Chagas Disease

Researchers are evaluating the safety, tolerability, and pharmacokinetics of single and multiple intravenous doses of BWC0977 in healthy adult volunteers. This Phase 1 randomized, double-blind, placebo-controlled trial involves 64 healthy participants divided into 8 cohorts. The study is focused on assessing how the body processes BWC0977 and monitoring for any adverse effects during treatment. The condition areas related to this research include infectious diseases and antimicrobial drug resistance. The study has two main phases: a single ascending dose (SAD) phase and a multiple ascending dose (MAD) phase. In the SAD phase, participants in the first two cohorts receive one dose of BWC0977 or placebo. In the MAD phase, participants in cohorts 3 to 7 receive multiple doses daily for 7 to 10 consecutive days. In both phases, doses increase sequentially across cohorts, and dosing is double-blind. Placebo groups are included in each cohort for comparison. Participants will attend multiple study visits for dosing and assessments. Researchers will perform physical examinations, vital signs checks, ECGs, and laboratory tests to monitor safety and tolerability. The primary outcome measured is the incidence of treatment-emergent adverse events, including serious events, during the study periods (up to 8 days for SAD and up to 16 days for MAD). Participants will also be monitored for pharmacokinetics and any other abnormalities. The entire participation includes screening, dosing, and follow-up visits according to the study schedule.

Researchers are evaluating the ability of the drug LXE408 to clear or reduce parasites in the blood of people with chronic Chagas disease who do not have severe organ dysfunction. This phase 2, proof-of-concept, randomized, participant- and investigator-blinded study compares LXE408 to placebo and the standard treatment benznidazole. The study aims to assess the effectiveness, safety, how the drug moves through and affects the body, and tolerability of LXE408 in adults with chronic indeterminate Chagas disease. The study includes four treatment groups with LXE408, placebo, and benznidazole administered orally. Participants receive the assigned treatments according to the study protocol. The study design is parallel group controlled, ensuring participants receive only their assigned treatment. Specific dosing details and treatment duration are outlined in the study plan. Participants will be monitored through visits that include blood tests using polymerase chain reaction (PCR) to check for parasite clearance at 2, 4, and 6 months after treatment starts. Safety and efficacy will be evaluated through clinical assessments, laboratory tests, and participant questionnaires. The study tracks how well participants adhere to the treatment and monitors for any side effects, with follow-up lasting at least six months to observe sustained parasite clearance.

Researchers are investigating the safety, pharmacokinetics (how the drug moves through the body), pharmacodynamics (the effects of the drug on the body), and early effectiveness of OriCAR-017, an anti-GPRC5D CAR-T cell therapy, in adults with relapsed or refractory multiple myeloma. This Phase I/II, open-label, multicenter trial aims to understand how this new cell therapy works in patients who have already received other treatments for their condition. The study includes a Phase I dose escalation stage where up to 18 participants will receive one of three escalating doses of OriCAR-017 as a single intravenous infusion. Following this, a dose expansion phase will enroll 10 to 15 participants to receive the selected dose, and then a Phase II stage will include up to 48 evaluable participants. The treatment is given once, and the study will monitor participants closely for safety and biological effects. Participants will undergo evaluations including safety monitoring, blood tests for pharmacokinetics and pharmacodynamics, and assessments of preliminary treatment response. The main outcomes measured are the maximum tolerated dose and any dose-limiting toxicities within 28 days after infusion. The study involves multiple visits and assessments over time to track these outcomes and ensure participant safety and treatment adherence.

Researchers are exploring a method to restore fertility in men who preserved testicular tissue as prepubertal boys before undergoing treatments that could harm their reproductive cells. This study focuses on men who have no sperm suitable for assisted reproduction despite freezing tissue containing spermatogonial stem cells. The goal is to perform the first autologous transplantation of this frozen testicular tissue to help restore sperm production and fertility. The intervention involves transplanting the patient's own previously frozen testicular tissue back into their body when no usable sperm are found in their semen. This procedure is intended to regenerate spermatogenesis and enable fertility restoration. If sperm are found in the ejaculate, standard fertility treatments like natural conception, intra-uterine insemination, in-vitro fertilization, or intra-cytoplasmic sperm injection may be pursued instead. Participants will be monitored through semen and blood analyses before and after transplantation. The main outcome measured is whether sperm cells are present in the grafted tissue one year after transplantation, at which point the graft will be removed. The study includes follow-up of participants and any children born after the procedure, ensuring safety and effectiveness are carefully evaluated over time.

Researchers are evaluating the ability of a smart wristband and home-based methods to measure chemical concentrations in sweat and saliva compared to traditional blood tests in patients with chronic or infectious diseases who are receiving medications. This study focuses on whether wearable sweat sensors can accurately detect medication and other substance levels despite the challenge of ultralow concentrations in sweat. The goal is to improve health monitoring and disease diagnosis with less invasive sampling techniques. Participants will provide sweat samples using the Macroduct Sweat Collection System, saliva, and blood samples within 24 hours after medication administration. They will also complete questionnaires lasting 5 to 10 minutes and have their medical charts reviewed. The study involves collecting these biospecimens and data to compare the new home-based sampling methods with the standard plasma measurements. Throughout the study, participants will be followed up periodically after completion. Researchers will assess the accuracy of the home-based methods in detecting chemical concentrations within 4 hours and observe plasma concentrations as the gold standard. The study also examines the ease of obtaining home-based samples and includes monitoring through questionnaires and chart reviews to support data collection and safety.

Researchers are investigating cardiac contractility modulation (CCM) as a potential treatment for patients with chronic Chagas cardiomyopathy (CCC), a condition affecting 30-50% of individuals infected with Trypanosoma cruzi. This study compares the effects of CCM to cardiac resynchronization therapy (CRT) in patients with advanced heart failure, severe systolic dysfunction, and non-left bundle branch block (non-LBB). The goal is to evaluate whether CCM provides better clinical and functional outcomes, as current treatments for heart failure in CCC show limited success and lack strong scientific evidence. The study involves implanting a CCM device in one group and using CRT in another. CCM delivers high-tension stimulation to the right ventricular septum to improve heart muscle contractility, potentially enhancing functional capacity. Participants have advanced heart failure with specific heart function criteria, including left ventricular ejection fraction below 35% and intraventricular desynchrony. The trial compares these two device-based treatments in patients meeting these criteria. Participants will be closely monitored for quality of life and myocardial contractility at baseline and after six months. Researchers will assess clinical evolution, including hospitalizations, functional class according to the New York Heart Association, and functional capacity. Various tests and evaluations will be done to measure heart function and patient well-being throughout the study, ensuring comprehensive assessment of the treatments' effects.

Researchers are evaluating the safety of CD7 CAR-T cell therapy followed by allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with relapsed or refractory CD7-positive malignant hematologic diseases. This single-arm, open-label, single-center phase I/II study aims to assess how well this combined treatment works and its risks in this patient group. Participants receive CD7 CAR-T cell injections designed to target their CD7-positive blood cancers. This treatment is used as a bridge to allo-HSCT, a procedure where patients receive stem cells from a donor to help restore healthy blood cells. The study follows patients from before treatment through the infusion of CD7 CAR-T cells and then on to transplantation. During the study, researchers monitor patients closely for adverse events (AEs) and serious adverse events (SAEs) from baseline until 28 days after CD7 CAR-T cell infusion. They also assess patients’ health status and treatment effects, including survival time and organ function. Participants must be followed for at least 12 weeks, and safety and response data are collected to understand the therapy’s impact and risks.

Researchers are evaluating combined platelet transfusion as a treatment for patients with hematologic diseases who need platelet transfusions. This single-arm, open, prospective, non-inferiority clinical trial aims to assess the efficacy of combined platelets, which are designed to allow ABO primary and secondary side compatible transfusions when ABO homotype platelets are not available. The study spans two years and includes adult participants aged 18 to 65 years. Participants receive combined platelet transfusions containing at least 2.5 × 10^11 platelets per therapeutic dose. During the study, ABO homotype platelet transfusions are preferred; however, if these are unavailable, combined platelets that are compatible on both primary and secondary ABO sides are used. The study includes 15 months for enrollment, a 6-month treatment period, and a 3-month follow-up phase after treatment ends. Throughout the study, researchers will evaluate the effectiveness of the combined platelet transfusions from the start of infusion through six months later. Participant involvement includes treatment with combined platelets, monitoring, and assessments during treatment and follow-up to measure outcomes and ensure safety. The total study duration for each participant is approximately 2 years, allowing thorough observation of treatment effects and post-treatment outcomes.

This research evaluates the use of continuous wireless monitoring of vital signs combined with artificial intelligence to improve detection and prediction of clinical deterioration in high-risk patients. It focuses on patients either at home or hospitalized with acute conditions or undergoing major elective surgery lasting more than one hour. The study aims to assess the system's implementation and effectiveness in monitoring patients and providing real-time alerts to clinical staff. Participants will have their vital signs continuously monitored using a new app designed for nurses to track data through a mobile device interface. The system, called WARD-Clinical Support System (WARD-CSS), integrates measurements from 10 physiological parameters and uses machine learning to interpret and predict health changes. This study will compare data quality and clinical user satisfaction between the early and later parts of the trial. During the study, participants' vital signs data quality and user satisfaction with the monitoring system will be assessed over 30 days. Researchers will monitor how well the system detects physiological changes and generates alerts. The study involves patients both at home and during hospitalization, with ongoing evaluations to understand the impact and usability of this wireless monitoring technology in real clinical settings.

Researchers are studying inherited bone marrow failure syndromes and related disorders (IBMFS-RD), which are rare diseases causing serious health problems and early death. These conditions often lack clear genetic explanations, and genomic testing is not widely accessible. The study aims to improve diagnosis by using whole genome sequencing (WGS) and whole transcriptome sequencing (WTS) to better understand these diseases and their genetic causes in a group of patients in Australia. The study involves performing whole genome and transcriptome sequencing on up to 350 patients suspected of having IBMFS-RD. This approach helps identify genetic mutations to provide a more precise diagnosis, differentiate inherited causes from acquired conditions, and guide treatment decisions including stem cell transplant options. Researchers also aim to evaluate the economic impact and challenges of applying these genomic tests in clinical care. Participants will undergo genetic testing and their health data will be monitored to confirm an IBMFS-RD diagnosis within 3 to 12 months after starting the study. The study collects detailed clinical information, and families may receive genetic counseling based on the results. The research seeks to improve diagnosis accuracy and treatment planning for patients and their relatives, with ongoing follow-up and data collection to assess outcomes and broader effects of genomic testing.