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
Start Date
7/27/2023
Completion Date
12/1/2028
Summary
Hypoxic-ischemic encephalopathy (HIE) affects approximately 4,000 to 12,000 persons annually in the United States. Mortality from HIE has been reported up to 60%, with at least 25% of survivors left with significant neurocognitive disability. Despite this vital unmet medical need, no pharmacological adjunct or alternative therapy has proven beneficial in improving outcomes in neonatal HIE. RLS-0071 is a novel peptide being developed for the treatment of neonatal HIE. This study is designed to evaluate the safety and tolerability of RLS-0071 in the treatment of newborns with moderate or severe HIE.
Detailed Description
This is a Phase 2, two-stage, multisite, randomized, double-blind, placebo-controlled, multiple-ascending dose study of RLS-0071 to assess the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy in newborns with moderate or severe HIE undergoing therapeutic hypothermia. In Stage 1, participants will receive either ascending doses of RLS-0071 or a matched volume of placebo for 72 hours in addition to standard of care treatment, including therapeutic hypothermia. During and after the dosing period, participants will be monitored and assessed for safety evaluations through Day 14. After completion of Stage 1, participants will transition to Stage 2 of the study for long-term observation until participants reach 24 months of age. The first cohort subsets, consisting of Cohort 1a (moderate HIE) and 1b (severe HIE), will receive a dose of 3 mg/kg RLS-0071 or a matched volume of placebo every 8 hours (q8h). A Data Safety Monitoring Board (DSMB) will review available clinical safety and PK data from Cohort 1 subsets with completed study intervention, and make a recommendation on whether to escalate the dose for moderate and severe HIE cohorts. The Sponsor will consider the DSMB recommendation to make their decision on dose escalation in addition to their own evaluation of all available safety and PK data. If the decision is made to escalate, Cohort 2 subsets (2a \[moderate\] and 2b \[severe\]) will be recruited to receive an escalated dose of RLS-0071 (10 mg/kg) or a matched volume of placebo. Following the completion of study intervention for each Cohort 2 subset (2a \[moderate\] or 2b \[severe\]), the DSMB will review available safety and PK data and make a recommendation whether to expand enrollment for Cohort 2+ (2a+ \[moderate\] or 2b+ \[severe\]) at 10 mg/kg RLS-0071 or a matched volume of placebo.
Eligibility Criteria
Age Range: No minimum to No maximum
Interventions
RLS-0071
Placebo
Conditions
Locations
Study Site 016
Little Rock, Arkansas 72202
United States
Study Site 013
Orange, California 92868
United States
Study Site 020
San Diego, California 92037
United States
Study Site 019
San Diego, California 92123
United States
Study Site 001
Gainesville, Florida 32608
United States
Study Site 018
Miami, Florida 33143
United States
Study Site 010
Orlando, Florida 32803
United States
Study Site 014
Indianapolis, Indiana 46202
United States
Study Site 012
Lexington, Kentucky 40536
United States
Study Site 002
Boston, Massachusetts 02115
United States
Study Site 006
St Louis, Missouri 63110
United States
Study Site 003
Durham, North Carolina 27710
United States
Study Site 021
Cleveland, Ohio 44195
United States
Study Site 022
Fort Worth, Texas 76104
United States
Study Site 005
Morgantown, West Virginia 26506
United States