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NCT05766839PHASE2Recruiting

Effect of Patiromer in Children Under 12 Years of Age With High Blood Potassium Levels

Vifor Pharma, Inc.

Start Date

4/6/2025

Completion Date

12/1/2030

Summary

A study to evaluate the pharmacodynamic effects, safety, and tolerability of patiromer in children under 12 years of age with hyperkalaemia.

Eligibility Criteria

Age Range: No minimum to 11 years

Inclusion Criteria: * Paediatric participants (\<12 years of age) with hyperkalaemia at screening. * Participant's age should not reach 12 years during the 28 days of the pharmacodynamic/dose-ranging period. * Participant is able to receive regular external feeding and medication, including via tubes, i.e., percutaneous endoscopic gastrostomy (PEG) or entero-gastric feeding tube. * At screening/baseline, the results from 2 separate and consecutive potassium assessments using the same measurement method (whole blood, plasma, or serum) need to be above the age-appropriate upper limit of normal (ULN). * In continuation to above criterion: At least 1 sample needs to be taken at screening/baseline and 1 sample should not be older than 30 days, i.e., a historical sample. The average of the 2 potassium values needs to be above the age-appropriate ULN of the measurement method plus 0.5 mEq/l (equivalent to 0.5 mmol/l). If the 2 samples are taken on the day of screening/baseline, the 2 individual potassium values must not differ from each other by more than 0.5 mEq/l. * In the opinion of the Investigator, the participant is expected to require treatment for hyperkalaemia for at least 28 days upon enrolment in the study. * If taking any renin-angiotensin aldosterone system inhibitors (RAASi), beta blockers, fludrocortisone, or diuretic medications, must be on a stable dose for at least 14 days prior to screening. * Parent(s) or legally acceptable representative(s) has provided the appropriate written informed consent, in accordance with local regulations. The assent of the child should also be obtained when appropriate or if requested by the Institutional Review Board (IRB)/Ethics Committee (EC)/Independent Ethics Committee (IEC). The written informed consent must be provided before any study specific procedures are performed including screening procedures. * Parent(s) or legally authorised representative(s) or another appropriate person delegated by the legally authorised representatives must be available to help the study-site personnel ensure follow-up; accompany the participant to the study site on each assessment day according to the Schedule of Events (Table 1, Table 2) (e.g., able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures); accurately and reliably dispense investigational product as directed. * Not applicable to Norway sites: Females of childbearing potential must be non-lactating, must have a negative pregnancy test at screening; and must have used an effective, acceptable form of contraception (e.g., abstinence) for at least 1 month before patiromer administration. Females of childbearing potential must agree to continue using contraception throughout the study and for 1 month after the last dose of patiromer. * If undergoing peritoneal dialysis, participants must be on a stable treatment plan for a minimum of 4 weeks prior to screening, or at least 8 weeks prior to screening if newly initiated on peritoneal dialysis. * For participants who have undergone organ transplantation, the general clinical condition must be stable for at least 4 weeks prior to Screening and is not expected to change during the first 4 weeks of the study. * For participants who have undergone organ transplantation, must have persistent hyperkalaemia requiring potassium-lowering therapy despite correction of reversible causes (e.g., high calcineurin inhibitor levels). Exclusion Criteria: * Preterm birth infants with \<37 weeks of gestation cannot be included in Cohort 3. * Participants who due to their general condition, e.g., anaemia or low body weight, are not suitable to have blood volume withdrawn as specified in the Schedule of Events. * Participants with pseudo-hyperkalaemia due to haemolysis or to abnormally high numbers of platelets (above ULN), leukocytes (above ULN), or erythrocytes (above ULN) at screening based on results obtained from the local laboratory. * Any participant with evidence of potential potassium-related 12-lead electrocardiogram (ECG) changes (i.e., changes consistent with hyper- or hypokalaemia) at screening. * Any participant with serum magnesium \<1.4 mg/dl (0.58 mmol/l) at screening/baseline. * Any of the following renal conditions: maintenance haemodialysis, renal artery stenosis, and acute kidney injury (defined by 2012 Kidney Disease Improving Global Outcomes) or a history of acute renal insufficiency in the past 3 months. Note: Chronic kidney disease (CKD) is not excluded. * A history of or current diagnosis of a severe gastrointestinal (GI) diagnosis or surgery that could affect GI transit of the drug (delayed gastric emptying), such as a severe swallowing disorder, severe gastroesophageal reflux, uncorrected pyloric stenosis, intussusception, any other intestinal obstruction (e.g., Hirschsprung disease, chronic intestinal pseudo-obstruction, clinically significant postsurgical abdominal adhesions) or any gut-shortening surgical procedure prior to screening. * In continuation to above criterion: Pre-gastric above-mentioned pathologies may be disregarded in case of existence of a PEG or entero-gastric feeding tube, as the PEG or entero-gastric feeding tube will serve for nutrition and investigational product administration. * Liver enzymes (alanine aminotransferase or aspartate aminotransferase) more than 3 times the ULN at screening, based on the local laboratory, as well as the participant's respective age. * Active cancer, currently on cancer treatment, or history of cancer in the past 2 years (except for non-melanoma skin cancer). * Scheduled for kidney transplant procedure during the first 28 days after Day 1. * History of sudden infant death in a sibling (only for participants \<2 years of age at screening). * Has severe hypoxaemia, respiratory acidosis, asphyxia, or hypotension 3 months before screening based on assessment of the Investigator. * Participants treated with sodium polystyrene sulphonate, calcium polystyrene sulphonate, sodium zirconium cyclosilicate, or patiromer within the last 48 hours prior to fulfilling the baseline baseline potassium assessments requested in 4th Inclusion Criterion. * Use of the following medications if doses have not been stable for at least 14 days prior to screening or if doses are anticipated to change during the 4-week pharmacodynamic/ dose-ranging period: digoxin, bronchodilators, theophylline, heparins (including low molecular heparins), tacrolimus, mycophenolate mofetil, cyclosporine, trimethoprim, or cotrimoxazole. * Use of any investigational product for an unapproved indication within 30 days prior to screening or within 5 half-lives, whichever is longer. * Known hypersensitivity to patiromer or its components. * In the opinion of the Investigator, parent(s) or legal representative(s) inability to comply with the protocol. * In the opinion of the Investigator, any medical condition, uncontrolled systemic disease, or serious intercurrent illness that would significantly decrease study compliance or jeopardise the safety of the participant or potentially affect the quality of the data such as: * In continuation to above criterion: hyperkalaemia at screening that requires emergency intervention; cardiovascular event or intervention within 3 months prior to screening; a haemodynamically unstable arrhythmia; hospitalisation for heart failure within the past 3 months; poorly controlled blood pressure; poorly controlled diabetes mellitus or frequent need for adjustment in insulin prescription or recent hospitalisation for treatment of hyper or hypoglycaemia. * If the child is being breastfed: 1. There is suspicion of current alcohol or substance misuse/abuse in breastfeeding mother. 2. The breastfeeding mother is taking potassium supplements

Interventions

DRUG

Patiromer

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Conditions

Hyperkalemia

Locations

Children's Hospital Colorado

Aurora, Colorado 80045

United States

UF Health Pediatric Multispecialty Center Site

Jacksonville, Florida 32207

United States

Miller School of Medicine, University of Miami

Miami, Florida 33124

United States

Arnold Palmer Hospital for Children

Orlando, Florida 32806

United States

Augusta University - Children's Hospital of Georgia

Augusta, Georgia 30912

United States

University of Illinois College of Medicine

Peoria, Illinois 61605

United States

Boston Children's Hospital

Boston, Massachusetts 02115

United States

Children's Mercy Hospitals and Clinics

Kansas City, Missouri 64108

United States

Northwell Health Cohen Children's Medical Center

New York, New York 11042

United States

Duke University Hospital

Durham, North Carolina 27710

United States

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania 19104

United States

Vanderbilt Children's Hospital Neurology

Nashville, Tennessee 37232

United States

Texas Tech University Health Sciences Center Amarillo

Amarillo, Texas 79106

United States

Monash Medical Centre Clayton

Clayton, 3168

Australia

The Royal Children's Hospital (RCH)

Parkville, 3052

Australia

Children's Hospital Westmead Centre for Kidney Research

Westmead, 2145

Australia

Universitair Ziekenhuis Gent

Ghent, 9000

Belgium

UZ Leuven

Leuven, 3000

Belgium

Helsingin Yliopistollinen Keskussairaala Uusi Lastensairaala

Helsinki, 00290

Finland

CHRU Montpellier - Arnaud de Villeneuve

Montpellier, 34090

France

Assistance Publique-Hopitaux de Paris Robert-Debre

Paris, 75019

France

Hôpital des Enfants - Toulouse

Toulouse, 31300

France

Ippokratio Thessaloniki General Hospital

Thessaloniki, Thessaloniki

Greece

Pan and Aglaia Kyriakou Children's Hospital

Athens,

Greece

Shaare Zedek Medical Center

Jerusalem, 9103102

Israel

Schneider Children's Medical Center of Israel

Petach Tikvah, 4920235

Israel

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122

Italy

IRCCS Ospedale Pediatrico Bambino Gesù

Rome, 00165

Italy

Helse Bergen HF Haukeland Universitetssjukehus

Bergen, 5009

Norway

Uniwersytecki Szpital Kliniczny we Wrocławiu

Wroclaw, 50-369

Poland

Unidade Local de Saude de Santo Antonio, E.P.E.

Porto, 4050-342

Portugal

Sidra Medicine

Doha,

Qatar

Spitalul Clinic de Urgenta pentru Copii Louis Turcanu Timisoara

Timișoara, 300011

Romania

King Saud University

Riyadh, 11451

Saudi Arabia

King Faisal Specialist Hospital & Research Centre

Riyadh, 12713

Saudi Arabia

King Abdulaziz Medical City

Riyadh, 14611

Saudi Arabia

Al Jalila Children's Hospital

Dubai,

United Arab Emirates