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Showing 1-7 of 7 trials
NCT04247906
The purpose of the EAP is to provide access to hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) patients who may benefit from treatment with narsoplimab based on the medical judgement of their respective physicians.
NCT05148299
The purpose of the study was to assess pharmacokinetics (PK), pharmacodynamics (PD), efficacy, and safety of pegcetacoplan in patients with TA-TMA after HSCT.
NCT07059026
Thrombotic Microangiopathy (TMA) Associated with Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) is a serious complication that is associated with increased morbidity, related to multiple organ failure, with increased mortality in transplant patients. The incidence and evolution of TMA, especially in the adult population, is unclear due to the lack of early systematic screening and clear criteria for its diagnosis. For this reason, we designed this protocol to study the incidence and evolution of TMA Associated with allogeneic HSCT in adult patients from Argentina.
NCT04743804
This study will investigate the efficacy and safety of ravulizumab compared to placebo in adult participants with thrombotic microangiopathy (TMA) associated with a trigger. Participants will be randomized to receive either ravulizumab plus best supportive care or placebo plus best supportive care. The treatment period is 26 weeks followed by a 26-week off-treatment follow-up period.
NCT05743400
Thymoglobulin is widely applied as serotherapy in order to prevent acute graft-versus-host disease (GvHD) and graft rejection in patients undergoing non-Human Leukocyte Antigen (HLA)-identical hematopoietic stem cell transplantations (HSCT), with a delicate balance between prevention of GvHD and the promotion of immune reconstitution. Thymoglobulin is known as a drug with high pharmacokinetic (PK) variability. This variability influences drug exposure, which in turn determines the drug response of pharmacodynamics (PD). In order to maintain efficacy while reducing adverse effects of drugs across the entire age range, identification of the PK/PD relationships and the effect of growth and maturation on the different PK and PD parameters involved are crucial. The investigators hypothesise that a better knowledge of Thymoglobulin PK and its covariates would help to individualise dosage regimen and would improve clinical outcomes, such as GvHD and immune reconstitution. The investigators aim to build a population PK model of Thymoglobulin in order to study PK variability and its covariates. This model will help in optimizing dosage regimen in an individually way.
NCT06065852
The goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research. The purpose of this research is to: * Develop Clinical Guidelines for specific rare kidney diseases. These are written recommendations on how to diagnose and treat a medical condition. * Audit treatments and outcomes. An audit makes checks to see if what should be done is being done and asks if it could be done better. * Further the development of future treatments. Participants will be invited to participate on clinical trials and other studies. The registry has the capacity to feedback relevant information to patients and in conjunction with Patient Knows Best (Home - Patients Know Best), allows patients to provide information themselves, including their own reported quality of life and outcome measures.
NCT00593229
This registry will collect clinical data and store biosamples (seru, plasma, urine, and DNA) annually from pediatric patients with thrombotic mcroangiopathy