Subacute Effect of Tolvaptan on Total Kidney Volume in Adult Patients With Autosomal Dominant Polycystic Kidney Disease (PoCKET)
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|ClinicalTrials.gov Identifier: NCT03596957|
Recruitment Status : Recruiting
First Posted : July 24, 2018
Last Update Posted : November 26, 2018
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.
|Condition or disease||Intervention/treatment||Phase|
|Autosomal Dominant Polycystic Kidney||Drug: Tolvaptan||Phase 4|
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 (Clinical Trial)|
|Estimated Enrollment :||90 participants|
|Intervention Model:||Parallel Assignment|
|Intervention 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 (Outcomes Assessor)|
|Masking Description:||The endpoint blinding will be assured since all the MRI scans will be forwarded to the Comparative Medicine Laboratory in Aarhus for evaluation|
|Official Title:||Subacute Effect of Tolvaptan on Total Kidney Volume in Adult Patients With Autosomal Dominant Polycystic Kidney Disease|
|Actual Study Start Date :||September 12, 2018|
|Estimated Primary Completion Date :||June 2019|
|Estimated Study Completion Date :||April 2020|
Active Comparator: Tolvaptan group
Treatment with tolvaptan for six weeks followed by six weeks observation without trial medication
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
Other Name: Jinarc
No Intervention: Control group
No tolvaptan treatment but following the same visit and investigation plan as the subjects in the tolvaptan group
- 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
- Changes in GFR [ Time Frame: Between baseline and six weeks and between baseline and 12 weeks ]The changes in GFR measured by Cr-EDTA clearance
- 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 ]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
- Changes in Quality of Life [ Time Frame: Between baseline and six weeks and between baseline and 12 weeks ]Questionnaire SF36 Health Survey - with 36 questions to subject's health and wellbeing
- Subject estimation of own health [ Time Frame: Between baseline and six weeks and between baseline and 12 weeks ]Estimated by a Visual Analogue Scale from 0 (worth wellbeing) to 100 (best wellbeing
- Changes in ASAT and ALAT [ Time Frame: Between baseline and six weeks and between baseline and 12 weeks ]Changes estimated from laboratory results
- Incidence of Adverse Events [ Time Frame: Between baseline and six weeks and between baseline and 12 weeks ]Evaluation of Adverse Events including severity, causality, outcome and seriousness assessments
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03596957
|Contact: Lisbet Brandi, MD DMSc MHM||+45 email@example.com|
|Contact: Clinical Project Manager||+45 firstname.lastname@example.org|
|Aarhus University Hospital - Site 43||Recruiting|
|Skejby, Aarhus N, Denmark, 8200|
|Contact: Henrik Birn, MD +45 6171 7870 email@example.com|
|Contact: Karin Hansen +45 4046 0831 Karin.firstname.lastname@example.org|
|Odense University Hospital - Site 45||Not yet recruiting|
|Odense, Odense C, Denmark, 5000|
|Contact: Helle Thiesson, MD +45 6541 1642 email@example.com|
|Contact: Kristian Bergholt Buhl, MD +456541 1642 firstname.lastname@example.org|
|Rigshospitalet - Site 42||Recruiting|
|Copenhagen, Denmark, 2100|
|Contact: Bo Feldt-Rasmussen, MD +45 3545 2135 Bo.Feldt-Rasmussen@regionh.dk|
|Contact: Tobias Bomholt, MD +45 35451838 email@example.com|
|Herlev, Denmark, 2730|
|Contact: Ditte Hansen, MD +45 3868 2056 firstname.lastname@example.org|
|Contact: Marie Moeller, MD +45 6169 5364 email@example.com|
|Nordsjaellands Hospital - Site 41||Recruiting|
|Hillerød, Denmark, 3400|
|Contact: Lisbet Brandi, MD +45 48295993 firstname.lastname@example.org|
|Contact: Charlotte Bjernved Nielsen +45 49294714 email@example.com|
|Sjællands University Hospital Roskilde||Not yet recruiting|
|Roskilde, Denmark, 4000|
|Contact: Bjarne Ørskov, MD +45 2966 2426 firstname.lastname@example.org|
|Principal Investigator:||Lisbet Brandi, MD DMSc MHM||KNEA, Nordsjaellands Hospital|