A Study in Children With Hyperkalaemia Between Birth and <18 Years of Age to Evaluate Doses of Sodium Zirconium Cyclosilicate (SZC) for Correction of Hyperkalaemia and Effectiveness of Same Dose to Maintain Normokalaemia

A Study in Children With Hyperkalaemia Between Birth and <18 Years of Age to Evaluate Doses of Sodium Zirconium Cyclosilicate (SZC) for Correction of Hyperkalaemia and Effectiveness of Same Dose to Maintain Normokalaemia

Sodium zirconium cyclosilicate has been shown to be effective and safe in adults for the treatment of hyperkalaemia, and therefore it is expected to be beneficial in children. This study will evaluate the efficacy, safety and tolerability of sodium zirconium cyclosilicate for the treatment of hyperkalaemia in children <18 years of age.

Primary Objective:28-day maintenance phase (MP) primary objective:
- To compare the effect of SZC vs placebo on maintaining normokalaemia during the MP.
- Correction phase (CP) primary objective:
- To evaluate SZC efficacy of different dose levels on achieving normokalaemia during the CP.

Overall design:
Approximately 140 participants will be enrolled at approximately 45 sites in locations including but not limited to Europe and North America for this study.

Treatment will include 3 phases: the CP, MP, and LTMP. Participants from 2 to <18 years are eligible to participate in all phases of the study. Participants <2 years of age will be included in the first 2 phases, namely the CP and the MP.

During the study, participants are recommended to be fasting prior to any visit pre-dose laboratory testing. All laboratory tests are taken pre-dose except select post dose K+ values during the CP. Electrocardiograms are to be taken prior to dosing. On visit days, the study treatment is to be taken at the site (during the CP this pertains only to the morning dose). For participants with diabetes, K+ measurements should be performed prior to insulin administration where possible.

For inclusion into the study i-STAT is used. For subsequent decisions on eligibility and determination of hyper and normokalaemia for entry into the MP and LTMP phases, local laboratory K+ is the determinant value.

Tolerability and safety will be assessed using AE reporting, results from laboratory testing, vital signs, physical examinations and ECG findings during the study. Care will be taken not to introduce bias when detecting AEs and/or SAEs. Open ended non leading verbal questioning of the participant or participant's legal representative, as appropriate, is the preferred method to inquire about AE occurrences.

Participants are allowed to discontinue the study treatment and assessments at any time or at the discretion of the Investigator(s). The independent Data Monitoring Committee (iDMC) will monitor data during all phases of the study including DL evaluations.


Study Type: Interventional (Clinical Trial)
Estimated Enrollment: 90 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Treatment will include 3 phases: the CP, MP, and LTMP. Participants from 2 to <18 years are eligible to participate in all phases of the study. Participants <2 years of age will be included in the first 2 phases, namely the CP and the MP.

Of the 140 participants enrolled, ≥5 participants should be in the birth to <2 years cohort. Additionally, the 90 (or greater) participants who enter into the MP should include ≥30 participants in the 12 to <18 years cohort, ≥30 participants in the 6 to <12 years cohort, and ≥10 participants in the 2 to <6 years cohort. At least 45 participants randomised in the MP should have a Screening i-STAT-K+ level (using the mean of the 2 consecutive i-STAT-K+ measurements at Screening) of >5.5 mmol/L. A minimum number of 75 participants must continue study treatment throughout the MP (≥25 participants in the 12 to <18 years cohort, ≥25 participants in the 6 to <12 years cohort, and ≥8 participants in the 2 to <6 years cohort).

Masking: Triple (Participant, Care Provider, Investigator)
Masking Description: This study consist of double blinded treatment as well as open label treatment.
Primary Purpose: Treatment
Official Title: A Phase 3, Dose-Escalating Study in Children With Hyperkalaemia Between Birth and <18 Years of Age to Evaluate Increasing Doses of Sodium Zirconium Cyclosilicate (SZC) Given Three Times Daily for the Correction of Hyperkalaemia and the Effectiveness of the Same Dose of SZC Given Once Daily to Maintain Normokalaemia Among Those Requiring Continuous Treatment.
Actual Study Start Date: April 2, 2019
Estimated Primary Completion Date: December 29, 2023
Estimated Study Completion Date: December 29, 2023

Arm:
- Experimental: Active Arm ( Sodium Zirconium Cyclosilicate SZC)
- Placebo Comparator: Placebo Arm

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