Currently, kidney disease affects up to 20% of the US population and is a strong contributor to morbidity and mortality in these patients. Specific therapies and diagnostic tools for kidney disease have been very slow to develop because of the absence of high quality samples and data that can be used for research studies. The purpose of this study is to develop a registry and sample bank called Poseidon (Prevention, Optimizing Safety, Early Intervention and DetectiOn in Nephrology) biobank that can be used for future research. In this study, patients can consent to donating their residual kidney tissue if they undergo a kidney biopsy and blood or urine during routine clinical care procedures. We will also collect blood and urine outside of clinical care.This study will allow biomedical research the ability to obtain specimens from patients with relevant diseases and appropriate clinical data. This study will collect patient demographic, clinical and historical data for a registry and patient samples will be deposited in the Poseidon biobank. Patients do not have to be diagnosed with Kidney Disease to be included in this study.
Overwhelming evidence exists that some types of proteinuric kidney diseases are caused by the factor(s) present in patients' blood and body fluids. Identification and characterization of such factor(s) would greatly help in better and noninvasive diagnosis of such conditions, development of better therapeutic options, and potentially revealing underlying pathogenic mechanisms.
Owing to the tremendous capabilities of the proteomics facility within the Division of Nephrology we developed experiments that we think will result in significant improvement in our knowledge of major kidney diseases.
If you have a glomerular disease, have had kidney biopsy in the last 5 years, are not on dialysis, and have not had a kidney transplant or was previously enrolled in the NEPTUNE study, you may be eligible to take part in this research study. The purpose of this study is to gather a group of patients with glomerular disease to create a source of information along with your kidney biopsy slides, blood, and urine samples so that researchers can easily and effectively study glomerular disease.
A device called the "Liposorber LA-15 System" has been approved by the
United States Food and Drug Administration for treating kids with focal
segmental glomerulosclerosis (FSGS). The "Liposorber LA-15 System" can only be used if other treatment options, like drugs, don't work or can't be used,
but the kidneys are still working okay. It can also be used if the subject
has had a kidney transplant and the FSGS comes back after the
transplant. Although the Liposorber System can be used for FSGS, we
are not sure how well the Liposorber System works. So, we are doing this
study to find out how well the treatment works in adults.
In this research study, there will be up to 5 adults who have FSGS
enrolled at MUSC. Subjects will come back for up to 12 treatments over 9
weeks and then 5 visits to their study doctor over the next 2 years.
This randomized, double-blind, placebo-controlled Phase 2/3 adaptive study involves an initial investigational blood test to determine if you have a specific variation related to kidney disease. The investigational blood test is to see if you have changes in your DNA of a gene called APOL1. People who have this gene variation may be at risk of losing their kidney function faster than others. If you have the variants (changes in DNA) you may be eligible to continue participation in the study. If you do not have the variants, you will not be eligible, and the study doctor will discuss your other options with you. If you decide to participate, there will be no cost to you and you will be compensated. This study will start by comparing two doses of VX-147 against placebo in subjects with APOL1-mediated kidney disease for 12 weeks. Subjects in Phase 2 will continue to Phase 3 once a dose for Phase 3 is selected. Then the Phase 3 dose of VX-147 will be evaluated for safety and effectiveness. If you meet the requirements and choose to take part in the study, you will be randomly assigned to a treatment group. You will not know which study treatment group you are assigned to and it is possible that you will receive placebo instead of VX-147. The study includes a screening, treatment, and follow-up period. The study will end after the last patient enrolled has completed 2 years in the study. This means some patients enrolling earlier could be in the study for up to 4 years.