Post-Marketing Surveillance Study of Tolvaptan in Patients With ADPKD

NCT02847624 | Completed Results

• 1 other identifier: 15600-003
• Study Type: observational
• Target: 1,802
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate the safety and effectiveness of tolvaptan in patients with autosomal dominant polycystic kidney disease (ADPKD) in the real world clinical setting in Japan.

Conditions

Polycystic Kidney, Autosomal Dominant

Outcome Measures

Primary Outcomes (1)

• Estimated Slope of Total Kidney Volume During Pre-administration and Administration

  Total Kidney Volume (TKV) was used as a biomarker to assess the progression of ADPKD. TKV was measured at each participating site based on imaging obtained via ultrasound, CT, or MRI. The Estimated Slope pre-administration was calculated for subjects who had TKV measurements both prior to and on the day of Tolvaptan initiation (baseline). The rate of change in TKV from the pre-treatment measurement date to the baseline was used to determine the Estimated Slope. Since the date of initiating tolvaptan administration is considered the start date of the study, the kidney volume measured prior to administration falls outside the study period (i.e., it is a value from before the study start date). The Estimated Slope during-treatment was calculated for subjects who had bilateral TKV measurements at baseline and during the treatment period. The rate of change in TKV from baseline to the measurement date during treatment was used to determine the Estimated Slope.

  Pre-administration: From the first pre-treatment measurement (up to 12.5 years prior) to the start of tolvaptan. During-treatment: From the start of tolvaptan to the final follow-up (up to 8.0 years later).

Secondary Outcomes (2)

• The Number of Cases in Which ALT Never Exceeded Three Times the Upper Limit of Normal (30 IU/L)

  From the date of Tolvaptan initiation to the earlier of either the date when ALT reached three times the upper limit of normal or the date of Tolvaptan discontinuation (up to 2789 days).

• The Number of Cases in Which AST Never Exceeded Three Times the Upper Limit of Normal (30 IU/L)

  From the date of Tolvaptan initiation to the earlier of either the date when AST reached three times the upper limit of normal or the date of Tolvaptan discontinuation (up to 2789 days).

Other Outcomes (4)

• Estimated Slope of Estimated Glomerular Filtration Rate (e-GFR) During Pre-administration and Administration

  Pre-administration: From the first pre-treatment measurement (up to 10 years prior) to the start of tolvaptan. During-treatment: From the start of tolvaptan to the final follow-up (up to 8.0 years later).

• The Number and Percentage of Adverse Events Observed in Patients Aged 65 Years and Older

  Adverse events were collected during the period from the start date to the end date of tolvaptan administration for each case (max. 8 years).

• Safety in Patients With Advanced ADPKD

  Adverse events were collected during the period from the start date to the end date of tolvaptan administration for each case (max. 8 years).

Study Arms (1)

ADPKD

Drug: tolvaptan

Interventions

tolvaptan DRUG

ADPKD

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

medical institutes all over Japan

You may qualify if:

• diagnosed ADPKD
• total kidney volume of 750 or more

Sponsors & Collaborators

• Otsuka Pharmaceutical Co., Ltd.: lead

Study Sites (1)

Otsuka Pharmaceutical Co., Ltd.

Tokyo, Japan

Location

Related Publications (1)

• Mochizuki T, Muto S, Suzue K, Komaniwa S, Tanaka T, Fukuta Y, Yamashige Y. Safety and efficacy of tolvaptan in real-world Japanese patients with autosomal dominant polycystic kidney disease: final results of SLOW-PKD surveillance. Clin Exp Nephrol. 2025 Jun;29(6):807-817. doi: 10.1007/s10157-025-02634-7. Epub 2025 Feb 14.

  PMID: 39953249 DERIVED

MeSH Terms

Conditions

Polycystic Kidney, Autosomal Dominant

Interventions

Tolvaptan

Condition Hierarchy (Ancestors)

Polycystic Kidney Diseases; Kidney Diseases, Cystic; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ciliopathies; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Benzazepines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Investigator in charge
• Organization: Pharmacovigilance Department Otsuka Pharmaceutical Co., Ltd

komaniwa.satoshi@otsuka.jp

+81-6-6943-7722

Study Officials

• Yasuhiko Fukuta, PhD

  Otsuka Pharmaceutical Co., Ltd.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 25, 2016
• First Posted: July 28, 2016
• Study Start: March 24, 2014
• Primary Completion: September 20, 2022
• Study Completion: September 20, 2022
• Last Updated: December 10, 2025
• Results First Posted: December 10, 2025

Record last verified: 2025-11

Locations

JP (1)