Autosomal Dominant Polycystic Kidney Disease （ADPKD)Phase 3NCT01280721

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 Phase 3 study testing tolvaptan in people with Autosomal Dominant Polycystic Kidney Disease （ADPKD), run by Otsuka Pharmaceutical Co., Ltd. at 7 sites 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

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). Read more about this study

Who is it for?: Ages 23 to 53 years
Conditions the study is for: Autosomal Dominant Polycystic Kidney Disease （ADPKD)
What is being tested?: tolvaptan
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 135 participants
Where: 7 sitesJapan
Study dates: Nov 2010to Aug 2014
Phase: Phase 3
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 23 to 53 years
All sexes
No healthy volunteers
Adult

Who may be able to join

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.

Who cannot join

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

Trial locations and contacts

7 sites in 1 country.

City, postcode or hospital

7 sites in 1 country

Japan7 sites

Study site
Chubu Region
Study site
Chugoku Region
Study site
Hokkaido Region
Study site
Kanto Region
Study site
Kinki Region
Study site
Kyushu Region
Study site
Tohoku Region

Other trials in Japan

About this study

Official title: 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).

Conditions: Autosomal Dominant Polycystic Kidney Disease （ADPKD)
Being tested: tolvaptan

What participants receive

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

tolvaptanDrug
Repeated oral administration twice daily (morning and evening) at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg.

Study groups in NCT01280721
Study group	Role	What they receive
tolvaptan	Experimental	Repeated oral administration twice daily (morning and evening) at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg. Receives: Drug: tolvaptan

What is being measured

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

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.
Measured: 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.
Measured: 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.
Measured: Baseline, Month 12, Month 24, and Month 36

Timeline

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

Nov 2010Aug 2014

First posted21 Jan 2011 (estimated)
Study startNov 2010
Primary completionAug 2014
Study completionAug 2014

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 Autosomal Dominant Polycystic Kidney Disease （ADPKD)Studies 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: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 135 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01280721
Sponsor's protocol ID: 156-10-003
Other IDs: JapicCTI-101362
Status last verified: Aug 2018
First submitted: 6 Dec 2010
Last updated: 17 Jan 2019
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

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.
PubMed 29123425

Study at a glance

From the registry record, last updated by the sponsor on 17 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: 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]
ClinicalTrials.gov ID: NCT01280721
Acronym / sponsor ID: 156-10-003
Conditions: Autosomal Dominant Polycystic Kidney Disease （ADPKD)
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 135 participants (actual)
Lead sponsor: Otsuka Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
Study start: Nov 2010
Primary completion: Aug 2014 (actual)
Study completion: Aug 2014 (actual)
First posted: 21 Jan 2011 (estimated)
Results: Posted 17 Jan 2019

Clinical summary

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

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 design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
tolvaptan	Experimental	Drug: tolvaptanRepeated oral administration twice daily (morning and evening) at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg.

Who may qualify

Ages: 23 to 53 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (3 listed)

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.

Key exclusion criteria (4 listed)

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

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
tolvaptanRepeated oral administration twice daily (morning and evening) at one of three split dose-regimens 45mg/15mg, 60mg/30mg or 90mg/30mg.	Drug	tolvaptan

Primary outcome

Total Kidney Volume
Time frame: Baseline, Month12, Month24, and Month36
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.
Renal Function Test (eGFR)
Time frame: Baseline, Month12, Month24, and Month36
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.
Renal Function Test (Cys-C)
Time frame: Baseline, Month 12, Month 24, and Month 36
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.

Results and safety

Results posted to the registry on 17 Jan 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Tolvaptan	135	113

Primary outcome: Total Kidney Volume

Time frame: Baseline, Month12, Month24, and Month36. Mean (Standard Deviation). Unit: mL

Measure	Baseline n = 135	Month 12 n = 123	Month 24 n = 120	Month 36 n = 27
Value	1812.38 (859.21)	1878.82 (950.60)	1934.14 (926.79)	2214.28 (842.87)

Primary outcome: Renal Function Test (eGFR)

Time frame: Baseline, Month12, Month24, and Month36. Mean (Standard Deviation). Unit: mL/min/1.73 m2

Measure	Baseline n = 108	Month 12 n = 100	Month 24 n = 99	Month 36 n = 22
Value	61.2 (21.7)	56.3 (21.7)	51.8 (20.1)	43.5 (20.3)

Primary outcome: Renal Function Test (Cys-C)

Time frame: Baseline, Month 12, Month 24, and Month 36. Mean (Standard Deviation). Unit: mg/L

Measure	Baselime n = 108	Month 12 n = 100	Month 24 n = 99	Month 36 n = 22
Value	1.016 (0.421)	1.114 (0.477)	1.129 (0.466)	1.281 (0.461)

Adverse events

Time frame: Through study completion, 2 to 3 years for major patients. Other adverse events reported at 2% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Tolvaptan	20 of 135 (14.8%)	134 of 135 (99.3%)	0 of 135

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 17 Jan 2019.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 29123425Muto 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.

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.

The record lists no contact. Use the official record to reach the sponsor.

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

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing tolvaptan in people with Autosomal Dominant Polycystic Kidney Disease （ADPKD), run by Otsuka Pharmaceutical Co., Ltd. at 7 sites 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 3 inclusion and 4 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 17 Jan 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites 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.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 17 Jan 2019). 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?

Total Kidney Volume (time frame: Baseline, Month12, Month24, and Month36). The study lists 3 primary outcomes in all. 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 NCT01280721, 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 17 Jan 2019.