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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
42
RLS-0071 (unit strength 10 mg/mL) will be administered by infusion for a dose level of 3 or 10 mg/kg. Planned infusion duration is 10 minutes for all dose levels.
Placebo control (commercial sterile saline) will be administered by infusion at a volume matched to RLS-0071 (3 or 10 mg/kg). Planned infusion duration is 10 minutes for all matched dose levels.
Study Site 016
Little Rock, Arkansas, United States
RECRUITINGStudy Site 013
Orange, California, United States
RECRUITINGStudy Site 020
San Diego, California, United States
RECRUITINGStudy Site 019
San Diego, California, United States
RECRUITINGStudy Site 001
Gainesville, Florida, United States
RECRUITINGStudy Site 018
Miami, Florida, United States
RECRUITINGStudy Site 010
Orlando, Florida, United States
RECRUITINGStudy Site 014
Indianapolis, Indiana, United States
RECRUITINGStudy Site 012
Lexington, Kentucky, United States
WITHDRAWNStudy Site 002
Boston, Massachusetts, United States
RECRUITING...and 5 more locations
Frequency and severity of adverse events (AEs) and serious adverse events (SAEs) by treatment group at Day 14
Number of participants with AEs and SAEs graded between Grade 1 (mild in severity) and Grade 5 (death related to AE).
Time frame: Day 1 to Day 14
Frequency and severity of events of special interest and SAEs by treatment group at 24 months
Number of participants with events of special interest and SAEs graded between Grade 1 (mild in severity) and Grade 5 (death related to AE). Events of special interest are: autoimmune disorder, persistent hypotension, persistent pulmonary hypertension, acute kidney injury, major venous thrombosis, severe intracranial hemorrhage, pulmonary hemorrhage, culture proven sepsis, necrotizing enterocolitis, severe thrombocytopenia, hepatic dysfunction, hyperbilirubinemia, coagulopathy, hypocalcemia, cerebral palsy, developmental or speech delay, learning disability, and visual or hearing impairment.
Time frame: Day 1 to 24 months
Frequency of premature discontinuation by treatment group due to AEs at Day 14
Number of participants who prematurely discontinue from the study due to AEs
Time frame: Day 1 to Day 14
Acute brain injury at Day 4, assessed through magnetic resonance imaging (MRI), using a standardized scoring system
Brain injury MRI score includes scoring extent of injury across 4 domains (Grey matter, White matter/cortex, Cerebellum, and Additional). A score of 0 indicates a normal brain MRI, whereas the maximum score of 57 indicates extensive bilateral injury.
Time frame: Day 4
Acute brain injury at Day 12, assessed through magnetic resonance imaging (MRI), using a standardized scoring system
Brain injury MRI score includes scoring extent of injury across 4 domains (Grey matter, White matter/cortex, Cerebellum, and Additional). A score of 0 indicates a normal brain MRI, whereas the maximum score of 57 indicates extensive bilateral injury.
Time frame: Day 12
Composite of mortality and neurodevelopmental impairment (NDI) at 24 months
Time frame: Day 1 to 24 months
Mortality at 3, 6, 12, 18, and 24 months
Number of participants alive at each timepoint
Time frame: Day 1 to 3, 6, 12, 18, and 24 months
Number of participants with clinically significant laboratory abnormalities, events of special interest, and SAEs at 3, 6, 12, and 18 months
Time frame: Day 1 to 3, 6, 12, and 18 months
Neurocognitive developmental outcome assessed by Bayley-4 at 24 months of age
The Bayley Scales of Infant and Toddler Development (4th Edition) consists of 5 subdomains: the Cognitive, Language, Motor, Social-Emotional, and Adaptive Behavior scales. Cognitive, Language, and Motor domains include a total of 264 items, each ranked between 0-2 (where 0 is not present and 2 is mastery); the Social-Emotional and Adaptive Behavior domains are assessed through caregiver questionnaires.
Time frame: 24 months
Neurodevelopmental growth impact: Diagnosis of cerebral palsy at 24 months of age
Number of participants diagnosed with cerebral palsy
Time frame: 24 months
Neurodevelopmental growth impact: Grading of cerebral palsy by using the Gross Motor Function Classification System-Expanded and Revised (GMFCS-E&R) at 24 months of age
The GMFCS is a 5-level classification system for children and young people with cerebral palsy, where Level 1 indicates ability to walk without limitations and Level 5 indicates reliance on a manual wheelchair.
Time frame: 24 months
Number of participants diagnosed with mild, moderate, or severe visual impairment and hearing impairment
Time frame: Day 1 to 24 months
Number of days of supplemental nutritional support required
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Time frame: Day 1 to 24 months
Seizure occurrence
Discrete number of seizures recorded
Time frame: Day 1 to Day 14
Total seizure burden (total number of minutes seizing as measured by continuous electroencephalogram [EEG]) during hospitalization
Time frame: Day 1 to Day 14
Electrical activity abnormality scoring as measured by EEG
Time frame: Day 1 to Day 14
Impact on infant and family wellness, assessed by the Mother-to-Infant Bonding Scale (MIBS)
The MIBS is a 9-item questionnaire, with total scores ranging from 0 to 27. A high score indicates weaker mother-to-infant bonding.
Time frame: 3 and 12 months
Impact on infant and family wellness, assessed by the Parenting Stress Index, 4th Edition Short Form (PSI-4-SF)
The PSI-4 is a 36-item questionnaire focusing on three domains: Parental Distress, Parent-Child Dysfunctional Interaction, and Difficult Child, which combine to form a Total Stress scale. Scores are assessed following conversion into percentile ranks. Scores falling in the 90th or higher percentile indicate clinically significant parenting stress.
Time frame: 3, 12, and 24 months
Quality of life assessment over the first 24 months of life
A quality of life questionnaire will be used to collect information regarding the care of the aging child over the first 24 months of life.
Time frame: Day 4 to 24 months