Patiromer for Treatment of Hyperkalaemia in Children Under 12 Years of Age

NCT05766839 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: RLY5016-208p2023-505252-21-00
• Study Type: interventional
• Target: 32
• Locations: 15 countries
• Sites: 37

Brief Summary

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

Conditions

Hyperkalemia

Outcome Measures

Primary Outcomes (1)

• Change in potassium levels (mmol/L)

  May be measured as serum, plasma, whole blood, potassium

  From baseline to Day 28

Secondary Outcomes (26)

• Change in potassium levels (mmol/L)

  From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

• Occurrence of treatment-emergent adverse events (TEAEs)

  Part 1: Day 1 up to end of treatment (Day 28 ±3 Days); Part 2: Day 1 up to follow-up visit (Up to 54 weeks)

• Occurrence of serious adverse events (SAEs)

  Part 1: Day 1 up to end of treatment (Day 28 ±3 Days); Part 2: Day 1 up to follow-up visit (Up to 54 weeks)

• Change from baseline in resting heart rate (beats per minute)

  From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

• Change from baseline in systolic blood pressure (mmHg)

  From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

Study Arms (1)

Patiromer

EXPERIMENTAL

4-week pharmacodynamic /dose-ranging period Cohort 1: 6 to less than(\<)12 years of age Cohort 2: 2 to \<6 years of age Cohort 3: 0 to \<2 years of age; In Cohort 3, a minimum of 3 study participants will be assessed in the subgroup of 0 to \<6 months and another 3 study participants in the subgroup 6 to \<24 months of age.

Drug: Patiromer

Interventions

Patiromer DRUG

Patiromer will be given once daily; In Cohort 3, depending on the dose and the study participant's age, the total daily dose might be split

Patiromer

Eligibility Criteria

• Age: 0 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• \- 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).
• \- 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 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; accurately and reliably dispense investigational product as directed.
• \- 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.

You may not qualify if:

• The following criteria exclude a participant from participating in this trial:
• \- 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.
• \- 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. 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.
• \- 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).
• \- 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.
• \- If the child is being breastfed:
• a)There is suspicion of current alcohol or substance misuse/abuse in breastfeeding mother
• b)The breastfeeding mother is taking potassium supplements

Sponsors & Collaborators

• Vifor Pharma, Inc.: lead

Study Sites (37)

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

NOT YET RECRUITING

UF Health Pediatric Multispecialty Center Site

Jacksonville, Florida, 32207, United States

TERMINATED

Miller School of Medicine, University of Miami

Miami, Florida, 33124, United States

RECRUITING

Arnold Palmer Hospital for Children

Orlando, Florida, 32806, United States

RECRUITING

Augusta University - Children's Hospital of Georgia

Augusta, Georgia, 30912, United States

RECRUITING

University of Illinois College of Medicine

Peoria, Illinois, 61605, United States

RECRUITING

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

Children's Mercy Hospitals and Clinics

Kansas City, Missouri, 64108, United States

RECRUITING

Duke University Hospital & Medical Center

Durham, North Carolina, 27710, United States

RECRUITING

Duke University Hospital

Durham, North Carolina, 27710, United States

RECRUITING

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Vanderbilt Children's Hospital Neurology

Nashville, Tennessee, 37232, United States

RECRUITING

Texas Tech University Health Sciences Center Amarillo

Amarillo, Texas, 79106, United States

TERMINATED

Monash Medical Centre Clayton

Clayton, 3168, Australia

RECRUITING

The Royal Children's Hospital (RCH)

Parkville, 3052, Australia

RECRUITING

Children's Hospital Westmead Centre for Kidney Research

Westmead, 2145, Australia

NOT YET RECRUITING

Universitair Ziekenhuis Gent

Ghent, 9000, Belgium

RECRUITING

UZ Leuven

Leuven, 3000, Belgium

RECRUITING

Helsingin Yliopistollinen Keskussairaala Uusi Lastensairaala

Helsinki, 00290, Finland

NOT YET RECRUITING

CHRU Montpellier - Arnaud de Villeneuve

Montpellier, 34090, France

RECRUITING

Assistance Publique-Hopitaux de Paris Robert-Debre

Paris, 75019, France

RECRUITING

Hôpital des Enfants - Toulouse

Toulouse, 31300, France

RECRUITING

Ippokratio Thessaloniki General Hospital

Thessaloniki, Thessaloniki, Greece

RECRUITING

Pan and Aglaia Kyriakou Children's Hospital

Athens, Greece

RECRUITING

Shaare Zedek Medical Center

Jerusalem, 9103102, Israel

RECRUITING

Schneider Children's Medical Center of Israel

Petach Tikvah, 4920235, Israel

RECRUITING

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

RECRUITING

IRCCS Ospedale Pediatrico Bambino Gesù

Rome, 00165, Italy

RECRUITING

Helse Bergen HF Haukeland Universitetssjukehus

Bergen, 5009, Norway

NOT YET RECRUITING

Uniwersytecki Szpital Kliniczny we Wrocławiu

Wroclaw, 50-369, Poland

NOT YET RECRUITING

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

Porto, 4050-342, Portugal

NOT YET RECRUITING

Sidra Medicine

Doha, Qatar

NOT YET RECRUITING

Spitalul Clinic de Urgenta pentru Copii Louis Turcanu Timisoara

Timișoara, 300011, Romania

RECRUITING

King Saud University

Riyadh, 11451, Saudi Arabia

NOT YET RECRUITING

King Faisal Specialist Hospital & Research Centre

Riyadh, 12713, Saudi Arabia

RECRUITING

King Abdulaziz Medical City

Riyadh, 14611, Saudi Arabia

NOT YET RECRUITING

Al Jalila Children's Hospital

Dubai, United Arab Emirates

RECRUITING

MeSH Terms

Conditions

Hyperkalemia

Interventions

patiromer

Condition Hierarchy (Ancestors)

Water-Electrolyte Imbalance; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Julian Platon, MD, PhD

  Vifor Pharma, Inc.

  STUDY DIRECTOR

Central Study Contacts

EMERALD-2 Clinical Study Team

CONTACT

+41 58 851 80 00; clinicaltrials@cslbehring.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 19, 2023
• First Posted: March 13, 2023
• Study Start: April 6, 2025
• Primary Completion (Estimated): December 1, 2029
• Study Completion (Estimated): December 1, 2030
• Last Updated: March 13, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, ICF, CSR
• Time Frame: Data from Vifor Pharma sponsored non-interventional and interventional clinical trials (phase 1-4) for medicines and indications approved in the US and the EU, will be shared if the evaluation of the request is positive and two years have elapsed since study completion or termination of the program.
• Access Criteria: Data is shared upon request from qualified scientific researchers under the prerequisite that the trial subjects' rights are kept fully protected, in particular with regards to Good Clinical Practice and Data Privacy, that the proposed analyses aim to yield an appropriate and valid scientific output and are not in conflict with the publication plan for the study. Requests should be submitted including a description of the data requested, a rationale for the proposed research, a Statistical Analysis Plan, a Publication Plan, qualifications and experience of the research team, disclosure of any conflicts of interests and competitive use of data, and source of research funding. If after evaluation the request is approved, the process of sharing the requested data will be initiated. As a prerequisite, a signed personal data processing and transfer agreement between Vifor Pharma and the data requestor is required.

Locations

FR (3); AE (1); BE (2); SA (3); NO (1); QA (1); FI (1); IT (2); AU (3); IL (2); PT (1); GR (2); PL (1); US (13); RO (1)