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A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of RLS-0071 in Newborns With Moderate or Severe Hypoxic-Ischemic Encephalopathy Undergoing Therapeutic Hypothermia (STAR)

2 november 2026 bijgewerkt door: ReAlta Life Sciences, Inc.

A Phase 2/3, Two-Stage, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of RLS-0071 in Newborns With Moderate or Severe Hypoxic-Ischemic Encephalopathy Undergoing Therapeutic Hypothermia With Long-Term Follow-Up

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 efficacy, safety, and tolerability of RLS-0071 in the treatment of newborns with moderate or severe HIE.

Studie Overzicht

Toestand

Werving

Conditie

Interventie / Behandeling

Gedetailleerde beschrijving

This is a Phase 2/3, two-stage, multisite, randomized, double-blind, placebo-controlled study to assess the safety, efficacy, and pharmacokinetics (PK) of RLS-0071 in newborns with moderate or severe HIE undergoing therapeutic hypothermia, with long-term follow-up of up to 24 months of age.

The study consists of 2 sequential stages for all participants in all cohorts. In Stage 1, participants will receive either ascending doses of RLS-0071 or a matched volume of placebo every 8 hours (q8h) 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 through Day 14, including magnetic resonance imaging (MRI) measurements of brain injury. After completion of Stage 1, participants will transition to Stage 2 of the study for long-term observation, including developmental assessments, until participants reach 24 months of age.

The Phase 2 portion of the STAR study is designed to inform dose escalation and correlation analysis to affirm that MRI measurement of brain injury in HIE babies is predictive of long-term neurocognitive outcomes at 24 months. The Phase 2 portion of the study consists of Cohort 1 and Cohort 2 subsets. The first recruited cohorts, 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. 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+.

The Phase 3 portion of the study is designed to generate data focusing on assessing the efficacy of RLS-0071 compared to placebo in reducing HIE brain injury as measured by MRI and improving the long-term neurocognitive outcomes at 24 months. The Phase 3 portion of the study consists of Cohort 2+ (2a+ [moderate] or 2b+ [severe]) and Cohort 3 (3a [moderate] or 3b [severe]), with participants recruited in these cohorts receiving 10 mg/kg RLS-0071 or a matched volume of placebo.

Studietype

Ingrijpend

Inschrijving (Geschat)

173

Fase

  • Fase 2
  • Fase 3

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studiecontact

Studie Locaties

  • Verenigde Staten
    • Arkansas
      • Little Rock, Arkansas, Verenigde Staten, 72202
        • Werving
        • Study Site 016
    • California
      • Orange, California, Verenigde Staten, 92868
        • Werving
        • Study Site 013
      • San Diego, California, Verenigde Staten, 92123
        • Werving
        • Study Site 019
      • San Diego, California, Verenigde Staten, 92037-7774
        • Werving
        • Study Site 020
    • Colorado
      • Aurora, Colorado, Verenigde Staten, 80045
        • Werving
        • Study Site 027
    • Florida
      • Gainesville, Florida, Verenigde Staten, 32608
        • Werving
        • Study Site 001
      • Miami, Florida, Verenigde Staten, 33143
        • Werving
        • Study Site 018
      • Orlando, Florida, Verenigde Staten, 32803
        • Werving
        • Study Site 010
    • Indiana
      • Indianapolis, Indiana, Verenigde Staten, 46202
        • Werving
        • Study Site 014
    • Kentucky
      • Lexington, Kentucky, Verenigde Staten, 40536
        • Ingetrokken
        • Study Site 012
      • Louisville, Kentucky, Verenigde Staten, 40202
        • Werving
        • Study Site 025
    • Massachusetts
      • Boston, Massachusetts, Verenigde Staten, 02115
        • Werving
        • Study Site 002
      • Boston, Massachusetts, Verenigde Staten, 02114
        • Werving
        • Study Site 029
    • Missouri
      • St Louis, Missouri, Verenigde Staten, 63110
        • Werving
        • Study Site 006
    • New York
      • Manhasset, New York, Verenigde Staten, 11030-3816
        • Werving
        • Study Site 024
      • New Hyde Park, New York, Verenigde Staten, 11040
        • Werving
        • Study Site 023
      • New York, New York, Verenigde Staten, 10032
        • Werving
        • Study Site 028
    • North Carolina
      • Durham, North Carolina, Verenigde Staten, 27710
        • Werving
        • Study Site 003
    • Ohio
      • Cleveland, Ohio, Verenigde Staten, 44195
        • Werving
        • Study Site 021
    • Texas
      • Fort Worth, Texas, Verenigde Staten, 76104
        • Werving
        • Study Site 022
      • Houston, Texas, Verenigde Staten, 77030
        • Werving
        • Study Site 030
    • West Virginia
      • Morgantown, West Virginia, Verenigde Staten, 26506
        • Werving
        • Study Site 005

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

Niet ouder dan 10 uur (Kind)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

Inclusion Criteria:

  1. ≥ 36 weeks gestation.

  2. Sentinel event prior to delivery such as abruption, tight nuchal cord, uterine rupture, profound bradycardia, shoulder dystocia, or cord prolapse or other acute event likely attributable to newborn depression at delivery or an acute change in the fetal status with a clinical presentation consistent with an acute sentinel event with no clearly defined etiology.

  3. Moderate or severe encephalopathy based on at least one risk of encephalopathy from criterion (a) and one clinical sign of encephalopathy from criterion (b):

    1. Risk of encephalopathy (either):

      • Blood gas drawn within 1 hour of birth, either arterial blood gas (ABG) or venous blood gas (VBG) (cord or infant) with pH ≤ 7.0 OR base deficit ≥ 16 mmol/L.

      OR

      • appearance, pulse, grimace, activity, and respiration (APGAR) score 2-5 at 10 minutes OR
      • The infant required assisted ventilation ≥ 10 minutes after birth (ie, endotracheal, mask ventilation, or continuous positive airway pressure [CPAP]).

    2. Clinical signs of encephalopathy (either/both):

      • Moderate/Severe encephalopathy on National Institute of Child Health and Human Development assessment.
      • Evidence of seizures (clinical and/or electroencephalogram).

  4. Be eligible to receive therapeutic hypothermia.

  5. Active whole-body cooling to be started prior to 6 hours of age (passive cooling is permitted prior to active whole-body cooling).

  6. Product of a singleton pregnancy.

  7. Written informed consent obtained from parent or legal guardian.

Exclusion Criteria:

  1. Inability to enroll in the study and initiate the first dose of RLS-0071 within 10 hours of life.
  2. Known major congenital and/or chromosomal abnormality(ies).
  3. Severe growth restriction (birth weight ≤ 1800 g).
  4. Prenatal diagnosis of brain abnormality or hydrocephalus.
  5. Participant's head circumference is < 30 cm.
  6. 10-minute APGAR score < 2, if available.
  7. Infants suspected of overwhelming sepsis or congenital infection based on the Investigator's clinical consideration at the time of enrollment.
  8. Persistent severe hypotension unresponsive to inotropic support (requiring >2 inotropes, not inclusive of hydrocortisone).
  9. Persistent severe hypoxia in the setting of 100% fraction of inspired oxygen (FiO₂) and unresponsive to nitric oxide or requiring extracorporeal membrane oxygenation (ECMO).
  10. Severe disseminated intravascular coagulation with clinical bleeding.
  11. Neonatal encephalopathy believed to be due to a cause other than perinatal hypoxia (ie, other than HIE).
  12. Moribund infants for whom withdrawal of care is being considered.
  13. Suspected or confirmed fetal alcohol syndrome or suspected substance withdrawal seizures.
  14. Any other condition that the investigator may consider would make the participant ineligible for the study or place the participant at an unacceptable risk, including a clinically significant (eg, Grade 3 or 4) intracranial hemorrhage.

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Verviervoudigen

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: RLS-0071

Doses of RLS-0071 to be administered every 8 hours (q8h), for a total of 10 doses over 72 hours.
Geneesmiddel: RLS-0071

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-vergelijker: Placebo

Doses of sterile saline (sodium chloride, 0.9%) to be administered every 8 hours (q8h), for a total of 10 doses over 72 hours.
Geneesmiddel: Placebo

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.

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Extent of acute brain injury at Day 4 (+3), assessed through magnetic resonance imaging (MRI), using a standardized scoring system
Tijdsspanne: Day 4 (up to Day 7)

Brain injury is assessed using the Weeke score (Weeke 2018), which 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.
Day 4 (up to Day 7)

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

ReAlta Life Sciences, Inc.

Medewerkers

Premier Research

Publicaties en nuttige links

De persoon die verantwoordelijk is voor het invoeren van informatie over het onderzoek stelt deze publicaties vrijwillig ter beschikking. Dit kan gaan over alles wat met het onderzoek te maken heeft.

Algemene publicaties

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

27 juli 2023

Primaire voltooiing (Geschat)

1 december 2026

Studie voltooiing (Geschat)

1 december 2028

Studieregistratiedata

Eerst ingediend

3 maart 2023

Eerst ingediend dat voldeed aan de QC-criteria

17 maart 2023

Eerst geplaatst (Werkelijk)

21 maart 2023

Updates van studierecords

Laatste update geplaatst (Werkelijk)

5 november 2026

Laatste update ingediend die voldeed aan QC-criteria

2 november 2026

Laatst geverifieerd

1 november 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • RLS-0071-202

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

NEE

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Ja

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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