Chagas Disease

Researchers are evaluating the safety, tolerability, and how the body processes (pharmacokinetics) single and multiple intravenous doses of a drug called BWC0977 in healthy adult volunteers. This Phase 1 study involves a total of 64 healthy adults aged 18 to 55 years and is designed as a randomized, double-blind, placebo-controlled trial with multiple dose groups. The study focuses on measuring any treatment-emergent adverse events and serious adverse events to understand the drug's safety profile. Participants will be divided into two main groups: single ascending dose (SAD) and multiple ascending dose (MAD) cohorts. In the SAD phase, volunteers receive one intravenous infusion of BWC0977 or placebo over 2 hours at doses of 750 mg or 1500 mg. In the MAD phase, participants receive multiple intravenous infusions of BWC0977 or placebo over 30 minutes to 2 hours daily for 7 to 10 consecutive days. Dose levels will increase sequentially based on safety and tolerability data collected during the study. During the study, participants will undergo various assessments including physical exams, vital signs, ECGs, laboratory tests, and blood sampling at specific times before, during, and after infusions to monitor safety and measure drug levels in the body. Researchers will track adverse events for up to 8 days after single dosing and up to 16 days after multiple dosing. Volunteers must comply with study visits and requirements throughout the trial, which lasts until August 2026.

Researchers are evaluating the ability of LXE408 to reduce or clear parasites in the blood of people living with chronic Chagas disease who do not have severe organ dysfunction. This phase 2, randomized, participant- and investigator-blinded study aims to assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of LXE408 compared to placebo and benznidazole. The study focuses on adults with chronic indeterminate Chagas disease without severe cardiac or gastrointestinal symptoms. Participants will be assigned to one of four treatment groups: LXE408 taken orally for 28 days, LXE408 for 14 days followed by placebo for 14 days, placebo for 28 days, or benznidazole for 60 days. This parallel group design allows comparison of LXE408 at different durations against placebo and an active standard-of-care drug. Treatments are all given by mouth, and the study includes a control arm to evaluate safety and tolerability alongside efficacy. During the study, participants will have blood tested by polymerase chain reaction (PCR) at months 2, 4, 6, and 12 to measure parasite levels and confirm clearance. Additional assessments include serology tests, pharmacokinetic blood sampling at multiple time points, and monitoring of adverse events throughout up to 48 months. Researchers will track time to parasite clearance and any treatment side effects while ensuring participants can comply with study visits and follow-up requirements.

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 I/II 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 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 new home-based methods to measure medication and chemical concentrations in sweat and saliva compared to standard blood tests in patients with chronic or infectious diseases who are receiving medications. The study aims to see if a smart wristband can accurately monitor substances like electrolytes and metabolites in sweat, which may help in health monitoring and disease diagnosis. This pilot observational study is focused on assessing the feasibility of these wearable sweat sensors. Participants provide sweat samples using a Macroduct Sweat Collection System, saliva samples, and blood samples within 24 hours after taking their medications. They also complete short questionnaires lasting 5 to 10 minutes and allow their medical records to be reviewed. The study collects samples and data to compare the new home-based methods against the standard liquid chromatography-mass spectrometry of plasma. During the study, patients are followed periodically after completing sample collection to monitor results and assess feasibility. Researchers measure how well the home-based sampling predicts medication levels and observe plasma concentrations within 4 hours. The study includes ongoing follow-up to evaluate the ease of obtaining home-based samples and to review medical data over time.

Chagas disease is a widespread condition in Latin America caused by the parasite Trypanosoma cruzi, affecting millions and leading to chronic Chagas cardiomyopathy (CCC) in 30-50% of cases. This serious heart condition often results in heart failure and high rates of illness and death. The trial investigates whether cardiac contractility modulation (CCM) devices provide better clinical and functional benefits compared to cardiac resynchronization therapy (CRT) for patients with advanced heart failure and CCC who do not have left bundle branch block. The study involves two groups: one receiving CCM device implantation and the other undergoing CRT implantation. CCM delivers high-tension electrical stimulation to improve heart muscle contraction during a specific phase of the heartbeat. The trial compares these two device-based treatments to evaluate their impact on heart function and patient quality of life. Participants will be monitored over six months with assessments including quality of life scores, heart strain measurements, functional capacity tests like the 6-minute walk test, heart failure classification, and hospitalization records. The study tracks these measures at the start and after six months to determine the effects of the treatments on heart performance and daily functioning in patients with CCC and severe heart failure.

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 I/II 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 × 10^11 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-A/B 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.

Researchers are evaluating the implementation and effectiveness of continuous wireless monitoring of vital signs with real-time alerts in patients who are either at home or hospitalized. This study focuses on high-risk patients with acute conditions or those undergoing major elective surgery, aiming to improve detection of physiological changes that may indicate clinical deterioration. The study builds on the WARD project, which integrates continuous measurements of multiple physiological signals with artificial intelligence to support clinical decision-making. Participants will have their vital signs continuously monitored using a specially developed app that collects data through a wireless device. The WARD-Clinical Support System (WARD-CSS) uses machine learning algorithms to interpret the data, predict potential health issues, and alert clinical staff via a mobile device with an easy-to-use interface. The study includes patients at home, during hospitalization, and after surgery, assessing how well the system works in these settings. During the study, participants' vital signs data quality and clinical user satisfaction will be tracked over 30 days. The researchers will evaluate how often patients are monitored with sufficient data quality and how satisfied clinical users are with the system. Participants may undergo assessments and monitoring through the app without interfering with their usual care. Safety and ease of use will be important aspects of the study's observations.

Researchers are studying inherited bone marrow failure syndromes and related disorders (IBMFS-RD), which are rare diseases causing serious health problems and early death. This research aims to improve diagnosis by using whole genome sequencing (WGS) and whole transcriptome sequencing (WTS) to better understand these conditions, which are often underdiagnosed due to limited genomic testing. The study will help clarify diagnosis, guide treatment choices, and inform genetic counseling for patients and their families. The study involves performing whole genome and transcriptome sequencing on up to 350 patients suspected of having IBMFS-RD. This observational study will analyze genetic data to increase the diagnostic rate and explore the health and economic impacts of genomic testing. The study will also investigate new ways to classify gene expression and gather data for a registry over a four-year period. Participants will provide samples for genomic analysis and share clinical information during the study. Researchers will review the diagnosis within 3 to 12 months after baseline and assess longer-term outcomes such as cost-effectiveness and health implementation over four years. The study will include genetic counseling and follow-up to monitor diagnostic results and help guide patient care. Participation duration varies as the study collects data and monitors outcomes over several years.