Published: June 23, 2026
Norton Children’s is currently enrolling neonatal intensive care unit (NICU) patients in a clinical trial evaluating RLS-0071, an investigational novel peptide being studied as a treatment for hypoxic-ischemic encephalopathy (HIE) in newborns. If proven effective, it would be the first pharmacological treatment approved for the condition.
The trial represents a collaboration between Norton Children’s Neonatology and Norton Children’s Neuroscience Institute, both affiliated with the UofL School of Medicine, and the NICU at Norton Children’s Hospital.
HIE affects an estimated 4,000 to 12,000 newborns annually in the U.S. and carries significant morbidity and mortality. Up to 60% of affected infants may not survive, and approximately 25% of survivors experience long-term neurocognitive disability. HIE occurs when a newborn is deprived of oxygen during or immediately after birth.
Current standard of care remains therapeutic hypothermia, with no approved pharmacological adjunct or alternative therapy.
The study recently received Food and Drug Administration authorization to transition directly from Phase 2 into Phase 3, a significant milestone granted following a favorable interim safety analysis. Rather than completing Phase 2 in full, submitting data and reopening as a new study, the trial will continue enrollment under a Phase 3 designation once the protocol amendment is approved. No safety concerns have been identified to date.
Enrolled newborns with moderate to severe HIE are randomized 2 to 1 to receive either RLS-0071 or placebo. The investigational drug is administered over 72 hours in conjunction with standard therapeutic hypothermia. Participants are monitored through day 14, then followed long-term through age 24 months.
Norton Children’s was activated as a study site April 24. The team enrolled and treated its first patient within one week of activation, with more enrolling thereafter.
The trial is led by principal investigator Margaret J. Means, M.D., pediatric neurologist with Norton Children’s Neuroscience Institute. Scott D. Duncan, M.D.; Tamina R. Singh, M.D.; and Kristi L. Havener, M.D.; neonatologists with Norton Children’s Neonatology, are subinvestigators. Rachel Welch Miles, R.N., BSN, clinical research nurse with Norton Children’s Research Institute, affiliated with the UofL School of Medicine, is the research coordinator overseeing day-to-day trial operations.
For more information on the clinical trial, visit ClinicalTrials.gov/study/NCT05778188.