Post-Marketing Surveillance Study of Tolvaptan in Patients With ADPKD in Japan
Conditions: Polycystic Kidney, Autosomal Dominant
Sponsor: Otsuka Pharmaceutical Co., Ltd.

Study ID: I15600-003 
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Samsca drug use-results survey (ADPKD) 
Study Protocol 
 
NCT Number: NCT02847624 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Otsuka Pharmaceutical Co., Ltd. 
 
Protocol Creation Date: February 21, 2014, Version 1.0 
Date of the latest version: September 2, 2021, Version 8.0

Study ID: I15600-003 
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Objective of 
survey 
To determine the safety and efficacy of Samsca used in routine clinical settings for suppression of 
ADPKD progression. 
Survey 
methods 
 
<All patients survey> 
All patients treated with Samsca must be registered in the survey until “CONDITIONS FOR 
APPROVAL” is removed. 
CRFs must be collected from all patients until the total number of patients registered in the survey 
reaches the planned number. 
Observation period: From start of Samsca administration through the end of survey 
In case of finish or discontinue of Samsca administration, collecting information of kidney 
volume, changes in renal function and hepatic dysfunction (outcome: not recovered) through 
the end of survey (8 years).  
Survey period 
8 years (March 24,2014 ~ March 23,2022) 
Planned 
number of 
patients to be 
surveyed 
1,600 
Depending on the onset of significant drug-induced liver injury post-marketing, the planned 
number of patients will be re-evaluated and necessity of additional safety measures will be also 
assessed. 
In addition, if there is a substantial change in expected number of patients with long-term 
treatment (≥4 years), the planned number of patients will be re-evaluated so as to collect at least 
800 patients with long-term treatment. 
Target patient 
population of 
survey 
All patients treated with Samsca after March 24, 2014 for the indication of “suppression of 
ADPKD progression with a rapid kidney volume increase in patients with increased kidney 
volume” 
Major items to 
be surveyed  
 
 
 
CRFs of drug use-results survey (for treatment period) 
1) Patient information  
Patient initials (last name, first name), patient ID, sex, any breast feeding, any pregnancy, 
date of birth (age), date of informed consent received (in written form) 
2) Patient demographics 
Indication for use, time of ADPKD diagnosis, diagnosis of ADPKD (severity of ADPKD, 
family history, number of cysts, complications (especially, hepatic cyst, pancreatic cyst, 
cerebral aneurysm, urinary calculus, any complication of cyst infection), past history, 
predisposition to hypersensitivity, history of drug adverse reactions 
3) Administration status of Samsca  
  Dosage form, time and dose, route of administration, period of administration (date started, 
date stopped),  
4) Usage status of Samsca 
Ongoing, completed, discontinued (reason for discontinuation → adverse events, 
insufficient efficacy, changing hospital, other), no visit 
5) Changes in kidney volume  
  In-patient/out-patient status, total kidney volume (measured or not, method of 
measurement), change of kidney volume 
6) Laboratory values and electrocardiography 
(1) Physical examination 

Study ID: I15600-003 
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Height, weight, body temperature, ECG finding 
(3) Blood chemistry tests  
AST (GOT), ALT (GPT), e-GFR 
(4) Urinalysis 
Urinary protein, urine sugar, bilirubin, urobilinogen, urine albumin, urine osmolality  
7) Adverse events after start of Samsca administration 
Specific adverse events, onset date, seriousness, reason for seriousness (if serious), date 
of outcome assessment, outcome, causal relationship to Samsca, other causative factors 
than Samsca, corrective treatment for adverse event 
8) Condition around the event onset 
Condition around the event onset, comments on the adverse events 
 
 
CRFs of drug use-results survey (for follow-up period) 
1) Patient information  
Patient initials (last name, first name), patient ID, sex, any breast feeding, any pregnancy, 
date of birth (age), date of informed consent received (in written form) 
2) Changes in kidney volume  
  In-patient/out-patient status, total kidney volume (measured or not, method of 
measurement), change of kidney volume,  
3) Laboratory values 
(1) Physical examination 
Height, weight 
(2) Blood chemistry tests  
AST (GOT), ALT (GPT), e-GFR 
4) Follow-up investigation of adverse events that remained unresolved or were of unknown 
status during the treatment period. 
Specific adverse events, onset date, seriousness, reason for seriousness (if serious), date 
of outcome assessment, outcome, causal relationship to Samsca, other causative factors 
than Samsca, corrective treatment for adverse event 
5) Condition around the event onset and comments on the adverse events 

Study ID: I15600-003 
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Items to be 
analyzed 
CRFs of drug use-results survey (for treatment period) 
1) Items related to constitution of patients 
Number of patients enrolled, number of patients withdrawn, number of CRFs collected, number of 
patients excluded, number of patients analyzed for safety, number of patients analyzed for efficacy 
2) Items related to efficacy 
Changes in kidney volume data, changes in renal function test data (e-GFR) 
3) Items related to safety 
(1) Safety in elderly patients 
(2) Safety of Samsca in advanced ADPKD patients (creatinine clearance <60 mL/min) 
(3) Adverse events of special interest (hepatic dysfunction, increases in transaminases 
(AST or ALT)) 
(4) Items to be closely monitored: hepatic dysfunction 
(5) Safety of Samsca in ADPKD patients after long-term treatment 
CRFs of drug use-results survey (for follow-up period) 
1) Changes in kidney volume, changes in renal function test data (e-GFR)