Patiromer for Treatment of Hyperkalaemia in Children From Birth to <6 Years of Age
Study Details
Study Description
Brief Summary
A study to evaluate the pharmacodynamic effects, safety, and tolerability of patiromer in children under 6 years of age with hyperkalaemia
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Patiromer Cohort A (2 to < 6 years old): Pharmacodynamics (PD)/dose-ranging period duration is 4 weeks Cohort B (0 to < 2 years old): Pharmacodynamics (PD)/dose-ranging period duration is 4 weeks |
Drug: Patiromer
Patiromer will be given once daily
|
Outcome Measures
Primary Outcome Measures
- Change in potassium levels (mmol/L) [From baseline to Day 28]
May be measured as serum, plasma, whole blood, or capillary blood potassium
Secondary Outcome Measures
- 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, or capillary 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
Eligibility Criteria
Criteria
Inclusion Criteria:
The following inclusion criteria must be met for each subject:
-
Paediatric subjects (<6 years of age) with hyperkalaemia at screening.
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Subject's age should not reach 6 years during the 28 days of the pharmacodynamic (PD)/dose-ranging period.
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Subject is able to receive regular external feeding and medication, including via tubes, e.g., percutaneous endoscopic gastrostomy (PEG).
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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.
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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.
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 B.
-
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 tube, as the PEG 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.
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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:
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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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Investigator Site 84003 | Miami | Florida | United States | 33124 |
Sponsors and Collaborators
- Vifor Pharma, Inc.
Investigators
- Study Director: Julian Platon, MD, PhD, Vifor Pharma, Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- RLY5016-208p
- 2022-501829-20