NCT02251275

Brief Summary

The purpose of the trial was to evaluate and describe the long term safety of tolvaptan in participants with autosomal dominant polycystic kidney disease (ADPKD).

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
1,803

participants targeted

Target at P75+ for phase_3

Timeline
Completed

Started Oct 2014

Typical duration for phase_3

Geographic Reach
20 countries

210 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 25, 2014

Completed
4 days until next milestone

First Posted

Study publicly available on registry

September 29, 2014

Completed
18 days until next milestone

Study Start

First participant enrolled

October 17, 2014

Completed
4.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 9, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 9, 2018

Completed
1 year until next milestone

Results Posted

Study results publicly available

November 27, 2019

Completed
Last Updated

November 27, 2019

Status Verified

November 1, 2019

Enrollment Period

4.1 years

First QC Date

September 25, 2014

Results QC Date

November 7, 2019

Last Update Submit

November 7, 2019

Conditions

Keywords

ADPKD

Outcome Measures

Primary Outcomes (1)

  • Number Of Participants Reporting Treatment-Emergent Adverse Events (TEAEs)

    An adverse event (AE) was as any untoward medical occurrence associated with the use of an investigational medicinal product (IMP), whether or not considered IMP related. A TEAE was an AE that started after trial drug treatment; or if the event was continuous from baseline and was serious, related to IMP, or resulted in death, discontinuation, interruption or reduction of trial therapy. A serious TEAE included any event that resulted in: death, life-threatening, persistent or significant incapacity, substantial disruption of ability to conduct normal life functions, required inpatient hospitalization, prolonged hospitalization, congenital anomaly/birth defect, or other medically significant events as per medical judgment, that jeopardized the participant and that required medical or surgical intervention. A severe TEAE was an inability to work or perform normal daily activity. A summary of serious and all other non-serious TEAEs, regardless of causality, is located in the AE section.

    Baseline through end of treatment (up to 42 months) and follow-up 7 days posttreatment(+ 7 days)

Study Arms (1)

Tolvaptan

EXPERIMENTAL

Tolvaptan was self-administered orally as split-dose regimens. The dose regimens used in this trial were 15/15 milligram (mg), 30/15 mg, 45/15 mg, 60/30 mg, or 90/30 mg. Starting doses were dependent upon the participant's previous trial as follows: * Trial 156-13-210: initiated on tolvaptan at a split-dose of 45/15 mg with upward titration every 3 to 4 days to 60/30 mg or 90/30 mg per day according to tolerability. * Trial 156-08-271: retained the last dose level of tolvaptan received in the trial (45/15 mg, 60/30 mg, or 90/30 mg) and started at that same dose in Trial 156-13-211. * Other Trials (156-04-251 and 156-09-290): initiated on tolvaptan at a split-dose of 45/15 mg with upward titration every 3 to 4 days to 60/30 mg or 90/30 mg per day according to tolerability.

Drug: Tolvaptan

Interventions

Tolvaptan tablets (15 or 30 mg) self-administered orally as split-dose regimens, once upon awakening and another approximately 8 to 9 hours later

Also known as: OPC-41061
Tolvaptan

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Male and female participants ≥ 18 years with confirmed diagnosis of ADPKD (during participation in prior tolvaptan trials) who have completed and transferred from the double-blind Trial 156-13-210 (12-month period including post treatment follow-up, regardless of whether this was on-treatment or off-treatment), or completed Trial 156-08-271 or a prior tolvaptan trial, or interrupted or discontinued treatment in a prior tolvaptan ADPKD trial other than Trial 156-13-210. Participants may be enrolled with the medical monitor approval, and additional close monitoring may be required at the beginning of the trial.
  • eGFR ≥ 20 milliliter (mL)/minute (min)/1.73 meter squared (m\^2) within 3 months prior to the baseline visit. Participants who have an eGFR ≤ 20 mL/min/1.73 m\^2 may be enrolled with medical monitor approval.

You may not qualify if:

  • Need for chronic diuretic use
  • Hepatic impairment based on liver function abnormalities other than that expected for ADPKD with cystic liver disease
  • Women of childbearing potential who do not agree to practice 2 different methods of birth control or remain abstinent during the trial and for 30 days after the last dose of investigational medicinal product (IMP)
  • Women who are breast-feeding and/or who have a positive pregnancy test result prior to receiving IMP.
  • Participants with contraindications to required trial assessments (contraindications to optional assessments, for example, magnetic resonance imaging \[MRI\] are not a limitation).
  • Participants who in the opinion of the investigator or the medical monitor, have a medical history or medical finding inconsistent with safety or trial compliance

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (210)

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Birmingham, Alabama, 35233, United States

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Huntsville, Alabama, 35805, United States

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Mobile, Alabama, 36617, United States

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Phoenix, Arizona, 85381, United States

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Tempe, Arizona, 85284, United States

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Tucson, Arizona, 85745, United States

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La Jolla, California, 92037, United States

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Los Angeles, California, 90022, United States

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Los Angeles, California, 90095, United States

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San Francisco, California, 94143, United States

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Stanford, California, 94305, United States

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Aurora, Colorado, 80045, United States

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Denver, Colorado, 80204, United States

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Denver, Colorado, 80210, United States

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New Haven, Connecticut, 06520, United States

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Hudson, Florida, 34667, United States

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Jacksonville, Florida, 32216, United States

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Miami, Florida, 33150, United States

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Ocala, Florida, 34471, United States

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Port Charlotte, Florida, 33952, United States

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Tampa, Florida, 33614, United States

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Atlanta, Georgia, 30322, United States

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Augusta, Georgia, 30909, United States

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Meridian, Idaho, 83642, United States

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Chicago, Illinois, 60611, United States

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Chicago, Illinois, 60637, United States

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Kansas City, Kansas, 66160, United States

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Wichita, Kansas, 67214, United States

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Baton Rouge, Louisiana, 70808, United States

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Baltimore, Maryland, 21201, United States

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Baltimore, Maryland, 21224, United States

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Greenbelt, Maryland, 20770, United States

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Rockville, Maryland, 20850, United States

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Boston, Massachusetts, 02111, United States

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Springfield, Massachusetts, 01107, United States

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Detroit, Michigan, 48202, United States

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Grand Rapids, Michigan, 49506, United States

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Kalamazoo, Michigan, 49007, United States

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Pontiac, Michigan, 48341, United States

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Roseville, Michigan, 48066, United States

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Rochester, Minnesota, 55905, United States

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St Louis, Missouri, 63110, United States

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Las Vegas, Nevada, 89106, United States

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Reno, Nevada, 89511, United States

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Eatontown, New Jersey, 07724, United States

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Voorhees Township, New Jersey, 08043, United States

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Buffalo, New York, 14219, United States

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Laurelton, New York, 11413, United States

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Mineola, New York, 11501, United States

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New York, New York, 10021, United States

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New York, New York, 10032, United States

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Asheville, North Carolina, 28801, United States

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Chapel Hill, North Carolina, 27599, United States

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Charlotte, North Carolina, 28207, United States

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Winston-Salem, North Carolina, 27103, United States

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Fargo, North Dakota, 58102, United States

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Akron, Ohio, 44302, United States

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Cincinnati, Ohio, 45206, United States

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Cincinnati, Ohio, 45229, United States

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Cleveland, Ohio, 44106, United States

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Columbus, Ohio, 43210, United States

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Portland, Oregon, 97210, United States

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Bethlehem, Pennsylvania, 18017, United States

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Doylestown, Pennsylvania, 18901, United States

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Philadelphia, Pennsylvania, 19104, United States

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Charleston, South Carolina, 29425, United States

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Columbia, South Carolina, 29203, United States

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Orangeburg, South Carolina, 29118, United States

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Knoxville, Tennessee, 37923, United States

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Nashville, Tennessee, 37205, United States

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Nashville, Tennessee, 37232, United States

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Arlington, Texas, 76015, United States

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Houston, Texas, 77004, United States

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Houston, Texas, 77030, United States

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McAllen, Texas, 78503, United States

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Burlington, Vermont, 05401, United States

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Arlington, Virginia, 22207, United States

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Charlottesville, Virginia, 22908, United States

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Norfolk, Virginia, 23507, United States

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Wenatchee, Washington, 98801, United States

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Morgantown, West Virginia, 26506, United States

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Ciudad Autonoma de Buenos Aires, Buenos Aires, C1093AAS, Argentina

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Ciudad Autonoma de Buenos Aires, Buenos Aires, C1425APQ, Argentina

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Ciudad Autonoma de Buenos Aires, Buenos Aires, C1429BWN, Argentina

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Ciudad Autonoma de Buenos Aires, Buenos Aires, C1431FWO, Argentina

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Junín, Buenos Aires, 6000, Argentina

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Pergamino, Buenos Aires, B2700CPM, Argentina

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Pilar, Buenos Aires, B1629ODT, Argentina

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Córdoba, X5000JHQ, Argentina

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Córdoba, X5016KEH, Argentina

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Córdoba, X5166BPW, Argentina

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Camperdown, New South Wales, 2050, Australia

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New Lambton Heights, New South Wales, 2305, Australia

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St Leonards, New South Wales, 2065, Australia

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Westmead, New South Wales, 2145, Australia

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Woolloongabba, Queensland, 4102, Australia

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Adelaide, South Australia, 5000, Australia

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Launceston, Tasmania, 7250, Australia

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Parkville, Victoria, 3050, Australia

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Reservoir, Victoria, 3073, Australia

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Richmond, Victoria, 3121, Australia

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Perth, Western Australia, 6000, Australia

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Aalst, 9300, Belgium

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Brussels, 1090, Belgium

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Brussels, 1200, Belgium

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Edegem, 2650, Belgium

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Ghent, 9000, Belgium

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Kortrijk, 8500, Belgium

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Leuven, 3000, Belgium

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Liège, 4000, Belgium

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Edmonton, Alberta, T6G 2B7, Canada

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Mount Pearl, Newfoundland and Labrador, A1N 3J5, Canada

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Scarborough Village, Ontario, M1H 3G4, Canada

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Toronto, Ontario, M4C 5T2, Canada

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Toronto, Ontario, M5G 2N2, Canada

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Montreal, Quebec, H4J 1C5, Canada

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Brno, 625 00, Czechia

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České Budějovice, 370 87, Czechia

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Hradec Králové, 500 05, Czechia

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Jihlava, 586 01, Czechia

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Jilemnice, 51401, Czechia

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Liberec, 460 63, Czechia

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Ostrava, 708 52, Czechia

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Prague, 128 08, Czechia

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Prague, 140 21, Czechia

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Aalborg, 9100, Denmark

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Aarhus N, 8200, Denmark

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Holstebro, 7500, Denmark

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Heidelberg, Baden-Wurttemberg, 69120, Germany

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Munich, Bavaria, 81675, Germany

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Nuremberg, Bavaria, 90471, Germany

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Wiesbaden, Hesse, 65191, Germany

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Düsseldorf, North Rhine-Westphalia, 40210, Germany

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Essen, North Rhine-Westphalia, 45147, Germany

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Dresden, Saxony, 01307, Germany

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Berlin, 10117, Germany

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Budapest, 1032, Hungary

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Pécs, 7623, Hungary

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Szeged, 6720, Hungary

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Ashkelon, 78278, Israel

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Jerusalem, 9112001, Israel

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Nahariya, 2210001, Israel

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Petah Tikva, 491004, Israel

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Ramat Gan, 52621, Israel

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Tel Aviv, 6423906, Israel

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Montichiari, Brescia, 25018, Italy

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Bari, 70124, Italy

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Lecco, 23900, Italy

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Milan, 20122, Italy

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Milan, 20132, Italy

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Modena, 41124, Italy

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Napoli, 80138, Italy

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Pavia, 27100, Italy

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Amsterdam, 1081 HV, Netherlands

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Groningen, 9713 GZ, Netherlands

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Nijmegen, 6525 GA, Netherlands

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Bergen, 5021, Norway

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Ciechanów, 06-400, Poland

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Gdansk, 80-952, Poland

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Golub-Dobrzyń, 87-400, Poland

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Krakow, 31-559, Poland

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Lodz, 92-213, Poland

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Lodz, 93-347, Poland

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Lublin, 20-954, Poland

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Szczecin, 70-111, Poland

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Warsaw, 04-749, Poland

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Wroclaw, 50-556, Poland

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Bucharest, 010731, Romania

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Bucharest, 011794, Romania

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Bucharest, 022328, Romania

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Oradea, 410469, Romania

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Kemerovo, 650002, Russia

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Krasnoyarsk, 660062, Russia

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Saint Petersburg, 19401344, Russia

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Saint Petersburg, 197022, Russia

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Yaroslavl, 150062, Russia

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Pretoria, Gauteng, 0002, South Africa

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Durban, KwaZulu-Natal, 4001, South Africa

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Cape Town, Western Cape, 7925, South Africa

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Barcelona, 08907, Spain

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Ciudad Real, 13005, Spain

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Madrid, 28041, Spain

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Madrid, 28046, Spain

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Valencia, 46017, Spain

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Gothenburg, 413 45, Sweden

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Linköping, 58185, Sweden

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Stockholm, 14186, Sweden

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Stockholm, 17176, Sweden

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Uppsala, 75185, Sweden

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Belfast, County Antrim, BT9 7AB, United Kingdom

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Exeter, Devon, EX2 5DW, United Kingdom

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Hull, East Riding Of Yorkshire, HU3 2JZ, United Kingdom

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Brighton, East Sussex, BN2 5BE, United Kingdom

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London, Greater London, NW3 2QG, United Kingdom

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London, Greater London, SE5 9RS, United Kingdom

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London, Greater London, SW17 0QT, United Kingdom

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Manchester, Greater Manchester, M13 9WL, United Kingdom

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Salford, Greater Manchester, M6 8HD, United Kingdom

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Stevenage, Hertfordshire, SG1 4AB, United Kingdom

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Inverness, Highland Region, IV2 3UJ, United Kingdom

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Leicester, Leicestershire, LE5 4PW, United Kingdom

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Edinburgh, Lothian Region, EH16 4SA, United Kingdom

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Liverpool, Norfolk, L7 8XP, United Kingdom

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Sheffield, South Yorkshire, S5 7AU, United Kingdom

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Stoke-on-Trent, Staffordshire, ST4 7LN, United Kingdom

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Newcastle upon Tyne, Tyne & Wear, NE7 7DN, United Kingdom

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Coventry, Warwickshire, CV2 2DX, United Kingdom

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Birmingham, West Midlands, B15 2GW, United Kingdom

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Middlesbrough, TS4 3BW, United Kingdom

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Swansea, SA6 6NL, United Kingdom

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Related Publications (3)

  • Chebib FT, Zhou X, Garbinsky D, Davenport E, Nunna S, Oberdhan D, Fernandes A. Tolvaptan and Kidney Function Decline in Older Individuals With Autosomal Dominant Polycystic Kidney Disease: A Pooled Analysis of Randomized Clinical Trials and Observational Studies. Kidney Med. 2023 Apr 14;5(6):100639. doi: 10.1016/j.xkme.2023.100639. eCollection 2023 Jun.

  • Alpers DH, Lewis JH, Hunt CM, Freston JW, Torres VE, Li H, Wang W, Hoke ME, Roth SE, Westcott-Baker L, Estilo A. Clinical Pattern of Tolvaptan-Associated Liver Injury in Trial Participants With Autosomal Dominant Polycystic Kidney Disease (ADPKD): An Analysis of Pivotal Clinical Trials. Am J Kidney Dis. 2023 Mar;81(3):281-293.e1. doi: 10.1053/j.ajkd.2022.08.012. Epub 2022 Sep 30.

  • Torres VE, Chapman AB, Devuyst O, Gansevoort RT, Perrone RD, Lee J, Hoke ME, Estilo A, Sergeyeva O. Multicenter Study of Long-Term Safety of Tolvaptan in Later-Stage Autosomal Dominant Polycystic Kidney Disease. Clin J Am Soc Nephrol. 2020 Dec 31;16(1):48-58. doi: 10.2215/CJN.10250620. Epub 2020 Dec 29.

MeSH Terms

Conditions

Polycystic Kidney, Autosomal Dominant

Interventions

Tolvaptan

Condition Hierarchy (Ancestors)

Polycystic Kidney DiseasesKidney Diseases, CysticKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCiliopathiesGenetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

BenzazepinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic Compounds

Results Point of Contact

Title
Global Clinical Development
Organization
Otsuka Pharmaceutical Development & Commercialization, Inc.

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 25, 2014

First Posted

September 29, 2014

Study Start

October 17, 2014

Primary Completion

November 9, 2018

Study Completion

November 9, 2018

Last Updated

November 27, 2019

Results First Posted

November 27, 2019

Record last verified: 2019-11

Data Sharing

IPD Sharing
Will share

Anonymized Individual participant data (IPD) that underlie the results of this study will be shared with researchers to achieve aims pre-specified in a methodologically sound research proposal. Small studies with less than 25 participants are excluded from data sharing.

Shared Documents
STUDY PROTOCOL, SAP, CSR
Time Frame
Data will be available after marketing approval in global markets, or beginning 1-3 years following article publication. There is no end date to the availability of the data.
Access Criteria
Otsuka will share data on an Otsuka-owned remotely accessible data sharing platform with Python and R analytical software. Research requests should be directed to clinicaltransparency@Otsuka-us.com

Locations