Autosomal Dominant Polycystic Kidney Disease (ADPKD)Phase 4ESSENTIALNCT03949894
Evaluating the Safety and effectivenesS in Adult KorEaN Patients Treated With Tolvaptan for Management of Autosomal domInAnt poLycystic Kidney Disease
A Phase 4 study testing Tolvaptan in people with Autosomal Dominant Polycystic Kidney Disease (ADPKD), run by Korea Otsuka Pharmaceutical Co., Ltd. at 18 sites in South Korea.
Trial 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. Read more about this study
- Who is it for?
- Ages 19 to 50 years
- Conditions the study is for
- Autosomal Dominant Polycystic Kidney Disease (ADPKD)
- What is being tested?
- Tolvaptan
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 118 participants
- Where
- 18 sitesSouth Korea
- Study dates
- 1 Jul 2019to 25 May 2022
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 19 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Subjects who voluntarily participate by giving written informed consent on this trial
- Male and female patients aged ≥ 19 to ≤ 50 years
- Subjects diagnosed with ADPKD based on the Unified Criteria for Ultrasonographic diagnosis of ADPKD (Pei-Ravine Criteria)
- Subjects with confirmed CKD stages 1-3 at the screening visit
- 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.
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- '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)
Who cannot join
- Patients with hyponatremia or hypernatremia
- Patients with anuria
- Patients with volume depletion
- Patients who are unable to sense or appropriately respond to thirst
- Patients with contraindications to MRI assessment [e.g., ferromagnetic metal prosthesis, aneurysm clips, severe claustrophobia, large tattoo on the abdomen or back, etc.]
- 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.]
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- 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.]
- 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.)
- Patients with a history of hypersensitivity and/or specific reactions to benzazepine or benzazepine derivatives (such as Benazepril), or tolvaptan
- Patients with hereditary problems of galactose intolerance, the Lapp lactose deficiency or glucose-galactose malabsorption, etc.
- Patients who need chronic diuretic use
- 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)]
- Patients who have received cyst decompression or sclerotherapy within 3 years from the screening visit
- Patients with a history of taking tolvaptan within 6 months from the screening visit
- Patients who received any investigational medicinal product in another trial within 30 days from the screening visit
- 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
- 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
Trial locations and contacts
18 sites in 1 country.
18 sites in 1 country
South Korea18 sites
Inje University Busan Paik Hospital
Busan
Kosin University Gospel Hospital
Busan
Hallym University Chuncheon Sacred Heart Hospital
Chuncheon
Keimyung University Dongsan Medical Center
Daegu
Kyungpook National University Hospital
Daegu
Chungnam National University Hospital
Daejeon
Chonnam National University Hospital
Gwangju
Gachon University Gil Medical Center
Incheon
Hallym University Medical Center
Pyeongchon
Seoul National University Bundang Hospital
Seongnam-si
Seoul National University Hospital
Seoul, 03080
Gangnam Severance Hospital
Seoul
Hallym University Kangnam Sacred Heart Hospital
Seoul
Hanyang University Seoul Hospital
Seoul
Kangbuk Samsung Hospital
Seoul
Severance Hospital
Seoul
SMG-SNU Boramae Medical Center
Seoul
The Catholic University of Korea Seoul St. Mary's Hospital
Seoul
About this study
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.
- Being tested
- Tolvaptan
- Medical subject headings
- Polycystic Kidney, Autosomal Dominant
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TolvaptanDrug
Also called Samsca
30mg and 15mg of Tolvaptan Tablet
| Study group | Role | What they receive |
|---|---|---|
| tolvaptan | Experimental | Not described Receives: Drug: Tolvaptan |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidences of TEAEs
The incidences of TEAEs
Measured: 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.
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
Measured: during the tolvaptan treatment period and up to 7 days after the date of last dosing
Secondary outcomes (3)
Total kidney volume (TKV) annual mean percent change rate [%/year]
Baseline TKV refers to the value measured during the screening period
Measured: from baseline to End of Treatment (Visit 25, Month 24)
eGFR CKD-EPI annual mean change [mL/min/1.73m^2] (off-treatment)
(off-treatment)
Measured: 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)
(on-treatment)
Measured: from Completion of Tolvaptan Titration Period (Visit 6, Week 4) to End of Treatment (Visit 25, Month 24)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2019
- Study start1 Jul 2019
- Primary completion25 May 2022
- Study completion25 May 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autosomal Dominant Polycystic Kidney Disease (ADPKD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 118 participants (actual)
Sponsor and investigators
- Lead sponsor
- Korea Otsuka Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03949894
- Sponsor's protocol ID
- 156-402-00144
- Acronym
- ESSENTIAL
- Status last verified
- Jun 2022
- First submitted
- 16 Apr 2019
- Last updated
- 16 Jun 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluating the Safety and effectivenesS in Adult KorEaN Patients Treated With Tolvaptan for Management of Autosomal domInAnt poLycystic Kidney Disease
- ClinicalTrials.gov ID
- NCT03949894
- Acronym / sponsor ID
- ESSENTIAL / 156-402-00144
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 118 participants (actual)
- Lead sponsor
- Korea Otsuka Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 1 Jul 2019 (actual)
- Primary completion
- 25 May 2022 (actual)
- Study completion
- 25 May 2022 (actual)
- First posted
- 14 May 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| tolvaptan | Experimental | Drug: Tolvaptan |
Who may qualify
- Ages
- 19 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (10 listed)
- Subjects who voluntarily participate by giving written informed consent on this trial
- Male and female patients aged ≥ 19 to ≤ 50 years
- Subjects diagnosed with ADPKD based on the Unified Criteria for Ultrasonographic diagnosis of ADPKD (Pei-Ravine Criteria)
- Subjects with confirmed CKD stages 1-3 at the screening visit
- Subjects with confirmed rapidly progressive typical ADPKD 'Typical ADPKD'
Key exclusion criteria (17 listed)
- Patients with hyponatremia or hypernatremia
- Patients with anuria
- Patients with volume depletion
- Patients who are unable to sense or appropriately respond to thirst
- Patients with contraindications to MRI assessment [e.g., ferromagnetic metal prosthesis, aneurysm clips, severe claustrophobia, large tattoo on the abdomen or back, etc.]
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TolvaptanAlso: Samsca30mg and 15mg of Tolvaptan Tablet | Drug | tolvaptan |
Primary outcome
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
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
3 secondary outcomes
Total kidney volume (TKV) annual mean percent change rate [%/year]
Time frame: 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))
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)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jun 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
18 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Tolvaptan in people with Autosomal Dominant Polycystic Kidney Disease (ADPKD), run by Korea Otsuka Pharmaceutical Co., Ltd. at 18 sites in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidences of TEAEs (time frame: during the tolvaptan treatment period and up to 7 days after the date of last dosing). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jun 2022.