Autosomal Dominant Polycystic KidneyPhase 4PoCKETNCT03596957
Subacute Effect of Tolvaptan on Total Kidney Volume in Adult Patients With Autosomal Dominant Polycystic Kidney Disease
A Phase 4 study testing Tolvaptan in people with Autosomal Dominant Polycystic Kidney, run by Lisbet Brandi at 6 sites in Denmark.
Trial summary
Investigator initiated controlled multi-centre trial in a Prospective, Randomised, Open, Blinded Endpoint (PROBE) design. Patients will be randomised in a 1:1 ratio either to treatment with tolvaptan for six weeks followed by six weeks observation without trial medication or no tolvaptan treatment, but following the… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Autosomal Dominant Polycystic Kidney
- What is being tested?
- Tolvaptan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 6 sitesDenmark
- Study dates
- 12 Sep 2018to Apr 2020
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult patients between 18 and 65 years
- Diagnosis of typical ADPKD
- tKV above or equal to 750 ml by MRI scanning
- Estimated GFR (e-GFR) by CKD-EPI formula of above or equal to 45 mL/min/1.73 m2
Who cannot join
- Kidney transplant recipient
- Known liver disease except for liver cysts relating to ADPKD
- ASAT and ALAT above upper normal level
- Current treatment with thiazide and thiazide-line diuretics, mineral corticoid receptor antagonists, amiloride or loop diuretics
- Evidence of urinary tract obstruction
- Current treatment with CYP3A4 inhibitors
- Active malignant disease
- Current or previous treatment with tolvaptan
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
Denmark6 sites, 6 recruiting
- Recruiting
Rigshospitalet - Site 42
Copenhagen, 2100
Bo Feldt-Rasmussen, MD, +45 3545 2135, Bo.Feldt-Rasmussen@regionh.dk
Tobias Bomholt, MD, +45 35451838, tobias.bomholt@regionh.dk
- Recruiting
Herlev Hospital
Herlev, 2730
Ditte Hansen, MD, +45 3868 2056, ditte.hansen@regionh.dk
Marie Moeller, MD, +45 6169 5364, marie.moeller@regioh.dk
- Recruiting
Nordsjaellands Hospital - Site 41
Hillerød, 3400
Lisbet Brandi, MD, +45 48295993, lisbet.brandi@regionh.dk
Charlotte Bjernved Nielsen, +45 49294714, charlotte.bjernved.nielsen@regionh.dk
- Not yet recruiting
Odense University Hospital - Site 45
Odense, Odense C, 5000
Helle Thiesson, MD, +45 6541 1642, helle.thiesson@rsyd.dk
Kristian Bergholt Buhl, MD, +456541 1642, kristian.bergholt.buhl@rsyd.dk
- Not yet recruiting
Sjællands University Hospital Roskilde
Roskilde, 4000
Bjarne Ørskov, MD, +45 2966 2426, bjaoe@regionsjalelland.dk
- Recruiting
Aarhus University Hospital - Site 43
Skejby, Aarhus N, 8200
Henrik Birn, MD, +45 6171 7870, hb@biomed.au.dk
Karin Hansen, +45 4046 0831, Karin.hansen@skejby.rm.dk
About this study
Investigator initiated controlled multi-centre trial in a Prospective, Randomised, Open, Blinded Endpoint (PROBE) design.
Patients will be randomised in a 1:1 ratio either to treatment with tolvaptan for six weeks followed by six weeks observation without trial medication or no tolvaptan treatment, but following the same visit and investigation plan as the subjects taking tolvaptan.
Read the detailed description
Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common genetic kidney disease and the fourth leading cause of end-stage renal disease in adults Worldwide.
The tolvaptan tablet has been approved by EMA (European Medicines Agency) with the indication of slowing the progression of cysts development and renal insufficiency in adults with ADPKD. It is the newest and only possible treatment for this patient group and could be initiated in patients with evidence for rapidly progressive disease Development.
There is however in Denmark and other countries both scientific and financial reluctance to initiate this expensive treatment for several reasons e.g. selection of patients who might benefit, effect on progression of kidney disease, side effects and tolerability.
Before deciding on implementation in Denmark, more knowledge is needed. The results of the PoCKET trial will contribute with guidance on this decision.
Foremost the trial is designed to address not only the change in kidney volume, but the change in kidney function, which is what matters to the patients and their prognosis in terms of postponing time to end stage renal disease. Furthermore, important data on side effects and tolerability will be generated.
- Conditions
- Autosomal Dominant Polycystic Kidney
- 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 Jinarc
At baseline the tolvaptan dosing will start with daily morning and afternoon doses of 45 mg and 15 mg respectively, with weekly increases to 60 mg and 30 mg and then to 90 mg and 30 mg according to subject tolerability
| Study group | Role | What they receive |
|---|---|---|
| Tolvaptan group | Active comparator | Treatment with tolvaptan for six weeks followed by six weeks observation without trial medication Receives: Drug: Tolvaptan |
| Control group | No intervention | No tolvaptan treatment but following the same visit and investigation plan as the subjects in the tolvaptan group |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in total Kidney Volume (tKV) measured by MRI scanning
The change in the total Kidney Volume after six and 12 weeks participation in the trial
Measured: Between baseline and six weeks and between six and 12 weeks
Secondary outcomes (6)
Changes in GFR
The changes in GFR measured by Cr-EDTA clearance
Measured: Between baseline and six weeks and between baseline and 12 weeks
Changes in relevant genetic and non-genetic biomarkers associated with CKD and ESRD
Prediction of change in progression of the disease over time in the genes PKD1, PKD2, PKHD1 and HNF1B. The following biomarkers will be determined: NGAL, UMOD, MCP-1, KIM-1, cystatin-C and copeptin
Measured: Between baseline and six weeks and between baseline and 12 weeks
Changes in Quality of Life
Questionnaire SF36 Health Survey - with 36 questions to subject's health and wellbeing
Measured: Between baseline and six weeks and between baseline and 12 weeks
Subject estimation of own health
Estimated by a Visual Analogue Scale from 0 (worth wellbeing) to 100 (best wellbeing
Measured: Between baseline and six weeks and between baseline and 12 weeks
Changes in ASAT and ALAT
Changes estimated from laboratory results
Measured: Between baseline and six weeks and between baseline and 12 weeks
Incidence of Adverse Events
Evaluation of Adverse Events including severity, causality, outcome and seriousness assessments
Measured: Between baseline and six weeks and between baseline and 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2018
- Study start12 Sep 2018
- Primary completionJun 2019 (estimated)
- Study completionApr 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autosomal Dominant Polycystic KidneyStudies 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
- Randomised
- Intervention model
- Parallel groups
- Model description
- Patients will be randomised in a 1:1 ration either to treatment with tolvaptan for six weeks followed by six weeks observation without trial medicaion or to the Control group receiving no tolvaptan treatment for 12 weeks
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Lisbet Brandi
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Lisbet Brandi, Nordsjaellands Hospital
- Investigators
- Lisbet Brandi, MD DMSc MHM, Principal investigator, KNEA, Nordsjaellands Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03596957
- Sponsor's protocol ID
- Acronym
- Status last verified
- Nov 2018
- First submitted
- 14 Feb 2018
- Last updated
- 26 Nov 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Nov 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Subacute Effect of Tolvaptan on Total Kidney Volume in Adult Patients With Autosomal Dominant Polycystic Kidney Disease
- ClinicalTrials.gov ID
- NCT03596957
- Acronym / sponsor ID
- PoCKET / PoCKET
- Conditions
- Autosomal Dominant Polycystic Kidney
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- Lisbet BrandiUniversity, hospital or other
- Study start
- 12 Sep 2018 (actual)
- Primary completion
- Jun 2019 (estimated)
- Study completion
- Apr 2020 (estimated)
- First posted
- 24 Jul 2018 (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.
Investigator initiated controlled multi-centre trial in a Prospective, Randomised, Open, Blinded Endpoint (PROBE) design.
Patients will be randomised in a 1:1 ratio either to treatment with tolvaptan for six weeks followed by six weeks observation without trial medication or no tolvaptan treatment, but following the same visit and investigation plan as the subjects taking tolvaptan.
Detailed description
Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common genetic kidney disease and the fourth leading cause of end-stage renal disease in adults Worldwide.
The tolvaptan tablet has been approved by EMA (European Medicines Agency) with the indication of slowing the progression of cysts development and renal insufficiency in adults with ADPKD. It is the newest and only possible treatment for this patient group and could be initiated in patients with evidence for rapidly progressive disease Development.
There is however in Denmark and other countries both scientific and financial reluctance to initiate this expensive treatment for several reasons e.g. selection of patients who might benefit, effect on progression of kidney disease, side effects and tolerability.
Before deciding on implementation in Denmark, more knowledge is needed. The results of the PoCKET trial will contribute with guidance on this decision.
Foremost the trial is designed to address not only the change in kidney volume, but the change in kidney function, which is what matters to the patients and their prognosis in terms of postponing time to end stage renal disease. Furthermore, important data on side effects and tolerability will be generated.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
- Model description
- Patients will be randomised in a 1:1 ration either to treatment with tolvaptan for six weeks followed by six weeks observation without trial medicaion or to the Control group receiving no tolvaptan treatment for 12 weeks
| Arm | Type | Receives |
|---|---|---|
| Tolvaptan group | Active comparator | Drug: TolvaptanTreatment with tolvaptan for six weeks followed by six weeks observation without trial medication |
| Control group | No intervention | –No tolvaptan treatment but following the same visit and investigation plan as the subjects in the tolvaptan group |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Adult patients between 18 and 65 years
- Diagnosis of typical ADPKD
- tKV above or equal to 750 ml by MRI scanning
- Estimated GFR (e-GFR) by CKD-EPI formula of above or equal to 45 mL/min/1.73 m2
Key exclusion criteria (8 listed)
- Kidney transplant recipient
- Known liver disease except for liver cysts relating to ADPKD
- ASAT and ALAT above upper normal level
- Current treatment with thiazide and thiazide-line diuretics, mineral corticoid receptor antagonists, amiloride or loop diuretics
- Evidence of urinary tract obstruction
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: JinarcAt baseline the tolvaptan dosing will start with daily morning and afternoon doses of 45 mg and 15 mg respectively, with weekly increases to 60 mg and 30 mg and then to 90 mg and 30 mg according to subject tolerability | Drug | Tolvaptan group |
Primary outcome
Change in total Kidney Volume (tKV) measured by MRI scanning
Time frame: Between baseline and six weeks and between six and 12 weeks
The change in the total Kidney Volume after six and 12 weeks participation in the trial
6 secondary outcomes
Changes in GFR
Time frame: Between baseline and six weeks and between baseline and 12 weeks
Changes in relevant genetic and non-genetic biomarkers associated with CKD and ESRD
Time frame: Between baseline and six weeks and between baseline and 12 weeks
Changes in Quality of Life
Time frame: Between baseline and six weeks and between baseline and 12 weeks
Subject estimation of own health
Time frame: Between baseline and six weeks and between baseline and 12 weeks
Changes in ASAT and ALAT
Time frame: Between baseline and six weeks and between baseline and 12 weeks
Incidence of Adverse Events
Time frame: Between baseline and six weeks and between baseline and 12 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Nov 2018.
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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.
Lisbet Brandi, MD DMSc MHM principal investigator
KNEA, Nordsjaellands Hospital
6 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, run by Lisbet Brandi at 6 sites in Denmark.
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 4 inclusion and 8 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: status not confirmed, as of the sponsor's last update on 26 Nov 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Denmark. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Lisbet Brandi, MD DMSc MHM (+45 48295993, lisbet.brandi@regionh.dk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in total Kidney Volume (tKV) measured by MRI scanning (time frame: Between baseline and six weeks and between six and 12 weeks). 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 26 Nov 2018.