Vicente Torres, M.D., Ph.D.

Closed for Enrollment

• A Phase 1b, Multicenter, Open-Label, Adaptive Design Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RGLS4326 Administered via Subcutaneous Injection to Patients with Autosomal Dominant Polycystic Kidney Disease (REGULUS 03) Rochester, Minn.

  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.

• A Phase 2, Open-Label, Multi-Center Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Lixivaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease Rochester, Minn.

  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.

• A Phase 3 Study of the Efficacy and Safety of Lixivaptan in Participants with Autosomal Dominant Polycystic Kidney Disease Consisting of a 1-year Double-blind, Placebo-controlled, Randomized Phase and a 1-year Open-Label Phase: The ACTION Study (ACTION 301) Rochester, Minn.

  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. 

• A Phase 3 Trial of Bardoxolone Methyl in Patients With Autosomal Dominant Polycystic Kidney Disease (FALCON) Rochester, Minn.

  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).

• A Phase 3b, Multi-Center, Open-Label Trial to Evaluate the Long Term Safety of Titrated Immediate-Release Tolvaptan (OPC 41061, 30 mg to 120 mg/Day, Split Dose) in Subjects with Autosomal Dominant Polycystic Kidney Disease Rochester, Minn.

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

• A Phase 3b, Multi-center, Randomized-withdrawal, Placebo-controlled, Double-blind, Parallel-group Trial to Compare the Efficacy and Safety of Tolvaptan (45 to 120 mg/Day, Split-dose) in Subjects With Chronic Kidney Disease Between Late Stage 2 to Early Stage 4 Due to Autosomal Dominant Polycystic Kidney Disease Rochester, Minn.

  The purpose of the study is to determine whether tolvaptan is effective and safe for the treatment of late-stage chronic kidney disease due to autosomal dominant polycystic kidney disease (ADPKD)

• An Extended Access Program to Assess Long Term Safety of Bardoxolone Methyl in Patients With Chronic Kidney Disease (EAGLE) Rochester, Minn.

  The purpose of this study is to assess the long-term safety and tolerability of bardoxolone methyl in qualified patients with chronic kidney disease (CKD) who previously participated in one of the qualifying clinical studies with bardoxolone methyl.

  Patients will remain in the study until bardoxolone methyl is available through commercial channels or until patient withdrawal, whichever is sooner.

• An Open-Label Study of Lixivaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease Who Previously Experienced Abnormal Liver Chemistry Test Results While Receiving Tolvaptan: The ALERT Study (ALERT) Rochester, Minn.

  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. 

• Multi-center, Open-label, Extension Study to Evaluate the Long-term Efficacy and Safety of Oral Tolvaptan Tablet Regimens in Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD) (TEMPO 4/4) Rochester, Minn.

  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.

• Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at Risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) (STAGED-PKD) Rochester, Minn.

  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).

• Randomized Controlled Trial of Caloric Restriction in Autosomal Dominant Polycystic Kidney Disease Rochester, Minn.

  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 Consortium for Radiologic Imaging Studies of Polycystic Kidney Disease (CRISP) 4 (CRISP 4) Rochester, Minn.

  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 can only be achieved through the study of human subjects.

  Through this study, knowledge will be gained about the natural history of the disease, biomarkers that can predict the subsequent course of the disease, and models to predict the risk and timing of development of kidney failure.