E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Autosomal dominant polycystic kidney disease |
Autosomaal dominante polycysteuze nierziekte |
|
E.1.1.1 | Medical condition in easily understood language |
Cystic kidney disease |
Cystenieren |
|
E.1.1.2 | Therapeutic area | Not possible to specify |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The main goal of this study is to determine if arterial stiffness is exacerbated due to a high-salt diet in patients with ADPKD. |
Het hoofddoel van deze studie is om aan te tonen of arteriële vaatstijfheid afhankelijk is van een hoog-zout dieet bij patiënten met ADPKD. |
|
E.2.2 | Secondary objectives of the trial |
The second goal is to determine whether treatment with amiloride prevents the effects of a high-salt diet. |
Het tweede doel is om te onderzoeken of amiloride het effect van een hoog-zout dieet teniet kan doen. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Inclusion Criteria: • Adults with ADPKD diagnosis based on Ravine criteria and/or with PKD1 or PKD2 mutation • CKD-EPI eGFR ≥ 60 ml/min/1.73m2 • Ability to provide informed consent |
Inclusie criteria: • Volwassenen met de diagnose ADPKD, gebaseerd op de Ravine criteria en/of een PKD1 of PKD2 mutatie • CKD-EPI eGFR ≥ 60 ml/min/1.73m2 • Vermogen om informed consent te geven |
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E.4 | Principal exclusion criteria |
Exclusion criteria: - Uncontrolled hypertension, defined as an office blood pressure of ≥160/ ≥90 mmHg with or without antihypertensive treatment - Concomitant use of ≥3 antihypertensive medications - Serum potassium levels >5.5 mmol/L (measured within last 6 months) - History of liver disease (excluding liver cysts due to ADPKD) - History of heart failure (cardiac ejection fraction < 35%) or cardiac arrhythmia - History of diabetes mellitus - Active infection or antibiotic therapy - Immunosuppressive therapy within the last year - Concomitant use of drugs that could influence blood pressure and/or disease progression (Tolvaptan/non-steroidal anti-inflammatory drugs (NSAIDs)/chemotherapy), excluding <3 antihypertensive drugs - Actual pregnancy or unwillingness to adhere to reproductive precautions during the duration of the study |
Exclusie criteria: - Niet goed behandelde hypertensie, gedefinieerd als een spreekkamer bloeddruk van ≥160/ ≥90 mmHg met/zonder antihypertensiva - Gebruik van ≥3 antihypertensive medicaties - Serum kalium gehalte van >5.5 mmol/L (gedurende de laatste 6 maanden) - Leverfalen (exclusief lever cysten door ADPKD) - Hartfalen (ejectie fractie < 35%) of hartritmestoornissen - Diabetes mellitus - Actieve infectie of antibiotica gebruik - Gebruik van immunosuppressiva gedurende het laatste jaar - Gebruik van medicatie dat bloeddruk of ziekte progressie van ADPKD beïnvloedt (Tolvaptan/NSAIDs/chemotherapie), behalve <3 antihypertsive medicaties - Actueel zwangerschap of onwil tot het nemen van anticonceptie maatregelen gedurende gehele studie |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary outcome of this study is a treatment group difference in non-invasively measured central PWV with or without high-salt diet. |
De primaire uitkomst van deze studie is het groepsverschil van non-invasief gemeten arteriële vaatstijfheid met of zonder hoog-zout dieet. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
At week 3, week 5 and at the end of the study |
In week 3, week 5 en op het eind van de studie |
|
E.5.2 | Secondary end point(s) |
Secondary outcomes include ambulatory (24-hour) blood pressures, markers of inflammation, salt tasting test and skin sodium content through 23Na-MRI. |
Secundaire uitkomstmaten zijn 24-uurs bloeddruk meting, markers van inflammatie, zoutsmaak gevoeligheidstesten en zoutopslag in de huid gemeten met 23Na-MRI. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
At week 3, week 5 and at the end of the study |
In week 3, week 5 en op het eind van de studie |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | No |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Een dubbelblinde, gerandomiseerde placebo gecontroleerde trial met open-label amiloride behandeling |
A double blinded, randomized placebo controlled trial with open-label treatment with amiloride |
|
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
LVLS |
Laatste visite van de laatste patient |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |