A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of RLS-0071 in Newborns With Moderate or Severe Hypoxic-Ischemic Encephalopathy Receiving Therapeutic Hypothermia

What this Trial Is About

Hypoxic-ischemic encephalopathy (HIE) is a serious condition affecting newborns and can lead to high mortality or significant brain-related disabilities. Researchers are studying RLS-0071, a new peptide, to assess its safety and tolerability in treating newborns with moderate or severe HIE who are receiving therapeutic hypothermia. This trial is a Phase 2, randomized, double-blind, placebo-controlled study designed to explore safety, drug behavior in the body, and early signs of effectiveness. Participants will receive multiple doses of either RLS-0071 or a placebo through infusions every 8 hours for a total of 10 doses over 72 hours, alongside standard care including cooling treatment. The study has two stages: Stage 1 involves dose escalation based on safety data, and Stage 2 involves long-term follow-up of participants until they reach 24 months old. The dose levels studied include 3 mg/kg and, if recommended by safety boards, an escalated dose of 10 mg/kg. During the study, newborns will be closely monitored through brain imaging (MRI) at days 4 and 12, safety assessments up to day 14, and ongoing evaluations of neurodevelopment, mortality, seizures, and family wellbeing up to 24 months. Various tests and scales will assess brain injury, developmental progress, motor function, vision, hearing, nutrition, and quality of life. Safety and adverse events will be carefully tracked throughout the study period and beyond.

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