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Minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), and Membranous nephropathy (MN), generate an enormous individual and societal financial burden, accounting for approximately 12% of prevalent end stage renal disease (ESRD) cases (2005) at an annual cost in the US of more than $3 billion. However, the clinical classification of these diseases is widely believed to be inadequate by the scientific community. Given the poor understanding of MCD/FSGS and MN biology, it is not surprising that the available therapies are imperfect. The therapies lack a clear biological basis, and as many families have experienced, they are often not beneficial, and in fact may be significantly toxic. Given these observations, it is essential that research be conducted that address these serious obstacles to effectively caring for patients. In response to a request for applications by the National Institutes of Health, Office of Rare Diseases (NIH, ORD) for the creation of Rare Disease Clinical Research Consortia, a number of affiliated universities joined together with The NephCure Foundation the NIDDK, the ORDR, and the University of Michigan in collaboration towards the establishment of a Nephrotic Syndrome (NS) Rare Diseases Clinical Research Consortium. Through this consortium the investigators hope to understand the fundamental biology of these rare diseases and aim to bank long-term observational data and corresponding biological specimens for researchers to access and further enrich.
The purpose of this study is to evaluate the safety and tolerability of atacicept in adult and adolescent participants and to measure the effect in reducing proteinuria and preserving renal function.
The morbidity of recurrence of focal segmental glomerulosclerosis (FSGS) and minimal change disease (MCD) after transplant is well-recognized and include contemporary reduction in quality of life, edema, early graft loss and mortality. Efforts to understand its mechanisms and improve its treatment have been limited by small sample sizes in single center studies and misclassification in registry studies. Recent advances in the understanding of the mechanisms of FSGS in the native kidney has reinvigorated the scientific community to develop a collaborative community to advance research into the epidemiology, mechanisms, interventions, and outcomes. The purpose of RESOLVE is to gather a group of people with FSGS and MCD that have had or will have a kidney transplant to create a bank of information and biospecimens so researchers can more effectively study these diseases.
This is a Phase 1/2, first in human, open-label, dose-escalation study to evaluate the safety, tolerability, and clinical activity of a single dose of JAG201 administered via intracerebroventricular (ICV) injection in pediatric and adult participants with SHANK3 haploinsufficiency resulting from SHANK3 loss of function mutations and chromosomal deletions encompassing the SHANK3 gene. Clinical data will be evaluated for safety, tolerability, and preliminary clinical activity of JAG201 in pediatric and adult participants with SHANK3 haploinsufficiency. The pediatric cohorts will start enrolling first and the enrollment for adult cohorts may be initiated at a later timepoint in the study.
The purpose of this study is to collect clinical, laboratory, and patient survey data from patients with Castleman disease to improve understanding, diagnosis, and treatment of the disease.
The goal of this clinical trial is to learn if serial amnioinfusions can improve the chances of survival for fetuses with severe kidney problems that cause low amniotic fluid (anhydramnios). Low amniotic fluid can affect lung development and may lead to serious health issues for the fetus. The main questions this study aims to answer are: * Can serial amnioinfusion increase the chances of survival for these fetuses? * Does this procedure improve chances of survival until dialysis and/or kidney transplant? Participants will: * Receive regular amnioinfusions, which is a procedure that adds fluid to the amniotic cavity. * Undergo monitoring to check the effects on the fetus and mother. This study will help researchers understand if amnioinfusion is a useful treatment for fetal kidney problems and may provide valuable information for similar cases in the future.
This is an adaptive prospective, multi-center, randomized, double-blind, placebo-controlled study to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of WAL0921 in subjects with glomerular kidney disease and proteinuria, including diabetic nephropathy and rare glomerular kidney diseases (primary focal segmental glomerulosclerosis \[FSGS\], treatment-resistant minimal change disease \[TR MCD\], primary immunoglobulin A nephropathy \[IgAN\], and primary membranous nephropathy \[PMN\]). Subjects in this study will be randomized to receive the investigational drug WAL0921 or placebo as an intravenous infusion once every 2 weeks for 7 total infusions. All subjects will be followed for 24 weeks after their last infusion.
To compare the efficacy and safety of remibrutinib versus teriflunomide in patients with relapsing multiple sclerosis (RMS)
To compare the efficacy and safety of remibrutinib versus teriflunomide in patients with relapsing multiple sclerosis (RMS)
To evaluate the safety, efficacy and tolerability of sparsentan oral suspension and tablets, and assess changes in proteinuria after once-daily dosing over 108 weeks.