UMIN-CTR Clinical Trial

Basic information
Public title	A study to evaluate effect and safety of Tolvaptan in Nephrogenic Diabetes Insipidus caused by mutations in the vasopressin type 2 receptor gene.
Acronym	A study to evaluate effect and safety of Tolvaptan in Nephrogenic Diabetes Insipidus caused by mutations in the vasopressin type 2 receptor gene.
Scientific Title	A study to evaluate effect and safety of Tolvaptan in Nephrogenic Diabetes Insipidus caused by mutations in the vasopressin type 2 receptor gene.
Scientific Title:Acronym	A study to evaluate effect and safety of Tolvaptan in Nephrogenic Diabetes Insipidus caused by mutations in the vasopressin type 2 receptor gene.
Region	Japan

Condition
Condition	Nephrogenic Diabetes Insipidus caused by mutations in the vasopressin type 2 receptor gene
Classification by specialty	Endocrinology and Metabolism Nephrology
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	The aim of this study is to evaluate the efficacy and safety of Tolvaptan in patients with Nephrogenic Diabetes Insipidus caused by mutations in the vasopressin type 2 receptor gene.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Urine Volume
Key secondary outcomes	Urine Osmolarity, amount of water drinking, Urine AQP2, safety

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Self control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	1) Administration of Tolvaptan once per day (60mg/day, 90mg/day, 120mg/day), each dose for two days. 2) Administration of Tolvaptan 120mg per day for 2 to 4 weeks in outpatient.
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	20 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	1) Over 20 years old 2) Patients who have been diagnosed with nephrogenic diabetes insipidus caused by mutations in the vasorepssin type 2 receptor gene. 3) Doctor's decision based on the tests at routine outpatient visits 4) Written informed consent after a detailed explanation of this study
Key exclusion criteria	1) History of allergy to tolvaptan 2) Pregnant women and women suspected of being pregnant 3) Severe Renaldysfunction (eGFR < 30 mL/min/1.73m2) 4) Severe liver dysfunction (AST > 100 U/L or ALT > 100 U/L) 5) Congestive heart failure 6) Active TBc 7) Substance use disorder 8) Use of investigational drug within 4 months before participation of this study 9) Amount of whole blood sampling > 200 mL within 4 weeks, amount of whole blood sampling > 400 mL within 12 weeks, or plasmapheresis / platelet apheresis within 2 weeks. 10) Doctor's decision
Target sample size	3

Research contact person
Name of lead principal investigator	1st name Noriko Middle name Last name Makita
Organization	The University of Tokyo Hospital
Division name	Nephrology and Endocrinology
Zip code	1138655
Address	7-3-1 Hongo, Bunkyo-ku, Tokyo, Japan
TEL	03-3815-5411
Email	norimaki-tky@umin.ac.jp

Public contact
Name of contact person	1st name Katsunori Middle name Last name Manaka
Organization	The University of Tokyo Hospital
Division name	Nephrology and Endocrinology
Zip code	1138655
Address	7-3-1 Hongo, Bunkyo-ku, Tokyo, Japan
TEL	03-3815-5411
Homepage URL
Email	manaka-tky@umin.ac.jp

Sponsor
Institute	Department of Nephrology and Endocrinology, The University of Tokyo Hospital
Institute
Department

Funding Source
Organization	Healthcare coopration
Organization
Division
Category of Funding Organization	Other
Nationality of Funding Organization

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

IRB Contact (For public release)
Organization	The University of Tokyo, Clinical Research Review Board
Address	7-3-1 Hongo, Bunkyo-ku, Tokyo, Japan
Tel	03-5841-0818
Email	ethics@m.u-tokyo.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

Other administrative information
Date of disclosure of the study information	2016 Year 04 Month 18 Day

Related information
URL releasing protocol
Publication of results	Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	Open public recruiting
Date of protocol fixation	2016 Year 02 Month 22 Day
Date of IRB	2016 Year 02 Month 22 Day
Anticipated trial start date	2016 Year 04 Month 18 Day
Last follow-up date	2024 Year 01 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2016 Year 03 Month 31 Day
Last modified on	2021 Year 10 Month 07 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000024903