High-dose Erythropoietin for Asphyxia and Encephalopathy (HEAL)
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ClinicalTrials.gov Identifier: NCT02811263 |
Recruitment Status :
Active, not recruiting
First Posted : June 23, 2016
Last Update Posted : January 15, 2021
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Sponsor:
University of California, San Francisco
Collaborators:
University of Washington
Mednax Center for Research, Education, Quality and Safety
University of Utah
Children's National Research Institute
University of Minnesota
University of Texas
Washington University School of Medicine
Indiana University
Stanford University
University of Pittsburgh
Children's Hospital Los Angeles
Nationwide Children's Hospital
Boston University
University of New Mexico
University of Chicago
University of North Carolina
Vanderbilt University
Children's Hospital Medical Center, Cincinnati
Johns Hopkins University
Cook Children's Medical Center
Children's Hospital of Philadelphia
Information provided by (Responsible Party):
Yvonne Wu, University of California, San Francisco
Tracking Information | |||||||
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First Submitted Date ICMJE | June 17, 2016 | ||||||
First Posted Date ICMJE | June 23, 2016 | ||||||
Last Update Posted Date | January 15, 2021 | ||||||
Actual Study Start Date ICMJE | January 2017 | ||||||
Estimated Primary Completion Date | January 2022 (Final data collection date for primary outcome measure) | ||||||
Current Primary Outcome Measures ICMJE |
Death or neurodevelopmental impairment [ Time Frame: Prior to final outcome assessment at 22-26 months of age ] Neurodevelopmental impairment defined as any of the following: a) Gross Motor Function Scale (GMFCS) level ≥ 1, or b) GMFCS = 0 or 0.5 and cerebral palsy (CP) (any type), or c) Bayley III Cognitive Score < 90
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Original Primary Outcome Measures ICMJE |
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Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures |
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Original Other Pre-specified Outcome Measures | Not Provided | ||||||
Descriptive Information | |||||||
Brief Title ICMJE | High-dose Erythropoietin for Asphyxia and Encephalopathy | ||||||
Official Title ICMJE | High-dose Erythropoietin for Asphyxia and Encephalopathy | ||||||
Brief Summary | Hypoxic-ischemic encephalopathy (HIE) occurs when a baby gets reduced blood flow and oxygen to the brain near the time of birth. This results in death or neurologic disabilities including cerebral palsy and cognitive impairment in up to half of affected infants. This clinical trial will determine if the drug erythropoietin (Epo) added to hypothermia (usual therapy) will improve outcomes for infants suffering from HIE. | ||||||
Detailed Description | Neonatal hypoxic-ischemic encephalopathy (HIE) refers to brain injury resulting from reduced blood and oxygen flow to a baby's brain near the time of birth. HIE affects up to 12,000 newborns each year in the U.S. Half of affected infants have a bad outcome including death, cerebral palsy and cognitive impairment despite receiving hypothermia, the only available treatment. Erythropoietin (Epo) is a cytokine with remarkable neuroprotective and neuroregenerative effects demonstrated in animal models of neonatal brain injury. In a phase I trial of Epo + hypothermia, the investigators found that Epo 1000 U/Kg/dose best reproduced the pharmacokinetics of neuroprotective dosing in animal models. Long term outcomes were better than expected based on entry criteria and MRI findings. A phase II trial compared 50 cooled infants randomized to receive Epo or placebo. Infants treated with hypothermia + Epo had less brain injury on early MRI, and better 12-month motor development. The investigators hypothesize that Epo given to cooled infants with moderate/severe HIE will reduce the combined primary outcome of death or neurodevelopmental impairment from 49 to 33%. This is a randomized, double-blind, placebo-controlled trial of Epo therapy in 500 infants with HIE undergoing hypothermia. Specific aims are 1) To determine if 5 doses of Epo 1000 U/kg IV reduces the rate of death, motor or cognitive deficits at 2 years; 2) To assess safety of Epo by evaluating clinical toxicity; and 3) To determine whether Epo decreases the severity of neonatal brain injury as evidenced by early MRI and circulating biomarkers of brain injury. The investigators anticipate that Epo will confer improved 2-year neurodevelopmental outcome, will be safe, and will decrease brain injury severity as determined by early biomarkers. | ||||||
Study Type ICMJE | Interventional | ||||||
Study Phase ICMJE | Phase 3 | ||||||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
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Intervention ICMJE |
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||
Recruitment Status ICMJE | Active, not recruiting | ||||||
Actual Enrollment ICMJE |
501 | ||||||
Original Estimated Enrollment ICMJE |
500 | ||||||
Estimated Study Completion Date ICMJE | September 2022 | ||||||
Estimated Primary Completion Date | January 2022 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | up to 24 Hours (Child) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||
Listed Location Countries ICMJE | United States | ||||||
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Administrative Information | |||||||
NCT Number ICMJE | NCT02811263 | ||||||
Other Study ID Numbers ICMJE | P0511976 | ||||||
Has Data Monitoring Committee | Yes | ||||||
U.S. FDA-regulated Product | Not Provided | ||||||
IPD Sharing Statement ICMJE |
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Responsible Party | Yvonne Wu, University of California, San Francisco | ||||||
Study Sponsor ICMJE | University of California, San Francisco | ||||||
Collaborators ICMJE |
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Investigators ICMJE |
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PRS Account | University of California, San Francisco | ||||||
Verification Date | January 2021 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |