Effect of patiromer in children under 12 years of age with high blood potassium levels
- Conditions
- HyperkalemiaMedDRA version: 21.1Level: LLTClassification code: 10020647Term: Hyperkalemia Class: 10027433Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- CTIS2023-505252-21-00
- Lead Sponsor
- Vifor Pharma Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 36
Paediatric subjects (<12 years of age) with hyperkalaemia at screening., Subject’s age should not reach 12 years during the 28 days of the PD/ dose-ranging period., Subject is able to receive regular external feeding and medication, including via tubes, e.g., 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). 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, In the opinion of the Investigator, the subject is expected to require treatment for hyperkalaemia for at least 28 days upon enrolment in the study., If taking any 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 IRB/EC/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., 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 prior to 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
Preterm birth infants with <37 weeks of gestation cannot be included in Cohort 3., 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, Has severe hypoxaemia, respiratory acidosis, asphyxia, or hypotension 3 months before screening based on assessment of the Investigator., Subjects treated with sodium polystyrene sulphonate, calcium polystyrene sulphonate, or sodium zirconium cyclosilicate within the last 48 hours prior to fulfilling the baseline potassium assessments requested in Inclusion Criterion 4., 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, 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 subject or potentially affect the quality of the data such as: 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: a) There is suspicion of current alcohol or substance misuse/abuse in breastfeeding mother b) The breastfeeding mother is taking potassium supplements, 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, Subjects 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 subject with evidence of potential potassium-related 12-lead electrocardiogram (ECG) changes (i.e., changes consistent with hyper- or hypokalaemia) at screening., Any subject with serum magnesium <1.4 mg/dl (0.58 mmol/l) at screening/baseline., 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: 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 o
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To assess change in potassium levels from baseline to Day 28 following administration of different doses of patiromer administered in children 0 to <12 years of age with hyperkalaemia;Secondary Objective: To assess the safety and tolerability of patiromer in children 0 to <12 years of age with hyperkalaemia.;Primary end point(s): Changes in potassium levels from baseline to Day 28.
- Secondary Outcome Measures
Name Time Method Secondary end point(s):Changes in potassium levels from baseline to Day 3, Day 7, and Day 14 and end of the study Part 1 and during the optional safety-extension period.;Secondary end point(s):Occurrence of TEAEs and SAEs.;Secondary end point(s):Change in vital signs, 12-lead ECG, and clinical safety laboratory evaluations.;Secondary end point(s):Laboratory safety endpoints of special interest including occurrence of blood potassium: - Below the LLN. - Above the ULN;Secondary end point(s):Occurrence of blood magnesium: - <1.4 mg/dl (<0.58 mmol/l) - <1.2 mg/dl (<0.49 mmol/l) - <1.0 mg/dl (<0.41 mmol/l);Secondary end point(s):Occurrence of serum calcium, phosphate, fluoride, creatinine, bicarbonate, and blood urea nitrogen levels that are outside of normal range of the respective age.