A Study to Investigate the Long-term Safety and Efficacy of Tolvaptan in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD) [Extension of Trial 156-04-251 in Japan]

A Multicenter, Open-label Extension Study to Investigate the Long-term Safety and Efficacy of Tolvaptan in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD) [Extension of Trial 156-04-251 in Japan]

ADPKD patients who enrolled in Trial 156-04-251 will receive repeated oral administration of tolvaptan twice daily (morning and evening: 45mg/15mg, 60mg/30mg, or 90mg/30mg).

Study Overview

• Status: Completed
• Conditions: Autosomal Dominant Polycystic Kidney Disease （ADPKD)
• Intervention / Treatment: Drug: tolvaptan

• Study Type: Interventional
• Enrollment (Actual): 135
• Phase: Phase 3

Contacts and Locations

Study Locations

• Japan
  • Chubu Region, Japan
  • Chugoku Region, Japan
  • Hokkaido Region, Japan
  • Kanto Region, Japan
  • Kinki Region, Japan
  • Kyushu Region, Japan
  • Tohoku Region, Japan

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 23 years to 53 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Patients who participated in Trial 156-04-251 in Japan and for whom CRF collection has been completed.
• Patients who completed 3-year repeated administration and who completed the second follow-up visit or patients whose treatment with the trial drug was interupted due to pregnancy and who completed out the second follow-up visit in Trial 156-04-251.
• Patients in whom any adverse events occurring in Trial 156-04-251 were resolved orstabilized and require no further follow-up.

Exclusion Criteria:

• Patients with eGFR of less than 15 mL/min/1.73 m2
• Pregnant, breast-feeding, or possibly pregnant women or women who are planning to become pregnant
• Patients who received any investigational drug other than Tolvaptan within 30 days prior to commencement of administration of tolvaptan
• Any patients who, in the opinion of the principle investigator or subinvestigators, should not participate in the study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: tolvaptan; Repeated oral administration twice daily (morning and evening) at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg.	Drug: tolvaptan; Repeated oral administration twice daily (morning and evening) at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Total Kidney Volume	Measured values of total kidney volume (sum of the volume of the left and right kidneys) during trial period. Twice-daily repeated oral administration of tolvaptan at daily doses of 60 to 120 mg. Number of participants analyzed at each time point represents number of participants with data at the specified time point. Patients who were withdrawn from trial or have no appropriate data (e.g., interruption of medication, protocol deviation, etc.) are excluded.	Baseline, Month12, Month24, and Month36
Renal Function Test (eGFR)	Estimated glomerular filtration rate calculated by Japanese equation for eGFR during trial period. Number of participants analyzed at each time point represents number of participants with data at the specified time point. Patients who were withdrawn from trial or have no appropriate data (e.g., interruption of medication, protocol deviation, etc.) are excluded.	Baseline, Month12, Month24, and Month36
Renal Function Test (Cys-C)	Measured values of serum cystatin C concentration during trial period. Number of participants analyzed at each time point represents number of participants with data at the specified time point. Patients who were withdrawn from trial or have no appropriate data (e.g., interruption of medication, protocol deviation, etc.) are excluded.	Baseline, Month 12, Month 24, and Month 36

Collaborators and Investigators

• Sponsor: Otsuka Pharmaceutical Co., Ltd.

Publications and helpful links

General Publications

• Muto S, Okada T, Yasuda M, Tsubouchi H, Nakajima K, Horie S. Long-term safety profile of tolvaptan in autosomal dominant polycystic kidney disease patients: TEMPO Extension Japan Trial. Drug Healthc Patient Saf. 2017 Oct 25;9:93-104. doi: 10.2147/DHPS.S142825. eCollection 2017. Erratum In: Drug Healthc Patient Saf. 2018 Jul 16;10:67-68.

Study record dates

Study Major Dates

• Study Start: November 1, 2010
• Primary Completion (Actual): August 1, 2014
• Study Completion (Actual): August 1, 2014

Study Registration Dates

• First Submitted: December 6, 2010
• First Submitted That Met QC Criteria: January 19, 2011
• First Posted (Estimate): January 21, 2011

Study Record Updates

• Last Update Posted (Actual): January 17, 2019
• Last Update Submitted That Met QC Criteria: August 15, 2018
• Last Verified: August 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urologic Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Joint Diseases; Musculoskeletal Diseases; Muscular Diseases; Musculoskeletal Abnormalities; Abnormalities, Multiple; Kidney Diseases, Cystic; Ciliopathies; Kidney Diseases; Polycystic Kidney Diseases; Polycystic Kidney, Autosomal Dominant; Arthrogryposis; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Natriuretic Agents; Antidiuretic Hormone Receptor Antagonists; Tolvaptan
• Other Study ID Numbers: 156-10-003; JapicCTI-101362 (Other Identifier: Japan Pharmaceutical Information Center)