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Patiromer for Treatment of Hyperkalaemia in Children From Birth to <6 Years of Age

Primary Purpose

Hyperkalemia

Status

Not yet recruiting

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Patiromer

About this trial

This is an interventional treatment trial for Hyperkalemia

Eligibility Criteria

undefined - 5 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria: The following inclusion criteria must be met for each subject: Paediatric subjects (<6 years of age) with hyperkalaemia at screening. Subject's age should not reach 6 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). 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. 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who due to their general condition, e.g., anaemia or low body weight, are not suitable to have blood volume withdrawn.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. 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. 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: 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

Sites / Locations

Investigator Site 84003
Investigator Site 84004

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Patiromer

Arm Description

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

Outcomes

Primary Outcome Measures

Change in potassium levels (mmol/L)

May be measured as serum, plasma, whole blood, or capillary blood potassium

Secondary Outcome Measures

Change in potassium levels (mmol/L)

May be measured as serum, plasma, whole blood, or capillary blood potassium

Occurrence of treatment-emergent adverse events (TEAEs)

Occurrence of serious adverse events (SAEs)

Change from baseline in resting heart rate (beats per minute)

Change from baseline in systolic blood pressure (mmHg)

Change from baseline in diastolic blood pressure (mmHg)

Change from baseline in body temperature (Celsius)

Number of patients with ECG abnormalities

Change from baseline in chemistry laboratory evaluation: Calcium (mg/dL)

Change from baseline in chemistry laboratory evaluation: Phosphate (mg/dL)

Change from baseline in chemistry laboratory evaluation: Magnesium (mg/dL)

Change from baseline in chemistry laboratory evaluation: Potassium (mEq/L)

Change from baseline in chemistry laboratory evaluation: Sodium (mEq/L)

Change from baseline in chemistry laboratory evaluation: Creatinine (mg/dL)

Change from baseline in chemistry laboratory evaluation: Serum bicarbonate (mEq/L)

Change from baseline in chemistry laboratory evaluation: Blood urea nitrogen (mEq/L)

Change from baseline in haematology laboratory evaluation: White blood cells (10^9/L)

Change from baseline in haematology laboratory evaluation: Red blood cells count (10^12/L)

Change from baseline in haematology laboratory evaluation: Haemoglobin (10^12/L)

Change from baseline in haematology laboratory evaluation: Haematocrit (%)

Change from baseline in haematology laboratory evaluation: Platelet count (10^9/L)

Change from baseline in haematology laboratory evaluation: Blood fluoride (ng/mL)

Occurrence of blood potassium below the lower limit of normal (LLN) (mmol/L)

Occurrence of blood potassium above the upper limit of normal (ULN) (mmol/L)

Occurrence of blood magnesium at levels specified in protocol (mmol/L)

Occurrence of abnormal clinical laboratory value findings

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

Full Information

NCT ID

NCT05766839

First Posted

January 19, 2023

Last Updated

May 11, 2023

Sponsor

Vifor Pharma, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT05766839

Brief Title

Patiromer for Treatment of Hyperkalaemia in Children From Birth to <6 Years of Age

Official Title

A 2-Part, Open-Label, Phase 2, Multiple Dose Study to Evaluate the Pharmacodynamic Effects, Safety, and Tolerability of Patiromer in Children Under 6 Years of Age With Hyperkalaemia (EMERALD 2)

Study Type

Interventional

2. Study Status

Record Verification Date

May 2023

Overall Recruitment Status

Not yet recruiting

Study Start Date

May 2023 (Anticipated)

Primary Completion Date

September 30, 2024 (Anticipated)

Study Completion Date

September 30, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Vifor Pharma, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. 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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hyperkalemia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

21 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Patiromer

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Patiromer

Intervention Description

Patiromer will be given once daily

Primary Outcome Measure Information:

Title

Change in potassium levels (mmol/L)

Description

May be measured as serum, plasma, whole blood, or capillary blood potassium

Time Frame

From baseline to Day 28

Secondary Outcome Measure Information:

Title

Change in potassium levels (mmol/L)

Description

May be measured as serum, plasma, whole blood, or capillary blood potassium

Time Frame

From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

Title

Occurrence of treatment-emergent adverse events (TEAEs)

Time Frame

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)

Title

Occurrence of serious adverse events (SAEs)

Time Frame

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)

Title

Change from baseline in resting heart rate (beats per minute)

Time Frame

From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

Title

Change from baseline in systolic blood pressure (mmHg)

Time Frame

From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

Title

Change from baseline in diastolic blood pressure (mmHg)

Time Frame

From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

Title

Change from baseline in body temperature (Celsius)

Time Frame

From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

Title

Number of patients with ECG abnormalities

Time Frame

From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

Title

Change from baseline in chemistry laboratory evaluation: Calcium (mg/dL)

Time Frame

From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

Title

Change from baseline in chemistry laboratory evaluation: Phosphate (mg/dL)

Time Frame

From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

Title

Change from baseline in chemistry laboratory evaluation: Magnesium (mg/dL)

Time Frame

From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

Title

Change from baseline in chemistry laboratory evaluation: Potassium (mEq/L)

Time Frame

From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

Title

Change from baseline in chemistry laboratory evaluation: Sodium (mEq/L)

Time Frame

From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

Title

Change from baseline in chemistry laboratory evaluation: Creatinine (mg/dL)

Time Frame

From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

Title

Change from baseline in chemistry laboratory evaluation: Serum bicarbonate (mEq/L)

Time Frame

From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

Title

Change from baseline in chemistry laboratory evaluation: Blood urea nitrogen (mEq/L)

Time Frame

From baseline to Day 3, Day 7, Day 14, Day 28, and during Part 2: up to 52 weeks

Title

Change from baseline in haematology laboratory evaluation: White blood cells (10^9/L)

Time Frame

From baseline to Day 7, Day 14, Day 28, and EoT visit for Part 2 (week 52)

Title

Change from baseline in haematology laboratory evaluation: Red blood cells count (10^12/L)

Time Frame

From baseline to Day 7, Day 14, Day 28, and EoT visit for Part 2 (week 52)

Title

Change from baseline in haematology laboratory evaluation: Haemoglobin (10^12/L)

Time Frame

From baseline to Day 7, Day 14, Day 28, and EoT visit for Part 2 (week 52)

Title

Change from baseline in haematology laboratory evaluation: Haematocrit (%)

Time Frame

From baseline to Day 7, Day 14, Day 28, and EoT visit for Part 2 (week 52)

Title

Change from baseline in haematology laboratory evaluation: Platelet count (10^9/L)

Time Frame

From baseline to Day 7, Day 14, Day 28, and EoT visit for Part 2 (week 52)

Title

Change from baseline in haematology laboratory evaluation: Blood fluoride (ng/mL)

Time Frame

From baseline to Day 7, Day 14, Day 28, and EoT visit for Part 2 (week 52)

Title

Occurrence of blood potassium below the lower limit of normal (LLN) (mmol/L)

Time Frame

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)

Title

Occurrence of blood potassium above the upper limit of normal (ULN) (mmol/L)

Time Frame

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)

Title

Occurrence of blood magnesium at levels specified in protocol (mmol/L)

Time Frame

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)

Title

Occurrence of abnormal clinical laboratory value findings

Description

Time Frame

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)

10. Eligibility

Sex

All

Maximum Age & Unit of Time

5 Years

Accepts Healthy Volunteers

Eligibility Criteria

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

EMERALD-2 Clinical Study Team

Phone

+41 58 851 80 00

EMERALD2.clinicaldevelopment@viforpharma.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Julian Platon, MD, PhD

Organizational Affiliation

Vifor Pharma, Inc.

Official's Role

Study Director

Facility Information:

Facility Name

Investigator Site 84003

City

Miami

State/Province

Florida

ZIP/Postal Code

33124

Country

United States

Facility Name

Investigator Site 84004

City

Kansas City

State/Province

Missouri

ZIP/Postal Code

64108

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

CSL Vifor acknowledges the importance of data transparency and commits to sharing, upon request from qualified scientific researchers, patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials for medicines and indications approved in the United States (US) and the European Union (EU).

IPD Sharing Time Frame

Data from Vifor Pharma sponsored non-interventional and interventional clinical trials (phase 1-4) for medicines and indications approved in the US and the EU, will be shared if the evaluation of the request is positive and two years have elapsed since study completion or termination of the program.

IPD Sharing Access Criteria

Data is shared upon request from qualified scientific researchers under the prerequisite that the trial subjects' rights are kept fully protected, in particular with regards to Good Clinical Practice and Data Privacy, that the proposed analyses aim to yield an appropriate and valid scientific output and are not in conflict with the publication plan for the study. Requests should be submitted including a description of the data requested, a rationale for the proposed research, a Statistical Analysis Plan, a Publication Plan, qualifications and experience of the research team, disclosure of any conflicts of interests and competitive use of data, and source of research funding. If after evaluation the request is approved, the process of sharing the requested data will be initiated. As a prerequisite, a signed personal data processing and transfer agreement between Vifor Pharma and the data requestor is required.

Learn more about this trial

Patiromer for Treatment of Hyperkalaemia in Children From Birth to <6 Years of Age

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