UMIN-CTR Clinical Trial

Basic information
Public title	Confirmatory study of patients with progressive autosomal dominant polycystic kidney disease (ADPKD) to establish the determinants of disease progression and response to tolvaptan treatment.
Acronym	ADPKD confirmatory study to establish the determinants of disease progression and response to tolvaptan treatment.
Scientific Title	Confirmatory study of patients with progressive autosomal dominant polycystic kidney disease (ADPKD) to establish the determinants of disease progression and response to tolvaptan treatment.
Scientific Title:Acronym	ADPKD confirmatory study to establish the determinants of disease progression and response to tolvaptan treatment.
Region	Japan

Condition
Condition	autosomal dominant polycystic kidney disease (ADPKD)
Classification by specialty	Nephrology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	We investigate the efficacy and safety of tolvaptan as a new therapeutic drug for progressive autosomal dominant polycystic kidney disease(ADPKD).
Basic objectives2	Others
Basic objectives -Others	We investigate the determinants of disease progression and response to tolvaptan treatment in progressive autosomal dominant polycystic kidney disease (ADPKD) .
Trial characteristics_1	Confirmatory
Trial characteristics_2	Others
Developmental phase	Not applicable

Assessment
Primary outcomes	change of kidney function (eGFR)
Key secondary outcomes	total kidney volume urine concentration plasma AVP concentration urine cAMP incidence and severity of symptom related with ADPKD (urine concentration ability, hypertension, kidney pain, kidney function, hematuria, kidney stone, kidney infection)

Base
Study type	Interventional

Study design
Basic design	Factorial
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Dose comparison
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	5
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	tolvaptan 60~120mg/day
Interventions/Control_2	tolvaptan less than 60mg/day (in case of minimize dose recommended, for example; kidney dysfunction)
Interventions/Control_3	tolvaptan less than 60mg/day (in case of minimize dose recommended, for example; kidney dysfunction)
Interventions/Control_4	no medication
Interventions/Control_5	tolvaptan less than 60mg/day of start dose (in case of minimize start dose recommended, for example; less than 20 years old or more than 65 years old)
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	15 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	1. ADPKD patient 2. total kidney volume is more than 750ml by CT or MRI, and also increased speed of total kidney volume is more than almost 5% per year. 3. The patient who was agreed informed consent.
Key exclusion criteria	(1) contraindicated patient of tolvaptan 1. patient who has past history of hypersensitivity to tolvaptan or similar compound (mozavaptan) 2. patient who is not able to feel thirst or is difficult to water intake. 3. hypernatremia 4. severe kidney dysfunction (eGFR less than 15mL/min/1.73m2) 5. present or past history of liver dysfunction by chronic hepatitis, or drug-induced liver dysfunction, excluding liver cyst in ADPKD 6. The pregnancy or the woman who may be pregnant. (2) The patient judged inappropriate by study responsibility doctor.
Target sample size	100

Research contact person
Name of lead principal investigator	1st name Kosaku Middle name Last name Nitta
Organization	Tokyo Women's Medical University
Division name	Department of Medicine four
Zip code	162-8666
Address	8-1, Kawada-cho, Shinjuku-ku, Tokyo 162-8666, Japan
TEL	0333538111
Email	mtoshi@twmu.ac.jp

Public contact
Name of contact person	1st name Toshio Middle name Last name Mochizuki
Organization	Tokyo Women's Medical University
Division name	Department of Nephrology
Zip code	162-8666
Address	Kawada-cho
TEL	0333538111
Homepage URL
Email	mtoshi@twmu.ac.jp

Sponsor
Institute	Department of Nephrology, Tokyo Women's Medical University
Institute
Department

Funding Source
Organization	None
Organization
Division
Category of Funding Organization	Self funding
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funder(s)

IRB Contact (For public release)
Organization	Ethics committee, Tokyo Women's Medical University
Address	8-1 kawada-cho, Shinjuku-ku, Tokyo
Tel	0333538111
Email	krinri.bm@twmu.ac.jp

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions	東京女子医科大学（東京都）

Other administrative information
Date of disclosure of the study information	2014 Year 09 Month 25 Day

Related information
URL releasing protocol	https://www.kireports.org/action/showPdf?pii=S2468-0249%2821%2901293-6
Publication of results	Published

Result
URL related to results and publications	https://www.kireports.org/action/showPdf?pii=S2468-0249%2821%2901293-6
Number of participants that the trial has enrolled	92
Results	The observation period was 2.4 years. U-AQP2 per milligram of urinary creatinine (U-AQP2/Cr) decreased from 67.8 to 20.7 fmol/mg urinary creatinine after 1 month of tolvaptan treatment. This initial change in U-AQP2/Cr was correlated with high baseline U-AQP2/Cr, low baseline eGFR, and a large initial change in eGFR (baseline to 1 month). The initial change in U-AQP2/Cr (baseline to 1 month) was strongly correlated with the annual change in TKV and eGFR in multivariable analysis.
Results date posted	2021 Year 09 Month 30 Day
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics	Patients with ADPKD who were administered tolvaptan in our hospital.
Participant flow	Follow up of patients with ADPKD who were administered tolvaptan in our hospital.
Adverse events	Liver dysfunction
Outcome measures	the annual change in total kidney volume (TKV) and estimated glomerular filtration rate (eGFR)
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	Completed
Date of protocol fixation	2014 Year 09 Month 10 Day
Date of IRB	2014 Year 08 Month 26 Day
Anticipated trial start date	2014 Year 09 Month 25 Day
Last follow-up date	2019 Year 03 Month 31 Day
Date of closure to data entry	2019 Year 03 Month 31 Day
Date trial data considered complete	2019 Year 03 Month 31 Day
Date analysis concluded	2021 Year 03 Month 31 Day

Other
Other related information

Management information
Registered date	2014 Year 09 Month 25 Day
Last modified on	2021 Year 09 Month 30 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000017722