Hemolytic Uremic Syndrome

This research focuses on kidney transplant patients to collect blood samples and clinical data for developing a non-invasive test that detects donor-derived cell-free DNA dd-cfDNA to assess the condition of transplanted kidneys. The study is prospective and multicenter, involving participants who have had a kidney transplant and are undergoing an indication biopsy. The goal is to improve monitoring of the transplanted organs status. Participants will provide whole blood samples at the time of their indication biopsy, before the biopsy procedure itself. Additionally, leftover de-identified retrospective genomic DNA gDNA samples from the kidney donors will be collected for paired analysis. This approach helps researchers study dd-cfDNA in a real-world transplant population. Participants will be involved through blood sample collection and clinical data gathering during their biopsy visits. Researchers will monitor the detection of donor-derived cell-free DNA in whole blood over an 18-month period. The study involves no investigational treatments, focusing on observation and sample analysis. Participation duration and follow-up details align with the biopsy schedule and sample collection requirements.

Congenital thrombotic thrombocytopenic purpura cTTP is a rare inherited blood disorder caused by a genetic problem that affects the bodys ability to produce an enzyme called ADAMTS13. This enzyme normally helps break down a larger protein called von Willebrand Factor vWF. People with cTTP have low ADAMTS13 levels, leading to buildup of large vWF forms that cause blood clots in small vessels, potentially blocking blood flow to vital organs. Researchers are studying patients with cTTP treated with Adzynma, a lab-made human ADAMTS13 protein, to better understand its safety and risks. This observational study collects and reviews medical records retrospectively for about 5 years from children and adults with cTTP who have received Adzynma treatment in routine clinical practice. The study focuses on risks of developing neutralizing antibodies against Adzynma and allergic reactions within 6 months and 7 days after initial treatment, respectively. It also aims to gather longer-term safety information and monitor pregnancy and infant outcomes in women treated with Adzynma during pregnancy. Participants medical data related to Adzynma treatment will be reviewed, including records of hypersensitivity reactions, antibody development, treatment-emergent adverse events, and pregnancy outcomes. The study evaluates these safety outcomes up to 5.5 years after treatment. No new treatments are given as part of the study. The research team monitors real-world safety data to better understand Adzynmas effects over time in patients with cTTP.

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 studying recombinant ADAMTS13, a treatment given by intravenous injection, for people with congenital thrombotic thrombocytopenic purpura cTTP in Japan. The main goal is to monitor side effects and see if this treatment helps prevent or improve cTTP. The study is observational, meaning the sponsor will not control how participants are treated but will guide how clinics record information. Participants will receive recombinant ADAMTS13 according to their clinics usual care. The study will follow these participants for up to 18 months to observe their treatment experience. During this time, the treatment is given intravenously as part of standard medical practice. Throughout the study, doctors will check for any treatment-related side effects and measure changes in platelet count, ADAMTS13 activity, and ADAMTS13 inhibitor levels. The study will also track the number of TTP events during periodic replacement therapy. Participants health and treatment outcomes will be monitored carefully during the 18-month period.

Researchers are investigating the relationship between the venous excess ultrasound grading system VExUS and important health outcomes in patients admitted to the intensive care unit ICU. The study focuses on how VExUS relates to acute kidney injury, mortality, and length of hospital stay. Additionally, the connection between VExUS and lung ultrasound scores will be explored as a secondary analysis to better understand fluid overload risks in ICU patients. The study is observational and involves monitoring patients admitted to the ICU who are 18 years or older and expected to stay more than 24 hours. There are no experimental treatments in this study instead, ultrasound assessments including VExUS and lung ultrasound scores will be performed to gather data. These ultrasound techniques are used to evaluate how the body handles fluids and to identify patients at risk of fluid overload. Participants will undergo ultrasound assessments and their health outcomes will be tracked for up to 30 days after ICU admission. Researchers will collect data on acute kidney injury rates, mortality, and major adverse kidney events within this period. No experimental interventions are given, and the study aims to observe natural outcomes while using ultrasound tools to improve fluid management understanding in the ICU setting.

This research aims to collect long-term safety and disease progression data on patients diagnosed with atypical hemolytic-uremic syndrome aHUS, including those treated or untreated with the drugs eculizumab or ravulizumab. The study is observational and involves multiple centers and countries, focusing on real-world information after these treatments have been marketed. Participants include patients of any age diagnosed with aHUS, regardless of whether they have identified complement genetic variants or antibodies. The study gathers data without administering new treatments, monitoring patients who may or may not have received eculizumab or ravulizumab. The registry collects information over extended periods to understand safety events and disease course. During the study, researchers track safety-related events over 10 years and the timing of these events within 5 years. Data collection involves reviewing patient health status and disease progression without altering their usual care. The study relies on informed consent and may include minors with appropriate assent. Participation duration varies, with continuous observation to gather comprehensive post-marketing safety data.

Researchers are investigating autologous testicular tissue transplantation as a method to restore fertility in men who had their testicular tissue frozen as prepubertal boys due to cancer or hematological diseases. This approach is considered when no sperm suitable for intra-cytoplasmic sperm injection ICSI is found in the ejaculate despite cryopreservation efforts. The goal is to restore spermatogenesis and fertility by transplanting the preserved tissue back into the patient. The study involves transplanting autologous testicular tissue that was previously frozen during childhood. Men who return for transplantation will first undergo semen and blood analyses. If no usable sperm are found, the transplantation procedure will be performed. Graft removal and histological studies will occur 12 months after grafting. Participants will be followed up with imaging, hormonal, biomarker, and complication assessments at 3, 6, 9, 12, and 15 months post-grafting. Participants will be monitored closely for up to 15 months after transplantation to assess restoration of sperm production and overall fertility. Evaluations include semen analysis for sperm presence in the graft, imaging, hormonal studies, and biomarker assessments at specified intervals. Safety and potential complications will also be tracked. The study aims to provide important information on the feasibility and outcomes of this novel fertility restoration method.

Researchers are evaluating the safety and effectiveness of BCMA-CD19 chimeric antigen receptor CAR-modified T cells in adults with multiple refractory autoimmune diseases that have not responded to other treatments. This open-label, single-center, single-arm Phase 2 study focuses on various autoimmune conditions including systemic lupus erythematosus, Sjogren syndrome, inflammatory myopathy, systemic sclerosis, Behcets disease, ANCA-associated vasculitis, antiphospholipid syndrome, acquired thrombotic thrombocytopenic purpura, and IgG4-related disease. The study aims to assess the complete remission rate and laboratory markers as primary outcomes, along with pharmacokinetics, long-term effectiveness, and safety as secondary outcomes. Participants receive a single intravenous dose of BCMA-CD19 targeted CAR-T cells at 3106 cells per kilogram of body weight, administered 1 to 2 days after preconditioning. The total volume infused ranges from approximately 10 to 70 mL. This single-dose treatment is followed by monitoring for clinical and laboratory improvements to evaluate treatment effects. The study spans up to 52 weeks depending on the specific autoimmune condition being treated. Throughout the study, participants undergo regular evaluations of clinical signs, laboratory tests, and pharmacokinetic assessments to track safety and efficacy. Outcome measures are collected from enrollment through either 24 or 52 weeks depending on the disease. Participants must adhere to stable doses of glucocorticoids and disease-modifying drugs during the trial. The study includes long-term safety monitoring and requires informed consent and contraception use for participants who may have children. Total participation lasts up to about one year from enrollment.

Researchers are evaluating the safety of a treatment involving CD7 CAR-T cells followed by allogeneic hematopoietic stem cell transplantation allo-HSCT for patients with CD7-positive relapsed or refractory malignant hematologic diseases. This single-arm, open-label, phase III study aims to assess safety outcomes and is sponsored by Zhejiang University. The trial focuses on patients with specific types of acute leukemia that are resistant to or have relapsed after standard treatments. Participants will receive an infusion of CD7 CAR-T cells designed to target cancer cells expressing CD7. This treatment is followed by allo-HSCT as a bridging therapy to further support recovery. The study includes monitoring of the CAR-T cell expression, cytokine levels related to CAR-T therapy, and various survival and remission outcomes over time, with evaluations scheduled from weeks to months after treatment. During the study, participants will undergo assessments including adverse event monitoring up to 28 days after CAR-T infusion, laboratory tests, and evaluations of remission and survival status up to two years post-treatment. Researchers will also monitor bone marrow transplantation success and minimal residual disease over extended follow-up periods. The total participation time and detailed evaluation schedules are designed to thoroughly understand the safety and effects of this combined treatment approach.

Researchers are evaluating combined platelet transfusion as a treatment for patients with hematologic diseases who need platelet transfusions. This study is a single-arm, open, prospective, non-inferiority trial aiming to assess whether combined platelet transfusion, which achieves ABO primary and secondary side compatible infusion, is effective and safe compared to traditional ABO homotype platelet transfusion. The study is sponsored by The General Hospital of Western Theater Command and focuses on improving platelet transfusion options when ABO-compatible platelets are not available. Participants will receive combined platelet transfusions when ABO homotype platelets are unavailable during the treatment period. Each therapeutic dose contains no less than 2.5 1011 platelets. The study includes 15 months of enrollment, followed by 6 months of treatment with combined platelets, and then a 3-month follow-up period. The combined platelet transfusion aims to reduce transfusion reaction risks associated with ABO incompatibility by using plasma-reduced or low anti-AB titer platelets. Throughout the study, participants will be monitored for efficacy and safety outcomes from the start of combined platelet infusion to 6 months later. Assessments include evaluation of treatment effectiveness, blood transfusion adverse reactions and events, safety, platelet antibody screening, waiting times, transfusion frequency, and bleeding events during hospitalization. The total participation time spans up to two years, including the treatment and follow-up periods to ensure thorough evaluation of the combined platelet transfusion approach.