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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/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. - The mean annualized change in eGFR was calculated using the Chronic Kidney Disease-Epidemiology (CKD-EPI) formula from pretreatment baseline to post-treatment follow-up, annualized (divided) by each subject's trial duration.
The baseline for the primary endpoint was defined as the average of up to 3 eGFR values observed during the screening and placebo run-in periods.
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 - To compare the efficacy of tolvaptan treatment in reducing the decline of annualized eGFR slope, as compared with placebo, in subjects with late-stage CKD due to ADPKD who tolerated tolvaptan during an initial run-in period, the annualized rate of eGFR change was derived from each individual subject's eGFR slope using the CKD-EPI formula.
The annualized eGFR change slope was derived from all eGFR observations from placebo-run-in, tolvaptan run-in, double-blind treatment and post-treatment follow-up periods using the linear mixed model of analysis. The mean annualized slope of eGFR change is presented.
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
Gender
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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,TAS,VIC,WA
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- Camperdown
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- Concord
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- New Lambton Heights
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- St. Leonards
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- Westmead
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- Woolloongabba
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- Adelaide
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- Launceston
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- Parkville
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- Reservoir
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- Richmond
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- Perth
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2050 - Camperdown
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2139 - Concord
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2305 - New Lambton Heights
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2065 - St. Leonards
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2145 - Westmead
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4102 - Woolloongabba
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5000 - Adelaide
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7250 - Launceston
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3050 - Parkville
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3073 - Reservoir
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3121 - Richmond
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6000 - Perth
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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/show/NCT02160145
Trial related presentations / publications
Public notes

Contacts
Principal investigator
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Address 0 0
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Email 0 0
Contact person for public queries
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Summary results
Other publications