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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Tolvaptan group | Active Comparator | Treatment 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 |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Tolvaptan | Drug | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | Between baseline and six weeks and between six and 12 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Changes in GFR | The changes in GFR measured by Cr-EDTA clearance | Between baseline and six weeks and between baseline and 12 weeks |
| Changes in relevant genetic and non-genetic biomarkers associated with CKD and ESRD |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Lisbet Brandi, MD DMSc MHM | Contact | +45 48295993 | lisbet.brandi@regionh.dk | |
| Clinical Project Manager | Contact | +45 48294714 | charlotte.bjernved.nielsen@regionh.dk |
| Name | Affiliation | Role |
|---|---|---|
| Lisbet Brandi, MD DMSc MHM | KNEA, Nordsjaellands Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Aarhus University Hospital - Site 43 | Recruiting | Skejby | Aarhus N | 8200 | Denmark |
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| ID | Term |
|---|---|
| D016891 | Polycystic Kidney, Autosomal Dominant |
| ID | Term |
|---|---|
| D007690 | Polycystic Kidney Diseases |
| D052177 | Kidney Diseases, Cystic |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
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| ID | Term |
|---|---|
| D000077602 | Tolvaptan |
| ID | Term |
|---|---|
| D001552 | Benzazepines |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D006571 | Heterocyclic Compounds |
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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
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The endpoint blinding will be assured since all the MRI scans will be forwarded to the Comparative Medicine Laboratory in Aarhus for evaluation
|
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
| 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 | 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 | Between baseline and six weeks and between baseline and 12 weeks |
| Changes in ASAT and ALAT | Changes estimated from laboratory results | 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 | Between baseline and six weeks and between baseline and 12 weeks |
| Odense University Hospital - Site 45 | Not yet recruiting | Odense | Odense C | 5000 | Denmark |
|
| Rigshospitalet - Site 42 | Recruiting | Copenhagen | 2100 | Denmark |
|
| Herlev Hospital | Recruiting | Herlev | 2730 | Denmark |
|
| Nordsjaellands Hospital - Site 41 | Recruiting | Hillerød | 3400 | Denmark |
|
| Sjællands University Hospital Roskilde | Not yet recruiting | Roskilde | 4000 | Denmark |
|
| D052776 |
| Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000072661 | Ciliopathies |
| D030342 | Genetic Diseases, Inborn |