Neonatal hypoxic and ischemic brain injury

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32Active trials110Specialists8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Neonatal hypoxic-ischemic brain injury, also known as hypoxic-ischemic encephalopathy (HIE) or neonatal hypoxic-ischemic encephalopathy, is a serious condition that occurs when a newborn's brain does not receive adequate oxygen and blood flow around the time of birth. This oxygen deprivation can result from events such as placental abruption, umbilical cord complications, uterine rupture, prolonged or obstructed labor, or severe maternal hypotension. The condition primarily affects the central nervous system, with the pattern and severity of brain injury depending on the duration and degree of the hypoxic-ischemic insult, as well as the gestational age of the infant. Clinical features vary based on severity and are often classified using the Sarnat staging system. Mild cases may present with irritability, hyperalertness, and mild changes in muscle tone. Moderate to severe cases can manifest with significant encephalopathy including seizures, depressed level of consciousness, abnormal tone (hypotonia or hypertonia), feeding difficulties, respiratory failure requiring ventilation, and multi-organ dysfunction affecting the kidneys, liver, and heart. Long-term neurological consequences may include cerebral palsy, intellectual disability, epilepsy, and visual or hearing impairment. The current standard of care for moderate to severe HIE is therapeutic hypothermia (cooling therapy), which involves reducing the infant's core body temperature to approximately 33.5°C for 72 hours, initiated within 6 hours of birth. This treatment has been shown to reduce mortality and improve neurodevelopmental outcomes. Supportive care includes management of seizures with anticonvulsant medications, maintenance of adequate ventilation and perfusion, correction of metabolic disturbances, and long-term neurodevelopmental follow-up. Research into adjunctive neuroprotective therapies, including erythropoietin and stem cell therapy, is ongoing.

Also known as:

Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

FDA & Trial Timeline

10 events
Oct 2026Sildenafil Plus Hypothermia to Treat Neonatal Encephalopathy

Assistance Publique - Hôpitaux de Paris — PHASE2

TrialNOT YET RECRUITING
Jun 2026Mesenchymal Stem Cells With Cooling Therapy for Infants With Hypoxic-Ischemic Encephalopathy

Mahidol University — PHASE1, PHASE2

TrialNOT YET RECRUITING
May 2026The PROTECT-HIE Trial

University of Alberta — PHASE2

TrialNOT YET RECRUITING
Mar 2026New Tools and Predictive Markers in the Prognosis of Hypoxic-ischemic Encephalopathy Following Cardiac Arrest

Cliniques universitaires Saint-Luc- Université Catholique de Louvain — NA

TrialRECRUITING
Mar 2026Intranasal AAV9-PHP.eB Gene Therapy in Cerebral Palsy (CP) & Hypoxic Ischemic Encephalopathy (HIE)

Healing Hope International

TrialENROLLING BY INVITATION
Mar 2026Regional Ventilation Evaluation During Neuro-injury Weaning Study

Assistance Publique - Hôpitaux de Paris

TrialNOT YET RECRUITING
Mar 2026Hydrogen In Neonatal Encephalopathy (HIE) Trial

Boston Children's Hospital — PHASE1

TrialNOT YET RECRUITING
Feb 2026Predictive Value of aEEG and Cerebral Oxygenation on Neurological Outcomes in Newborns With Mild Hypoxic-Ischemic Encephalopathy

Uludag University

TrialNOT YET RECRUITING
Feb 2026Genetics of Neonatal Encephalopathy and Related Disorders

Boston Children's Hospital

TrialRECRUITING
Jan 2026Treatment of Seizures in Neonate With HIE

Mansoura University — PHASE4

TrialNOT YET RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Neonatal hypoxic and ischemic brain injury.

20 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

20 recruitingView all trials with filters →
Phase 31 trial
Neonatal Brain Ultrasound With CEUS and Elastography
Phase 3
Actively Recruiting
PI: Tiina Laurikainen (Turku University Hospital) · Sites: Turku, Southwestern Finland
Phase 41 trial
Hydrocortisone Therapy Optimization During Hypothermia Treatment in Asphyxiated Neonates
Phase 4
Actively Recruiting
PI: Kata Kovacs, MD, PhD (Semmelweis University) · Sites: Budapest, Pest County
Phase 23 trials
A Dose Escalation Study of Levetiracetam in the Treatment of Neonatal Seizures
Phase 2
Actively Recruiting
PI: Sonya G Wang, M.D. (University of Minnesota) · Sites: San Diego, California; Minneapolis, Minnesota +3 more
Assess Safety and Efficacy of Sovateltide in Hypoxic-ischemic Encephalopathy
Phase 2
Actively Recruiting
PI: Anil Gulati (Chairman and CEO) · Sites: Mangalore, Karnataka; Ludhiana, Punjab +2 more
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of RLS-0071 in Newborns With Moderate or Severe Hypoxic-Ischemic Encephalopathy Undergoing Therapeutic Hypothermia
Phase 2
Actively Recruiting
· Sites: Little Rock, Arkansas; Orange, California +13 more
Phase 11 trial
Role of Citicoline in Treatment of Neonates With Hypoxic Ischemic Encephalopathy
Phase 1
Actively Recruiting
PI: arshad Khushdil, FCPS (PEMH) · Sites: Rawalpindi
Other13 trials
Florida Neonatal Neurologic Network
Enrolling by Invitation
PI: Michael D Weiss, MD (University of Florida) · Sites: Gainesville, Florida
Healthy Little Eyes
Actively Recruiting
PI: Pelin Cengiz, MD (University of Wisconsin, Madison) · Sites: Madison, Wisconsin
Investigating Cerebral Oxygenation in the Newborn
Active
PI: Helen L Turner, MBChB (Hons) (University of Edinburgh/NHS Lothian) · Sites: Edinburgh, Lothian
Cool Prime Comparative Effectiveness Study for Mild HIE
Actively Recruiting
PI: Lina Chalak, MD (University of Texas Southwestern Medical Center) · Sites: Dallas, Texas
Intranasal AAV9-PHP.eB Gene Therapy in Cerebral Palsy (CP) & Hypoxic Ischemic Encephalopathy (HIE)
Enrolling by Invitation
PI: Dr. Anna Lara Kattan, MD: Regenerative Medicine (Stem Solutions) · Sites: Monterrey, Nuevo León · Age: 265 yrs
Biomarkers Study in Infants With Prior Neonatal Brain Injury
Actively Recruiting
PI: Melisa Carrasco McCaul, MD, PhD (University of Wisconsin, Madison) · Sites: Madison, Wisconsin
Stillbirth Risk Identification Using Multiparametric Ultrasound
Actively Recruiting
PI: Robert Rohling, PhD (The University of British Columbia) · Sites: Vancouver, British Columbia · Age: 1999 yrs
Neonatal Seizure Registry, GEnetics of Post-Neonatal Epilepsy
Active
PI: Hannah C Glass, MDCM, MAS (University of California, San Francisco) · Sites: San Francisco, California; Washington D.C., District of Columbia +6 more
Functional Imaging of Baby Brains
Actively Recruiting
· Sites: Hamilton, Ontario
Speech of Kids After Neonatal Encephalopathy
Actively Recruiting
· Sites: Budapest · Age: 07 yrs
Predicting Mortality in Patients With Return of Spontaneous Circulation After Cardiac Arrest
Active
· Sites: Diyarbakır, kayapinar · Age: 1899 yrs
Neurodevelopmental Outcome in Newborn With Hypoxic-ischemic Encephalopathy Treated With Therapeutic Hypothermia
Actively Recruiting
PI: Domenico M Romeo, MD,PHD (ChatolicUIT) · Sites: Rome; Rome
Prognostic Factors to Regain Consciousness
Actively Recruiting
PI: Jens D Rollnik, MD (BDH-Clinic Hessisch Oldendorf) · Sites: Hessisch Oldendorf · Age: 1885 yrs

Specialists

Showing 25 of 110View all specialists →
YM
Yvonne W Wu, MD, MPH
ORANGE, CA
Specialist
PI on 2 active trials
SM
Sudhin Thayyil, MD
Specialist
PI on 1 active trial
AM
Abbot Laptook, MD
PROVIDENCE, RI
Specialist
PI on 1 active trial1 Neonatal hypoxic and ischemic brain injury publication
GM
Guilherme Sant'Anna, MD
Specialist
PI on 1 active trial1 Neonatal hypoxic and ischemic brain injury publication
CM
Chatchay Prempunpong, MD
Specialist
PI on 1 active trial
EM
Elwyn Chomba, MBChB, DCH, MRCP
Specialist
PI on 1 active trial
MP
Miklós Szabó, MD, PhD
Specialist
PI on 3 active trials
UP
Unoke Meder, MD, PhD
Specialist
PI on 2 active trials
PM
Pablo Sanchez, MD
Specialist
PI on 1 active trial
KP
Kata Kovacs, MD, PhD
ASHLAND, KY
Specialist
PI on 1 active trial
CM
Cheo Lian Yeo, MBBS
Specialist
PI on 1 active trial1 Neonatal hypoxic and ischemic brain injury publication
SM
Samuel Rajadurai, MBBS
Specialist
PI on 1 active trial
JM
Jiun Lee, MBBS
Specialist
PI on 1 active trial
MP
Melisa Carrasco McCaul, MD, PhD
Specialist
PI on 2 active trials
KP
Katherine Steele, PHD
Specialist
PI on 1 active trial
HM
Hannah C. Glass, MDCM, MAS
Specialist
PI on 1 active trial
SP
Stacey C Dusing, PhD, PT
LOS ANGELES, CA
Specialist
PI on 1 active trial
BP
Barbara Sargent, PhD, PT
LOS ANGELES, CA
Specialist
PI on 1 active trial
WM
Waldemar A Carlo, MD
BIRMINGHAM, AL
Specialist
PI on 5 active trials1 Neonatal hypoxic and ischemic brain injury publication
MM
Michele C Walsh, MD
Specialist
PI on 4 active trials
LM
Lina Chalak, MD
LITTLE ROCK, AR
Specialist
PI on 3 active trials1 Neonatal hypoxic and ischemic brain injury publication
SM
Seetha Shankaran, MD
DETROIT, MI
Specialist
PI on 8 active trials
MM
Mitchell S Cairo, MD
HAWTHORNE, NY
Specialist
PI on 12 active trials
AM
Abbot R. Laptook, MD
Birmingham, Alabama
Specialist

Rare Disease Specialist

PI on 11 active trials1 Neonatal hypoxic and ischemic brain injury publication
JK
Joanne Kurtzberg
DURHAM, NC
Specialist
PI on 2 active trials16 Neonatal hypoxic and ischemic brain injury publications

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Neonatal hypoxic and ischemic brain injury.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Neonatal hypoxic and ischemic brain injury

Disease timeline:

New recruiting trial: Stillbirth Risk Identification Using Multiparametric Ultrasound

A new clinical trial is recruiting patients for Neonatal hypoxic and ischemic brain injury

New recruiting trial: Cord Blood S100B Protein Concentration in Neonates With Fetal Growth Restriction

A new clinical trial is recruiting patients for Neonatal hypoxic and ischemic brain injury

New recruiting trial: Early Cessation of Sedation and TTM in Patients With a Favourable EEG After Cardiac Arrest

A new clinical trial is recruiting patients for Neonatal hypoxic and ischemic brain injury

New recruiting trial: Pediatric Influence of Cooling Duration on Efficacy in Cardiac Arrest Patients (P-ICECAP)

A new clinical trial is recruiting patients for Neonatal hypoxic and ischemic brain injury

New recruiting trial: buRst-supprESsion TO Stop Refractory Status Epilepticus Post-cardiac Arrest

A new clinical trial is recruiting patients for Neonatal hypoxic and ischemic brain injury

New recruiting trial: Early WAKE-up Predictors After Out-of-Hospital Cardiac Arrest

A new clinical trial is recruiting patients for Neonatal hypoxic and ischemic brain injury

New recruiting trial: Predictors for Survival and Good Neurological Outcome in E-CPR and Non CPR Treated Patients

A new clinical trial is recruiting patients for Neonatal hypoxic and ischemic brain injury

New recruiting trial: Hypertonic Lactate After Cardiac Arrest

A new clinical trial is recruiting patients for Neonatal hypoxic and ischemic brain injury

New recruiting trial: Brain-injured Patients Extubation Readiness Study

A new clinical trial is recruiting patients for Neonatal hypoxic and ischemic brain injury

New recruiting trial: Role of Citicoline in Treatment of Neonates With Hypoxic Ischemic Encephalopathy

A new clinical trial is recruiting patients for Neonatal hypoxic and ischemic brain injury

Caregiver Resources

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Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Neonatal hypoxic and ischemic brain injury

What is Neonatal hypoxic and ischemic brain injury?

Neonatal hypoxic-ischemic brain injury, also known as hypoxic-ischemic encephalopathy (HIE) or neonatal hypoxic-ischemic encephalopathy, is a serious condition that occurs when a newborn's brain does not receive adequate oxygen and blood flow around the time of birth. This oxygen deprivation can result from events such as placental abruption, umbilical cord complications, uterine rupture, prolonged or obstructed labor, or severe maternal hypotension. The condition primarily affects the central nervous system, with the pattern and severity of brain injury depending on the duration and degree of

How is Neonatal hypoxic and ischemic brain injury inherited?

Neonatal hypoxic and ischemic brain injury follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Neonatal hypoxic and ischemic brain injury typically begin?

Typical onset of Neonatal hypoxic and ischemic brain injury is neonatal. Age of onset can vary across affected individuals.

Are there clinical trials for Neonatal hypoxic and ischemic brain injury?

Yes — 20 recruiting clinical trials are currently listed for Neonatal hypoxic and ischemic brain injury on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Neonatal hypoxic and ischemic brain injury?

25 specialists and care centers treating Neonatal hypoxic and ischemic brain injury are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.