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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
90
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
Aarhus University Hospital - Site 43
Skejby, Aarhus N, Denmark
RECRUITINGOdense University Hospital - Site 45
Odense, Odense C, Denmark
NOT_YET_RECRUITINGRigshospitalet - Site 42
Copenhagen, Denmark
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
Time frame: Between baseline and six weeks and between six and 12 weeks
Changes in GFR
The changes in GFR measured by Cr-EDTA clearance
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
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
Time frame: 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
Time frame: 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
Time frame: Between baseline and six weeks and between baseline and 12 weeks
Changes in ASAT and ALAT
Changes estimated from laboratory results
Time frame: 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
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Herlev Hospital
Herlev, Denmark
RECRUITINGNordsjaellands Hospital - Site 41
Hillerød, Denmark
RECRUITINGSjællands University Hospital Roskilde
Roskilde, Denmark
NOT_YET_RECRUITINGTime frame: Between baseline and six weeks and between baseline and 12 weeks