Neonatal AnemiaPhase 2OETNANCT02075970
Optimized Erythropoietin (EPO) Treatment
A Phase 2 study testing Epoetin Alpha in people with Neonatal Anemia, run by University of Iowa at 1 site in United States.
Trial summary
Neonatal anemia is the most commonly encountered hematologic problem among all neonates cared for in the neonatal intensive care unit (NICU). This project seeks to better understand the pathophysiology and treatment of this challenging and important condition, especially as it affects premature, critically ill very… Read more about this study
- Who is it for?
- Ages Up to 2 days
- Conditions the study is for
- Neonatal Anemia
- What is being tested?
- Epoetin Alpha
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2014to Dec 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 2 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Post-menstrual age at birth less than 37 wk;
- birth weight of 1,001 to 1,500 g;
- postnatal age <48 h;
- respiratory distress requiring ventilation;
- signed consent by parent or guardian.
Who cannot join
- Anticipated survival <72 h;
- Hemolytic anemia due to alloimmune disease (including due to ABO), and other hemolytic disease processes;
- Major anomalies that are life-threatening during the neonatal and infant periods (central nervous system, cardiac, metabolic chromosomal including, but not limited to, trisomies, deletions, and trinucleotide repeats);
- Clinical seizures;
- Congenital thrombotic or hemorrhagic conditions including disseminated intravascular coagulation;
- Positive blood or spinal fluid bacterial or fungal culture, or other laboratory and/or clinical data indicative of sepsis, including TORCH infections, prior to 48 h of age;
Show all 10Show fewer
- Hematocrit >50%;
- Platelet count >400,000 per µL in first 48 h of life;
- Hypertension with systolic blood pressure >100 mm Hg.
- Any condition, in the opinion of the investigators, that would compromise the well being of the subject or the study, or prevent the subject from meeting or performing study requirements.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
About this study
Official title: Optimized EPO Treatment of Neonatal Anemia
Neonatal anemia is the most commonly encountered hematologic problem among all neonates cared for in the neonatal intensive care unit (NICU). This project seeks to better understand the pathophysiology and treatment of this challenging and important condition, especially as it affects premature, critically ill very low birth weight (VLBW) infants who require intensive laboratory blood monitoring leading to the need for multiple red blood cell (RBC) transfusions (RBCTX). In the research strategy proposed in Study 1, Aims 1, 2 and 3, recombinant human erythropoietin (Epoetin Alpha, PROCRIT, provided by Janssen Scientific Affairs) will first be administered to 1.0 to 1.5 kg VLBW infants; then comprehensive pharmacokinetics (PK) and pharmacodynamics (PD) data will be systematically gathered and analyzed to identify clinical and laboratory covariate parameters differentiating the infants based on their level of Epoetin Alpha responsiveness. Finally the Epoetin Alpha responsiveness predictors thus determined will be applied prospectively in the Aim 4 Study, a 2 x 2 design in which VLBW infants will be identified as good or poor Epoetin Alpha responders, based on the predictors, and then randomly assigned to receive Epoetin Alpha treatment or no treatment. This will test the central hypothesis: RBCTX can be eliminated in the majority of good Epoetin Alpha responders by optimal administration of Epoetin Alpha, but only marginal reductions in RBCTx will occur in the poor Epoetin Alpha responders. This project challenges the prevailing thinking that the efficacy of Epoetin Alpha dosing in stimulating erythropoiesis is insufficient to eliminate the need for RBC transfusions in VLBW infants. Based on extensive preclinical and clinical PK/PD studies by our PPG team, we contend that previous Erythropoietin treatment studies in VLBW infants were not able to realize the full potential of Erythropoietin to eliminate RBCTX (in contrast to the very successful use of Erythropoietin in adult renal failure patients) because previous VLBW studies were conducted 1) without Epoetin Alpha dosing individualized for the complexities of neonatal erythropoiesis and PK/PD of Epoetin Alpha and 2) without consistent criteria for RBC transfusion, Epoetin Alpha dosing, and patient enrollment. Net Epoetin Alpha responsiveness as reflected in Hb level depends on two components: Epoetin Alpha PD and RBC lifespan (Fig 15). By determining RBC lifespan, we will explain inter-subject variability of Epoetin Alpha responsiveness resulting from one of these components. The fetal lifespan data will be examined for its correlation with gestational age. If the correlation is statistically significant, gestational age will be included in the final selection of covariates for the population PK/PD model to be developed at the end of Infant Study 1. To fully understand the correlation of RBC lifespan with gestational age infants ranging from 22-42 weeks gestational age will be studied. The overall impact of Project 1 will be significant and potentially transformative: the development of a personalized, mechanism-driven approach built on sound principles will improve understanding of neonatal anemia and will be applicable to the care of premature, anemic infants.
RELEVANCE Project 1 results confirming our hypothesis that PK/PD optimized Epo treatment is effective in eliminating RBC transfusions administered to a select sub-group of NICU infants will provide fundamental knowledge about neonatal anemia that will reduce the burden of illness and disability caused by this condition. In addition, our results will stimulate researchers to extend our findings to other sub-groups with neonatal anemia, ie, smaller and sicker infants, and will stimulate novel treatments with similar, new biotechnology-produced protein drugs.
Read the detailed description
SPECIFIC AIMS Erythropoietin (Epo) stimulates red blood cell (RBC) production. Erythropoietin treatment of anemic, very low birth weight (VLBW) preterm infants has not been as effective as originally anticipated in eliminating multiple RBC transfusions (RBCTX). This conclusion is based on Epoetin Alpha dosing studies employing inconsistent RBCTX criteria, and study designs that did not fully consider Epoetin Alpha's complex pharmacokinetics (PK) and pharmacodynamics (PD). The hypothesis of this project challenges the conclusion that Epoetin Alpha therapy cannot reduce RBCTX in VLBW infants to a clinically important extent. This challenge is based on our novel comprehensive determinations of the PD of endogenous Epo in VLBW infants, and on our recent computer Epo simulation modeling results predicting that RBCTX can be eliminated in a select group of VLBW infants.
Study Objective To develop a pharmacodynamically-based, individualized medicine approach capable of completely eliminating RBCTX in an identifiable group of VLBW infants by optimally administering Epoetin Alpha.
Central Hypotheses Infants with good Epoetin Alpha responsiveness can be identified by a mechanism-driven, individualized prediction model. Optimized Epoetin Alpha treatment of the predicted good responders that is based on sound, evidence based PK/PD principles will eliminate the need for RBCTX.
Epoetin Alpha responsiveness is determined by two key components: 1) RBC production, which depends on Epoetin Alpha PD. 2) RBC lifespan. Individualized covariate-based (ie, patient specific characteristics) prediction of these two components is critical for the development of an individualized prediction model of Epoetin Alpha responsiveness that will be used for testing the Central Hypothesis. These important predictors of Epoetin Alpha responsiveness will be investigated in the following Specific Aims (SA) and Hypotheses (HY):
INFANT STUDY 1 (Years 1-3) performed in VLBW infants with birth weights from 1.0 to 1.5 kg SA 1 Determine clinical and laboratory covariates (ie, patient-specific characteristics) controlling the large inter-subject variability in Epoetin Alpha's PD using data from Epoetin Alpha dosing of VLBW infants HY 1 The inter-subject variability in Epoetin Alpha's PD is predictable by several covariates that are identifiable by our PK/PD modeling approach.
SA 2 Determine the lifespan of fetal RBC in the gestational age spectrum of the study infants.
HY 2 There exists a significant inter-subject variability in the lifespan of fetal RBCs in VLBW infants that is predictable based on gestational age.
SA 3 Derive an individualized, optimal Epoetin Alpha dosing algorithm and an individualized prediction model for the Epoetin Alpha responsiveness in VLBW infants from the results in Specific Aims 1 and 2.
HY 3 Crossvalidation-type computer simulations based on individualized and optimized Epoetin Alpha dosing in a subgroup of VLBW infants with good Epoetin Alpha responsiveness, identified by the prediction model, will indicate that RBCTX can be avoided in a select group of VLBW infants.
INFANT STUDY 2 (Years 3-4, to be addressed as a modification after completion of study 1) also performed in VLBW infant with birth weights from 1.0 to 1.5 kg SA 4 Apply the optimal Epoetin Alpha dosing algorithm and Epoetin Alpha responsiveness prediction model developed in Infant Study 1 to an Epoetin Alpha dosing study to test the Central Hypothesis.
HY 4 The subgroup of VLBW infants with good Epoetin Alpha responsiveness will have a higher proportion of infants that will not have any RBCTX compared to those with poor Epoetin Alpha responsiveness.
Expected Outcomes SA 1 Among the covariates considered in Specific Aims 1 (e.g. clinical neonatal/maternal factors, blood cell parameters, cytokines linked to erythropoiesis, inflammation biomarkers, oxidative stress, iron status, and genetic factors), we will identify several with statistically significant correlations to Epoetin Alpha's PD.
SA2 The lifespan of fetal RBCs will show similar relationship to gestational age as that which was observed by us in the ovine fetus.
SA3 The model will accurately predict individual Epoetin Alpha responsiveness to optimized Epo dosing and will provide strong support for our hypothesis that RBCTX can be completely eliminated in a select group of VLBW infants.
SA4 Infant Study 2 will show that the select group of VLBW infants that are predicted to be good Epoetin Alpha responders by the combined use of an individualized Epoetin Alpha responsiveness prediction model and an optimal, individualized Epo dosing algorithm will have a greater proportion of infants who do not require any RBCTX compared to those predicted to have poor Epoetin Alpha responsiveness.
A successful completion of the proposed research is potentially transformative and is likely to be applied to the care of VLBW infants, resulting in a significant overall impact. Moreover, a successful demonstration of the utility and power of these principles will lead to improvements in the complex pharmacotherapy of VLBW infants, who are among the most difficult to study of any patient group.
- Conditions
- Neonatal Anemia
- Being tested
- Epoetin Alpha
- Keywords
- Neonatal, Anemia, Transfusion, Erythropoetin, Epoetin Alpha, Red Blood Cell, Survival, Pharmacodynamics, Model
- Medical subject headings
- Anemia, Neonatal, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Epoetin AlphaDrug
Also called Procrit
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.
| Study group | Role | What they receive |
|---|---|---|
| Epoetin Alpha study 1 | Experimental | 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. Receives: Drug: Epoetin Alpha |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Red Blood Cell Transfusions
The count of the number of packed red blood cell transfusions an infant receives during the entire course of their initial hospital stay.
Measured: From date of randomization through day of life 28 or death, whichever comes first
Pharmacokinetic Parameter: First-order Absorption Rate Constant
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.
Measured: Plasma concentrations collected at multiple time points within 48 hours after each dose during the first 28 days of life.
Pharmacokinetic Parameter: Bioavailability
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.
Measured: Plasma concentrations measured at multiple time points within 48 hours after each dose during the first 28 days of life.
Pharmacokinetic Parameter: Central Volume of Distribution
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.
Measured: Plasma concentrations measured at multiple time points within 48 hours after each dose during the first 28 days of life.
Pharmacokinetic Parameter: Peripheral Volume of Distribution
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.
Measured: Plasma concentrations measured at multiple time points within 48 hours after each dose during the first 28 days of life.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2014 (estimated)
- Study startJun 2014
- Primary completionJun 2019
- Study completionDec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Neonatal AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Iowa
- Sponsor type
- University, hospital or other
- Collaborators
- Department of Health and Human Services, National Heart, Lung, and Blood Institute (NHLBI), Janssen Scientific Affairs, LLC
- Responsible party
- Sponsor
- Investigators
- John A Widness, MD, Principal investigator, University of Iowa
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02075970
- Sponsor's protocol ID
- 201306729
- Other IDs
- 3P01HL046925
- Acronym
- OETNA
- Status last verified
- Jun 2026
- First submitted
- 30 May 2013
- Last updated
- 23 Jul 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Optimized EPO Treatment of Neonatal Anemia
- ClinicalTrials.gov ID
- NCT02075970
- Acronym / sponsor ID
- OETNA / 201306729
- Conditions
- Neonatal Anemia
- Keywords
- Neonatal, Anemia, Transfusion, Erythropoetin, Epoetin Alpha, Red Blood Cell, Survival, Pharmacodynamics
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- University of IowaUniversity, hospital or other
- Collaborators
- Department of Health and Human Services, National Heart, Lung, and Blood Institute (NHLBI), Janssen Scientific Affairs, LLC
- Study start
- Jun 2014 (actual)
- Primary completion
- Jun 2019 (actual)
- Study completion
- Dec 2019 (actual)
- First posted
- 3 Mar 2014 (estimated)
- Results
- Posted 23 Jul 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Neonatal anemia is the most commonly encountered hematologic problem among all neonates cared for in the neonatal intensive care unit (NICU). This project seeks to better understand the pathophysiology and treatment of this challenging and important condition, especially as it affects premature, critically ill very low birth weight (VLBW) infants who require intensive laboratory blood monitoring leading to the need for multiple red blood cell (RBC) transfusions (RBCTX). In the research strategy proposed in Study 1, Aims 1, 2 and 3, recombinant human erythropoietin (Epoetin Alpha, PROCRIT, provided by Janssen Scientific Affairs) will first be administered to 1.0 to 1.5 kg VLBW infants; then comprehensive pharmacokinetics (PK) and pharmacodynamics (PD) data will be systematically gathered and analyzed to identify clinical and laboratory covariate parameters differentiating the infants based on their level of Epoetin Alpha responsiveness. Finally the Epoetin Alpha responsiveness predictors thus determined will be applied prospectively in the Aim 4 Study, a 2 x 2 design in which VLBW infants will be identified as good or poor Epoetin Alpha responders, based on the predictors, and then randomly assigned to receive Epoetin Alpha treatment or no treatment. This will test the central hypothesis: RBCTX can be eliminated in the majority of good Epoetin Alpha responders by optimal administration of Epoetin Alpha, but only marginal reductions in RBCTx will occur in the poor Epoetin Alpha responders. This project challenges the prevailing thinking that the efficacy of Epoetin Alpha dosing in stimulating erythropoiesis is insufficient to eliminate the need for RBC transfusions in VLBW infants. Based on extensive preclinical and clinical PK/PD studies by our PPG team, we contend that previous Erythropoietin treatment studies in VLBW infants were not able to realize the full potential of Erythropoietin to eliminate RBCTX (in contrast to the very successful use of Erythropoietin in adult renal failure patients) because previous VLBW studies were conducted 1) without Epoetin Alpha dosing individualized for the complexities of neonatal erythropoiesis and PK/PD of Epoetin Alpha and 2) without consistent criteria for RBC transfusion, Epoetin Alpha dosing, and patient enrollment. Net Epoetin Alpha responsiveness as reflected in Hb level depends on two components: Epoetin Alpha PD and RBC lifespan (Fig 15). By determining RBC lifespan, we will explain inter-subject variability of Epoetin Alpha responsiveness resulting from one of these components. The fetal lifespan data will be examined for its correlation with gestational age. If the correlation is statistically significant, gestational age will be included in the final selection of covariates for the population PK/PD model to be developed at the end of Infant Study 1. To fully understand the correlation of RBC lifespan with gestational age infants ranging from 22-42 weeks gestational age will be studied. The overall impact of Project 1 will be significant and potentially transformative: the development of a personalized, mechanism-driven approach built on sound principles will improve understanding of neonatal anemia and will be applicable to the care of premature, anemic infants.
RELEVANCE Project 1 results confirming our hypothesis that PK/PD optimized Epo treatment is effective in eliminating RBC transfusions administered to a select sub-group of NICU infants will provide fundamental knowledge about neonatal anemia that will reduce the burden of illness and disability caused by this condition. In addition, our results will stimulate researchers to extend our findings to other sub-groups with neonatal anemia, ie, smaller and sicker infants, and will stimulate novel treatments with similar, new biotechnology-produced protein drugs.
Detailed description
SPECIFIC AIMS Erythropoietin (Epo) stimulates red blood cell (RBC) production. Erythropoietin treatment of anemic, very low birth weight (VLBW) preterm infants has not been as effective as originally anticipated in eliminating multiple RBC transfusions (RBCTX). This conclusion is based on Epoetin Alpha dosing studies employing inconsistent RBCTX criteria, and study designs that did not fully consider Epoetin Alpha's complex pharmacokinetics (PK) and pharmacodynamics (PD). The hypothesis of this project challenges the conclusion that Epoetin Alpha therapy cannot reduce RBCTX in VLBW infants to a clinically important extent. This challenge is based on our novel comprehensive determinations of the PD of endogenous Epo in VLBW infants, and on our recent computer Epo simulation modeling results predicting that RBCTX can be eliminated in a select group of VLBW infants.
Study Objective To develop a pharmacodynamically-based, individualized medicine approach capable of completely eliminating RBCTX in an identifiable group of VLBW infants by optimally administering Epoetin Alpha.
Central Hypotheses Infants with good Epoetin Alpha responsiveness can be identified by a mechanism-driven, individualized prediction model. Optimized Epoetin Alpha treatment of the predicted good responders that is based on sound, evidence based PK/PD principles will eliminate the need for RBCTX.
Epoetin Alpha responsiveness is determined by two key components: 1) RBC production, which depends on Epoetin Alpha PD. 2) RBC lifespan. Individualized covariate-based (ie, patient specific characteristics) prediction of these two components is critical for the development of an individualized prediction model of Epoetin Alpha responsiveness that will be used for testing the Central Hypothesis. These important predictors of Epoetin Alpha responsiveness will be investigated in the following Specific Aims (SA) and Hypotheses (HY):
INFANT STUDY 1 (Years 1-3) performed in VLBW infants with birth weights from 1.0 to 1.5 kg SA 1 Determine clinical and laboratory covariates (ie, patient-specific characteristics) controlling the large inter-subject variability in Epoetin Alpha's PD using data from Epoetin Alpha dosing of VLBW infants HY 1 The inter-subject variability in Epoetin Alpha's PD is predictable by several covariates that are identifiable by our PK/PD modeling approach.
SA 2 Determine the lifespan of fetal RBC in the gestational age spectrum of the study infants.
HY 2 There exists a significant inter-subject variability in the lifespan of fetal RBCs in VLBW infants that is predictable based on gestational age.
SA 3 Derive an individualized, optimal Epoetin Alpha dosing algorithm and an individualized prediction model for the Epoetin Alpha responsiveness in VLBW infants from the results in Specific Aims 1 and 2.
HY 3 Crossvalidation-type computer simulations based on individualized and optimized Epoetin Alpha dosing in a subgroup of VLBW infants with good Epoetin Alpha responsiveness, identified by the prediction model, will indicate that RBCTX can be avoided in a select group of VLBW infants.
INFANT STUDY 2 (Years 3-4, to be addressed as a modification after completion of study 1) also performed in VLBW infant with birth weights from 1.0 to 1.5 kg SA 4 Apply the optimal Epoetin Alpha dosing algorithm and Epoetin Alpha responsiveness prediction model developed in Infant Study 1 to an Epoetin Alpha dosing study to test the Central Hypothesis.
HY 4 The subgroup of VLBW infants with good Epoetin Alpha responsiveness will have a higher proportion of infants that will not have any RBCTX compared to those with poor Epoetin Alpha responsiveness.
Expected Outcomes SA 1 Among the covariates considered in Specific Aims 1 (e.g. clinical neonatal/maternal factors, blood cell parameters, cytokines linked to erythropoiesis, inflammation biomarkers, oxidative stress, iron status, and genetic factors), we will identify several with statistically significant correlations to Epoetin Alpha's PD.
SA2 The lifespan of fetal RBCs will show similar relationship to gestational age as that which was observed by us in the ovine fetus.
SA3 The model will accurately predict individual Epoetin Alpha responsiveness to optimized Epo dosing and will provide strong support for our hypothesis that RBCTX can be completely eliminated in a select group of VLBW infants.
SA4 Infant Study 2 will show that the select group of VLBW infants that are predicted to be good Epoetin Alpha responders by the combined use of an individualized Epoetin Alpha responsiveness prediction model and an optimal, individualized Epo dosing algorithm will have a greater proportion of infants who do not require any RBCTX compared to those predicted to have poor Epoetin Alpha responsiveness.
A successful completion of the proposed research is potentially transformative and is likely to be applied to the care of VLBW infants, resulting in a significant overall impact. Moreover, a successful demonstration of the utility and power of these principles will lead to improvements in the complex pharmacotherapy of VLBW infants, who are among the most difficult to study of any patient group.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Epoetin Alpha study 1 | Experimental | Drug: Epoetin AlphaEpoetin 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. |
Who may qualify
- Ages
- Up to 2 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Post-menstrual age at birth less than 37 wk;
- birth weight of 1,001 to 1,500 g;
- postnatal age <48 h;
- respiratory distress requiring ventilation;
- signed consent by parent or guardian.
Key exclusion criteria (10 listed)
- Anticipated survival <72 h;
- Hemolytic anemia due to alloimmune disease (including due to ABO), and other hemolytic disease processes;
- Major anomalies that are life-threatening during the neonatal and infant periods (central nervous system, cardiac, metabolic chromosomal including, but not limited to, trisomies, deletions, and trinucleotide repeats);
- Clinical seizures;
- Congenital thrombotic or hemorrhagic conditions including disseminated intravascular coagulation;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Epoetin AlphaAlso: ProcritInfant 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. | Drug | Epoetin Alpha study 1 |
Primary outcome
Number of Red Blood Cell Transfusions
Time frame: From date of randomization through day of life 28 or death, whichever comes first
The count of the number of packed red blood cell transfusions an infant receives during the entire course of their initial hospital stay.
Pharmacokinetic Parameter: First-order Absorption Rate Constant
Time frame: 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.
Pharmacokinetic Parameter: Bioavailability
Time frame: 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.
Pharmacokinetic Parameter: Central Volume of Distribution
Time frame: 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.
Pharmacokinetic Parameter: Peripheral Volume of Distribution
Time frame: 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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jul 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John A Widness, MD principal investigator
University of Iowa
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Epoetin Alpha in people with Neonatal Anemia, run by University of Iowa at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Jul 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Red Blood Cell Transfusions (time frame: From date of randomization through day of life 28 or death, whichever comes first). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jul 2026.