Hemolytic Anemia

This NIH-sponsored study aims to characterize three biomarkers derived from 129Xe gas exchange MRI and understand how they change in response to interventions. The study focuses on markers derived from the interaction of 129Xe with pulmonary capillary red blood cells RBCs, specifically RBC transfer MRI, cardiogenic oscillations in 129Xe-RBC signal amplitude, and the 129Xe-RBC chemical shift. The study population includes healthy volunteers, patients scheduled to undergo transfusion or phlebotomy, patients with dyspnea, interstitial lung disease ILD, idiopathic pulmonary fibrosis IPF, non-specific interstitial pneumonias NSIP, chronic hypersensitivity pneumonitis cHP, sarcoid, chronic thromboembolic pulmonary hypertension CTEPH, and acute pulmonary embolism.

Researchers are investigating the minimum effective dose of 131I-apamistamab as part of a conditioning regimen for patients with advanced sickle cell disease SCD undergoing hematopoietic stem cell transplantation. This Phase 1, single-center study focuses on patients aged 12 to 50 years with sickle cell anemia and an available HLA-matched sibling donor. The study aims to replace the typical conditioning regimen, which includes chemotherapy, total body irradiation, and Campath antibody, with 131I-apamistamab to potentially reduce long-term side effects such as secondary cancers, infertility, cataracts, and lung toxicity. Participants will receive 131I-apamistamab as an intravenous infusion approximately ten days before receiving donor stem cell infusion. The dose of 131I-apamistamab will be personalized and prepared in advance, with target dose levels of either 100 mCi or 150 mCi. Additional treatments may include oral sirolimus to suppress immune response and intravenous Campath antibody. Other procedures such as total body irradiation and red blood cell exchange transfusions may also be part of the overall treatment process, following institutional practices. During the study, participants will undergo various assessments including blood tests, hormone level measurements, and imaging scans to monitor drug absorption and treatment effects. Researchers will evaluate outcomes such as graft failure rate 42 days after transplant, event-free survival, engraftment times for neutrophils and platelets, incidence of graft-versus-host disease, transplant-related toxicities and infections, donor cell presence, and immune recovery. Hormonal evaluations related to fertility will be measured at study start and one year post-transplant. The study includes long-term follow-up of up to seven years to assess these outcomes and safety.

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 studying an experimental drug called ALN-CFB for adults with Paroxysmal Nocturnal Hemoglobinuria PNH who continue to have anemia despite treatment with a complement component C5 inhibitor. This study aims to evaluate the safety, tolerability, and initial effectiveness of ALN-CFB compared to a placebo. The study also examines how ALN-CFB affects levels of Complement Factor B protein in the blood and how the drug is processed in the body. Participants will receive either ALN-CFB or a placebo in a randomized, double-blind manner. The study includes a single-ascending dose escalation design to find the appropriate dosing. The protocol will be updated after initial data analysis to describe further parts of the study. Treatment duration and dosing schedules are defined by the study protocol. During the study, participants will undergo regular evaluations including blood tests to measure drug levels and Complement Factor B concentrations, and will be monitored for side effects for up to 365 days. Researchers will assess the occurrence and severity of treatment-emergent adverse events. The study spans several years, with the primary completion expected in late 2029 and final completion by mid-2031.

This research aims to evaluate the long-term safety and effectiveness of CS-101 in participants who previously received CS-101 in an earlier study CS-101-03. It focuses on individuals with beta-thalassemia who underwent treatment with this base-edited autologous hematopoietic stem cell transplantation. The study is led by the Childrens Hospital of Fudan University and monitors participants up to two years after their infusion. Participants in this follow-up study have completed the initial CS-101-03 trial where they received the CS-101 infusion. This current study tracks their health and treatment outcomes for up to two years after the last six-month follow-up visit post-infusion. The study does not introduce new treatments but observes the long-term effects of the prior CS-101 therapy. During the study, participants will undergo evaluations for serious adverse events, death, new cancers or blood disorders, and whether they achieve transfusion independence for at least 12 consecutive months. Researchers will also monitor changes in fetal and total hemoglobin levels, and the genetic modification presence in blood and bone marrow cells. All assessments occur from the time of consent through two years post-infusion, with safety and efficacy carefully observed throughout this period.

This research follows patients with Hemoglobinopathy and Sickle Cell Disease who previously received BEAM-101, a gene-edited cell treatment, in an earlier study. The trial is a long-term observational follow-up to monitor safety and health outcomes over an extended period. It aims to gather important information about the long-term effects and risks after receiving BEAM-101 treatment. Participants in this study have received a single dose of BEAM-101 by intravenous infusion after a conditioning treatment with busulfan. This follow-up study will track their health for 13 years, adding up to 15 years from the initial treatment. Study visits will happen annually for the first 5 years, then every 3 years until year 11, with a final visit at year 15. Additionally, virtual or phone check-ins will occur every 6 months for the first 5 years, then annually thereafter. During the study, participants will undergo regular safety and efficacy assessments to monitor their health, including how well they remain free of severe sickle cell crises and their blood hemoglobin levels. Researchers will review long-term safety data and mortality over the 13 years. The study includes blood tests and other evaluations at scheduled visits and check-ins. Participation will last up to 15 years, with ongoing monitoring to understand the lasting impact of BEAM-101 treatment.

Researchers are conducting a real-world study to evaluate the impact of iptacopan on adult patients with paroxysmal nocturnal hemoglobinuria PNH in China. The study aims to assess treatment-related outcomes, disease management, and healthcare resource use, considering new standards for PNH care and the introduction of iptacopan. It includes patients who are either new to complement inhibitor therapy or those stable on C5 complement inhibitors like eculizumab. The study involves two patient groups one with PNH patients never treated with complement inhibitors who are starting iptacopan, and another with patients stable on C5 complement inhibitors who will switch to iptacopan after its approval in China. Treatments are oral capsules of LNP023 iptacopan, and patients must start iptacopan within 60 days of consenting. Participants must have documented vaccinations against Neisseria meningitidis and Streptococcus pneumoniae before starting treatment. Participants will be followed for at least 12 months, with assessments including hemoglobin levels, lactate dehydrogenase LDH, absolute reticulocyte count ARC, bilirubin, PNH clone size, and signs or symptoms related to PNH. Researchers will monitor treatment effects, adverse events, medication use, transfusions, and healthcare visits. Patient fatigue and work productivity will be evaluated using specific questionnaires, with data collected at baseline and regular intervals to understand iptacopans real-world impact on disease and quality of life.

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.

Diffuse large B-cell lymphoma DLBCL is the most common and aggressive subtype of non-Hodgkin lymphoma, with rapid tumor growth and symptoms varying by tumor location. The disease is classified using advanced methods like immunohistochemistry and molecular testing to guide prognosis and personalized treatment. Despite standard immunochemotherapy curing 60-70% of patients, many experience treatment failure or relapse, and options after second-line therapies remain limited. This observational study examines treatment patterns, demographic, and clinical characteristics of DLBCL patients in the Middle East and Africa. It includes two patient groups those newly diagnosed and eligible for treatment, and those with relapsed or refractory DLBCL who have failed previous therapies. The study aims to explore access to novel therapies and understand real-world treatment approaches in these regions. Participants will be observed over 22 months, during which researchers will document treatment regimens, patient risk profiles, and access to new treatments. Data collection includes patient demographics, disease characteristics, and treatment outcomes. The study does not involve experimental treatments but gathers detailed information to inform future care strategies for DLBCL patients.

Researchers are evaluating 9MW3011, a drug being studied in patients with non-transfusion-dependent beta-thalassemia, a condition affecting blood production. This phase Ib, randomized, double-blind, placebo-controlled study aims to assess the safety, tolerability, how the drug moves and acts in the body, and immune responses to 9MW3011. The study involves 40 patients divided into four dose groups to explore these effects carefully. Participants will receive either 9MW3011 or a placebo through intravenous infusion in a 41 ratio within each dose group. The study has multiple ascending dose cohorts to test increasing doses of 9MW3011. This design helps researchers understand the drugs impact at different dose levels and compare it to a placebo under blinded conditions. During the study, participants will be monitored up to day 169 for adverse events, vital signs, physical exams, ECG results, and lab tests to evaluate safety. Researchers will measure drug levels in the blood, effects on iron metabolism markers, immune responses, and liver iron concentration. Participants will be involved in scheduled visits for assessments, tests, and safety monitoring throughout the study duration.