Active clinical trials for "Polycystic Kidney Diseases"

Triptolide has been approve effective in animal model.

This study will analyze the germline and somatic mutations underlying the development of ADPKD in order to better understand the genetic mechanism responsible for the cystic transformation. Once identified, these mutations could help us understand better the mechanism leading to the development of this disease and may explain at least in part the phenotypic variability.

Primary sarcopenia is used to describe aging and progressed with the physiologic decline. Secondary sarcopenia is associated many chronic disease, including acquired immune deficiency syndrome, cancer, chronic heart failure, chronic lung disease, liver cirrhosis, chronic kidney disease and rheumatoid arthritis. In the past, nutrition status is evaluated by body mass index, mid-upper -arm circumference and serum albumin. Bioelectrical impedance analysis is also a common method to measured body composition, but bioelectrical impedance analysis will be affected by tissue edema and ascites. In contrast, cross-section imaging, such as computed tomography and magnetic resonance can analyzed abdominal muscle and fat accurately. Since computed tomography and magnetic resonance imaging can evaluate the severity of polycystic liver and kidney disease. Investigators can use cross section imaging at 3rd lumber level to separate skeletal muscle and fat tissue. Previous studies showed the quantity and quality of abdominal muscle are important prognostic factor after liver transplantation. Besides, chronic kidney disease and receiving renal placement therapy lead protein catabolism and make patients with end stage renal disease have sarcopenia. Finally, patients with polycystic liver and kidney disease have organomegaly, which causes abdominal distention and poor appetite. Therefore, the aim of this study is to observe the association between skeletal muscle mass and the severity of disease and to study whether change in hepatic and renal volumes is associated with change in muscle mass.

Funding Source - FDA OOPD Pioglitazone is currently used in clinical practice to treat diabetes and this study will examine the potential use of a low dose of the same drug for the treatment of polycystic kidney disease. The purpose of this study is to determine whether the diabetes drug pioglitazone (Actos) is a safe and effective treatment of autosomal dominant polycystic kidney disease when treated in its early stages. Pioglitazone is approved by the FDA for the treatment of diabetes. Pre-clinical models of polycystic kidney disease have shown that low dose treatment with pioglitazone decreases the growth of the cysts. The studies also suggest that effective pioglitazone dosing for polycystic kidney disease may be lower than that used to treat diabetes. The purpose of this study is to see if pioglitazone might slow cyst disease in humans.

DRINK is an open-label randomised controlled feasibility trial of high versus ad libitum water intake in ADPKD.

To evaluate the safety and therapeutic effectiveness of tolvaptan when administered to slow the progression of cyst development and renal function insufficiency in adult Korean patients diagnosed with rapidly progressive ADPKD who have chronic kidney disease (CKD) stages 1-3 at initiation of treatment.

The proposed research will determine the effectiveness of blocking aldosterone for improving the health and function of arteries in patients with autosomal dominant polycystic kidney disease (ADPKD). The study also will provide insight into how blocking aldosterone improves artery health by determining the physiological mechanisms (biological reasons) involved. Overall, the proposed research will provide important new scientific evidence upon which physicians can base recommendations to patients with ADPKD to decrease risk of developing cardiovascular diseases.

Rationale: Polycystic liver disease (PLD) is a rare disorder characterized by >20 fluid-filled hepatic cysts. Polycystic livers are present in the combination with renal cysts as a manifestation of autosomal dominant polycystic kidney disease (ADPKD), or isolated in the absence of renal cysts as autosomal dominant polycystic liver disease (ADPLD or PCLD). PLD patients are confronted with symptoms caused by the mass effect of their polycystic liver every day for the rest of their life. There is no standard therapeutic option for symptomatic PLD patients. Current options are fairly invasive or their efficacy is only moderate. Preliminary data in our research lab have shown that ursodeoxycholic acid (UDCA) inhibited the proliferation of polycystic human cholangiocytes in vitro through the normalization of the intracellular calcium levels in cystic cholangiocytes. The investigators also found that daily oral administration of UDCA for 5 months to polycystic kidney disease (PCK) rats, an animal model of ARPKD that spontaneously develops hepato-renal cystogenesis, resulted in inhibition of hepatic cystogenesis. The investigators hypothesize that UDCA is an effective therapeutic tool in reducing liver volume in PLD. Objective: First, to demonstrate whether UDCA-therapy is effective in reducing total liver volume in PLD patients. Second, the investigators want to assess if UDCA modifies quality of life. Finally, the investigators want to assess safety and tolerability. Study design: International, multicenter, randomized, controlled trial Study population: 34 subjects (18 ≤age ≤ 80 years) suffering from symptomatic polycystic liver disease with underlying diagnosis of (PCLD or ADPKD), defined as ≥ 20 liver cysts on CT-scan and liver volume of ≥ 2500. Symptomatic is defined as Eastern Cooperative Oncology Group- Performance Score (ECOG-PS) ≥ 1 and having at least three out of ten PLD symptoms. Intervention: The patients will be randomized (1:1) into two groups. One group of patients will receive 15-20mg/kg/day UDCA for 24 weeks. The other group will receive standard care. Main study endpoint: Proportional change of total liver volume in UDCA treated patients versus non treated patients, as assessed by CT at baseline and 6 months.

The investigators multicenter randomized open-labelled study will investigate the efficacy of an everolimus based immunosuppression in reducing total native kidney volume in kidney recipients with autosomal dominant polycystic kidney disease compared to a calcineurin inhibitor-based immunosuppression.

The goal of this pilot study is to evaluate the feasibility of administering niacinamide to patients with autosomal dominant polycystic kidney disease, to develop methods to assess the biological efficacy of niacinamide, and to perform a preliminary exploration of its clinical effect on kidney cyst growth and kidney function.