Trial Outcomes & Findings for Triferic Pediatric Pharmacokinetic Protocol (NCT NCT02595437)

NCT ID: NCT02595437

Last Updated: 2018-10-25

Results Overview

The PK will be done by assessing the mean absolute and baseline-corrected Cmax of total iron with an IV infusion of Triferic at 0.07 mg iron/kg during a single dialysis session. The absolute Cmax includes the concentration of iron that was present in the serum prior to dosing as well the iron administered, while the baseline-corrected Cmax factors out the iron present in the serum prior to dosing and includes the administered iron only.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

22 participants

Primary outcome timeframe

0, 1, 2, 4, 4.5, 5, 6, 8, 10 hrs

Results posted on

2018-10-25

Participant Flow

Participant milestones

Participant milestones
Measure
Triferic Via IV and Hemodialysate
Triferic was administered via IV infusion on study Day 1, and then mixed with the liquid bicarbonate and administered via hemodialysate on study Day 3.
Triferic Via IV
STARTED
22
Triferic Via IV
COMPLETED
21
Triferic Via IV
NOT COMPLETED
1
Triferic Via Hemodialysate
STARTED
21
Triferic Via Hemodialysate
COMPLETED
21
Triferic Via Hemodialysate
NOT COMPLETED
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Triferic Via IV and Hemodialysate
Triferic was administered via IV infusion on study Day 1, and then mixed with the liquid bicarbonate and administered via hemodialysate on study Day 3.
Triferic Via IV
Withdrawal by Subject
1

Baseline Characteristics

Triferic Pediatric Pharmacokinetic Protocol

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Safety Population
n=22 Participants
All patients enrolled in the study who received any amount of Triferic, regardless of whether the dose was administered intravenously or via dialysate.
Age, Continuous
11.8 years
STANDARD_DEVIATION 4.98 • n=99 Participants
Sex: Female, Male
Female
11 Participants
n=99 Participants
Sex: Female, Male
Male
11 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
9 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
12 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
1 Participants
n=99 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=99 Participants
Race (NIH/OMB)
Asian
1 Participants
n=99 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=99 Participants
Race (NIH/OMB)
Black or African American
8 Participants
n=99 Participants
Race (NIH/OMB)
White
8 Participants
n=99 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=99 Participants
Race (NIH/OMB)
Unknown or Not Reported
5 Participants
n=99 Participants

PRIMARY outcome

Timeframe: 0, 1, 2, 4, 4.5, 5, 6, 8, 10 hrs

Population: Pharmacokinetic Group: all patients who received at least one dose of study drug and have sufficient PK samples (a sample at the end of infusion and at least 3 samples during the elimination phase)

The PK will be done by assessing the mean absolute and baseline-corrected Cmax of total iron with an IV infusion of Triferic at 0.07 mg iron/kg during a single dialysis session. The absolute Cmax includes the concentration of iron that was present in the serum prior to dosing as well the iron administered, while the baseline-corrected Cmax factors out the iron present in the serum prior to dosing and includes the administered iron only.

Outcome measures

Outcome measures
Measure
Triferic 0.07 mg/kg Iron Intravenous: Absolute Value
n=21 Participants
Patients were administered Triferic intravenously 0.07 mg/kg at the same time that they received their standard of care hemodialysis. The results for this group are the absolute values.
Triferic 0.07 mg/kg Iron Intravenous: Baseline Corrected
n=21 Participants
Patients were administered Triferic intravenously 0.07 mg/kg at the same time that they received their standard of care hemodialysis. The results for this group are baseline-corrected.
Pharmacokinetics (PK) of Triferic Iron Administered IV in Pediatric CKD-5HD Patients: Cmax.
217 microgram/deciliter
Geometric Coefficient of Variation 28.0
114 microgram/deciliter
Geometric Coefficient of Variation 53.7

PRIMARY outcome

Timeframe: 0, 1, 2, 4, 4.5, 5, 6, 8, 10 hours

Population: Pharmacokinetic Group: all patients who received at least one dose of study drug and have sufficient PK samples (a sample at the end of infusion and at least 3 samples during the elimination phase)

The PK will be done by assessing the mean absolute and baseline-corrected AUC(last) of total iron with an IV infusion of Triferic at 0.07 mg iron/kg during a single dialysis session. The absolute AUC(last) includes iron that was present in the serum prior to dosing as well the iron administered, while the baseline-corrected AUC(last) factors out the iron present in the serum prior to dosing and includes the administered iron only.

Outcome measures

Outcome measures
Measure
Triferic 0.07 mg/kg Iron Intravenous: Absolute Value
n=21 Participants
Patients were administered Triferic intravenously 0.07 mg/kg at the same time that they received their standard of care hemodialysis. The results for this group are the absolute values.
Triferic 0.07 mg/kg Iron Intravenous: Baseline Corrected
n=21 Participants
Patients were administered Triferic intravenously 0.07 mg/kg at the same time that they received their standard of care hemodialysis. The results for this group are baseline-corrected.
Pharmacokinetics (PK) of Triferic Iron Administered IV in Pediatric CKD-5HD Patients: AUC(Last).
1324 hours* micrograms/ deciliters
Geometric Coefficient of Variation 36.5
419 hours* micrograms/ deciliters
Geometric Coefficient of Variation 101.6

PRIMARY outcome

Timeframe: 0, 1, 2, 4, 4.5, 5, 6, 8, 10 hours

Population: Pharmacokinetic Group: all patients who received at least one dose of study drug and have sufficient PK samples (a sample at the end of infusion and at least 3 samples during the elimination phase)

The PK will be done by assessing the mean absolute and baseline-corrected AUC(0-end) of total iron with an IV infusion of Triferic at 0.07 mg iron/kg during a single dialysis session. The absolute AUC (0-end) includes iron that was present in the serum prior to dosing as well the iron administered, while the baseline-corrected AUC (0-end) factors out the iron present in the serum prior to dosing and includes the administered iron only.

Outcome measures

Outcome measures
Measure
Triferic 0.07 mg/kg Iron Intravenous: Absolute Value
n=21 Participants
Patients were administered Triferic intravenously 0.07 mg/kg at the same time that they received their standard of care hemodialysis. The results for this group are the absolute values.
Triferic 0.07 mg/kg Iron Intravenous: Baseline Corrected
n=21 Participants
Patients were administered Triferic intravenously 0.07 mg/kg at the same time that they received their standard of care hemodialysis. The results for this group are baseline-corrected.
Pharmacokinetics (PK) of Triferic Iron Administered IV in Pediatric CKD-5HD Patients: AUC(0-end).
648 hours* micrograms/ deciliters
Geometric Coefficient of Variation 29.3
237 hours* micrograms/ deciliters
Geometric Coefficient of Variation 76.8

SECONDARY outcome

Timeframe: 0, 1, 2, 4, 4.5, 5, 6, 8, 10 hours

Population: Pharmacokinetic Group: all patients who received at least one dose of study drug and have sufficient PK samples (a sample at the end of infusion and at least 3 samples during the elimination phase)

The PK will be done by assessing the mean absolute and baseline-corrected Cmax of total iron. The absolute Cmax includes the concentration of iron that was present in the serum prior to dosing as well the iron administered, while the baseline-corrected Cmax factors out the iron present in the serum prior to dosing and includes the administered iron only.

Outcome measures

Outcome measures
Measure
Triferic 0.07 mg/kg Iron Intravenous: Absolute Value
n=21 Participants
Patients were administered Triferic intravenously 0.07 mg/kg at the same time that they received their standard of care hemodialysis. The results for this group are the absolute values.
Triferic 0.07 mg/kg Iron Intravenous: Baseline Corrected
n=21 Participants
Patients were administered Triferic intravenously 0.07 mg/kg at the same time that they received their standard of care hemodialysis. The results for this group are baseline-corrected.
Pharmacokinetics (PK) of Triferic Iron Administered Via the Hemodialysate in Pediatric CKD-5HD Patients: Cmax.
261 micrograms/ deciliters
Geometric Coefficient of Variation 26.4
166 micrograms/ deciliters
Geometric Coefficient of Variation 54.3

SECONDARY outcome

Timeframe: 0, 1, 2, 4, 4.5, 5, 6, 8, 10 hours

Population: Pharmacokinetic Group: all patients who received at least one dose of study drug and have sufficient PK samples (a sample at the end of infusion and at least 3 samples during the elimination phase)

The PK will be done by assessing the mean absolute and baseline-corrected AUC(last) of total iron. The absolute AUC (last) includes the iron that was present in the serum prior to dosing as well the iron administered, while the baseline-corrected Cmax factors out the iron present in the serum prior to dosing and includes the administered iron only.

Outcome measures

Outcome measures
Measure
Triferic 0.07 mg/kg Iron Intravenous: Absolute Value
n=21 Participants
Patients were administered Triferic intravenously 0.07 mg/kg at the same time that they received their standard of care hemodialysis. The results for this group are the absolute values.
Triferic 0.07 mg/kg Iron Intravenous: Baseline Corrected
n=21 Participants
Patients were administered Triferic intravenously 0.07 mg/kg at the same time that they received their standard of care hemodialysis. The results for this group are baseline-corrected.
Pharmacokinetics (PK) of Triferic Iron Administered Via the Hemodialysate in Pediatric CKD-5HD Patients: AUC(Last).
1453 hours* micrograms/ deciliters
Geometric Coefficient of Variation 59.6
682 hours* micrograms/ deciliters
Geometric Coefficient of Variation 82.9

SECONDARY outcome

Timeframe: 0, 1, 2, 4, 4.5, 5, 6, 8, 10 hours

Population: Pharmacokinetic Group: all patients who received at least one dose of study drug and have sufficient PK samples (a sample at the end of infusion and at least 3 samples during the elimination phase)

The PK will be done by assessing the mean absolute and baseline-corrected AUC(0-end) of total iron. The absolute AUC (0-end) includes the of iron that was present in the serum prior to dosing as well the iron administered, while the baseline-corrected Cmax factors out the iron present in the serum prior to dosing and includes the administered iron only.

Outcome measures

Outcome measures
Measure
Triferic 0.07 mg/kg Iron Intravenous: Absolute Value
n=21 Participants
Patients were administered Triferic intravenously 0.07 mg/kg at the same time that they received their standard of care hemodialysis. The results for this group are the absolute values.
Triferic 0.07 mg/kg Iron Intravenous: Baseline Corrected
n=21 Participants
Patients were administered Triferic intravenously 0.07 mg/kg at the same time that they received their standard of care hemodialysis. The results for this group are baseline-corrected.
Pharmacokinetics (PK) of Triferic Iron Administered Via the Hemodialysate in Pediatric CKD-5HD Patients: AUC(0-end).
763 hours* micrograms/ deciliters
Geometric Coefficient of Variation 38.5
387 hours* micrograms/ deciliters
Geometric Coefficient of Variation 79.6

OTHER_PRE_SPECIFIED outcome

Timeframe: 1.5 weeks

Population: Safety Population: all patients who received any amount of study medication are included in the safety population.

The incidence of treatment-emergent AEs (TEAEs) and treatment-emergent serious AEs (TESAEs) will be grouped by body system. Adverse events were recorded from study Day 1 through the following up visit (approximately 1.5 weeks).

Outcome measures

Outcome measures
Measure
Triferic 0.07 mg/kg Iron Intravenous: Absolute Value
n=22 Participants
Patients were administered Triferic intravenously 0.07 mg/kg at the same time that they received their standard of care hemodialysis. The results for this group are the absolute values.
Triferic 0.07 mg/kg Iron Intravenous: Baseline Corrected
n=21 Participants
Patients were administered Triferic intravenously 0.07 mg/kg at the same time that they received their standard of care hemodialysis. The results for this group are baseline-corrected.
Treatment-emergent Adverse Events (TEAEs)
4 Participants
3 Participants

Adverse Events

IV Exposure

Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths

IV and Hemodialysate Exposure

Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
IV Exposure
n=22 participants at risk
Patients in this population had received Triferic via IV infusion only at the time of onset of the adverse event.
IV and Hemodialysate Exposure
n=21 participants at risk
Patients in this population had received Triferic via IV infusion and via hemodialysate at the time of onset of the adverse event.
Gastrointestinal disorders
Nausea
9.1%
2/22 • Number of events 2 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
0.00%
0/21 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
Gastrointestinal disorders
Abdominal Pain
4.5%
1/22 • Number of events 1 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
0.00%
0/21 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
Gastrointestinal disorders
Abdominal pain upper
4.5%
1/22 • Number of events 1 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
0.00%
0/21 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
Gastrointestinal disorders
Constipation
0.00%
0/22 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
4.8%
1/21 • Number of events 1 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
Cardiac disorders
Tachycardia
0.00%
0/22 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
4.8%
1/21 • Number of events 1 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
General disorders
Axillary Pain
0.00%
0/22 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
4.8%
1/21 • Number of events 1 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
Musculoskeletal and connective tissue disorders
Back Pain
4.5%
1/22 • Number of events 1 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
0.00%
0/21 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
Nervous system disorders
Dizziness
4.5%
1/22 • Number of events 1 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
0.00%
0/21 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
Respiratory, thoracic and mediastinal disorders
Dyspnoea
4.5%
1/22 • Number of events 1 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
0.00%
0/21 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
Vascular disorders
Hypotension
0.00%
0/22 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.
4.8%
1/21 • Number of events 1 • 1.5 weeks
Adverse events were collected from date of enrollment through the date of final study visit. The total time period of collection was approximately one and a half weeks.

Additional Information

Clinical Project Manager

Rockwell Medical, Inc

Phone: 248-960-9009

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place

Restriction type: GT60