Clinical Trials

The purpose of this study is to determine whether patients with autosomal dominant polycystic kidney disease (ADPKD) present with abnormal endothelial function, increased levels of NOX4 activity and mitochondrial abnormalities, contributing to oxidative stress from early stages that correlate with disease severity.

The purpose of the study is to evaluate and describe the long term safety of tolvaptan in patients with autosomal dominant polycystic kidney disease.

The overall objective of this study is to define genes causing cystic kidney disease and to determine the degree to which variants at these cystic kidney disease genes underlie the clinical diversity of these disorders. This will provide an insight into the disease mechanisms, and explore novel mutation screening methods.  A combination of mutation screening in clinically characterized human cystic kidney disease populations, gene hunting and exploration of incompletely penetrant alleles in humans will provide a comprehensive picture of how mutations cause the resulting phenotype.

The natural course of autosomal dominant polycystic kidney disease (ADPKD) is highly variable and typically characterized by progressive enlargement of cysts within the kidneys.  ADPKD is a leading cause of end-stage renal disease.  Recently, a new medication called Tolvaptan has been approved by the FDA to slow progression of ADPKD.  Accurate, MR based kidney volume measurements are the best parameter for following the progression of ADPKD, and are suggested be acquired to measure progression and effects of Tolvaptan.  However, making these slice-by-slice volume measurements can be extremely time consuming when done manually.  The purpose of this work is to evaluate an AI algorithm to make the ...

The purpose of this study is to evaluate clinical complications, pattern of kidney function decline and identification of clinical variables related to progression of Autosomal Dominant Polycystic Kidney Disease (ADPKD).

To demonstrate whether tolvaptan modifies ADPKD progression as measured by changes from baseline (from trial 156-04-251) in total kidney volume (TKV) and renal function.

The goal of the study is to compare and evaluate safety and efficacy of tesevatinib 50mg versus placebo in patients with ADPKD.

The purpose of this study is to investigate the biological variation in baseline levels of proteins (uELV PC1 and PC2) in individual Autosomal Dominant Polycystic Kidney Disease (ADPKD) patients within a 14 day period that will enable the further optimization of biomarker assays for measuring uELV PC1 and PC2.

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is a devastating genetic disorder that causes numerous cysts in the kidneys, and remains a leading cause of renal failure. However, the only FDA-approved therapy for ADPKD (tolvaptan) is limited to slowing-down disease progression, and has associated side effects. Furthermore, the mechanisms that contribute to cyst formation and further damage to the kidney are still uncertain. A better understanding of these processes may assist in development of new therapies with fewer adverse effects and improve the quality of life of these patients.

Another critical problem in the care of patients with ...

This research study is about developing a rapid, accurate urine test to help diagnose and perhaps monitor ADPKD (Autosomal Dominant Polycystic Kidney Disease).

The purpose of this study is to test the hypothesis that patients with PKD have abnormal endothelial function and increased oxidative stress from early stages (eGFR > 60 mL/min/m2, BP<130 0="" mmhg),="" and="" these="" correlate="" with="" defects="" in="" isolated="" endothelial="">

This study will develop a complete dataset of outcomes on the largest prospectively and rigorously phenotyped ADPKD cohort with ~20 years of follow-up and accompanying biospecimen repository, which will be a valuable and unique resource for biomarker discovery, validation, and model building for the current proposal and for the greater PKD scientific community.

This study proposes to continue follow-up of the CRISP observational cohort of patients with autosomal dominant polycystic kidney disease (ADPKD), and to follow-up patients from the HALT Study A. Since the goal of this study is to elucidate the natural history of ADPKD in patients, this ...

The purpose of this study is to conduct a controlled, unblinded RCT comparing mild CR to a control diet in patients with rapidly progressive ADPKD (class 1C-E by the lrazabal classification), under conditions of standardized hydration and sodium intake, to determine whether it slows the rate of growth of the kidneys (primary endpoint), is well tolerated and safe, lowers levels of IGF-1 in serum and the excretion of inflammation (MCP-1) and fibrosis (TGF-􀁇) biomarkers in urine, improves kidney texture, slows the decline of GFR and the rate of growth of the liver, and improves the quality of life.

The primary objective of this study is to determine the safety, plasma pharmacokinetics, and maximum tolerated dose (MTD) of KD019 when administered to subjects with ADPKD.

The primary purose of this study is to determine the effect of venglustat on the rate of total kidney volume (TKV) growth and estimated glomerular filtration rate (eGFR) decline in patients at risk of rapidly progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD).

The purpose of this study is to determine whether patients with ADPKD present with abnormal levels of fumarate that correlates with disease severity and this increase in fumarate results in upregulation of Nrf2 signaling leading to increased cellular proliferation and contributing to cystogenesis.

The primary objective of this study is to assess the dose response relationship between RGLS4326 and ADPKD biomarkers.  Secondary objectives of this study are to characterize the pharmacokinetic (PK) properties of RGLS4326 in plasma and urine, and to assess the safety and tolerability of RGLS4326.

The purpose of this study is to evaluate the safety, tolerability, and effectiveness of bardoxolone methyl in qualified patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD).

The primary objective of this study is to evaluate the use of Super-resolution ultrasound (SRU) to assess the intrarenal microvasculature in patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD).

The purpose of this study is to evaluate the pharmacokinetics, pharmacodynamics, safety and tolerability of multiple doses of lixivaptan in Autosomal Dominant Polycystic Kidney Disease subjects with chronic kidney disease in stages CKD1, CKD2 or CKD3.

The purpose of this study is to establish normal Magnetic Resonance quantitative values (tissues stiffness, Apparent Diffusion Coefficient values and Blood Oxygen Level Determination values for both renal cortex and medullary tissues and total renal blood flow) for young Autosomal Dominant Polycystic Kidney Disease patients with normal renal function, and normal young adult controls without Autosomal Dominant Polycystic Kidney Disease and normal renal function.

Hypothesis: Newer Magnetic Resonance quantitative imaging parameters (tissue stiffness, Apparent Diffusion Coefficient, Blood Oxygen Level Determination levels, Magnetization Transfer and renal blood flow) will have different values in young adult ADPKD patients as compared to normal volunteers.

The purpose of this trial will assess the effectiveness and safety of lixivaptan in a broad population of adult participants with Autosomal Dominant Polycystic Kidney Disease (ADPKD).

The vasopressin V2 receptor antagonist lixivaptan has also been shown to ameliorate polycystic disease manifestations in animal models of the disease. Evidence from quantitative systems toxicology modeling and simulation have demonstrated that lixivaptan does not have the same potential for liver injury as tolvaptan. 

The purpose of this study is to assess homocysteine metabolism and endothelial function at early stages of ADPKD and how these influence microvascular function and renal disease severity and progression. 

The purpose of this study is to assess liver safety, non-liver safety, and effectiveness in subjects who previously experienced liver chemistry test abnormalities while treated with tolvaptan and were permanently discontinued from the drug for that reason. 

The purpose of this study is to perform a comprehensive metabolic fingerprinting of pediatric-adolescents patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD) and healthy volunteers.

This research study is being done to create a collection of biospecimens (blood, urine, cyst fluid, bile, kidney or liver tissue, etc.) that can be used to facilitate the conduction of research on polycystic kidney and/or liver disease.

The purpose of ths study is to assess the safety and tolerability of RGLS8429. Addiitionally, to assess the impact of RGLS8429 on ADPKD biomarkers

Other objectives are o assess the impact of RGLS8429 on height-adjusted total kidney volume (htTKV) and to characterize the pharmacokinetic (PK) properties of RGLS8429 -To assess the impact of RGLS8429 on renal function.

The purpose of this study is to characterize urinary crystalluria, supersaturation, cytokines, and extracellular vehicles from activated renal tubular epithelial cells, interstitial monocytes, and pro-and anti-inflammatory macrophages in a cohort of male and female pediatric patients with autosomal dominant polycystic kidney disease (ADPKD). Also, to determine cross-sectional associations between cyst burden/kidney function and urinary crystalluria, supersaturation, cytokines, and extracellular vehicles (EVs).

The purpose of this study is to assess the long term safety, tolerability, and effectiveness of tesevatinib for patients with autosomal dominant polycystic kidney disease, who started and completed 24 months of treatment for previous study KD019-101.

The purpose of this study is to determine the impact of foam sclerotherapy of large, dominant kidney/liver cysts on quality of life outcomes and kidney/liver cyst volumes at up to 12 months of follow-up in patients with autosomal dominant polycystic kidney disease (ADPKD) and autosomal dominant polycystic liver disease (ADPLD).
 

The objectives of this study are to evaluate the effectiveness and safety of PB in the treatment of patients with hereditary nephrogenic diabetes insipidus, to evaluate the effectiveness and safety of PB in polyuric patients with autosomal dominant polycystic kidney disease treated with tolvaptan, and to evaluate the effectiveness and safety of PB in polyuric patients previously treated with lithium.

The purpose of this research study is to improve our understanding of normal liver and gallbladder function and liver and gallbladder diseases including PBC, PSC, ADPKD (Autosomal Dominant Polycystic Kidney Disease), and ARPKD (Autosomal Recessive Polycystic Kidney Disease) and gallbladder inflammation (cholecystitis).