PAEAN - Erythropoietin for Hypoxic Ischaemic Encephalopathy in Newborns (PAEAN)
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|ClinicalTrials.gov Identifier: NCT03079167|
Recruitment Status : Recruiting
First Posted : March 14, 2017
Last Update Posted : July 8, 2019
|Condition or disease||Intervention/treatment||Phase|
|Hypoxic-Ischemic Encephalopathy||Drug: Epoetin Alfa Drug: Normal saline||Phase 3|
A lack of oxygen (hypoxia) or low blood supply (ischaemia) before or during birth can destroy cells in a newborn baby's brain. The damage caused by the lack of oxygen continues for some time afterwards. One way to try to reduce this damage is to induce hypothermia cooling the baby or just the baby's head for hours to days. Erythropoietin (Epo) given in the first week after birth shows promise as a treatment that may also help. This study is to find out whether Epo plus induced hypothermia (cooling) of near-term newborn babies who have suffered from low blood or oxygen supply to the brain at birth reduces death and disability in survivors at two years of age.
The target population is 300 newborn term or near term infants (greater than or equal to 35+0 weeks gestation) with hypoxic ischaemic encephalopathy who are receiving, or planned to receive hypothermia and who are able to be recruited in time to allow study treatment to commence before 24 hours of age.
This is a double blind, placebo controlled, parallel, 2 arm randomised, phase III multicentre trial, stratified by study site and by severity of encephalopathy at study entry.
The treatment group of 150 infants will receive human recombinant Epo, 1000 IU/kg IV on days 1, 2, 3, 5 & 7 of life. The control group will receive 0.9% sodium chloride as a placebo on days 1, 2, 3, 5 & 7 of life.
Families will be followed up every 6 months until the primary assessment of death and disability at 2 years of age.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||300 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Preventing Adverse Outcomes of Neonatal Hypoxic Ischaemic Encephalopathy With Erythropoietin: A Phase III Randomised Placebo Controlled Multicentre Clinical Trial|
|Actual Study Start Date :||May 14, 2016|
|Estimated Primary Completion Date :||December 2021|
|Estimated Study Completion Date :||December 2021|
Erythropoietin (epoetin alfa) 1000 IU/kg birth weight (capped at 4000IU daily) IV infusion, on Days 1, 2, 3, 5 and 7 of age
Drug: Epoetin Alfa
Placebo Comparator: Placebo
IV normal saline (equiv. volume), on Days 1, 2, 3, 5 and 7 of age
Drug: Normal saline
Other Name: 0.9% NaCl
- Composite measure of death or moderate/severe disability [ Time Frame: 2 years of age ]Moderate/severe disability is defined as any cerebral palsy and a Gross Motor Function Classification Scale (GMFCS) score greater than or equal to 1), or Bayley Scale of Infant Development III (BSDIII) less than or equal to 80
- Death [ Time Frame: Any time from Day 1 of treatment to 2 years of age ]
- Cerebral palsy (CP), assessed by paediatric assessment [ Time Frame: 2 years of age ]
- Moderate/severe motor deficit [ Time Frame: 2 years of age ]Composite of any incidence of CP (any of quadriparesis, CP, hemiparesis or diparesis) AND any level of functional impairment using the GMFCS greater than or equal to 1.0
- Moderate/severe cognitive deficit [ Time Frame: 2 years of age ]Defined as a BSDIII cognitive score less than or equal to 80
- Need for supplemental respiratory support (includes tracheostomy, ventilator, high flow nasal cannula, CPAP or oxygen dependency) [ Time Frame: 2 years of age ]
- Need for nutritional support (includes gastrostomy or nasogastric feeds) [ Time Frame: 2 years of age ]
- Major cortical visual impairment by paediatric examination [ Time Frame: 2 years of age ]
- Hearing impairment status by paediatric examination - requirement for hearing aids [ Time Frame: 2 years of age ]
- Autism spectrum disorder, assessed by Modified Checklist for Autism in Toddlers (M-CHAT) parent questionnaire [ Time Frame: 2 years of age ]
- Epilepsy (history of 2 or more afebrile unprovoked seizures since discharge from neonatal unit where PAEAN study treatment was provided, or use of anticonvulsants at 2 years of age). [ Time Frame: 2 years of age ]
- Cost of healthcare and service utilisation [ Time Frame: 2 years of age ]Defined as a composite of parent completed questionnaire data and Medicare service use
- Frequency of selected adverse events (AEs) of interest, including deaths [ Time Frame: Up to 30 days post study treatment ]
- Distribution of overall disability [ Time Frame: 2 years of age ]Distribution of overall severity across 4 domains: 1) normal, 2) mild motor or cognitive deficit, 3) moderate/severe motor or cognitive deficit, and 4) death
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03079167
|Contact: PAEAN Coordinatorfirstname.lastname@example.org|
|Study Chair:||Helen Liley, BHB, MBChB||University of Sydney|