Trial Review

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT02160145




Registration number
NCT02160145
Ethics application status
Date submitted
6/06/2014
Date registered
10/06/2014
Date last updated
8/08/2018

Titles & IDs
Public title
Efficacy and Safety of Tolvaptan in Subjects With Chronic Kidney Disease Between Late Stage 2 to Early Stage 4 Due to Autosomal Dominant Polycystic Kidney Disease
Scientific title
A Phase 3b, Multi-center, Randomized-withdrawal, Placebo-controlled, Double-blind, Parallel-group Trial to Compare the Efficacy and Safety of Tolvaptan (45 to 120 mg/Day, Split-dose) in Subjects With Chronic Kidney Disease Between Late Stage 2 to Early Stage 4 Due to Autosomal Dominant Polycystic Kidney Disease
Secondary ID [1] 0 0
156-13-210
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Chronic Kidney Disease 0 0
Autosomal Dominant Polycystic Kidney Disease 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Renal and Urogenital 0 0 0 0
Kidney disease
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Tolvaptan (OPC-41061)
Treatment: Drugs - Placebo

Experimental: Tolvaptan - Tolvaptan (OPC-41061)

Placebo Comparator: Placebo - Placebo


Treatment: Drugs: Tolvaptan (OPC-41061)
Tolvaptan tablets (15 or 30 mg) will be self-administered orally as split-dose regimens, once upon awakening and another approximately 8 to 9 hours later.

Treatment: Drugs: Placebo
Matching placebo tablets will be self-administered orally as split-dose regimens, once upon awakening and another approximately 8 to 9 hours later
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
The Mean Annualized Change in eGFR From Pretreatment Baseline to Post-treatment Follow-up.
Timepoint [1] 0 0
Pretreatment baseline to post-treatment follow-up (up to 61 weeks).
Secondary outcome [1] 0 0
Mean Annualized Slope of eGFR Change
Timepoint [1] 0 0
Pretreatment baseline to post-treatment follow-up (up to 61 weeks).

Eligibility
Key inclusion criteria
- Male and female subjects with eGFR between 25-65 mL/min/1.73m2 (if aged 18 to55) or
eGFR between 25-44 mL/min/1.73m2 (if aged 56 to <66)

- Tolvaptan naïve

- Diagnosis of ADPKD by modified pei-Ravine criteria 1) 3 cysts per kidney by sonography
or 5 cysts by CT or MRI with family history of ADPKD or 2) 10 cysts per kidney by any
radiologic method and exclusion of other cystic kidney diseases if without family
history
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- 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 prior to
receiving IMP

- Need for chronic diuretic use

- Hepatic impairment or liver function abnormalities other than that expected for ADPKD
with typical cystic liver disease

- Advanced diabetes, evidence of additional significant renal disease, renal cancer,
single kidney, recent renal surgery or acute kidney injury

- Contraindications to required trial assessments

- Medical history or medical findings inconsistent with safety or compliance with trial
assessments

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/05/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
18/04/2017
Sample size
Target
Accrual to date
Final
1370
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,TAS,VIC,WA
Recruitment hospital [1] 0 0
- Camperdown
Recruitment hospital [2] 0 0
- Concord
Recruitment hospital [3] 0 0
- New Lambton Heights
Recruitment hospital [4] 0 0
- St. Leonards
Recruitment hospital [5] 0 0
- Westmead
Recruitment hospital [6] 0 0
- Woolloongabba
Recruitment hospital [7] 0 0
- Adelaide
Recruitment hospital [8] 0 0
- Launceston
Recruitment hospital [9] 0 0
- Parkville
Recruitment hospital [10] 0 0
- Reservoir
Recruitment hospital [11] 0 0
- Richmond
Recruitment hospital [12] 0 0
- Perth
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2139 - Concord
Recruitment postcode(s) [3] 0 0
2305 - New Lambton Heights
Recruitment postcode(s) [4] 0 0
2065 - St. Leonards
Recruitment postcode(s) [5] 0 0
2145 - Westmead
Recruitment postcode(s) [6] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [7] 0 0
5000 - Adelaide
Recruitment postcode(s) [8] 0 0
7250 - Launceston
Recruitment postcode(s) [9] 0 0
3050 - Parkville
Recruitment postcode(s) [10] 0 0
3073 - Reservoir
Recruitment postcode(s) [11] 0 0
3121 - Richmond
Recruitment postcode(s) [12] 0 0
6000 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Colorado
Country [5] 0 0
United States of America
State/province [5] 0 0
Connecticut
Country [6] 0 0
United States of America
State/province [6] 0 0
Florida
Country [7] 0 0
United States of America
State/province [7] 0 0
Georgia
Country [8] 0 0
United States of America
State/province [8] 0 0
Idaho
Country [9] 0 0
United States of America
State/province [9] 0 0
Illinois
Country [10] 0 0
United States of America
State/province [10] 0 0
Kansas
Country [11] 0 0
United States of America
State/province [11] 0 0
Louisiana
Country [12] 0 0
United States of America
State/province [12] 0 0
Maryland
Country [13] 0 0
United States of America
State/province [13] 0 0
Massachusetts
Country [14] 0 0
United States of America
State/province [14] 0 0
Michigan
Country [15] 0 0
United States of America
State/province [15] 0 0
Minnesota
Country [16] 0 0
United States of America
State/province [16] 0 0
Missouri
Country [17] 0 0
United States of America
State/province [17] 0 0
Nevada
Country [18] 0 0
United States of America
State/province [18] 0 0
New Jersey
Country [19] 0 0
United States of America
State/province [19] 0 0
New York
Country [20] 0 0
United States of America
State/province [20] 0 0
North Carolina
Country [21] 0 0
United States of America
State/province [21] 0 0
North Dakota
Country [22] 0 0
United States of America
State/province [22] 0 0
Ohio
Country [23] 0 0
United States of America
State/province [23] 0 0
Oregon
Country [24] 0 0
United States of America
State/province [24] 0 0
Pennsylvania
Country [25] 0 0
United States of America
State/province [25] 0 0
South Carolina
Country [26] 0 0
United States of America
State/province [26] 0 0
Tennessee
Country [27] 0 0
United States of America
State/province [27] 0 0
Texas
Country [28] 0 0
United States of America
State/province [28] 0 0
Vermont
Country [29] 0 0
United States of America
State/province [29] 0 0
Virginia
Country [30] 0 0
United States of America
State/province [30] 0 0
Washington
Country [31] 0 0
United States of America
State/province [31] 0 0
West Virginia
Country [32] 0 0
United States of America
State/province [32] 0 0
Wisconsin
Country [33] 0 0
Argentina
State/province [33] 0 0
Buenos Aires
Country [34] 0 0
Argentina
State/province [34] 0 0
Cordoba
Country [35] 0 0
Belgium
State/province [35] 0 0
Aalst
Country [36] 0 0
Belgium
State/province [36] 0 0
Antwerpen
Country [37] 0 0
Belgium
State/province [37] 0 0
Bruxelles
Country [38] 0 0
Belgium
State/province [38] 0 0
Gent
Country [39] 0 0
Belgium
State/province [39] 0 0
Kortrijk
Country [40] 0 0
Belgium
State/province [40] 0 0
Leuven
Country [41] 0 0
Belgium
State/province [41] 0 0
Liège
Country [42] 0 0
Canada
State/province [42] 0 0
Alberta
Country [43] 0 0
Canada
State/province [43] 0 0
Newfoundland and Labrador
Country [44] 0 0
Canada
State/province [44] 0 0
Ontario
Country [45] 0 0
Canada
State/province [45] 0 0
Quebec
Country [46] 0 0
Czechia
State/province [46] 0 0
Brno
Country [47] 0 0
Czechia
State/province [47] 0 0
Ceske Budejovice
Country [48] 0 0
Czechia
State/province [48] 0 0
Hradec Kralove
Country [49] 0 0
Czechia
State/province [49] 0 0
Jihlava
Country [50] 0 0
Czechia
State/province [50] 0 0
Jilemnice
Country [51] 0 0
Czechia
State/province [51] 0 0
Liberec
Country [52] 0 0
Czechia
State/province [52] 0 0
Ostrava
Country [53] 0 0
Czechia
State/province [53] 0 0
Praha 2
Country [54] 0 0
Czechia
State/province [54] 0 0
Praha 4
Country [55] 0 0
Czechia
State/province [55] 0 0
Tabor
Country [56] 0 0
Denmark
State/province [56] 0 0
Aalborg
Country [57] 0 0
Denmark
State/province [57] 0 0
Aarhus C
Country [58] 0 0
Denmark
State/province [58] 0 0
Holstebro
Country [59] 0 0
Denmark
State/province [59] 0 0
Odense C
Country [60] 0 0
Denmark
State/province [60] 0 0
Viborg
Country [61] 0 0
France
State/province [61] 0 0
Finistere
Country [62] 0 0
France
State/province [62] 0 0
Gironde
Country [63] 0 0
France
State/province [63] 0 0
Haute Garonne
Country [64] 0 0
France
State/province [64] 0 0
Herault
Country [65] 0 0
France
State/province [65] 0 0
Isere
Country [66] 0 0
France
State/province [66] 0 0
Loire
Country [67] 0 0
France
State/province [67] 0 0
Marne
Country [68] 0 0
France
State/province [68] 0 0
Meurthe Et Moselle
Country [69] 0 0
France
State/province [69] 0 0
Rhone
Country [70] 0 0
Germany
State/province [70] 0 0
Baden Wuerttemberg
Country [71] 0 0
Germany
State/province [71] 0 0
Bayern
Country [72] 0 0
Germany
State/province [72] 0 0
Niedersachsen
Country [73] 0 0
Germany
State/province [73] 0 0
Nordrhein Westfalen
Country [74] 0 0
Germany
State/province [74] 0 0
Sachsen
Country [75] 0 0
Hungary
State/province [75] 0 0
Budapest
Country [76] 0 0
Hungary
State/province [76] 0 0
Pecs
Country [77] 0 0
Hungary
State/province [77] 0 0
Szeged
Country [78] 0 0
Israel
State/province [78] 0 0
Ashkelon
Country [79] 0 0
Israel
State/province [79] 0 0
Jerusalem
Country [80] 0 0
Israel
State/province [80] 0 0
Nahariya
Country [81] 0 0
Israel
State/province [81] 0 0
Petach Tikva
Country [82] 0 0
Israel
State/province [82] 0 0
Peta? Tiqwa
Country [83] 0 0
Israel
State/province [83] 0 0
Ramat-Gan
Country [84] 0 0
Israel
State/province [84] 0 0
Tel Aviv
Country [85] 0 0
Italy
State/province [85] 0 0
Ancona
Country [86] 0 0
Italy
State/province [86] 0 0
Brescia
Country [87] 0 0
Italy
State/province [87] 0 0
Bari
Country [88] 0 0
Italy
State/province [88] 0 0
Bologna
Country [89] 0 0
Italy
State/province [89] 0 0
Lecco
Country [90] 0 0
Italy
State/province [90] 0 0
Milano
Country [91] 0 0
Italy
State/province [91] 0 0
Modena
Country [92] 0 0
Italy
State/province [92] 0 0
Napoli
Country [93] 0 0
Italy
State/province [93] 0 0
Pavia
Country [94] 0 0
Netherlands
State/province [94] 0 0
Groningen
Country [95] 0 0
Netherlands
State/province [95] 0 0
Nijmegen
Country [96] 0 0
Norway
State/province [96] 0 0
Bergen
Country [97] 0 0
Norway
State/province [97] 0 0
Oslo
Country [98] 0 0
Norway
State/province [98] 0 0
Stavanger
Country [99] 0 0
Poland
State/province [99] 0 0
Ciechanow
Country [100] 0 0
Poland
State/province [100] 0 0
Golub Dobrzyn
Country [101] 0 0
Poland
State/province [101] 0 0
Kraków
Country [102] 0 0
Poland
State/province [102] 0 0
Lodz
Country [103] 0 0
Poland
State/province [103] 0 0
Lublin
Country [104] 0 0
Poland
State/province [104] 0 0
Warszawa
Country [105] 0 0
Poland
State/province [105] 0 0
Wroclaw
Country [106] 0 0
Poland
State/province [106] 0 0
Lódz
Country [107] 0 0
Puerto Rico
State/province [107] 0 0
San Juan
Country [108] 0 0
Romania
State/province [108] 0 0
Bucuresti
Country [109] 0 0
Romania
State/province [109] 0 0
Oradea
Country [110] 0 0
Russian Federation
State/province [110] 0 0
Krasnoyarsk
Country [111] 0 0
Russian Federation
State/province [111] 0 0
St. Petersburg
Country [112] 0 0
Russian Federation
State/province [112] 0 0
Yaroslavl
Country [113] 0 0
South Africa
State/province [113] 0 0
Gauteng
Country [114] 0 0
South Africa
State/province [114] 0 0
KwaZulu-Natal
Country [115] 0 0
South Africa
State/province [115] 0 0
Western Cape
Country [116] 0 0
Spain
State/province [116] 0 0
Barcelona
Country [117] 0 0
Spain
State/province [117] 0 0
Ciudad Real
Country [118] 0 0
Spain
State/province [118] 0 0
Madrid
Country [119] 0 0
Spain
State/province [119] 0 0
Sevilla
Country [120] 0 0
Spain
State/province [120] 0 0
Valencia
Country [121] 0 0
Sweden
State/province [121] 0 0
Göteborg
Country [122] 0 0
Sweden
State/province [122] 0 0
Linköping
Country [123] 0 0
Sweden
State/province [123] 0 0
Stockholm
Country [124] 0 0
Sweden
State/province [124] 0 0
Uppsala
Country [125] 0 0
Sweden
State/province [125] 0 0
Örebro
Country [126] 0 0
United Kingdom
State/province [126] 0 0
Devon
Country [127] 0 0
United Kingdom
State/province [127] 0 0
East Riding Of Yorkshire
Country [128] 0 0
United Kingdom
State/province [128] 0 0
East Sussex
Country [129] 0 0
United Kingdom
State/province [129] 0 0
Greater London
Country [130] 0 0
United Kingdom
State/province [130] 0 0
Greater Manchester
Country [131] 0 0
United Kingdom
State/province [131] 0 0
Hertfordshire
Country [132] 0 0
United Kingdom
State/province [132] 0 0
Highland Region
Country [133] 0 0
United Kingdom
State/province [133] 0 0
Leicestershire
Country [134] 0 0
United Kingdom
State/province [134] 0 0
Lothian Region
Country [135] 0 0
United Kingdom
State/province [135] 0 0
Merseyside
Country [136] 0 0
United Kingdom
State/province [136] 0 0
North Yorkshire
Country [137] 0 0
United Kingdom
State/province [137] 0 0
South Yorkshire
Country [138] 0 0
United Kingdom
State/province [138] 0 0
Staffordshire
Country [139] 0 0
United Kingdom
State/province [139] 0 0
Tyne & Wear
Country [140] 0 0
United Kingdom
State/province [140] 0 0
Warwickshire
Country [141] 0 0
United Kingdom
State/province [141] 0 0
Swansea

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Otsuka Pharmaceutical Development & Commercialization, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of the study is to determine whether tolvaptan is effective and safe for the
treatment of late-stage chronic kidney disease due to autosomal dominant polycystic kidney
disease (ADPKD)
Trial website
https://clinicaltrials.gov/ct2/show/NCT02160145
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT02160145