156-13-210: Trial to Compare the Efficacy and Safety of Tolvaptan
Research type
Research Study
Full title
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
IRAS ID
154401
Contact name
Neil Turner
Contact email
Sponsor organisation
Otsuka Pharmaceutical Development & Commercialization, Inc.
Eudract number
2014-000226-38
Duration of Study in the UK
2 years, 4 months, 2 days
Research summary
Otsuka Pharmaceutical Development & Commercialization, Inc. (OPDC) is studying an investigational drug called tolvaptan. Tolvaptan (Samsca®) is a drug approved for use to treat hyponatremia (low amount of sodium or salt in the blood) in the following countries: United States, Europe, Australia, Canada, China, Hong Kong, Indonesia, Japan, Philippines, Republic of Korea, Taiwan, Thailand, and Turkey. It is also approved in Japan for volume overload in heart failure, liver cirrhosis and recently for treatment of autosomal dominant polycystic kidney disease (ADPKD).
ADPKD is a disease that causes kidney cysts (cysts are like fluid-filled balloons), worsening kidney function, blood in the urine, kidney pain, high blood pressure, kidney stones, kidney infections, and cysts in the liver or other parts of the body and sometimes heart or blood vessel abnormalities. For people with ADPKD, the kidneys respond abnormally to the hormone vasopressin that may be involved in cyst development or growth in humans. Tolvaptan interferes with the effect of vasopressin on the kidney, and reduces cyst growth when given to animal models of ADPKD. Its effects will continued to be studied in humans to further test how useful tolvaptan will be in treating ADPKD.
ADPKD is a devastating, progressive disease that places a tremendous burden on patients and their families. Tolvaptan is potentially the first therapy that offers patients a treatment option to slow their disease progression; however, as of January 2014, it has not yet been approved for this indication. The treatment risks are well characterised, manageable, and must be weighed against the consequences of no treatment.
Tolvaptan will be studied in this trial as a possible treatment for ADPKD.
Approximately 1300 people globally will take in this study and it is expected that participation will last for approximately 15 months for the majority of participants.
REC name
Scotland A: Adults with Incapacity only
REC reference
14/SS/1048
Date of REC Opinion
8 Jan 2015
REC opinion
Further Information Favourable Opinion