Trial Outcomes & Findings for Optimized Erythropoietin (EPO) Treatment (NCT NCT02075970)
NCT ID: NCT02075970
Last Updated: 2026-07-23
Results Overview
The count of the number of packed red blood cell transfusions an infant receives during the entire course of their initial hospital stay.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
33 participants
Primary outcome timeframe
From date of randomization through day of life 28 or death, whichever comes first
Results posted on
2026-07-23
Participant Flow
Participant milestones
| Measure |
Epoetin Alpha Study 1
Epoetin Alpha Tthe number of separate doses (per kg) to be given over 4 weeks by day of age will not exceed 10. These will be administered as follows: day of life (DOL) 2 (1200 U), DOL 4 (600 U), DOL 5 (600 U), DOL 6 (600U), DOL 7 (600 U), DOL 9 (600 U), DOL 14 (600 U), DOL 15 (600 U), DOL 16 (1200 U), and DOL 28 (600 U). The greatest dose in any 1 wk period is 3600 U.
Infant study 1 Drug Intervention: all infants will be treated with Epoetin Alpha during the first four weeks of life to provide data for determining: 1) the PK/PD model determined optimized Epoetin Alpha dosing schedule; and 2) clinical and laboratory covariates predictive of which infants are good and poor Epoetin Alpha responder.
Epoetin Alpha: Infant study 1: VLBW infant study subjects (weighing 1.0 to 1.5 kg at birth) who are otherwise receiving standard clinical care will be treated with Epoetin Alpha according to a dosing algorithm.
Infant study 2:
Dosing algorithm will be determined by results of Infant Study 1.
|
|---|---|
|
Overall Study
STARTED
|
33
|
|
Overall Study
COMPLETED
|
27
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
Epoetin Alpha Study 1
Epoetin Alpha Tthe number of separate doses (per kg) to be given over 4 weeks by day of age will not exceed 10. These will be administered as follows: day of life (DOL) 2 (1200 U), DOL 4 (600 U), DOL 5 (600 U), DOL 6 (600U), DOL 7 (600 U), DOL 9 (600 U), DOL 14 (600 U), DOL 15 (600 U), DOL 16 (1200 U), and DOL 28 (600 U). The greatest dose in any 1 wk period is 3600 U.
Infant study 1 Drug Intervention: all infants will be treated with Epoetin Alpha during the first four weeks of life to provide data for determining: 1) the PK/PD model determined optimized Epoetin Alpha dosing schedule; and 2) clinical and laboratory covariates predictive of which infants are good and poor Epoetin Alpha responder.
Epoetin Alpha: Infant study 1: VLBW infant study subjects (weighing 1.0 to 1.5 kg at birth) who are otherwise receiving standard clinical care will be treated with Epoetin Alpha according to a dosing algorithm.
Infant study 2:
Dosing algorithm will be determined by results of Infant Study 1.
|
|---|---|
|
Overall Study
2 infants transferred to other hospitals prior to end of required 28-day EPO treatment period
|
2
|
|
Overall Study
Received Insufficient dose of Epo
|
4
|
Baseline Characteristics
Optimized Erythropoietin (EPO) Treatment
Baseline characteristics by cohort
| Measure |
Epoetin Alpha Study 1
n=33 Participants
Epoetin Alpha Tthe number of separate doses (per kg) to be given over 4 weeks by day of age will not exceed 10. These will be administered as follows: day of life (DOL) 2 (1200 U), DOL 4 (600 U), DOL 5 (600 U), DOL 6 (600U), DOL 7 (600 U), DOL 9 (600 U), DOL 14 (600 U), DOL 15 (600 U), DOL 16 (1200 U), and DOL 28 (600 U). The greatest dose in any 1 wk period is 3600 U.
Infant study 1 Drug Intervention: all infants will be treated with Epoetin Alpha during the first four weeks of life to provide data for determining: 1) the PK/PD model determined optimized Epoetin Alpha dosing schedule; and 2) clinical and laboratory covariates predictive of which infants are good and poor Epoetin Alpha responder.
Epoetin Alpha: Infant study 1: VLBW infant study subjects (weighing 1.0 to 1.5 kg at birth) who are otherwise receiving standard clinical care will be treated with Epoetin Alpha according to a dosing algorithm.
Infant study 2:
Dosing algorithm will be determined by results of Infant Study 1.
|
|---|---|
|
Age, Categorical
<=18 years
|
33 Participants
n=9 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=9 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=9 Participants
|
|
Age, Continuous
|
45.1 hours
STANDARD_DEVIATION 15.5 • n=9 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=9 Participants
|
|
Sex: Female, Male
Male
|
24 Participants
n=9 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=9 Participants
|
|
Race (NIH/OMB)
White
|
26 Participants
n=9 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
6 Participants
n=9 Participants
|
|
Region of Enrollment
United States
|
33 participants
n=9 Participants
|
PRIMARY outcome
Timeframe: From date of randomization through day of life 28 or death, whichever comes firstThe count of the number of packed red blood cell transfusions an infant receives during the entire course of their initial hospital stay.
Outcome measures
| Measure |
Epoetin Alpha Study 1
n=27 Participants
Epoetin Alpha Tthe number of separate doses (per kg) to be given over 4 weeks by day of age will not exceed 10. These will be administered as follows: day of life (DOL) 2 (1200 U), DOL 4 (600 U), DOL 5 (600 U), DOL 6 (600U), DOL 7 (600 U), DOL 9 (600 U), DOL 14 (600 U), DOL 15 (600 U), DOL 16 (1200 U), and DOL 28 (600 U). The greatest dose in any 1 wk period is 3600 U.
Infant study 1 Drug Intervention: all infants will be treated with Epoetin Alpha during the first four weeks of life to provide data for determining: 1) the PK/PD model determined optimized Epoetin Alpha dosing schedule; and 2) clinical and laboratory covariates predictive of which infants are good and poor Epoetin Alpha responder.
Epoetin Alpha: Infant study 1: VLBW infant study subjects (weighing 1.0 to 1.5 kg at birth) who are otherwise receiving standard clinical care will be treated with Epoetin Alpha according to a dosing algorithm.
|
|---|---|
|
Number of Red Blood Cell Transfusions
|
1.2 transfusions per participant
Standard Deviation 1.0
|
PRIMARY outcome
Timeframe: Plasma concentrations collected at multiple time points within 48 hours after each dose during the first 28 days of life.First-order absorption rate constant (Ka) estimated using a population pharmacokinetic model. The estimate was derived from plasma erythropoietin concentration data collected at multiple time points following dosing throughout the 28-day study period.
Outcome measures
| Measure |
Epoetin Alpha Study 1
n=27 Participants
Epoetin Alpha Tthe number of separate doses (per kg) to be given over 4 weeks by day of age will not exceed 10. These will be administered as follows: day of life (DOL) 2 (1200 U), DOL 4 (600 U), DOL 5 (600 U), DOL 6 (600U), DOL 7 (600 U), DOL 9 (600 U), DOL 14 (600 U), DOL 15 (600 U), DOL 16 (1200 U), and DOL 28 (600 U). The greatest dose in any 1 wk period is 3600 U.
Infant study 1 Drug Intervention: all infants will be treated with Epoetin Alpha during the first four weeks of life to provide data for determining: 1) the PK/PD model determined optimized Epoetin Alpha dosing schedule; and 2) clinical and laboratory covariates predictive of which infants are good and poor Epoetin Alpha responder.
Epoetin Alpha: Infant study 1: VLBW infant study subjects (weighing 1.0 to 1.5 kg at birth) who are otherwise receiving standard clinical care will be treated with Epoetin Alpha according to a dosing algorithm.
|
|---|---|
|
Pharmacokinetic Parameter: First-order Absorption Rate Constant
|
0.0738 1/hour
Interval 0.06 to 0.088
|
PRIMARY outcome
Timeframe: Plasma concentrations measured at multiple time points within 48 hours after each dose during the first 28 days of life.Bioavailability estimated using a population pharmacokinetic target-mediated drug disposition (TMDD) model. Bioavailability represents the fraction of the administered dose reaching the systemic circulation and is reported as a unitless proportion ranging from 0 to 1. The estimate was derived from plasma erythropoietin concentration data collected at multiple time points following dosing throughout the 28-day study period.
Outcome measures
| Measure |
Epoetin Alpha Study 1
n=27 Participants
Epoetin Alpha Tthe number of separate doses (per kg) to be given over 4 weeks by day of age will not exceed 10. These will be administered as follows: day of life (DOL) 2 (1200 U), DOL 4 (600 U), DOL 5 (600 U), DOL 6 (600U), DOL 7 (600 U), DOL 9 (600 U), DOL 14 (600 U), DOL 15 (600 U), DOL 16 (1200 U), and DOL 28 (600 U). The greatest dose in any 1 wk period is 3600 U.
Infant study 1 Drug Intervention: all infants will be treated with Epoetin Alpha during the first four weeks of life to provide data for determining: 1) the PK/PD model determined optimized Epoetin Alpha dosing schedule; and 2) clinical and laboratory covariates predictive of which infants are good and poor Epoetin Alpha responder.
Epoetin Alpha: Infant study 1: VLBW infant study subjects (weighing 1.0 to 1.5 kg at birth) who are otherwise receiving standard clinical care will be treated with Epoetin Alpha according to a dosing algorithm.
|
|---|---|
|
Pharmacokinetic Parameter: Bioavailability
|
0.78 unitless
Interval 0.65 to 0.9
|
PRIMARY outcome
Timeframe: Plasma concentrations measured at multiple time points within 48 hours after each dose during the first 28 days of life.Central volume of distribution (Vc) estimated using a population pharmacokinetic target-mediated drug disposition (TMDD) model. The estimate was derived from plasma erythropoietin concentration data collected at multiple time points following dosing throughout the 28-day study period.
Outcome measures
| Measure |
Epoetin Alpha Study 1
n=27 Participants
Epoetin Alpha Tthe number of separate doses (per kg) to be given over 4 weeks by day of age will not exceed 10. These will be administered as follows: day of life (DOL) 2 (1200 U), DOL 4 (600 U), DOL 5 (600 U), DOL 6 (600U), DOL 7 (600 U), DOL 9 (600 U), DOL 14 (600 U), DOL 15 (600 U), DOL 16 (1200 U), and DOL 28 (600 U). The greatest dose in any 1 wk period is 3600 U.
Infant study 1 Drug Intervention: all infants will be treated with Epoetin Alpha during the first four weeks of life to provide data for determining: 1) the PK/PD model determined optimized Epoetin Alpha dosing schedule; and 2) clinical and laboratory covariates predictive of which infants are good and poor Epoetin Alpha responder.
Epoetin Alpha: Infant study 1: VLBW infant study subjects (weighing 1.0 to 1.5 kg at birth) who are otherwise receiving standard clinical care will be treated with Epoetin Alpha according to a dosing algorithm.
|
|---|---|
|
Pharmacokinetic Parameter: Central Volume of Distribution
|
0.080 Liters
Interval 0.055 to 0.1
|
PRIMARY outcome
Timeframe: Plasma concentrations measured at multiple time points within 48 hours after each dose during the first 28 days of life.Peripheral volume of distribution (Vp) estimated using a population pharmacokinetic target-mediated drug disposition (TMDD) model. The estimate was derived from plasma erythropoietin concentration data collected at multiple time points following dosing throughout the 28-day study period.
Outcome measures
| Measure |
Epoetin Alpha Study 1
n=27 Participants
Epoetin Alpha Tthe number of separate doses (per kg) to be given over 4 weeks by day of age will not exceed 10. These will be administered as follows: day of life (DOL) 2 (1200 U), DOL 4 (600 U), DOL 5 (600 U), DOL 6 (600U), DOL 7 (600 U), DOL 9 (600 U), DOL 14 (600 U), DOL 15 (600 U), DOL 16 (1200 U), and DOL 28 (600 U). The greatest dose in any 1 wk period is 3600 U.
Infant study 1 Drug Intervention: all infants will be treated with Epoetin Alpha during the first four weeks of life to provide data for determining: 1) the PK/PD model determined optimized Epoetin Alpha dosing schedule; and 2) clinical and laboratory covariates predictive of which infants are good and poor Epoetin Alpha responder.
Epoetin Alpha: Infant study 1: VLBW infant study subjects (weighing 1.0 to 1.5 kg at birth) who are otherwise receiving standard clinical care will be treated with Epoetin Alpha according to a dosing algorithm.
|
|---|---|
|
Pharmacokinetic Parameter: Peripheral Volume of Distribution
|
0.20 Liters
Interval 0.079 to 0.32
|
Adverse Events
Epoetin Alpha Study 1
Serious events: 1 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Epoetin Alpha Study 1
n=27 participants at risk
Epoetin Alpha Tthe number of separate doses (per kg) to be given over 4 weeks by day of age will not exceed 10. These will be administered as follows: day of life (DOL) 2 (1200 U), DOL 4 (600 U), DOL 5 (600 U), DOL 6 (600U), DOL 7 (600 U), DOL 9 (600 U), DOL 14 (600 U), DOL 15 (600 U), DOL 16 (1200 U), and DOL 28 (600 U). The greatest dose in any 1 wk period is 3600 U.
Infant study 1 Drug Intervention: all infants will be treated with Epoetin Alpha during the first four weeks of life to provide data for determining: 1) the PK/PD model determined optimized Epoetin Alpha dosing schedule; and 2) clinical and laboratory covariates predictive of which infants are good and poor Epoetin Alpha responder.
Epoetin Alpha: Infant study 1: VLBW infant study subjects (weighing 1.0 to 1.5 kg at birth) who are otherwise receiving standard clinical care will be treated with Epoetin Alpha according to a dosing algorithm.
Infant study 2:
Dosing algorithm will be determined by results of Infant Study 1.
Deaths and adverse events were not collected separately for each dose
|
|---|---|
|
Nervous system disorders
intraventicular hemorrhage
|
3.7%
1/27 • Number of events 1 • 1 month
|
Other adverse events
| Measure |
Epoetin Alpha Study 1
n=27 participants at risk
Epoetin Alpha Tthe number of separate doses (per kg) to be given over 4 weeks by day of age will not exceed 10. These will be administered as follows: day of life (DOL) 2 (1200 U), DOL 4 (600 U), DOL 5 (600 U), DOL 6 (600U), DOL 7 (600 U), DOL 9 (600 U), DOL 14 (600 U), DOL 15 (600 U), DOL 16 (1200 U), and DOL 28 (600 U). The greatest dose in any 1 wk period is 3600 U.
Infant study 1 Drug Intervention: all infants will be treated with Epoetin Alpha during the first four weeks of life to provide data for determining: 1) the PK/PD model determined optimized Epoetin Alpha dosing schedule; and 2) clinical and laboratory covariates predictive of which infants are good and poor Epoetin Alpha responder.
Epoetin Alpha: Infant study 1: VLBW infant study subjects (weighing 1.0 to 1.5 kg at birth) who are otherwise receiving standard clinical care will be treated with Epoetin Alpha according to a dosing algorithm.
Infant study 2:
Dosing algorithm will be determined by results of Infant Study 1.
Deaths and adverse events were not collected separately for each dose
|
|---|---|
|
Blood and lymphatic system disorders
anemia
|
14.8%
4/27 • Number of events 6 • 1 month
|
|
Nervous system disorders
Intraventricular or Intraparenchymal Hemorrhage
|
3.7%
1/27 • Number of events 1 • 1 month
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place