Patiromer for Treatment of Hyperkalaemia in Children Under 12 Years of Age
- Registration Number
- NCT05766839
- Lead Sponsor
- Vifor Pharma, Inc.
- Brief Summary
A study to evaluate the pharmacodynamic effects, safety, and tolerability of patiromer in children under 12 years of age with hyperkalaemia
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 36
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.
The following criteria exclude a subject from participating in this trial:
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Preterm birth infants with <37 weeks of gestation cannot be included in Cohort 3.
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Participants who due to their general condition, e.g., anaemia or low body weight, are not suitable to have blood volume withdrawn.
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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.
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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.
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Active cancer, currently on cancer treatment, or history of cancer in the past 2 years (except for non-melanoma skin cancer).
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Recipient of any organ transplant requiring treatment with immunosuppressive therapy or scheduled for kidney transplant procedure during the first 28 days after Day 1.
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History of sudden infant death in a sibling (only for participants <2 years of age at screening).
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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.
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Use of any investigational product for an unapproved indication within 30 days prior to screening or within 5 half-lives, whichever is longer.
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Known hypersensitivity to patiromer or its components.
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If the child is being breastfed:
- There is suspicion of current alcohol or substance misuse/abuse in breastfeeding mother
- The breastfeeding mother is taking potassium supplements
Other protocol defined Inclusion/Exclusion criteria may apply
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Patiromer Patiromer 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.
- Primary Outcome Measures
Name Time Method Change in potassium levels (mmol/L) From baseline to Day 28 May be measured as serum, plasma, whole blood, potassium
- Secondary Outcome Measures
Name Time Method 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 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
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 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) 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 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 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 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 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 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: Serum bicarbonate (mEq/L) From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks 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) 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 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: Haematocrit (%) From baseline to Day 7, Day 14, Day 28, and EoT visit for Part 2 (week 52) 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 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: 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: Platelet count (10^9/L) From baseline to Day 7, Day 14, Day 28, and EoT visit for Part 2 (week 52) 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) 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 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
Trial Locations
- Locations (6)
UF Health Pediatric Multispecialty Center
🇺🇸Jacksonville, Florida, United States
Miller School of Medicine, University of Miami
🇺🇸Miami, Florida, United States
Shaare Zedek Medical Center
🇮🇱Jerusalem, Israel
Schneider Children's Medical Center of Israel
🇮🇱Petach Tikvah, Israel
Children's Mercy Hospitals and Clinics
🇺🇸Kansas City, Missouri, United States
Duke University Hospital & Medical Center
🇺🇸Durham, North Carolina, United States