Trial Outcomes & Findings for Pharmacodynamic & Safety of Patiromer in Children & Adolescents (2-<18 Yrs) With Chronic Kidney Disease and Hyperkalemia (NCT NCT03087058)

NCT ID: NCT03087058

Last Updated: 2022-10-19

Results Overview

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

23 participants

Primary outcome timeframe

from Baseline to Day 14

Results posted on

2022-10-19

Participant Flow

Participant milestones

Participant milestones
Measure	Cohort 1 Patiromer administrated to pediatric participants (12 to \<18 years of age), with chronic kidney disease (CKD) and hyperkalemia. The starting dose levels of Patiromer for Cohort 1 were: 4.2 g/day, 8.4 g/day and 16.8 g/day, starting with the lowest dose level, depending on the participant's median weights. Once-daily administration up to 26 weeks including screening at Day 1 followed by 14-days (Pharmacodynamic dose-finding phase), 22 weeks (Long-term treatment phase), and a 2-week follow-up period consisting of 1 follow-up visit and 1 follow-up phone call.	Cohort 2 Patiromer administrated to pediatric participants (6 to \<12 years of age), with chronic kidney disease (CKD) and hyperkalemia. The starting dose levels of Patiromer for Cohort 2 were: 2 g/day, 4 g/day and 8 g/day, starting with the lowest dose level, depending on the participant's median weights. Once-daily administration up to 26 weeks including screening at Day 1 followed by 14-days (Pharmacodynamic dose-finding phase), 22 weeks (Long-term treatment phase), and a 2-week follow-up period consisting of 1 follow-up visit and 1 follow-up phone call.
Overall Study STARTED	14	9
Overall Study COMPLETED	12	9
Overall Study NOT COMPLETED	2	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Cohort 1 Patiromer administrated to pediatric participants (12 to \<18 years of age), with chronic kidney disease (CKD) and hyperkalemia. The starting dose levels of Patiromer for Cohort 1 were: 4.2 g/day, 8.4 g/day and 16.8 g/day, starting with the lowest dose level, depending on the participant's median weights. Once-daily administration up to 26 weeks including screening at Day 1 followed by 14-days (Pharmacodynamic dose-finding phase), 22 weeks (Long-term treatment phase), and a 2-week follow-up period consisting of 1 follow-up visit and 1 follow-up phone call.	Cohort 2 Patiromer administrated to pediatric participants (6 to \<12 years of age), with chronic kidney disease (CKD) and hyperkalemia. The starting dose levels of Patiromer for Cohort 2 were: 2 g/day, 4 g/day and 8 g/day, starting with the lowest dose level, depending on the participant's median weights. Once-daily administration up to 26 weeks including screening at Day 1 followed by 14-days (Pharmacodynamic dose-finding phase), 22 weeks (Long-term treatment phase), and a 2-week follow-up period consisting of 1 follow-up visit and 1 follow-up phone call.	Cohort 3 Patiromer was planned to be administrated to pediatric participants (2 to \<6 years of age), with chronic kidney disease (CKD) and hyperkalemia. However, the trial was terminated by sponsor decision on the 13 July 2022. The PD effects, safety and tolerability of patiromer in the population aged from 0 to \<6 will be assessed in a new trial with the protocol number RLY5016-208p.
Overall Study Withdrawal by Subject	2	0	0

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort 1 n=14 Participants Patiromer administrated to pediatric participants (12 to \<18 years of age), with chronic kidney disease (CKD) and hyperkalemia. The starting dose levels of Patiromer for Cohort 1 were: 4.2 g/day, 8.4 g/day and 16.8 g/day, starting with the lowest dose level, depending on the participant's median weights. Once-daily administration up to 26 weeks including screening at Day 1 followed by 14-days (Pharmacodynamic dose-finding phase), 22 weeks (Long-term treatment phase), and a 2-week follow-up period consisting of 1 follow-up visit and 1 follow-up phone call.	Cohort 2 n=9 Participants Patiromer administrated to pediatric participants (6 to \<12 years of age), with chronic kidney disease (CKD) and hyperkalemia. The starting dose levels of Patiromer for Cohoer1 1 were: 2 g/day, 4 g/day and 8 g/day, starting with the lowest dose level, depending on the participant's median weights. Once-daily administration up to 26 weeks including screening at Day 1 followed by 14-days (Pharmacodynamic dose-finding phase), 22 weeks (Long-term treatment phase), and a 2-week follow-up period consisting of 1 follow-up visit and 1 follow-up phone call.	Total n=23 Participants Total of all reporting groups
Age, Categorical <=18 years	14 Participants n=5 Participants	9 Participants n=7 Participants	23 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	14.5 years STANDARD_DEVIATION 1.99 • n=5 Participants	8.0 years STANDARD_DEVIATION 2.00 • n=7 Participants	12.0 years STANDARD_DEVIATION 3.78 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	6 Participants n=7 Participants	9 Participants n=5 Participants
Sex: Female, Male Male	11 Participants n=5 Participants	3 Participants n=7 Participants	14 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	13 Participants n=5 Participants	9 Participants n=7 Participants	22 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	2 participants n=5 Participants	0 participants n=7 Participants	2 participants n=5 Participants
Region of Enrollment Ukraine	6 participants n=5 Participants	7 participants n=7 Participants	13 participants n=5 Participants
Region of Enrollment Poland	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants
Region of Enrollment Georgia	3 participants n=5 Participants	1 participants n=7 Participants	4 participants n=5 Participants
Region of Enrollment Germany	2 participants n=5 Participants	0 participants n=7 Participants	2 participants n=5 Participants

PRIMARY outcome

Timeframe: from Baseline to Day 14

Population: The efficacy population includes all subjects who have taken at least 1 dose of patiromer. As the safety population and efficacy population are the same in this study, all analyses are displayed with 1 population.

Outcome measures

Outcome measures
Measure	Cohort 1 n=14 Participants Patiromer administrated to pediatric participants (12 to \<18 years of age), with chronic kidney disease (CKD) and hyperkalemia. The starting dose levels of Patiromer for Cohort 1 were: 4.2 g/day, 8.4 g/day and 16.8 g/day, starting with the lowest dose level, depending on the participant's median weights. Once-daily administration up to 26 weeks including screening at Day 1 followed by 14-days (Pharmacodynamic dose-finding phase), 22 weeks (Long-term treatment phase), and a 2-week follow-up period consisting of 1 follow-up visit and 1 follow-up phone call.	Cohort 2 n=9 Participants Patiromer administrated to pediatric participants (6 to \< 12 years of age), with chronic kidney disease (CKD) and hyperkalemia. The starting dose levels of Patiromer for Cohoer1 1 were: 2 g/day, 4 g/day and 8 g/day, starting with the lowest dose level, depending on the participant's median weights. Once-daily administration up to 26 weeks including screening at Day 1 followed by 14-days (Pharmacodynamic dose-finding phase), 22 weeks (Long-term treatment phase), and a 2-week follow-up period consisting of 1 follow-up visit and 1 follow-up phone call.
Change in Serum Potassium Levels	-0.50 mEq/l Standard Deviation 0.542	-0.14 mEq/l Standard Deviation 0.553

SECONDARY outcome

Timeframe: Day 14 and Week 26

Day 14: Initial PD / Dose Finding Phase. Week 26: Long-Term Treatment Phase.

Outcome measures

Outcome measures
Measure	Cohort 1 n=14 Participants Patiromer administrated to pediatric participants (12 to \<18 years of age), with chronic kidney disease (CKD) and hyperkalemia. The starting dose levels of Patiromer for Cohort 1 were: 4.2 g/day, 8.4 g/day and 16.8 g/day, starting with the lowest dose level, depending on the participant's median weights. Once-daily administration up to 26 weeks including screening at Day 1 followed by 14-days (Pharmacodynamic dose-finding phase), 22 weeks (Long-term treatment phase), and a 2-week follow-up period consisting of 1 follow-up visit and 1 follow-up phone call.	Cohort 2 n=9 Participants Patiromer administrated to pediatric participants (6 to \< 12 years of age), with chronic kidney disease (CKD) and hyperkalemia. The starting dose levels of Patiromer for Cohoer1 1 were: 2 g/day, 4 g/day and 8 g/day, starting with the lowest dose level, depending on the participant's median weights. Once-daily administration up to 26 weeks including screening at Day 1 followed by 14-days (Pharmacodynamic dose-finding phase), 22 weeks (Long-term treatment phase), and a 2-week follow-up period consisting of 1 follow-up visit and 1 follow-up phone call.
Proportion of Subjects With Serum Potassium Levels in the Range of 3.8 - 5.0 mEq/L Day 14	50.0 percentage of participants (%) Interval 23.0 to 77.0	12.5 percentage of participants (%) Interval 0.3 to 52.7
Proportion of Subjects With Serum Potassium Levels in the Range of 3.8 - 5.0 mEq/L Week 26	81.8 percentage of participants (%) Interval 48.2 to 97.7	22.2 percentage of participants (%) Interval 2.8 to 60.0

Adverse Events

Cohort 1

Serious events: 0 serious events

Other events: 10 other events

Deaths: 0 deaths

Cohort 2

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

EMERALD Clinical Study Team

Vifor Pharma, Inc.

Phone: +41 588 518 000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Cohort 1 n=14 participants at risk Patiromer administrated to pediatric participants (12 to \< 18 years of age), with chronic kidney disease (CKD) and hyperkalemia. The starting dose levels of Patiromer for Cohort 1 were: 4.2 g/day, 8.4 g/day and 16.8 g/day, starting with the lowest dose level, depending on the participant's median weights. Once-daily administration up to 26 weeks including screening at Day 1 followed by 14-days (Pharmacodynamic dose-finding phase), 22 weeks (Long-term treatment phase), and a 2-week follow-up period consisting of 1 follow-up visit and 1 follow-up phone call.	Cohort 2 n=9 participants at risk Patiromer administrated to pediatric participants (6 to \< 12 years of age), with chronic kidney disease (CKD) and hyperkalemia. The starting dose levels of Patiromer for Cohoer1 1 were: 2 g/day, 4 g/day and 8 g/day, starting with the lowest dose level, depending on the participant's median weights. Once-daily administration up to 26 weeks including screening at Day 1 followed by 14-days (Pharmacodynamic dose-finding phase), 22 weeks (Long-term treatment phase), and a 2-week follow-up period consisting of 1 follow-up visit and 1 follow-up phone call.
Gastrointestinal disorders Diarrhoea	21.4% 3/14 • Number of events 3 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Gastrointestinal disorders Flatulence	14.3% 2/14 • Number of events 2 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Gastrointestinal disorders Nausea	14.3% 2/14 • Number of events 2 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Gastrointestinal disorders Abdominal pain	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Gastrointestinal disorders Constipation	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Gastrointestinal disorders Dyspepsia	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Gastrointestinal disorders Frequent bowel movements	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Infections and infestations Nasopharyngitis	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	22.2% 2/9 • Number of events 2 • During treatment period up to 26 weeks after randomization.
Infections and infestations Human herpesvirus 6 infection	0.00% 0/14 • During treatment period up to 26 weeks after randomization.	11.1% 1/9 • Number of events 1 • During treatment period up to 26 weeks after randomization.
Infections and infestations Infection	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Infections and infestations Otitis externa	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Infections and infestations Severe acute respiratory syndrome	0.00% 0/14 • During treatment period up to 26 weeks after randomization.	11.1% 1/9 • Number of events 1 • During treatment period up to 26 weeks after randomization.
Infections and infestations Upper respiratory tract infection	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Metabolism and nutrition disorders Decreased appetite	14.3% 2/14 • Number of events 2 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Metabolism and nutrition disorders Hypokalaemia	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	11.1% 1/9 • Number of events 1 • During treatment period up to 26 weeks after randomization.
Metabolism and nutrition disorders Metabolic acidosis	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Investigations Blood calcium decreased	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Investigations Blood calcium increased	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Investigations Blood creatinine increased	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Investigations Body temperature increased	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Investigations Urine output increased	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Renal and urinary disorders Renal impairment	21.4% 3/14 • Number of events 3 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	21.4% 3/14 • Number of events 3 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Respiratory, thoracic and mediastinal disorders Epistaxis	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Blood and lymphatic system disorders Anaemia	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	11.1% 1/9 • Number of events 1 • During treatment period up to 26 weeks after randomization.
Injury, poisoning and procedural complications Medication error	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Injury, poisoning and procedural complications Skin abrasion	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Nervous system disorders Dizziness	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Nervous system disorders Parosmia	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Nervous system disorders Somnolence	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Psychiatric disorders Dysphoria	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Psychiatric disorders Psychomotor retardation	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
Cardiac disorders Arrhythmia	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.
General disorders Fatigue	7.1% 1/14 • Number of events 1 • During treatment period up to 26 weeks after randomization.	0.00% 0/9 • During treatment period up to 26 weeks after randomization.