Evaluating the Safety and effectivenesS in Adult KorEaN Patients Treated With Tolvaptan for Management of Autosomal domInAnt poLycystic Kidney Disease (ESSENTIAL)

June 15, 2022 updated by: Korea Otsuka Pharmaceutical Co., Ltd.
To evaluate the safety and therapeutic effectiveness of tolvaptan when administered to slow the progression of cyst development and renal function insufficiency in adult Korean patients diagnosed with rapidly progressive ADPKD who have chronic kidney disease (CKD) stages 1-3 at initiation of treatment.

Study Overview

Status

Completed

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

118

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Busan, Korea, Republic of
        • Inje University Busan Paik Hospital
      • Busan, Korea, Republic of
        • Kosin University Gospel Hospital
      • Chuncheon, Korea, Republic of
        • Hallym University Chuncheon Sacred Heart Hospital
      • Daegu, Korea, Republic of
        • Keimyung University Dongsan Medical Center
      • Daegu, Korea, Republic of
        • Kyungpook National University Hospital
      • Daejeon, Korea, Republic of
        • Chungnam National University Hospital
      • Gwangju, Korea, Republic of
        • Chonnam National University Hospital
      • Incheon, Korea, Republic of
        • Gachon University Gil Medical Center
      • Pyeongchon, Korea, Republic of
        • Hallym University Medical Center
      • Seongnam-si, Korea, Republic of
        • Seoul National University Bundang Hospital
      • Seoul, Korea, Republic of, 03080
        • Seoul National University Hospital
      • Seoul, Korea, Republic of
        • The Catholic University of Korea Seoul St. Mary's Hospital
      • Seoul, Korea, Republic of
        • Gangnam Severance Hospital
      • Seoul, Korea, Republic of
        • Severance Hospital
      • Seoul, Korea, Republic of
        • SMG-SNU Boramae Medical Center
      • Seoul, Korea, Republic of
        • Kangbuk Samsung Hospital
      • Seoul, Korea, Republic of
        • Hanyang University Seoul Hospital
      • Seoul, Korea, Republic of
        • Hallym University Kangnam Sacred Heart Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

19 years to 50 years (ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Subjects who voluntarily participate by giving written informed consent on this trial
  2. Male and female patients aged ≥ 19 to ≤ 50 years
  3. Subjects diagnosed with ADPKD based on the Unified Criteria for Ultrasonographic diagnosis of ADPKD (Pei-Ravine Criteria)
  4. Subjects with confirmed CKD stages 1-3 at the screening visit
  5. Subjects with confirmed rapidly progressive typical ADPKD 'Typical ADPKD'

    • refers to bilateral and diffuse distribution, with mild, moderate or severe replacement of kidney tissue by cysts, where all cysts contribute similarly to TKV.

'rapidly progressive ADPKD'

  • Patients will be defined as 'rapidly progressive ADPKD' if they meet any of the following criteria:

    • Mayo class 1C, 1D or 1E

      • Truncating PKD1 mutation confirmed by genetic testing before participating this trial ③ PRO-PKD score > 6 ④ Patients with ADPKD with a decline in Estimated glomerular filtration rate(eGFR) ≥ 5 mL/min/1.73 m2 within 1 year from the screening visit or with an average annual decline in eGFR ≥ 2.5 mL/min/1.73 m2 over a period of 5 years (excluding patients with an eGFR decline due to factors other than ADPKD, such as uncontrolled type 2 diabetes, early diabetic glomerular disease or immune-mediated glomerulonephritis)

Exclusion Criteria:

  1. Patients with hyponatremia or hypernatremia
  2. Patients with anuria
  3. Patients with volume depletion
  4. Patients who are unable to sense or appropriately respond to thirst
  5. Patients with contraindications to MRI assessment [e.g., ferromagnetic metal prosthesis, aneurysm clips, severe claustrophobia, large tattoo on the abdomen or back, etc.]
  6. Patients with severe renal impairment [e.g., patients with currently active glomerulonephritis, kidney cancer, having a single kidney, history of renal surgery within the last 3 years, etc.]
  7. Patients with severe hepatic impairment [e.g., cirrhosis, viral hepatitis, unspecified liver function test abnormalities (ALT or Aspartate aminotransferase(AST)) > 3 x ULN or Total Bilirubin > 2 x ULN), etc.]
  8. Patients with eGFR decline due to factors other than ADPKD (e.g., uncontrolled type 2 diabetes, early diabetic glomerular disease or immune-mediated glomerulonephritis, etc.)
  9. Patients with a history of hypersensitivity and/or specific reactions to benzazepine or benzazepine derivatives (such as Benazepril), or tolvaptan
  10. Patients with hereditary problems of galactose intolerance, the Lapp lactose deficiency or glucose-galactose malabsorption, etc.
  11. Patients who need chronic diuretic use
  12. Patients who are receiving any experimental (not marketed) or approved therapies that may affect the treatment of ADPKD within 6 months from the screening visit [e.g., anti-sense RNA therapy, rapamycin, sirolimus, everolimus and somatostatin analogs (octreotide, sandostatin), vasopressin antagonist (mozavaptan, conivaptan), vasopressin agonist (desmopressin)]
  13. Patients who have received cyst decompression or sclerotherapy within 3 years from the screening visit
  14. Patients with a history of taking tolvaptan within 6 months from the screening visit
  15. Patients who received any investigational medicinal product in another trial within 30 days from the screening visit
  16. Fertile women who are currently pregnant or breat feeding, or not willing to use or capable of using acceptable contraceptive methods (abstinence, oral, implanted or injected hormonal methods of contraception, intrauterine device or barrier methods of contraception, such as condom, contraceptive diaphragm and spermicidal agents) to avoid pregnancy until completion of the trial
  17. Patients who are, in the opinion of the investigator, unable to comply with the administration of the Investigational Medicinal Product(IMP) or the trial procedures

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: tolvaptan
30mg and 15mg of Tolvaptan Tablet
Other Names:
  • Samsca

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The incidences of TEAEs
Time Frame: during the tolvaptan treatment period and up to 7 days after the date of last dosing
The incidences of TEAEs
during the tolvaptan treatment period and up to 7 days after the date of last dosing
The incidences of TEAEs meeting any of the followings will be summarized.
Time Frame: during the tolvaptan treatment period and up to 7 days after the date of last dosing
Liver injury [ALT or AST elevation (>3 x ULN) or Total Bilirubin elevation (>2 x ULN), etc.], AEs leading to death, Serious AEs, AEs leading to treatment discontinuation, AEs whose causal relationship with the IMP cannot be ruled out, Severe AEs, Dehydration, Effects on Sodium, Creatinine
during the tolvaptan treatment period and up to 7 days after the date of last dosing

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Total kidney volume (TKV) annual mean percent change rate [%/year]
Time Frame: from baseline to End of Treatment (Visit 25, Month 24)
Baseline TKV refers to the value measured during the screening period
from baseline to End of Treatment (Visit 25, Month 24)
eGFR CKD-EPI annual mean change [mL/min/1.73m^2] (off-treatment)
Time Frame: from baseline to the Follow-up visit (Visit 26, 7 days after End of Treatment(Visit 25, Month 24))
(off-treatment)
from baseline to the Follow-up visit (Visit 26, 7 days after End of Treatment(Visit 25, Month 24))
eGFR CKD-EPI annual mean change [mL/min/1.73m^2] (on-treatment)
Time Frame: from Completion of Tolvaptan Titration Period (Visit 6, Week 4) to End of Treatment (Visit 25, Month 24)
(on-treatment)
from Completion of Tolvaptan Titration Period (Visit 6, Week 4) to End of Treatment (Visit 25, Month 24)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

July 1, 2019

Primary Completion (ACTUAL)

May 25, 2022

Study Completion (ACTUAL)

May 25, 2022

Study Registration Dates

First Submitted

April 16, 2019

First Submitted That Met QC Criteria

May 13, 2019

First Posted (ACTUAL)

May 14, 2019

Study Record Updates

Last Update Posted (ACTUAL)

June 16, 2022

Last Update Submitted That Met QC Criteria

June 15, 2022

Last Verified

June 1, 2022

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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