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

Vifor

Status and phase: Enrolling; Phase 2

Conditions: Hyperkalemia

Treatments: Drug: Patiromer

Study type: Interventional

Funder types: Industry

Identifiers

NCT05766839

RLY5016-208p

2023-505252-21-00 (Other Identifier)

Details and patient eligibility

About

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

Enrollment: 36 estimated patients

Sex: All

Ages: Under 11 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

The following inclusion criteria must be met for each subject:

• Paediatric subjects (<12 years of age) with hyperkalaemia at screening.
• Subject's age should not reach 12 years during the 28 days of the pharmacodynamic (PD)/dose-ranging period.
• Subject is able to receive regular external feeding and medication, including via tubes, e.g., 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 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 child bearing 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 child bearing potential must agree to continue using contraception throughout the study and for 1 month after the last dose of patiromer.

Exclusion criteria

The following criteria exclude a subject 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who due to their general condition, e.g., anaemia or low body weight, are not suitable to have blood volume withdrawn.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 or peritoneal dialysis, 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).

• Recipient of any organ transplant requiring treatment with immunosuppressive therapy or 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 PD/ 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:

  1. There is suspicion of current alcohol or substance misuse/abuse in breastfeeding mother
  2. The breastfeeding mother is taking potassium supplements

Other protocol defined Inclusion/Exclusion criteria may apply

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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36 participants in 1 patient group

Patiromer

Experimental group

Description:

4-week pharmacodynamic (PD)/dose-ranging period Cohort 1: 6 to \<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 sub-group of 0 to \<6 months and another 3 study participants in the sub-group 6 to \<24 months of age.

Treatment:

Drug: Patiromer