Overview
Sponsor-declared trial summary
Autosomal Dominant Polycystic Kidney Disease
We aim to determine if arterial stiffness is exacerbated due to a high-salt diet in patients with ADPKD and whether treatment with amiloride prevents these effects. We divided the main objective into three sub-objectives: 1. To determine if arterial stiffness is exacerbated due to a high-salt diet in patients with ADPK…
Key facts
- Sponsor
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Trial duration
- completed 16 May 2025
- Decision date (initial)
- 2024-05-24
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- No
External identifiers
- EU CT number
- 2024-512544-27-00
- EudraCT number
- 2020-000433-40
- ClinicalTrials.gov
- NCT05228574
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy
We aim to determine if arterial stiffness is exacerbated due to a high-salt diet in patients with ADPKD and whether treatment with amiloride prevents these effects.
We divided the main objective into three sub-objectives:
1. To determine if arterial stiffness is exacerbated due to a high-salt diet in patients with
ADPKD.
2. To investigate whether amiloride treatment could reduce the effect of high salt on arterial
stiffness in the group with salt supplement.
3. To investigate whether treatment with amiloride further reduces arterial stiffness in the
setting of a low-salt diet.
Conditions and MedDRA coding
Autosomal Dominant Polycystic Kidney Disease
Study design 1 period
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Intervention period (4 weeks) This is a single-center, double-blinded, randomized and placebo-controlled study (with sodium chloride / placebo capsules) with an open-label treatment part (with amiloride) in subjects with ADPKD with preserved renal function.
|
Randomised Controlled | Double | [{"id":58820,"code":3,"name":"Monitor"},{"id":58823,"code":2,"name":"Investigator"},{"id":58824,"code":1,"name":"Subject"},{"id":58822,"code":5,"name":"Carer"},{"id":58821,"code":4,"name":"Analyst"}] | Group 1: Group 1: receive sodium chloride capsules (6 grams/day) after wash-out period till the end of the study (in total 4 weeks). Amiloride will be started in the last two weeks of the study (together with sodium chloride capsules) till the end. Group 2: Placebo capsules: Group 2: receive sodium placebo capsules (6 grams/day) after wash-out period till the end of the study (in total 4 weeks). Amiloride will be started in the last two weeks of the study (together with sodium chloride capsules) till the end. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 3
- Adults with typical ADPKD diagnosed based on Ravine criteria and/or a documented Pkd 1 or 2 mutation
- CKD-EPI eGFR ≥60 ml/min/1.73m2
- Ability to provide informed consent
Exclusion criteria 11
- Uncontrolled hypertension, defined as an office blood pressure of ≥160/ ≥90 mmHg with or without antihypertensive treatment
- Concomitant use of ≥ 3 antihypertensive medications
- When antihypertensive treatment is prescribed for any other treatment indication than hypertension (e.g. cardia arrhythmia)
- 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 £ 2 antihypertensive drugs
- Actual pregnancy or unwillingness to adhere to reproductive precautions during the duration of the study
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- The three primary outcomes of this study are the differences in pulse wave velocity (PWV) in high salt versus placebo group, and before versus after amiloride treatment in both groups.
Secondary endpoints 5
- Ambulatory (24-hours) blood pressure measurements
- Serum markers of inflammation
- Other relevant serum markers for inflammation and endothelial dysfunction
- 23Na-MRI scans measuring interstitial sodium accumulation in a subgroup of patients
- Salt tasting sensitivity
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 2
SUB05433MIG · Substance
- Active substance
- Amiloride
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 20 mg milligram(s)
- Max total dose
- 560 mg milligram(s)
- Max treatment duration
- 2 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB12581MIG · Substance
- Active substance
- Sodium Chloride
- Pharmaceutical form
- CAPSULE, HARD
- Route of administration
- ORAL
- Max daily dose
- 23 g gram(s)
- Max total dose
- 644 g gram(s)
- Max treatment duration
- 4 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Placebo 2
SUB12237MIG · Substance
- Active substance
- Potato Starch
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 23 g gram(s)
- Max total dose
- 644 g gram(s)
- Max treatment duration
- 4 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Sponsor organisation
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Address
- Dr. Molewaterplein 40
- City
- Rotterdam
- Postcode
- 3015 GD
- Country
- Netherlands
Scientific contact point
- Organisation
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Contact name
- M. Salih
Public contact point
- Organisation
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Contact name
- M. Salih
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Netherlands | Ended | 54 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-04-30 | Netherlands | Acceptable 2024-05-24
|
2024-05-24 |