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

NCT ID: NCT03949894

Last Updated: 2022-06-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 118 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-07-01
• Study Completion Date: 2022-05-25

Brief Summary

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.

Conditions

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

tolvaptan

Group Type: EXPERIMENTAL

Tolvaptan

Intervention Type: DRUG

30mg and 15mg of Tolvaptan Tablet

Interventions

Tolvaptan

30mg and 15mg of Tolvaptan Tablet

Intervention Type: DRUG

Other Intervention Names

Samsca

Eligibility Criteria

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

• Minimum Eligible Age: 19 Years
• Maximum Eligible Age: 50 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Korea Otsuka Pharmaceutical Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Inje University Busan Paik Hospital

Busan, , South Korea

Kosin University Gospel Hospital

Busan, , South Korea

Hallym University Chuncheon Sacred Heart Hospital

Chuncheon, , South Korea

Keimyung University Dongsan Medical Center

Daegu, , South Korea

Kyungpook National University Hospital

Daegu, , South Korea

Chungnam National University Hospital

Daejeon, , South Korea

Chonnam National University Hospital

Gwangju, , South Korea

Gachon University Gil Medical Center

Incheon, , South Korea

Hallym University Medical Center

Pyeongchon, , South Korea

Seoul National University Bundang Hospital

Seongnam-si, , South Korea

Seoul National University Hospital

Seoul, , South Korea

Gangnam Severance Hospital

Seoul, , South Korea

Hallym University Kangnam Sacred Heart Hospital

Seoul, , South Korea

Hanyang University Seoul Hospital

Seoul, , South Korea

Kangbuk Samsung Hospital

Seoul, , South Korea

Severance Hospital

Seoul, , South Korea

SMG-SNU Boramae Medical Center

Seoul, , South Korea

The Catholic University of Korea Seoul St. Mary's Hospital

Seoul, , South Korea

Countries

South Korea

Other Identifiers

156-402-00144

Identifier Type: -

Identifier Source: org_study_id