Subacute Effect of Tolvaptan on Total Kidney Volume in Adult Patients With Autosomal Dominant Polycystic Kidney Disease

NCT ID: NCT03596957

Last Updated: 2018-11-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE4

Total Enrollment

90 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-09-12

Study Completion Date

2020-04-30

Brief Summary

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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.

Detailed Description

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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

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Autosomal Dominant Polycystic Kidney

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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
Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors
The endpoint blinding will be assured since all the MRI scans will be forwarded to the Comparative Medicine Laboratory in Aarhus for evaluation

Study Groups

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Tolvaptan group

Treatment with tolvaptan for six weeks followed by six weeks observation without trial medication

Group Type ACTIVE_COMPARATOR

Tolvaptan

Intervention Type 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

Control group

No tolvaptan treatment but following the same visit and investigation plan as the subjects in the tolvaptan group

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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Tolvaptan

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

Intervention Type DRUG

Other Intervention Names

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Jinarc

Eligibility Criteria

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Inclusion Criteria

* 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

Exclusion Criteria

* 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
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Lisbet Brandi

OTHER

Sponsor Role lead

Responsible Party

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Lisbet Brandi

Head of Department for Endrocrinology and Nephrology, MD, DMSc, MHM

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

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Lisbet Brandi, MD DMSc MHM

Role: PRINCIPAL_INVESTIGATOR

KNEA, Nordsjaellands Hospital

Locations

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Aarhus University Hospital - Site 43

Skejby, Aarhus N, Denmark

Site Status RECRUITING

Odense University Hospital - Site 45

Odense, Odense C, Denmark

Site Status NOT_YET_RECRUITING

Rigshospitalet - Site 42

Copenhagen, , Denmark

Site Status RECRUITING

Herlev Hospital

Herlev, , Denmark

Site Status RECRUITING

Nordsjaellands Hospital - Site 41

Hillerød, , Denmark

Site Status RECRUITING

Sjællands University Hospital Roskilde

Roskilde, , Denmark

Site Status NOT_YET_RECRUITING

Countries

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Denmark

Central Contacts

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Lisbet Brandi, MD DMSc MHM

Role: CONTACT

+45 48295993

Clinical Project Manager

Role: CONTACT

+45 48294714

Facility Contacts

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Henrik Birn, MD

Role: primary

+45 6171 7870

Karin Hansen

Role: backup

+45 4046 0831

Helle Thiesson, MD

Role: primary

+45 6541 1642

Kristian Bergholt Buhl, MD

Role: backup

+456541 1642

Bo Feldt-Rasmussen, MD

Role: primary

+45 3545 2135

Tobias Bomholt, MD

Role: backup

+45 35451838

Ditte Hansen, MD

Role: primary

+45 3868 2056

Marie Moeller, MD

Role: backup

+45 6169 5364

Lisbet Brandi, MD

Role: primary

+45 48295993

Charlotte Bjernved Nielsen

Role: backup

+45 49294714

Bjarne Ørskov, MD

Role: primary

+45 2966 2426

Other Identifiers

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PoCKET

Identifier Type: -

Identifier Source: org_study_id

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