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- Essai clinique NCT07646028
R-3750 in Patients With Late-Stage Acute Respiratory Distress Syndrome (ARDS) (R-3750 ARDS)
A Phase 1, Open-Label Study to Evaluate the Safety, Tolerability, and Immunomodulatory Effects of R-3750 in Patients With Late-Stage Acute Respiratory Distress Syndrome (ARDS)
The goal of this clinical trial is to learn if R-3750 is safe in [in patients with late-stage, non-resolving Acute Respiratory Distress Syndrome (ARDS) . The main question[s] it aims to answer are:
- the favorable safety profile
- clinical improvement that includes reduced ventilator dependence and improved lung function.
Participants will be given oral capsules daily and/or enteral nasogastric or orogastric (NG/OG tube) if necessary.
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Description détaillée
Acute Respiratory Distress Syndrome (ARDS) is a severe inflammatory lung condition characterized by diffuse alveolar damage, impaired gas exchange, respiratory failure, and significant morbidity and mortality. Patients with late-stage, non-resolving ARDS often remain dependent on mechanical ventilation and have limited therapeutic options beyond supportive care. Persistent inflammation and immune dysregulation are believed to contribute to ongoing lung injury, delayed recovery, and prolonged ventilator dependence.
R-3750 is an investigational orally administered biotherapeutic designed to modulate immune responses and promote restoration of immune homeostasis. This study will evaluate the safety, tolerability, and preliminary clinical activity of R-3750 in patients with late-stage, non-resolving ARDS.
The primary objective of this clinical trial is to assess the safety and tolerability of R-3750 when administered daily to hospitalized patients with persistent ARDS. Safety evaluations will include the assessment of adverse events (AEs), serious adverse events (SAEs), laboratory parameters, vital signs, and other clinically significant findings throughout the study period.
Secondary and exploratory objectives include evaluating potential clinical benefits associated with R-3750 treatment, including:
Reduction in ventilator dependence and duration of mechanical ventilation. Improvement in pulmonary function and respiratory status. Improvement in oxygenation parameters. Changes in inflammatory and immune-related biomarkers. Assessment of overall clinical recovery and disease progression. Evaluation of survival and other clinically relevant outcomes.
Participants will receive R-3750 once daily as oral capsules. For participants unable to swallow capsules, study treatment may be administered enterally through a nasogastric (NG) or orogastric (OG) feeding tube in accordance with study procedures. Participants will undergo regular safety assessments, clinical evaluations, and laboratory monitoring throughout the treatment and follow-up periods.
The results of this study will provide important information regarding the safety profile of R-3750 and its potential to improve clinical outcomes in patients with late-stage, non-resolving ARDS, a population with substantial unmet medical need.
Type d'étude
Inscription (Estimé)
Phase
- La phase 1
Contacts et emplacements
Coordonnées de l'étude
- Nom: Christian F Freguia, PhD
- Numéro de téléphone: 215-923-1818
- E-mail: cfreguia@risetherapeutics.com
Sauvegarde des contacts de l'étude
- Nom: Janet L Stephens, PhD
- Numéro de téléphone: 650-417-8556
- E-mail: jstephens@risetherapeutics.com
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
- Adulte
- Adulte plus âgé
Accepte les volontaires sains
La description
Inclusion Criteria:
- Age ≥18 years at the time of consent.
- Acute Respiratory Distress Syndrome (ARDS) meeting the contemporary clinical definition including all of the following occurring within a 24-hour interval:
- Hypoxemia defined as PaO2/FiO2 ≤300 mmHg, or SpO2/FiO2 ≤315 if SpO2 is ≤97%;
- Bilateral pulmonary infiltrates/opacities on chest imaging consistent with pulmonary edema or fibroproliferative lung injury and not fully explained by pleural effusions, lobar collapse, or atelectasis;
- Requirement for positive pressure respiratory support, including invasive mechanical ventilation via endotracheal tube;
- Respiratory failure not fully explained by left atrial hypertension or cardiogenic pulmonary edema; if measured, pulmonary artery wedge pressure should be ≤18 mmHg.
- Late-stage, non-resolving ARDS, defined as:
- At least 7 days and no more than 28 days since ARDS onset at the time of enrollment; and
- Persistent bilateral infiltrates since ARDS onset; and
- Continued need for substantial respiratory support since ARDS onset.
- On the day of enrollment, ongoing respiratory impairment demonstrated by:
- PaO2/FiO2 ≤300 mmHg, or
- SpO2/FiO2 ≤315 if SpO2 is ≤97%.
- Subject is receiving invasive mechanical ventilation, or other protocol-defined intensive respiratory support as approved by the Sponsor/Medical Monitor.
- The treating team considers the subject an appropriate candidate for enteral administration of study drug, including by nasogastric (NG), orogastric (OG), or other enteral tube, and there is an intention to provide enteral nutrition or medication administration through the gastrointestinal tract.
- For subjects unable to provide consent, legally authorized representative (LAR) consent is obtainable in accordance with local regulations and Institutional Review Board (IRB)/Ethics Committee requirements.
- The investigator judges that the subject is likely to remain under protocol-compatible management and follow-up for the duration of the treatment and key follow-up period.
Exclusion Criteria:
- Age <18 years.
- More than 28 days since onset of ARDS at the time of enrollment.
- ARDS that is clearly resolving rapidly, in the judgment of the investigator, such that enrollment in a 28-day rescue-therapy study is not clinically appropriate.
- Pregnancy or breastfeeding.
- Participation in another interventional investigational drug or biologic study within 30 days prior to enrollment, or within 5 half-lives of the investigational product, whichever is longer, unless approved by the Sponsor and Medical Monitor.
- Extracorporeal support for gas exchange at the time of study entry, including ECMO.
Inability to use the gastrointestinal tract for study drug administration, including but not limited to:
- Continuous gastric suction/drainage that would preclude dosing,
- Bowel discontinuity preventing enteral delivery,
- Open abdomen,
- Dependence on total parenteral nutrition with no enteral access,
- Other conditions that, in the investigator's judgment, make enteral dosing infeasible.
- Known or suspected bowel ischemia, gastrointestinal perforation, uncontrolled GI bleeding, or other severe gastrointestinal disorder that would substantially increase risk from enteral dosing.
- Not committed to full supportive care, with the exception that subjects with a do-not-resuscitate order may be eligible if all other indicated intensive supportive treatments are being provided.
- AIDS by CDC criteria, including documented AIDS-defining illness or CD4 count <200 cells/mm³, if known.
- Severe immunosuppression, including any of the following:
- Cytotoxic chemotherapy within 3 weeks prior to enrollment,
- High-dose corticosteroid exposure defined as cumulative prednisone ≥300 mg equivalent within 21 days prior to enrollment,
- Ongoing systemic corticosteroid therapy >15 mg/day prednisone equivalent within 7 days prior to enrollment, unless justified as part of standard ARDS care and approved by the Medical Monitor,
- Other severe acquired or iatrogenic immunodeficiency considered clinically significant by the investigator.
- Active uncontrolled infection outside the lung that, in the judgment of the investigator, would confound safety assessment or increase subject risk.
- Severe chronic respiratory disease likely to confound ARDS assessment or outcomes, including:
- Advanced COPD requiring chronic home oxygen,
- Clinically significant pulmonary fibrosis or interstitial lung disease,
- Other severe chronic lung, chest wall, or neuromuscular disorders causing chronic respiratory failure.
- Diffuse alveolar hemorrhage due to vasculitis.
- Malignancy or other irreversible chronic condition for which estimated 6-month mortality is ≥50%, in the investigator's judgment.
- Morbid obesity or body habitus that would substantially impair protocol assessments or ventilatory interpretation, if deemed clinically significant by the investigator.
- Any condition that, in the opinion of the investigator or Medical Monitor, would make participation unsafe, interfere with interpretation of study results, or be inconsistent with study objectives.
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: Non randomisé
- Modèle interventionnel: Affectation séquentielle
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
|---|---|
|
Expérimental: Cohort 1: 9E9 CFU/day
Participants with late-stage, non-resolving Acute Respiratory Distress Syndrome (ARDS) will receive R-3750 at a dose of 9 × 10⁹ CFU/day administered once daily.
Study treatment may be administered orally as capsules or enterally via nasogastric (NG) or orogastric (OG) tube if required.
|
R-3750 is an investigational live biotherapeutic product administered once daily at either 9 × 10⁹ CFU/day or 9 × 10¹⁰ CFU/day.
Study treatment may be administered orally as capsules or enterally via nasogastric (NG) or orogastric (OG) tube when oral administration is not feasible.
|
|
Expérimental: Cohort 2: 9E10 CFU/day
Participants with late-stage, non-resolving Acute Respiratory Distress Syndrome (ARDS) will receive R-3750 at a dose of 9 × 10¹⁰ CFU/day administered once daily.
Study treatment may be administered orally as capsules or enterally via nasogastric (NG) or orogastric (OG) tube if required.
|
R-3750 is an investigational live biotherapeutic product administered once daily at either 9 × 10⁹ CFU/day or 9 × 10¹⁰ CFU/day.
Study treatment may be administered orally as capsules or enterally via nasogastric (NG) or orogastric (OG) tube when oral administration is not feasible.
|
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
To assess the safety and tolerability of R-3750 administered to participants with late-stage, non-resolving Acute Respiratory Distress Syndrome (ARDS)
Délai: From Day 1 through Day 90, including the 28-day treatment period and subsequent follow-up assessments.
|
To assess the number of participants with adverse events (AEs), serious adverse events (SAEs), with abnormal laboratory parameters, abnormal vital signs, abnormal physical examination findings throughout the study.
|
From Day 1 through Day 90, including the 28-day treatment period and subsequent follow-up assessments.
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
All-cause mortality
Délai: Day 90
|
Proportion of participants who are alive at Day 90.
|
Day 90
|
|
Ventilator-free days
Délai: Day 28
|
Number of days alive and free from invasive mechanical ventilation.
|
Day 28
|
|
Organ failure-free days
Délai: Day 28
|
Number of days alive and free from organ failure.
|
Day 28
|
Collaborateurs et enquêteurs
Parrainer
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Estimé)
Achèvement primaire (Estimé)
Achèvement de l'étude (Estimé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- RISE-3750-01AR
Informations sur les médicaments et les dispositifs, documents d'étude
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