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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 novembre 2026 mis à jour par: 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.

Aperçu de l'étude

Statut

Recrutement

Les conditions

Intervention / Traitement

Description détaillée

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.

Type d'étude

Interventionnel

Inscription (Estimé)

173

Phase

  • Phase 2
  • Phase 3

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Coordonnées de l'étude

Lieux d'étude

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

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

Pas plus vieux que 10 heures (Enfant)

Accepte les volontaires sains

Non

Sexes éligibles pour l'étude

Tout

La description

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.

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: Randomisé
  • Modèle interventionnel: Affectation parallèle
  • Masquage: Quadruple

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: RLS-0071

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

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Extent of acute brain injury at Day 4 (+3), assessed through magnetic resonance imaging (MRI), using a standardized scoring system
Délai: 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)

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

ReAlta Life Sciences, Inc.

Collaborateurs

Premier Research

Publications et liens utiles

La personne responsable de la saisie des informations sur l'étude fournit volontairement ces publications. Il peut s'agir de tout ce qui concerne l'étude.

Publications générales

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

27 juillet 2023

Achèvement primaire (Estimé)

1 décembre 2026

Achèvement de l'étude (Estimé)

1 décembre 2028

Dates d'inscription aux études

Première soumission

3 mars 2023

Première soumission répondant aux critères de contrôle qualité

17 mars 2023

Première publication (Réel)

21 mars 2023

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

5 novembre 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

2 novembre 2026

Dernière vérification

1 novembre 2026

Plus d'information

Termes liés à cette étude

Mots clés

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • RLS-0071-202

Plan pour les données individuelles des participants (IPD)

Prévoyez-vous de partager les données individuelles des participants (DPI) ?

NON

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Oui

Étudie un produit d'appareil réglementé par la FDA américaine

Non

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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