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 drugs 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 LXE408 in adults with chronic Chagas disease without severe organ dysfunction. This study is a phase 2, randomized, participant- and investigator-blinded, controlled trial with four treatment groups. The goal is to assess the effectiveness, safety, tolerability, pharmacokinetics, and pharmacodynamics of LXE408 compared to placebo and benznidazole in clearing or reducing parasites in the blood. Participants are randomly assigned to one of four 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. The study monitors parasite clearance using PCR tests at multiple time points, including early clearance at days 7, 14, and 28, and sustained clearance for up to 12 months. Pharmacokinetic measurements of LXE408 are also taken during treatment. Participants will undergo regular assessments including PCR testing for parasite levels at months 2, 4, 6, and 12, serology tests at months 6 and 12, and monitoring for adverse events up to 48 months. Safety evaluations and pharmacokinetic blood samples are collected during treatment. The total study duration extends up to 6 years including long-term follow-up to track treatment impact and participant health.

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 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.

Researchers are studying patients with Chagas cardiomyopathy CCC, a serious heart condition caused by the parasite Trypanosoma cruzi, which affects millions primarily in Latin America and increasingly worldwide due to migration. This trial compares two implantable devices to improve heart function in patients with advanced heart failure, severe systolic dysfunction, and specific electrical heart patterns, focusing on how cardiac contractility modulation CCM compares to cardiac resynchronization therapy CRT. Participants receive either a CCM device implanted to enhance heart muscle contraction or a CRT device designed to improve heart rhythm coordination. The study evaluates the effects of these devices over a six-month period, tracking quality of life and heart performance. CCM works by stimulating the heart muscle during a specific phase of its activity to boost contraction strength, while CRT targets rhythm synchronization. Both groups are monitored to assess clinical and functional responses. Throughout the study, participants undergo assessments including quality of life questionnaires, heart function tests like global longitudinal strain GLS, six-minute walk tests, and heart failure classification evaluations at the start and after six months. Researchers also record hospitalizations related to heart failure during the study. This comprehensive monitoring aims to understand which device better supports patients heart function and daily living over the trial timeline ending in December 2027.

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.

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.

Researchers are evaluating the safety, tolerability, and effectiveness of adding metformin to standard multidrug therapy MDT for patients newly diagnosed with multibacillary leprosy. This double-blind, placebo-controlled Phase 2 trial aims to see if metformin can reduce leprosy reactions, nerve damage, and corticosteroid use while assessing immunological responses. The study is supported by the Leprosy Research Initiative and conducted in Indonesia with participants aged 18 to 65. Participants will be randomly assigned to receive either metformin or a placebo once daily. Metformin dosing starts at 500mg for two weeks, then increases to 1000mg for 22 weeks, alongside 48 weeks of standard MDT. The placebo group receives matching tablets to maintain blinding. The trial plans to enroll 166 patients to compare clinical outcomes between the two groups. During the 48-week study, participants will be regularly monitored for leprosy reactions, adverse events, nerve function, corticosteroid use, and quality of life through clinical assessments and questionnaires. The primary outcomes include the proportion of patients experiencing leprosy reactions and adverse events within specified timeframes. Secondary outcomes focus on reaction severity, timing, frequency, and impact on daily living. Safety and tolerability will also be closely observed throughout the study period.