Denne siden ble automatisk oversatt og nøyaktigheten av oversettelsen er ikke garantert. Vennligst referer til engelsk versjon for en kildetekst.

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 oppdatert av: 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.

Studieoversikt

Status

Rekruttering

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

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

Intervensjonell

Registrering (Antatt)

173

Fase

  • Fase 2
  • Fase 3

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Studiesteder

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

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

Ikke eldre enn 10 timer (Barn)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

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.

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Randomisert
  • Intervensjonsmodell: Parallell tildeling
  • Masking: Firemannsrom

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: RLS-0071

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

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Extent of acute brain injury at Day 4 (+3), assessed through magnetic resonance imaging (MRI), using a standardized scoring system
Tidsramme: 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)

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

ReAlta Life Sciences, Inc.

Samarbeidspartnere

Premier Research

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Generelle publikasjoner

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

27. juli 2023

Primær fullføring (Antatt)

1. desember 2026

Studiet fullført (Antatt)

1. desember 2028

Datoer for studieregistrering

Først innsendt

3. mars 2023

Først innsendt som oppfylte QC-kriteriene

17. mars 2023

Først lagt ut (Faktiske)

21. mars 2023

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

5. november 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

2. november 2026

Sist bekreftet

1. november 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • RLS-0071-202

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

NEI

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

Abonnere