Safety and Efficacy Study of GKL-006RTU in Moderate to Severe Acute Respiratory Distress Syndrome (ARDS)

NCT07706855 | Not Yet Recruiting Phase 1

• 1 other identifier: GKL006RTUST01
• Study Type: interventional
• Target: 52
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a Phase I/II, multicenter study designed to evaluate the safety, tolerability, and preliminary efficacy of GKL-006RTU injection in participants with moderate to severe Acute Respiratory Distress Syndrome (ARDS). The study consists of two parts: Phase I is a single-arm, open-label study. Eligible participants will receive a single intravenous infusion of GKL-006RTU injection (1 bag or 3 bags, containing approximately 5.0±0.5×10\^8 invariant natural killer T (iNKT) cells per bag) in addition to standard background treatment. The primary objective of this phase is to assess the safety and tolerability of the investigational product. Phase II is a randomized, double-blind, placebo-controlled study. Based on the results from Phase I, participants will receive GKL-006RTU injection or placebo via intravenous infusion, in addition to standard background treatment. The primary objective of this phase is to evaluate the efficacy of GKL-006RTU injection in treating moderate to severe ARDS.

Conditions

ARDS (Acute Respiratory Distress Syndrome)

Outcome Measures

Primary Outcomes (2)

• Phase I: Incidence and Severity of Adverse Events and Serious Adverse Events

  To evaluate the safety and tolerability of GKL-006RTU injection in participants with moderate-to-severe Acute Respiratory Distress Syndrome (ARDS). Safety will be assessed throughout the study duration, from Day 1 to Day 180.

  Day 1 to Day 180

• Phase II: Difference in Ventilator-Free Days within 28 Days Compared to Placebo

  To evaluate the efficacy of GKL-006RTU injection compared to placebo in participants with moderate-to-severe ARDS. The primary efficacy endpoint is the difference in ventilator-free days (days alive and free from mechanical ventilation) within 28 days post-treatment between the GKL-006RTU injection group and the placebo group.

  Day 1 to Day 28

Secondary Outcomes (17)

• Phase I: All-cause Mortality at Day 1 to Day 28, Day 90, and Day 180

  Day 1 to Day 28, Day 90, and Day 180

• Phase I: Ventilator-Free Days, ICU-Free Days, and Organ Support-Free Days within Day 1 to Day 28

  Day 1 to Day 28

• Phase I: Change in Arterial Partial Pressure of Oxygen/Fraction of Inspired Oxygen Ratio (PaO2/FiO2) from Baseline

  Day 1 to Day 28

• Phase I: Change in Arterial Potential of Hydrogen (pH) from Baseline

  Day 1 to Day 28

• Phase I: Change in Arterial Partial Pressure of Oxygen (PaO2) from Baseline

  Day 1 to Day 28

Other Outcomes (4)

• [Phase I & II] Immunogenicity Assessment: Anti-MHC Antibodies and Donor-Specific Antibodies (DSA)

  Day 1, Day 14, Day 28, and Day 90

• [Phase I & II] Pharmacokinetics (PK): Count of Allogeneic Invariant Natural Killer T (iNKT) Cells

  Day 1 to Day 4, Day 7, Day 14, Day 28, and Day 90

• [Phase I & II] Pharmacodynamics (PD): Inflammatory Cytokines and Acute Phase Reactants

  Day 1, Day 4, Day 7, Day 14, Day 28, and Day 90

Study Arms (3)

Phase I: GKL-006RTU injection (1 or 3 Bags)

EXPERIMENTAL

A single-arm, open-label dose-escalation study. Dose Level 1: 1 bag of GKL-006RTU injection (25 mL/bag, containing 5.0±0.5×10\^8 iNKT cells). Dose Level 2: 3 bags of RTU injection (25 mL/bag, containing 5.0±0.5×10\^8 iNKT cells per bag). Dose escalation follows the '3+3' design based on dose-limiting toxicities (DLTs) observed within a 28-day safety window. All participants will receive standard of care background therapy as per clinical guidelines, including restrictive fluid management and lung-protective ventilation strategies. Supportive care medications (for example, antibiotics, albumin, diuretics) and organ support will be administered at the investigator's discretion based on individual patient needs.

Drug: GKL-006RTU injection

Phase II: GKL-006RTU Injection

EXPERIMENTAL

Participants in this arm will receive the Recommended Phase II Dose of GKL-006RTU injection . This is a randomized, double-blind portion of the study. All participants will receive standard of care background therapy as per clinical guidelines, including restrictive fluid management and lung-protective ventilation strategies. Supportive care medications (for example, antibiotics, albumin, diuretics) and organ support will be administered at the investigator's discretion based on individual patient needs.

Drug: GKL-006RTU injection

Phase II: Placebo

PLACEBO COMPARATOR

Participants in this arm will receive matching placebo. They will be randomized in a 1:1 ratio to the GKL-006RTU injection arm. The study is double-blind. All participants will receive standard of care background therapy as per clinical guidelines, including restrictive fluid management and lung-protective ventilation strategies. Supportive care medications (for example, antibiotics, albumin, diuretics) and organ support will be administered at the investigator's discretion based on individual patient needs.

Drug: Placebo

Interventions

GKL-006RTU injection DRUG

iNKT cell injection

Phase I: GKL-006RTU injection (1 or 3 Bags); Phase II: GKL-006RTU Injection

Placebo DRUG

Matching placebo for GKL-006RTU injection

Phase II: Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female, aged 18 to 80 years (inclusive).
• Participants (or their legally authorized representatives, if the participant is unable to provide informed consent) who fully understand the nature of the study, voluntarily sign the informed consent form, and are willing to comply with and complete all study procedures during the study period.
• Clinically diagnosed with Acute Respiratory Distress Syndrome (ARDS) according to the diagnostic criteria, with a time from diagnosis to enrollment not exceeding 96 hours.
• The etiology of ARDS is confirmed to be infectious.
• Received active treatment (including anti-infective therapy and lung-protective ventilation strategies) for at least 24 hours; and arterial blood gas analysis results within 6 hours prior to enrollment support the diagnosis of moderate-to-severe ARDS (defined as Arterial Partial Pressure of Oxygen/Fraction of Inspired Oxygen Ratio (PaO2/FiO2) ≤ 200 mmHg and Positive End-Expiratory Pressure \[PEEP\] ≥ 5 cmH2O).
• Participants and their partners must have no plans for reproduction, sperm donation, or egg donation for at least 6 months after the last dose of study drug, and must voluntarily adopt effective contraceptive measures deemed appropriate by the investigator.

You may not qualify if:

• Positive screening for infectious diseases meeting any of the following: a)Active Hepatitis B virus infection (defined as \[positive Hepatitis B Surface Antigen (HBsAg) or positive Hepatitis B Core Antibody (HBcAb)\] with Hepatitis B Virus DNA \[HBV-DNA\] above the upper limit of normal); b) Hepatitis C virus (HCV) infection (defined as positive HCV antibody with HCV-RNA above the upper limit of normal); c) Human Immunodeficiency Virus (HIV) infection (defined as positive HIV antibody); d) Syphilis infection (defined as positive Treponema pallidum antibody).
• Known immune system dysfunction (e.g., primary immunodeficiency, acquired immunodeficiency) or currently receiving systemic immunosuppressive therapy.
• Expected survival time of less than 72 hours.
• Occurrence of a cerebrovascular or cardiovascular event (unstable angina, congestive heart failure, myocardial infarction, or stroke) within the past 6 months; or severe cardiovascular disease at screening: New York Heart Association (NYHA) Functional Classification Class III or higher; uncontrolled myocarditis or valvular disease; hemodynamic instability, severe cardiac dysfunction, malignant arrhythmias, or severe rhythm/conduction abnormalities requiring drug therapy within the past 6 months.
• Currently receiving or expected to receive Extracorporeal Membrane Oxygenation (ECMO) during the trial period.
• Severe hepatic or renal dysfunction at screening: long-term hemodialysis and known severe renal impairment with an estimated Glomerular Filtration Rate (eGFR) \< 30 mL/min/1.73 m² (calculation method detailed in Appendix 6), currently requiring Continuous Renal Replacement Therapy (CRRT); or moderate to severe liver failure (Child-Pugh score \> 12 ).
• Severe hematological abnormalities at screening: evidence of bleeding with an International Normalized Ratio (INR) ≥ 2.0; severe anemia (Hemoglobin \[Hb\] \< 60 g/L); moderate or greater thrombocytopenia (Platelets \[PLT\] \< 50×10⁹/L); Disseminated Intravascular Coagulation (DIC); leukemia; or other hematological abnormalities deemed unsuitable for enrollment.
• Severe end-stage respiratory diseases at screening (e.g., COPD with respiratory failure, pulmonary fibrosis, pulmonary hypertension with right heart failure, etc., excluding ARDS).
• History of deep vein thrombosis or pulmonary embolism within 6 months prior to enrollment.
• History of solid organ or hematopoietic stem cell transplantation.
• Severe cardiopulmonary malformations at screening that significantly affect cardiopulmonary function.
• Severe neuropsychiatric disorders (e.g., Alzheimer's disease, schizophrenia).
• ARDS caused by other etiologies (e.g., trauma, aspiration).
• Pregnant or lactating women.
• Cumulative corticosteroid use equivalent to \> 400 mg of prednisone within 3 weeks prior to screening.

Sponsors & Collaborators

• Beijing Gene Key Life Technology Co., Ltd: lead

MeSH Terms

Conditions

Acute Lung Injury

Condition Hierarchy (Ancestors)

Lung Injury; Lung Diseases; Respiratory Tract Diseases

Central Study Contacts

Bin Du, M.D.

CONTACT

+86-10-69151188; dubin98@gmail.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 29, 2026
• First Posted: July 16, 2026
• Study Start: July 10, 2026
• Primary Completion (Estimated): July 30, 2027
• Study Completion (Estimated): July 30, 2027
• Last Updated: July 16, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share