Impact of Patiromer treatment on dietary Potassium intake restriction and health-related quality of life and nutrition in patients on Chronic Dialysis Therapy: a Double-Blind, Prospective, Randomized, Placebo-Controlled, Pilot Trial

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Istituto Di Ricerche Farmacologiche Mario Negri

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

Trial duration

31 Jul 2025 → ongoing

Decision date (initial)

2025-07-08

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Vifor

External identifiers

EU CT number

2025-521378-33-00

ClinicalTrials.gov

NCT06858280

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others

To assess whether treatment with the oral potassium binder patiromer as compared to placebo allows withdrawal or down-titration of potassium dietary restriction considered as a dichotomous variable (YES/NO) without increasing the risk of hyperkalemia (serum potassium >5.5 mEq/L) in chronic dialysis patients with pre-dialysis serum potassium 4 to 5.5 mEq/L ) achieved and maintained by dietary restriction of potassium reach aliments

Secondary objectives 8

1. To assess whether patiromer treatment as compared to placebo - Allows withdrawal of any potassium dietary restriction considered as a single endpoint without increasing the risk of hyperkalemia
2. To assess whether patiromer treatment as compared to placebo allows reducing dietary restriction considered as a continuous variable as assessed by a dietary score (to be defined): 0 = No restriction; 1 = Moderate restriction; 2 = Strict restriction
3. To assess whether patiromer treatment as compared to placebo reduces pre-dialysis serum potassium level considered as a continuous variable
4. To assess whether patiromer treatment as compared to placebo reduces the need for rescue therapy with potassium-binding resins such as Sodium polystyrene sulfonate (SPS, Kayexalate®, Sanofi-Aventis S.p.A) or supplementary dialysis sessions to treat intercurrent hyperkalemia
5. To assess whether patiromer treatment as compared to placebo allows introducing or optimizing RAS inhibitor therapy to control arterial blood pressure or heart failure and/or prevent cardiovascular events without increasing the risk of hyperkalemia
6. To assess whether patiromer treatment as compared to placebo reduces the risk of cardiac arrhythmias or other symptoms (muscle weakness, fatigue, hand of feet numbness or tingling, vomiting, flaccid paralysis and others) potentially related to hyperkalemia
7. To assess whether patiromer treatment as compared to placebo does not increase the risk of hypokalemia (serum potassium <3.5 mEq/L) and/or of cardiac arrhythmias potentially related to hypokalemia
8. To assess whether patiromer treatment as compared to placebo improves patient-perceived health-related quality of life (hrQoL: as assessed by submission of a standardized questionnaire) in particular concerning withdrawn/down-titration of potassium dietary restriction and improved diet palatability (as evaluated by submission of a standardized questionnaire)

Conditions and MedDRA coding

End Stage Kidney Disease on chronic dialysis therapy

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. More than 18-year-old
2. Chronic and stable dialysis therapy with three weekly dialysis sessions for at least three months because of ESKD
3. Pre-dialysis (in the long interdialytic period) serum potassium 4 to 5.5 mEq/L confirmed in two consecutive weeks, without any clinical signs or symptoms of hyperkalemia
4. Stable therapy (since at least 3 months) with RAS inhibitors or MRAs. No treatment with potassium sparing diuretics On standardized and stable (moderately or strictly restricted) low-potassium diet
5. Compliance with recommended diet
6. Written informed consent

Exclusion criteria 18

1. Hyperkalemia (pre-dialysis potassium >5.5 mEq/L during the long interdialytic period)
2. Hypomagnesemia (serum magnesium <1.7 mg/dL)
3. Hypercalcemia (serum calcium >10.5mg/dl)
4. Ongoing treatment with potassium binding medications including Sodium polystyrene sulfonate (SPS, Kayexalate®, Sanofi-Aventis S.p.A) or Sodium zirconium cyclosilate (Lokelma®, Astra Zeneca S.p.A.)
5. Ongoing treatment with potassium-sparing diuretics
6. Pre-dialysis potassium <4.0 mEq/L during the long interdialytic period
7. One or two weekly dialysis session
8. Poor compliance to prescribed potassium-restricted diet
9. History of bowel obstruction or major gastrointestinal surgery, severe gastrointestinal disorders, or swallowing disorders
10. Previous history of cardiac arrhythmias potentially related to hypokalemia
11. Known hypersensitivity to the active ingredient or any of the excipients of the study drug
12. Inability to fully understand the potential risks and benefits related to study participation
13. Concomitance of clinical conditions that could jeopardize the completion of the treatment period and/or confound data interpretation including: • Cancer (except non-metastatic cutaneous cancers) • Active systemic autoimmune diseases • Concomitant treatment with steroids or any other immunosuppressive agent • Severe/unstable heart failure requiring hospitalization or changes in pharmacological therapy or supplementary dialysis sessions over the last three months • Refractory severe hypertension (BP >180/100 mmHg despite optimized pharmacological treatment with at least three blood pressure-lowering medications) • Known to be positive for human immunodeficiency virus • Drug or alcohol abuse
14. Pregnancy, lactation, or intention to become pregnant before or during the study period, or within 90 days of the last dose of study treatment
15. Intention to donate ova or sperm over the same period
16. Childbearing potential without highly effective contraception methods according to the 2020 CTFG Recommendations related to contraception and pregnancy testing in clinical trials (https://www.hma.eu/fileadmin/dateien/Human_Medicines/01About_HMA/Working_Groups/CTFG/2020_09_HMA_CTFG_Contraception_guidance_Version_1.1_updated.pdf)
17. Involvement in the study planning and/or conduct
18. Participation in another clinical study with an investigational product during the last month

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Proportion of participants with low-potassium diet withdrawal and/or down-titration at completion of the treatment period compared to diet at inclusion

Secondary endpoints 8

1. Proportion of participants with low potassium diet withdrawal at completion of the treatment period, considered as a single endpoint
2. Reduction in dietary restriction considered as a continuous variable as assessed by a dietary score (to be defined): 0 = No restriction; 1 = Moderate restriction; 2 = Strict restriction
3. Changes in pre-dialysis serum potassium level considered as a continuous variable
4. Reduced need for rescue therapy with potassium binding resins such as Sodium polystyrene sulfonate (SPS, Kayexalate®, Sanofi-Aventis S.p.A) or supplementary dialysis sessions to treat intercurrent hyperkalemia
5. Introduction or optimization of RAS inhibitor therapy to control arterial blood pressure or heart failure and/or prevent cardiovascular events without increasing the risk of hyperkalemia
6. Proportion of patients with cardiac arrhythmias or other symptoms (muscle weakness, fatigue, hand of feet numbness or tingling, vomiting, flaccid paralysis and others) potentially related to hyperkalemia
7. Proportion of patients with hypokalemia (serum potassium <3.5 mEq/L) and/or of cardiac arrhythmias potentially related to hypokalemia
8. Participant’s quality of life change from baseline at the end of the intervention, measured using the Italian translation of the Kidney Disease Quality of Life Instrument (KDQOL) Version 1.31 and the standard SF-36 quality of life questionnaire Italian Version 1.6

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Istituto Di Ricerche Farmacologiche Mario Negri

Sponsor organisation

Istituto Di Ricerche Farmacologiche Mario Negri

Address

Via Gian Battista Camozzi 3

City

Ranica

Postcode

24020

Country

Italy

Scientific contact point

Organisation

Istituto Di Ricerche Farmacologiche Mario Negri

Contact name

Ufficio Attività Regolatorie

Public contact point

Organisation

Istituto Di Ricerche Farmacologiche Mario Negri

Contact name

Ufficio Attività Regolatorie

Locations

1 EU/EEA country · 5 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 22 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications