Infant, NewbornPhase 2/3EPONCT01203514
The Effects of Erythropoietin (EPO) on the Transfusion Requirements of Very Low Birth Weight Infants
A Phase 2/3 study testing Erythropoietin in people with Infant, Newborn and Infant, Low Birth Weight, run by NICHD Neonatal Research Network at 9 sites in United States.
Trial summary
This study tested the safety and efficacy of transfusing erythropoietin (Epo) and iron in infants of <1,250g birth weight. For infants 401-1,000g birth weight, we tested whether early erythropoietin (Epo) and iron therapy would decrease the number of transfusions received. Read more about this study
- Who is it for?
- Ages 24 hours to 96 daysHealthy volunteers can take part
- Conditions the study is for
- Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age
- What is being tested?
- Erythropoietin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 318 participants
- Where
- 9 sitesUnited States
- Study dates
- Aug 1997to Aug 2000
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
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 24 hours to 96 days
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- Infants with a birth weight of 4010-1250g, <32 weeks' gestation, and 24-96 hours old at the time of study entry
- Likely to survive >72 hours
- Informed consent from a parent or guardian.
Who cannot join
- Major congenital anomaly
- A positive direct antiglobulin test
- Evidence of coagulopathy
- Clinical seizures
- Systolic blood pressure >100 mm Hg (in the absence of pressor support)
- Absolute neutrophil count (ANC) of <=500/micro-L
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
University of New Mexico
Albuquerque, New Mexico, 87131
Emory University
Atlanta, Georgia, 30303
Harvard University
Cambridge, Massachusetts, 02138
Cincinnati Children's Medical Center
Cincinnati, Ohio, 45267
Wayne State University
Detroit, Michigan, 48201
Indiana University
Indianapolis, Indiana, 46202
University of Tennessee
Memphis, Tennessee, 38163
Yale University
New Haven, Connecticut, 06504
George Washington University
Washington D.C., District of Columbia, 20052
About this study
Official title: The Effects of Erythropoietin (EPO) on the Transfusion Requirements of Preterm Infants 401-1250 Grams: Two Multi-Center, Randomized, Double-Masked, Placebo Controlled Studies
This study tested the safety and efficacy of transfusing erythropoietin (Epo) and iron in infants of <1,250g birth weight. For infants 401-1,000g birth weight, we tested whether early erythropoietin (Epo) and iron therapy would decrease the number of transfusions received. For infants 1,001-1,250g birth weight, we tested whether early erythropoietin (Epo) and iron therapy would decrease the percentage of infants who received any transfusions.
Read the detailed description
Critically ill preterm infants experience in the first 1-2 weeks after birth daily blood losses that may equal 5-10% of their total blood volume. Such losses and associated anemia typically result in multiple erythrocyte transfusions. This iatrogenic anemia commonly is followed by the anemia of prematurity, prompting additional transfusions.
This study tested the safety and efficacy of transfusing erythropoietin (Epo) and iron in infants of <1,250g birth weight. For infants 401-1,000g birth weight (Trial 1), we tested whether early erythropoietin (Epo) and iron therapy would decrease the number of transfusions received. For infants 1,001-1,250g birth weight (Trial 2), we tested whether early erythropoietin (Epo) and iron therapy would decrease the percentage of infants who received any transfusions.
Therapy was initiated by day of life 4 and continued through the 35th postmenstrual week. Infants were randomized to receive either Epo and iron therapy or a sham procedure. Treated infants received 400 U/kg Epo 3 times weekly and a weekly intravenous infusion of 5 mg/kg iron dextran until they had an enteral intake of 60 mL/kg/d. Infants in the placebo/control group received sham subcutaneous injections when intravenous access was not available. Complete blood and reticulocyte counts were measured weekly, and ferritin concentrations were measured monthly.
Transfusions were administered according to protocol. Infants did not receive a transfusion solely to replace blood lost through phlebotomy. Infants who met transfusion criteria received a transfusion of 15 mL/kg packed red blood cells (PRBC) for a hematocrit of >25% or 20 mL/kg PRBC for a hematocrit of <=25%. Blood losses and transfusion data were recorded.
Trial 2 was terminated after enrollment of 118 infants after the Data and Safety Monitoring Committee reviewed the final results of Trial 1 and preliminary results of Trial 2. On the basis of these data, the Committee concluded that it was statistically unlikely that Trial 2 would demonstrate a significant decrease in the percentage of infants who would receive a transfusion during the study.
- Conditions
- Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature, Anemia, Neonatal
- Being tested
- Erythropoietin, Sham Comparator
- Keywords
- NICHD Neonatal Research Network, Extremely Low Birth Weight (ELBW), Prematurity, Erythrocyte transfusions, Erythropoietin (Epo), Blood loss, Phlebotomy
- Medical subject headings
- Premature Birth, Anemia, Neonatal, Hemorrhage
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ErythropoietinDrug
Also called Human recombinant erythropoietin
Infants received 400 U/kg Erythropoietin (Epo) 3 times weekly and a weekly intravenous infusion of 5 mg/kg iron dextran until they had an enteral intake of 60 mL/kg/d.
Sham ComparatorOther
Infants in the placebo/control group received sham subcutaneous injections when intravenous access was not available.
| Study group | Role | What they receive |
|---|---|---|
| Trial 1 Experimental | Experimental | Infants 401-1,000g birthweight Receives: Drug: Erythropoietin |
| Trial 1: Sham Comparator | Sham comparator | Infants 401-1,000g birthweight Receives: Other: Sham Comparator |
| Trial 2: Experimental | Experimental | Infants 1,001-1,250g birth weight Receives: Drug: Erythropoietin |
| Trial 2: Sham Comparator | Sham comparator | Infants 1,001-1,250g birth weight Receives: Other: Sham Comparator |
What is being measured
The primary outcome is the main question the study is designed to answer.
Erythrocyte transfusions in infants 401-1,000g birthweight
Measured: Hospital discharge or 35 weeks postmenstrual age
Blood transfusions
Measured: Hospital discharge or 35 weeks postmenstrual age
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2010 (estimated)
- Study startAug 1997
- Primary completionAug 1998
- Study completionAug 2000
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Infant, NewbornStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Outcomes assessor
- Enrolment
- 318 participants (actual)
Sponsor and investigators
- Lead sponsor
- NICHD Neonatal Research Network
- Sponsor type
- Research network
- Collaborators
- National Center for Research Resources (NCRR)
- Investigators
- Robin K. Ohls, MD, Study director, University of New MexicoEdward F. Donovan, MD, Principal investigator, Children's Hospital Medical Center, CincinnatiBarbara J. Stoll, MD, Principal investigator, Emory UniversityAnn R. Stark, MD, Principal investigator, Brigham and Women's HospitalJames A. Lemons, MD, Principal investigator, Indiana UniversitySheldon B. Korones, MD, Principal investigator, University of TennesseeSeetha Shankaran, MD, Principal investigator, Wayne State UniversityRichard A. Ehrenkranz, MD, Study director, Yale University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01203514
- Sponsor's protocol ID
- NICHD-NRN-0017
- Other IDs
- U10HD027853, M01RR008084, U10HD027851, M01RR000039, U10HD034167, M01RR002635, M01RR002172, M01RR001032
- Acronym
- EPO
- Status last verified
- Sep 2017
- First submitted
- 15 Sep 2010
- Last updated
- 26 Sep 2017
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Ohls RK, Ehrenkranz RA, Wright LL, Lemons JA, Korones SB, Stoll BJ, Stark AR, Shankaran S, Donovan EF, Close NC, Das A. Effects of early erythropoietin therapy on the transfusion requirements of preterm infants below 1250 grams birth weight: a multicenter, randomized, controlled trial. Pediatrics. 2001 Oct;108(4):934-42. doi: 10.1542/peds.108.4.934.
PubMed 11581447Ohls RK, Ehrenkranz RA, Das A, Dusick AM, Yolton K, Romano E, Delaney-Black V, Papile LA, Simon NP, Steichen JJ, Lee KG; National Institute of Child Health and Human Development Neonatal Research Network. Neurodevelopmental outcome and growth at 18 to 22 months' corrected age in extremely low birth weight infants treated with early erythropoietin and iron. Pediatrics. 2004 Nov;114(5):1287-91. doi: 10.1542/peds.2003-1129-L.
PubMed 15520109
Study at a glance
From the registry record, last updated by the sponsor on 26 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effects of Erythropoietin (EPO) on the Transfusion Requirements of Preterm Infants 401-1250 Grams: Two Multi-Center, Randomized, Double-Masked, Placebo Controlled Studies
- ClinicalTrials.gov ID
- NCT01203514
- Acronym / sponsor ID
- EPO / NICHD-NRN-0017
- Conditions
- Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature, Anemia, Neonatal
- Keywords
- NICHD Neonatal Research Network, Extremely Low Birth Weight (ELBW), Prematurity, Erythrocyte transfusions, Erythropoietin (Epo), Blood loss, Phlebotomy
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 318 participants (actual)
- Lead sponsor
- NICHD Neonatal Research NetworkResearch network
- Collaborators
- National Center for Research Resources (NCRR)
- Study start
- Aug 1997
- Primary completion
- Aug 1998 (actual)
- Study completion
- Aug 2000 (actual)
- First posted
- 16 Sep 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study tested the safety and efficacy of transfusing erythropoietin (Epo) and iron in infants of <1,250g birth weight. For infants 401-1,000g birth weight, we tested whether early erythropoietin (Epo) and iron therapy would decrease the number of transfusions received. For infants 1,001-1,250g birth weight, we tested whether early erythropoietin (Epo) and iron therapy would decrease the percentage of infants who received any transfusions.
Detailed description
Critically ill preterm infants experience in the first 1-2 weeks after birth daily blood losses that may equal 5-10% of their total blood volume. Such losses and associated anemia typically result in multiple erythrocyte transfusions. This iatrogenic anemia commonly is followed by the anemia of prematurity, prompting additional transfusions.
This study tested the safety and efficacy of transfusing erythropoietin (Epo) and iron in infants of <1,250g birth weight. For infants 401-1,000g birth weight (Trial 1), we tested whether early erythropoietin (Epo) and iron therapy would decrease the number of transfusions received. For infants 1,001-1,250g birth weight (Trial 2), we tested whether early erythropoietin (Epo) and iron therapy would decrease the percentage of infants who received any transfusions.
Therapy was initiated by day of life 4 and continued through the 35th postmenstrual week. Infants were randomized to receive either Epo and iron therapy or a sham procedure. Treated infants received 400 U/kg Epo 3 times weekly and a weekly intravenous infusion of 5 mg/kg iron dextran until they had an enteral intake of 60 mL/kg/d. Infants in the placebo/control group received sham subcutaneous injections when intravenous access was not available. Complete blood and reticulocyte counts were measured weekly, and ferritin concentrations were measured monthly.
Transfusions were administered according to protocol. Infants did not receive a transfusion solely to replace blood lost through phlebotomy. Infants who met transfusion criteria received a transfusion of 15 mL/kg packed red blood cells (PRBC) for a hematocrit of >25% or 20 mL/kg PRBC for a hematocrit of <=25%. Blood losses and transfusion data were recorded.
Trial 2 was terminated after enrollment of 118 infants after the Data and Safety Monitoring Committee reviewed the final results of Trial 1 and preliminary results of Trial 2. On the basis of these data, the Committee concluded that it was statistically unlikely that Trial 2 would demonstrate a significant decrease in the percentage of infants who would receive a transfusion during the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Trial 1 Experimental | Experimental | Drug: ErythropoietinInfants 401-1,000g birthweight |
| Trial 1: Sham Comparator | Sham comparator | Other: Sham ComparatorInfants 401-1,000g birthweight |
| Trial 2: Experimental | Experimental | Drug: ErythropoietinInfants 1,001-1,250g birth weight |
| Trial 2: Sham Comparator | Sham comparator | Other: Sham ComparatorInfants 1,001-1,250g birth weight |
Who may qualify
- Ages
- 24 hours to 96 days
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Infants with a birth weight of 4010-1250g, <32 weeks' gestation, and 24-96 hours old at the time of study entry
- Likely to survive >72 hours
- Informed consent from a parent or guardian.
Key exclusion criteria (6 listed)
- Major congenital anomaly
- A positive direct antiglobulin test
- Evidence of coagulopathy
- Clinical seizures
- Systolic blood pressure >100 mm Hg (in the absence of pressor support)
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 |
|---|---|---|
| ErythropoietinAlso: Human recombinant erythropoietinInfants received 400 U/kg Erythropoietin (Epo) 3 times weekly and a weekly intravenous infusion of 5 mg/kg iron dextran until they had an enteral intake of 60 mL/kg/d. | Drug | Trial 1 Experimental, Trial 2: Experimental |
| Sham ComparatorInfants in the placebo/control group received sham subcutaneous injections when intravenous access was not available. | Other | Trial 1: Sham Comparator, Trial 2: Sham Comparator |
Primary outcome
Erythrocyte transfusions in infants 401-1,000g birthweight
Time frame: Hospital discharge or 35 weeks postmenstrual age
Blood transfusions
Time frame: Hospital discharge or 35 weeks postmenstrual age
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Sep 2017.
- Results publication: PubMed 11581447Ohls RK, Ehrenkranz RA, Wright LL, Lemons JA, Korones SB, Stoll BJ, Stark AR, Shankaran S, Donovan EF, Close NC, Das A. Effects of early erythropoietin therapy on the transfusion requirements of preterm infants below 1250 grams birth weight: a multicenter, randomized, controlled trial. Pediatrics. 2001 Oct;108(4):934-42. doi: 10.1542/peds.108.4.934.
- Results publication: PubMed 15520109Ohls RK, Ehrenkranz RA, Das A, Dusick AM, Yolton K, Romano E, Delaney-Black V, Papile LA, Simon NP, Steichen JJ, Lee KG; National Institute of Child Health and Human Development Neonatal Research Network. Neurodevelopmental outcome and growth at 18 to 22 months' corrected age in extremely low birth weight infants treated with early erythropoietin and iron. Pediatrics. 2004 Nov;114(5):1287-91. doi: 10.1542/peds.2003-1129-L.
- NICHD Neonatal Research NetworkLink provided by the sponsor.
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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.
Robin K. Ohls, MD study director
University of New Mexico
Edward F. Donovan, MD principal investigator
Children's Hospital Medical Center, Cincinnati
Barbara J. Stoll, MD principal investigator
Emory University
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Erythropoietin in people with Infant, Newborn and Infant, Low Birth Weight, run by NICHD Neonatal Research Network at 9 sites 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 3 inclusion and 6 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 26 Sep 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Erythrocyte transfusions in infants 401-1,000g birthweight (time frame: Hospital discharge or 35 weeks postmenstrual age). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01203514, which also lists 2 linked publications.
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 26 Sep 2017.