CompletedNCT01280721Updated Jan 17, 2019Results posted

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 interventional study of tolvaptan in Autosomal Dominant Polycystic Kidney Disease （ADPKD), sponsored by Otsuka Pharmaceutical Co., Ltd.. Completed at 7 sites in Japan. Open to participants aged 23 Years to 53 Years. Per ClinicalTrials.gov, last updated 2019-01-17.

Sponsored by Otsuka Pharmaceutical Co., Ltd. · Phase 3, Interventional, and Treatment

Phase: Phase 3
Study type: Interventional
Enrollment: 135
Allocation: Not applicable
Ages: 23 Years to 53 Years
Sex: All

Study 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).

Conditions studied

Autosomal Dominant Polycystic Kidney Disease （ADPKD)

Browse trials for

In context

Arthrogryposis

85 studies on the registry are indexed under Arthrogryposis; 10 are open to participants now.

This study's enrollment of 135 is above the median of 66 across 63 interventional studies indexed under Arthrogryposis.

Browse Arthrogryposis studies →

Lead sponsor

Otsuka Pharmaceutical Co., Ltd. is the lead sponsor of 140 studies on the registry; 11 are open to participants now.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 23 Years to 53 Years
Sexes eligible: All
Accepts healthy volunteers: No

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

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 design

Phase: Phase 3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrollment: 135 participants (actual)

Study arms

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

Interventions

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

What researchers measure

Primary outcomes

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.
Time frame: 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.
Time frame: 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.
Time frame: Baseline, Month 12, Month 24, and Month 36

Results

Posted Jan 17, 2019

Participant flow

Participant flow — Overall Study
Milestone	Tolvaptan
Started	135
Completed	113
Not completed	22
Withdrew: Adverse event	4
Withdrew: Physician decision	3
Withdrew: Withdrawal by subject	6
Withdrew: Egfr under the withdrawal criteria	7
Withdrew: No tolerability for the lowest dose	2

Outcome measures

PrimaryTotal 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.

Time frame:: Baseline, Month12, Month24, and Month36
Reported as:: Mean · mL

Total Kidney Volume
mL	Baseline	Month 12	Month 24	Month 36
Total Kidney Volume	1812.38 ± 859.21	1878.82 ± 950.60	1934.14 ± 926.79	2214.28 ± 842.87

PrimaryRenal 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.

Time frame:: Baseline, Month12, Month24, and Month36
Reported as:: Mean · mL/min/1.73 m2

Renal Function Test (eGFR)
mL/min/1.73 m2	Baseline	Month 12	Month 24	Month 36
Renal Function Test (eGFR)	61.2 ± 21.7	56.3 ± 21.7	51.8 ± 20.1	43.5 ± 20.3

PrimaryRenal 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.

Time frame:: Baseline, Month 12, Month 24, and Month 36
Reported as:: Mean · mg/L

Renal Function Test (Cys-C)
mg/L	Baselime	Month 12	Month 24	Month 36
Renal Function Test (Cys-C)	1.016 ± 0.421	1.114 ± 0.477	1.129 ± 0.466	1.281 ± 0.461

Adverse events

Collected over Through study completion, 2 to 3 years for major patients. Non-serious events are listed at a 2% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Tolvaptan	0/135 (0%)	20/135 (14.8%)	134/135 (99.3%)

Most frequent serious events

Showing 10 of 25

Most frequent serious events
Event	Tolvaptan
Renal cyst infectionInfections and infestations	4/135
Renal cyst haemorrhageRenal and urinary disorders	2/135
Cardiac failure acuteCardiac disorders	1/135
Angina pectorisCardiac disorders	1/135
IleusGastrointestinal disorders	1/135
Umbilical herniasGastrointestinal disorders	1/135
AppendicitisInfections and infestations	1/135
SepsisInfections and infestations	1/135
Hepatic cyst infectionInfections and infestations	1/135
Ligament ruptureInjury, poisoning and procedural complications	1/135

Most frequent other events

Showing 10 of 93

Most frequent other events
Event	Tolvaptan
ThirstGeneral disorders	104/135
NasopharyngitisInfections and infestations	94/135
PollakiuriaRenal and urinary disorders	77/135
PolyuriaRenal and urinary disorders	52/135
Back painMusculoskeletal and connective tissue disorders	30/135
HyperuricaemiaMetabolism and nutrition disorders	29/135
HeadacheNervous system disorders	26/135
DiarrhoeaGastrointestinal disorders	21/135
Upper respiratory tract inflammationRespiratory, thoracic and mediastinal disorders	21/135
InfluenzaInfections and infestations	20/135

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)	Tolvaptan
<=18 years	0
Between 18 and 65 years	135
>=65 years	0

Age, Continuous
Age, Continuous(years)	Tolvaptan
Mean	42.7 ± 5.9

Sex: Female, Male
Sex: Female, Male(Participants)	Tolvaptan
Female	77
Male	58

Region of Enrollment
Region of Enrollment(participants)	Tolvaptan
Japan	135

Study locations

7 sites

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

References and documents

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. doi: 10.2147/DHPS.S175655. PubMed 29123425 ↗

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Jan 17, 2019, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT01280721
Lead sponsor: Otsuka Pharmaceutical Co., Ltd.
Responsible party: Sponsor
First posted: Jan 21, 2011
Start date: Nov 2010
Primary completion: Aug 2014
Completion: Aug 2014
Results posted: Jan 17, 2019
Last update: Jan 17, 2019

Oversight

Data monitoring committee: No

View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Aug 2018. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Arthrogryposis→

Other studies from this sponsor

Otsuka Pharmaceutical Co., Ltd.

Start the discussion