcoma who have not previously received systemic anti-tumor treatment. This is a phase 2, prospective, randomized, controlled clinical trial focusing on patients aged 14 to 40 years with confirmed Ewings sarcoma. The study compares the use of Trilaciclib combined with standard VDCIE chemotherapy versus chemotherapy alone. Participants will be randomly assigned to one of two groups the experimental group receiving Trilaciclib along with alternating VDCIE chemotherapy every 3 weeks for up to 17 cycles, or the control group receiving only the alternating VDCIE chemotherapy on the same schedule. The chemotherapy regimen includes vincristine, doxorubicin or actinomycin D after a certain cumulative dose, cyclophosphamide, ifosfamide, and etoposide. Trilaciclib is given as an intravenous infusion following chemotherapy administration in the experimental group. Supportive care is allowed in both groups as needed. During the treatment, participants will be monitored for myelosuppression effects such as neutropenia and thrombocytopenia through laboratory tests. The primary outcome measures how long severe neutropenia lasts in the first treatment cycle, with additional outcomes tracking recovery times, incidence of febrile neutropenia, anemia, and serious infections across multiple cycles. The study will also assess event-free survival up to 24 months. Treatment continues until disease progression, unacceptable side effects, or withdrawal. The total study duration includes up to 17 cycles of treatment and follow-up assessments.

Researchers are studying the safety and tolerability of elritercept in adults with anemia linked to very low, low, or intermediate risk Myelodysplastic Syndromes MDS. The study aims to understand how elritercept affects red blood cell production and the progression of MDS, including how well participants tolerate different doses of the drug. This phase 2, open-label trial focuses on anemia associated with lower-risk MDS and evaluates the impact on healthy red blood cell production. Participants receive elritercept as a subcutaneous injection every 4 weeks, with doses ranging from 0.75 mgkg to 5.0 mgkg during an initial period of up to 4 cycles each 28 days. Following this, participants continue treatment with elritercept every 4 weeks for up to 24 cycles, with dose adjustments based on individual response. Different cohorts include participants with or without ring sideroblasts, those requiring red blood cell transfusions, and those with chronic myelomonocytic leukemia CMML. Some participants may enter a long-term extension phase receiving elritercept every 4 weeks for up to about 10 years. During the study, participants undergo regular monitoring including blood tests, assessments of red blood cell parameters, and tracking of adverse events and disease progression. Researchers measure treatment-emergent adverse events, progression to higher-risk MDS or acute leukemia, transfusion independence, and hematologic improvements over up to 11 years. The study includes follow-ups to evaluate the duration and timing of responses, safety, and overall effects on anemia and MDS progression.

This research aims to evaluate whether starting treatment with emapalumab can improve treatment planning and increase the effectiveness of standard therapies for pediatric patients with severe aplastic anemia sAA. The study focuses on children and young adults under 25 years old who have been newly diagnosed with sAA characterized by severe cytopenias and hypocellular bone marrow. The trial is sponsored by Memorial Sloan Kettering Cancer Center and funded by the FDAs Office of Orphan Products Development. Participants will initially receive emapalumab, an antibody that blocks interferon gamma, for six weeks. After this initial treatment, they will either receive standard immunosuppressive therapy IST with equine anti-thymocyte globulin and cyclosporin along with a reduced dose of emapalumab, or they will proceed to a standard hematopoietic stem cell transplant HCT. These two treatment paths are both experimental arms within the study. During the study, participants will be closely monitored to assess their response to treatment at six weeks. Evaluations include clinical assessments and laboratory tests to measure blood counts and marrow function. Researchers will track the best response to therapy and monitor safety throughout the process. The total study duration and follow-up extend up to May 2029, allowing for long-term observation of outcomes.

Researchers are evaluating mavorixafor, a drug being studied for people aged 12 and older who have congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders. These conditions cause low neutrophil levels and lead to recurrent or serious infections. The study aims to show if mavorixafor can improve clinical outcomes and increase neutrophil counts, while also assessing its safety and tolerability. Participants will continue their current treatment during the study, which may include therapies like granulocyte-colony stimulating factor G-CSF, immunoglobulin replacement, antibiotics, or no active treatment. They will be randomly assigned to receive either mavorixafor or a placebo orally once daily for up to 52 weeks. The study uses a quadruple-blind design to compare these groups in parallel. During the trial, participants will have regular assessments to monitor infection rates and severity, neutrophil counts, antibiotic use, oral ulcers, and fatigue levels using questionnaires. An independent committee will review infections to ensure accuracy. Safety and treatment effects will be followed throughout the 52-week treatment period. The total participation may last until the study ends in late 2027.

Researchers are evaluating the safety, pharmacokinetics, pharmacodynamics, and preliminary effectiveness of an anti-GPRC5D CAR-T cell product called OriCAR-017 in adults with relapsed or refractory multiple myeloma. This Phase III open-label study is the first clinical trial of OriCAR-017 in the United States by OriCell Therapeutics Co., Ltd., aiming to find suitable dosing and assess early treatment results in this patient group. The study includes a Phase I dose escalation stage with three different doses given as a single intravenous infusion to up to 18 participants. This is followed by a dose expansion stage with 10-15 participants and then a Phase II stage that may include up to 48 participants. Each participant receives one infusion of OriCAR-017 to evaluate its effects and safety. Participants will be closely monitored for up to two years after treatment. Researchers will assess the maximum tolerated dose and dose-limiting toxicities within 28 days after infusion. They will also study how the drug moves through and affects the body, measure response duration, progression-free survival, overall survival, and other response rates. Regular evaluations include laboratory tests, clinical assessments, and safety monitoring throughout the study period.

Researchers are evaluating the safety and clinical activity of different doses of belantamab mafodotin combined with lenalidomide, dexamethasone, and nirogacestat in patients with newly diagnosed multiple myeloma who are not able to undergo a transplant. This phase 12, open-label study aims to find the recommended dose for future studies and to explore how to manage eye-related side effects. About 36 participants will be enrolled, with follow-up lasting up to 3 to 4 years after enrollment ends. Belantamab mafodotin is given intravenously every other 28-day cycle at doses ranging from 1.0 to 1.9 mgkg. Lenalidomide is taken orally daily on days 1 to 21 of each 28-day cycle. Dexamethasone is given either orally or intravenously on days 1, 8, 15, and 22 of each cycle, with dose adjustments based on age. Nirogacestat is taken twice daily starting three days before the first dose and on the same days as belantamab mafodotin. The study has two parts dose finding to establish the best dose, followed by dose expansion with random assignment to different dose modification guidelines for eye side effects. Participants will undergo regular assessments to monitor side effects, including eye exams, and overall response to treatment. Researchers will track dose-limiting toxicities, adverse events, ocular toxicity, and response rates over up to four years. Additional evaluations include measuring drug levels, disease progress, and survival. Participants must provide consent and will be closely monitored throughout the study period, which is expected to last approximately four years in total.

Researchers are evaluating allogeneic hematopoietic stem cell transplantation HSCT as a treatment for patients with mutations in the GATA2 gene or the clinical syndrome known as MonoMAC. This condition causes immune system deficiencies and increases the risk of serious infections and blood cancers like myelodysplastic syndrome MDS and leukemia. The study aims to determine if this transplant approach can restore normal blood cell production and reverse disease symptoms by one year after transplant. Participants will receive stem cell transplants from donors matched by specific immune system markers HLA. There are different treatment plans depending on donor matching, involving chemotherapy and radiation conditioning before receiving the donor stem cells. After the transplant, patients will receive medications to prevent graft-versus-host disease GVHD. The transplant and post-transplant care include detailed drug regimens and monitoring, with hospital stays until the patient stabilizes. During the trial, participants will undergo physical exams, blood tests, imaging, and other assessments before and after transplant. Frequent monitoring will occur especially in the first six months, with less frequent follow-ups afterward. Researchers will measure successful engraftment, immune system recovery, disease outcomes, and safety over several years. The total participation duration may extend up to five years for long-term survival and health evaluations.

This research aims to study children, adolescents, and young adults up to 30 years old who have malignant or non-malignant blood disorders and are undergoing allogeneic stem cell transplantation AlloSCT. The trial focuses on selecting stem cells using alphabeta T cell and CD19 B cell depletion techniques. The purpose is to evaluate outcomes related to this specialized cell selection method in these patients. Participants will receive conditioning treatment before transplantation that may be full intensity, reduced intensity, or reduced toxicity based on their disease, status, organ function, and performance. They will then undergo AlloSCT with stem cells processed using alphabeta CD3CD19 cell depletion via the Prodigy system. Standard pre-conditioning and post-transplant monitoring will be provided throughout the study. During the study, participants will be monitored for engraftment success, immune system recovery, graft-versus-host disease GVHD, and quality of life. Researchers will track adverse events related to the cell-depleted stem cell administration over one year. The total involvement includes regular evaluations and follow-up to assess treatment effects and safety over a 12-month period.