Pharmacodynamic & Safety of Patiromer in Children & Adolescents (2-<18 Yrs) With Chronic Kidney Disease and Hyperkalemia (EMERALD)

A Phase 2, Open-Label, Multiple Dose Study to Evaluate the Pharmacodynamic Effects, Safety, and Tolerability of Patiromer for Oral Suspension in Children and Adolescents 2 to < 18 Years of Age With Chronic Kidney Disease and Hyperkalemia

The purpose of this study is to evaluate the change in serum potassium levels from start of treatment to Day 14, when patiromer is administered at different doses, once daily, in children 2 - < 18 years of age with chronic kidney disease (CKD) and hyperkalemia (too much potassium in the blood). Another purpose of the study is to evaluate the safety and tolerability of patiromer in children 2 - < 18 years of age with CKD and hyperkalemia.

Study Overview

• Status: Terminated
• Conditions: Hyperkalemia
• Intervention / Treatment: Drug: Patiromer; Drug: Patiromer; Drug: Patiromer

Detailed Description

Up to 54 subjects, 2 - < 18 years of age with CKD (estimated glomerular filtration rate [eGFR] < 90 mL/min/1.73 m2 ) and hyperkalemia (two potassium measurements of 5.1 to < 6.5 mEq/L performed on separate days) will be enrolled in this open-label, multiple-dose, Phase 2 study.

The study will include two treatment phases: Pharmacodynamic (PD; drug effect on potassium) / Dose Finding Phase consisting of the initial 14-day dose finding period followed by an up to 5.5-month Long-Term Treatment Phase for a total study participation duration for individual subjects of up to 6.5 months (includes a 2 week follow up period).

• Study Type: Interventional
• Enrollment (Actual): 23
• Phase: Phase 2

Contacts and Locations

Study Locations

• Bulgaria
  • Sofia, Bulgaria, 1606
    • Investigator Site 1401
• Canada
  • British Columbia
    • Vancouver, British Columbia, Canada, V5Z 4H4
      • Investigator Site 1902
• Georgia
  • Tbilisi, Georgia, 0121
    • Investigator Site 3915
  • Tbilisi, Georgia, 0144
    • Investigator Site 3913
  • Tbilisi, Georgia, 0159
    • Investigator Site 3911
  • Tbilisi, Georgia, 0159
    • Investigator Site 3912
  • Tbilisi, Georgia, 0159
    • Investigator Site 3914
• Germany
  • Essen, Germany, 45147
    • Investigator Site 4312
  • Hannover, Germany, 30625
    • Investigator Site 4313
  • Heidelberg, Germany, 69120
    • Investigator Site 4311
  • Köln, Germany, 50937
    • Investiagor Site 4314
• Poland
  • Bialystok, Poland, 15-274
    • Investigator Site 5401
  • Gdansk, Poland, 80-952
    • Investigator Site 5404
  • Krakow, Poland, 30-663
    • Investigator Site 5406
  • Lodz, Poland, 93-338
    • Investigator Site 5402
  • Lublin, Poland, 20-093
    • Investigator Site 5403
  • Warsaw, Poland, 02-091
    • Investigator Site 5405
• Ukraine
  • Dnipropetrovs'k, Ukraine, 49000
    • Investigator Site 7906
  • Kharkiv, Ukraine, 61075
    • Investigator Site 7903
  • Kyiv, Ukraine, 04050
    • Investigator Site 7904
• United States
  • California
    • Palo Alto, California, United States, 94394
      • Investigator Site 1107
  • Kansas
    • Kansas City, Kansas, United States, 64108
      • Investigator Site 1101
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • Investigator Site 1103
  • New York
    • Bronx, New York, United States, 10467
      • Investigator Site 1102
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Investigator Site 1105
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15224
      • Investigator Site 1108
  • Texas
    • Amarillo, Texas, United States, 79106
      • Investigator Site 1104
    • Dallas, Texas, United States, 75235
      • Investigator Site 1109
    • Houston, Texas, United States, 77030
      • Investigator Site 1113
  • Wisconsin
    • Madison, Wisconsin, United States, 53792
      • Investigator Site 1106

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 17 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Written assent (when applicable) and written informed consent by a legally authorized representative provided prior to participation in the study
• Age 2 - <18 years old
• CKD defined by the estimated glomerular filtration rate (eGFR) <90 mL/min/1.73m2, including renal transplant subjects, based on local creatinine measurement at screening
• Two potassium measurements of 5.1 to < 6.5 mEq/L performed on separate days
• In the opinion of the study doctor, is expected to require treatment for hyperkalemia for at least 6 month
• If taking any renin-angiotensin-aldosterone system inhibitors (RAASi) beta blockers or diuretic medications, must be on a stable dose for at least 28 days prior to Screening
• Negative pregnancy test in females of child-bearing potential

Exclusion Criteria:

• Pseudohyperkalemia due to hemolysis or to abnormally high numbers of platelets (>500,000/mm3), leukocytes (>70,000/mm3), or erythrocytes (hematocrit >55%) at Screening based on results obtained locally
• Evidence of potassium-related electrocardiogram (ECG) changes at Screening
• Any of the following kidney conditions: maintenance hemodialysis or peritoneal dialysis, renal artery stenosis, and acute kidney injury or a history of acute renal insufficiency in the past 3 months
• Severe disorder of stomach or intestines including surgery that could affect gastrointestinal transit of the drug
• Increased liver enzymes (ALT, AST > 3 times upper limit of normal) at Screening
• Active cancer, currently on cancer treatment or history of cancer in the past 2 years (except for non-melanoma skin cancer)
• Heart or liver transplant, or anticipated need for transplant during the study treatment period including a scheduled kidney transplant recipient. (Note: patients currently on a kidney transplant wait list are not excluded unless there is an identified donor).
• Alcohol abuse or substance use disorder within 1 year of Screening
• Subjects currently being treated with or having taken any one of the following medications (includes resins) in the 7 days prior to Screening: sodium or calcium polystyrene sulfonate, drospirenone
• Use of certain medications that can affect blood potassium levels if doses have not been stable for at least 14 days prior to Screening or if doses are anticipated to change during the 14-day PD / Dose Finding Phase
• Use of investigational product within 30 days of screening or within 5 half-lives, whichever is longer
• Known hypersensitivity to patiromer or its components
• In the opinion of the Investigator, any medical condition, uncontrolled systemic disease, or serious intercurrent illness that would significantly decrease study compliance or jeopardize the safety of the subject or potentially affect the quality of the data

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1; Patiromer for age 12 to < 18 years	Drug: Patiromer; 4.2 g/day, 8.4 g/day and 16.8 g/day; Other Names: Veltassa; RLY5016 for Oral Suspension; Drug: Patiromer; 2 g/day, 4 g/day and 8 g/day; Other Names: Veltassa; RLY5016 for Oral Suspension; Drug: Patiromer; 1 g/day, 2 g/day and 4 g/day; Other Names: Veltassa; RLY5016 for Oral Suspension
Experimental: Cohort 2; Patiromer for age 6 to < 12 years	Drug: Patiromer; 4.2 g/day, 8.4 g/day and 16.8 g/day; Other Names: Veltassa; RLY5016 for Oral Suspension; Drug: Patiromer; 2 g/day, 4 g/day and 8 g/day; Other Names: Veltassa; RLY5016 for Oral Suspension; Drug: Patiromer; 1 g/day, 2 g/day and 4 g/day; Other Names: Veltassa; RLY5016 for Oral Suspension
Experimental: Cohort 3; Patiromer for age 2 to < 6 years	Drug: Patiromer; 4.2 g/day, 8.4 g/day and 16.8 g/day; Other Names: Veltassa; RLY5016 for Oral Suspension; Drug: Patiromer; 2 g/day, 4 g/day and 8 g/day; Other Names: Veltassa; RLY5016 for Oral Suspension; Drug: Patiromer; 1 g/day, 2 g/day and 4 g/day; Other Names: Veltassa; RLY5016 for Oral Suspension

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change in Serum Potassium Levels	from Baseline to Day 14

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of Subjects With Serum Potassium Levels in the Range of 3.8 - 5.0 mEq/L	Day 14: Initial PD / Dose Finding Phase. Week 26: Long-Term Treatment Phase.	Day 14 and Week 26

Collaborators and Investigators

• Sponsor: Vifor Pharma, Inc.
• Investigators: Study Director: Julian Platon, MD, PhD, Vifor Pharma, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): July 7, 2017
• Primary Completion (Actual): January 13, 2021
• Study Completion (Actual): April 30, 2021

Study Registration Dates

• First Submitted: March 16, 2017
• First Submitted That Met QC Criteria: March 16, 2017
• First Posted (Actual): March 22, 2017

Study Record Updates

• Last Update Posted (Actual): October 19, 2022
• Last Update Submitted That Met QC Criteria: October 17, 2022
• Last Verified: October 1, 2022

More Information

Terms related to this study

• Keywords: Chronic Kidney Disease; Potassium; Treatment of Hyperkalemia; Hyperkalemia
• Additional Relevant MeSH Terms: Metabolic Diseases; Urologic Diseases; Renal Insufficiency; Water-Electrolyte Imbalance; Kidney Diseases; Renal Insufficiency, Chronic; Hyperkalemia
• Other Study ID Numbers: RLY5016-206p; 2016-002785-31 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No