Polycystic Kidney, Autosomal DominantNCT02847624

Post-Marketing Surveillance Study of Tolvaptan in Patients With ADPKD

An observational study of Polycystic Kidney, Autosomal Dominant, run by Otsuka Pharmaceutical Co., Ltd. at 1 site in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Any age
Conditions the study is for: Polycystic Kidney, Autosomal Dominant
What is being tested?: tolvaptan
How many people: 1,802 participants
Where: 1 siteJapan
Study dates: 24 Mar 2014to 20 Sep 2022
Phase: Observational
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Study population: medical institutes all over Japan

Who may be able to join

diagnosed ADPKD
total kidney volume of 750 or more

Who cannot join

Trial locations and contacts

1 site in 1 country.

Japan1 site

Otsuka Pharmaceutical Co., Ltd.
Tokyo

Other trials in Japan

About this study

Official title: Post-Marketing Surveillance Study of Tolvaptan in Patients With ADPKD in Japan

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
Being tested: tolvaptan
Medical subject headings: Polycystic Kidney, Autosomal Dominant

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

tolvaptanDrug

Study groups in NCT02847624
Study group	Role	What they receive
ADPKD		Not described Receives: Drug: tolvaptan

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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)
Among cases included in the safety analysis, we counted and listed the number of patients whose ALT levels increased after starting tolvaptan but never exceeded three times the upper limit of normal (30 IU/L) at a specific time point (day) during treatment.
Cases in which Tolvaptan treatment was discontinued were considered as dropouts on the day of discontinuation.
The number of participants analyzed in each row of the data table is the same as the Overall Number of Participants Analyzed (n=1,672)
Measured: 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)
Among cases included in the safety analysis, we counted and listed the number of patients whose AST levels increased after starting tolvaptan but never exceeded three times the upper limit of normal (30 IU/L) at a specific time point (day) during treatment.
Cases in which Tolvaptan treatment was discontinued were considered as dropouts on the day of discontinuation.
The number of participants analyzed in each row of the data table is the same as the Overall Number of Participants Analyzed (n=1,672)
Measured: 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).

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

24 Mar 201420 Sep 2022

First posted28 Jul 2016 (estimated)
Study start24 Mar 2014
Primary completion20 Sep 2022
Study completion20 Sep 2022

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Polycystic Kidney, Autosomal DominantStudies of the same condition, open and under way.
More trials run by Otsuka Pharmaceutical Co., Ltd.The sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Observational
Observational model: Cohort
Time perspective: Prospective
Enrolment: 1,802 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02847624
Sponsor's protocol ID: 15600-003
Status last verified: Nov 2025
First submitted: 25 Jul 2016
Last updated: 10 Dec 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
Individual data sharing: Undecided

Publications

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.
PubMed 39953249

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
Hypertension in Children and Young People at Risk of Autosomal Dominant Polycystic Kidney Disease
Autosomal Dominant Polycystic Kidney Disease (ADPKD
Active, not recruitingObservational study
Role of ROS and cAMP-PKA Biomarkers in ADPKD
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A Study to Investigate the Pharmacokinetics of JMKX003142 in Healthy Participants and Participants With Hepatic Impairment
ADPKD (Autosomal Dominant Polycystic Kidney Disease), Renal Edema
RecruitingPhase 1
Absorption, Distribution, Metabolism, and Excretion of 14C-VX-407 After a Single Oral Dose
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
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Rare Disorders, Undiagnosed Disorders

All Polycystic Kidney, Autosomal Dominant trials

Study at a glance

From the registry record, last updated by the sponsor on 10 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Post-Marketing Surveillance Study of Tolvaptan in Patients With ADPKD in Japan
ClinicalTrials.gov ID: NCT02847624
Acronym / sponsor ID: 15600-003
Conditions: Polycystic Kidney, Autosomal Dominant
Study type: Observational
Recruitment status: Completed
Enrolment: 1,802 participants (actual)
Lead sponsor: Otsuka Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
Study start: 24 Mar 2014 (actual)
Primary completion: 20 Sep 2022 (actual)
Study completion: 20 Sep 2022 (actual)
First posted: 28 Jul 2016 (estimated)
Results: Posted 10 Dec 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Study design

Observational model: Cohort
Time perspective: Prospective
Number of arms: 1

Arm	Type	Receives
ADPKD	–	Drug: tolvaptan

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult
Study population: medical institutes all over Japan

Key inclusion criteria (2 listed)

diagnosed ADPKD
total kidney volume of 750 or more

Key exclusion criteria (1 listed)

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
tolvaptan	Drug	ADPKD

Primary outcome

Estimated Slope of Total Kidney Volume During Pre-administration and Administration
Time frame: 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).
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.

2 secondary outcomes

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

Results and safety

Results posted to the registry on 10 Dec 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
ADPKD	1,802	1,672

Primary outcome: Estimated Slope of Total Kidney Volume During Pre-administration and Administration

Time frame: 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).. Mean (Standard Deviation). Unit: %/year

Measure	ADPKD n = 1672
Pre-administration	6.7517 (0.2183)
During administration	3.6811 (0.9514)

Population analysed: Pre-administration: Estimated slope was calculated in cases with both pre-treatment and baseline TKV data. During-treatment: Estimated slope was calculated in cases with both baseline and on-treatment TKV data. Because this study used real-world clinical data, not all patients had both sets of measurements required for either group. As a result, the number of cases included in the pre-administration and during-treatment groups was smaller than the total of 1,672 cases analyzed for efficacy.

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse events were collected during the period from the start date to the end date of tolvaptan administration for each case (max. 8 years).. Other adverse events reported at 2% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
ADPKD	253 of 1,672 (15.1%)	1,086 of 1,672 (65%)	21 of 1,672

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 10 Dec 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 39953249Mochizuki 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.
Individual participant dataSharing planned: Undecided

Related trials for Polycystic Kidney, Autosomal Dominant

RecruitingObservational study
Hypertension in Children and Young People at Risk of Autosomal Dominant Polycystic Kidney Disease
Autosomal Dominant Polycystic Kidney Disease (ADPKD
Active, not recruitingObservational study
Role of ROS and cAMP-PKA Biomarkers in ADPKD
Autosomal Dominant Polycystic Kidney Disease
Not yet recruitingPhase 1
A Study to Investigate the Pharmacokinetics of JMKX003142 in Healthy Participants and Participants With Hepatic Impairment
ADPKD (Autosomal Dominant Polycystic Kidney Disease), Renal Edema
RecruitingPhase 1
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Autosomal Dominant Polycystic Kidney Disease (ADPKD)
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Yasuhiko Fukuta, PhD study director

Otsuka Pharmaceutical Co., Ltd.

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

An observational study of Polycystic Kidney, Autosomal Dominant, run by Otsuka Pharmaceutical Co., Ltd. at 1 site in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 10 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Yes. Results are posted in the registry (first posted 10 Dec 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Estimated Slope of Total Kidney Volume During Pre-administration and Administration (time frame: 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).). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02847624, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Dec 2025; AzTrials last refreshed its copy on 8 Nov 2026.