Hyperkalemia
Conditions
Brief summary
A study to evaluate the pharmacodynamic effects, safety, and tolerability of patiromer in children under 12 years of age with hyperkalaemia.
Interventions
DRUGPatiromer
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
Sponsors
Vifor Pharma, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
0 Years to 11 Years
Healthy volunteers
No
Inclusion criteria
* The following inclusion criteria must be met for each participant: * \- 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.
Exclusion criteria
* 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 * Other protocol defined Inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in potassium levels (mmol/L) | From baseline to Day 28 | May be measured as serum, plasma, whole blood, potassium |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | May be measured as serum, plasma, whole blood, potassium |
| 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 | — |
| Change from baseline in diastolic blood pressure (mmHg) | From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks | — |
| Change from baseline in body temperature (Celsius) | From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks | — |
| Number of patients with ECG abnormalities | From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks | — |
| Change from baseline in chemistry laboratory evaluation: Calcium (mg/dL) | From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks | — |
| Change from baseline in chemistry laboratory evaluation: Phosphate (mg/dL) | From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks | — |
| Change from baseline in chemistry laboratory evaluation: Magnesium (mg/dL) | From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks | — |
| Change from baseline in chemistry laboratory evaluation: Potassium (mEq/L) | From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks | — |
| Change from baseline in chemistry laboratory evaluation: Sodium (mEq/L) | From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks | — |
| Change from baseline in chemistry laboratory evaluation: Creatinine (mg/dL) | From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks | — |
| Change from baseline in chemistry laboratory evaluation: Serum bicarbonate (mEq/L) | From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks | — |
| Change from baseline in chemistry laboratory evaluation: Blood urea nitrogen (mEq/L) | From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks | — |
| Change from baseline in haematology laboratory evaluation: White blood cells (10^9/L) | From baseline to Day 7, Day 14, Day 28, and EoT visit for Part 2 (week 52) | — |
| Change from baseline in haematology laboratory evaluation: Red blood cells count (10^12/L) | From baseline to Day 7, Day 14, Day 28, and EoT visit for Part 2 (week 52) | — |
| Change from baseline in haematology laboratory evaluation: Haemoglobin (10^12/L) | From baseline to Day 7, Day 14, Day 28, and EoT visit for Part 2 (week 52) | — |
| Change from baseline in haematology laboratory evaluation: Haematocrit (%) | From baseline to Day 7, Day 14, Day 28, and EoT visit for Part 2 (week 52) | — |
| Change from baseline in haematology laboratory evaluation: Platelet count (10^9/L) | From baseline to Day 7, Day 14, Day 28, and EoT visit for Part 2 (week 52) | — |
| Change from baseline in haematology laboratory evaluation: Blood fluoride (ng/mL) | From baseline to Day 7, Day 14, Day 28, and EoT visit for Part 2 (week 52) | — |
| Occurrence of blood potassium below the lower limit of normal (LLN) (mmol/L) | Part 1: Screening up to end of treatment (Day 28 ±3 Days); Part 2: Day 1 up to end of treatment (Up to 52 weeks) | — |
| Occurrence of blood potassium above the upper limit of normal (ULN) (mmol/L) | Part 1: Screening up to end of treatment (Day 28 ±3 Days); Part 2: Day 1 up to end of treatment (Up to 52 weeks) | — |
| Occurrence of blood magnesium at levels specified in protocol (mmol/L) | Part 1: Screening up to end of treatment (Day 28 ±3 Days); Part 2: Day 1 up to end of treatment (Up to 52 weeks) | — |
| Occurrence of abnormal clinical laboratory value findings | Part 1: Screening up to end of treatment (Day 28 ±3 Days); Part 2: Day 1 up to end of treatment (Up to 52 weeks) | Occurrence of clinical laboratory value findings that are outside of normal range of the respective age for: serum calcium, phosphate, fluoride, creatinine, bicarbonate, and blood urea nitrogen levels |
Countries
Australia, Belgium, Finland, France, Greece, Israel, Italy, Norway, Poland, Portugal, Qatar, Romania, Saudi Arabia, United Arab Emirates, United States
Contacts
CONTACTEMERALD-2 Clinical Study Team
STUDY_DIRECTORJulian Platon, MD, PhD
Vifor Pharma, Inc.
Outcome results
None listed