- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg 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.
Studieoversigt
Status
Betingelser
Intervention / Behandling
Detaljeret beskrivelse
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.
Undersøgelsestype
Tilmelding (Anslået)
Fase
- Fase 1
Kontakter og lokationer
Studiekontakt
- Navn: Christian F Freguia, PhD
- Telefonnummer: 215-923-1818
- E-mail: cfreguia@risetherapeutics.com
Undersøgelse Kontakt Backup
- Navn: Janet L Stephens, PhD
- Telefonnummer: 650-417-8556
- E-mail: jstephens@risetherapeutics.com
Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
- Voksen
- Ældre voksen
Tager imod sunde frivillige
Beskrivelse
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.
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: Ikke-randomiseret
- Interventionel model: Sekventiel tildeling
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
|
Eksperimentel: 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.
|
|
Eksperimentel: 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.
|
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
To assess the safety and tolerability of R-3750 administered to participants with late-stage, non-resolving Acute Respiratory Distress Syndrome (ARDS)
Tidsramme: 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.
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
All-cause mortality
Tidsramme: Day 90
|
Proportion of participants who are alive at Day 90.
|
Day 90
|
|
Ventilator-free days
Tidsramme: Day 28
|
Number of days alive and free from invasive mechanical ventilation.
|
Day 28
|
|
Organ failure-free days
Tidsramme: Day 28
|
Number of days alive and free from organ failure.
|
Day 28
|
Samarbejdspartnere og efterforskere
Sponsor
Datoer for undersøgelser
Studer store datoer
Studiestart (Anslået)
Primær færdiggørelse (Anslået)
Studieafslutning (Anslået)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Nøgleord
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- RISE-3750-01AR
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
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