Clinical Trial of Lyophilized Powder for Inhalation of Recombinant Human Keratinocyte Growth Factor-2 (Rh-KGF-2) in The Treatment of Patients With Acute Respiratory Distress Syndrome

A Single Dose, Dose Escalation Clinical Trial on the Safety, Tolerability and Efficacy of Lyophilized Powder for Inhalation of Recombinant Human Keratinocyte Growth Factor-2 (Rh-KGF-2) in The Treatment of Patients With Acute Respiratory Distress Syndrome

This study is a randomized, blank-controlled, open-label, single-dose, dose-escalation clinical study of rhKGF-2 in patients with ARDS.

The trial is designed with three dose groups (5 mg, 10 mg, and 15 mg), which will be escalated sequentially from the lowest dose group to the highest dose group. Each dose group will enroll 8 subjects, randomized in a 6:2 ratio according to the order of enrollment, to receive either the corresponding dose of rhKGF-2 (6 subjects) or serve as a blank control (2 subjects). Each subject will receive a single dose, administered once via a disposable bronchoscopic catheter.

All subjects will receive the trial intervention on top of standard ARDS treatment (see Concomitant Medications for details). Following the completion of drug administration, subjects will enter a 28-day follow-up period. Outcome measures include adverse events (AE), vital signs, laboratory parameters, oxygenation index (PFR), chest imaging changes, etc., to evaluate the safety, tolerability, and efficacy of the treatment.

Study Overview

• Status: Not yet recruiting
• Conditions: ARDS (Acute Respiratory Distress Syndrome); ARDS (Moderate or Severe)
• Intervention / Treatment: Drug: Recombinant Human Keratinocyte Growth Factor-2 for Inhalation (Lyophilized Powder)

• Study Type: Interventional
• Enrollment (Estimated): 24
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Yi Wei; Phone Number: +86 18872984075; Email: yiwei4075@163.com

Study Locations

• China
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China, 200000
      • ShanghaiZhongshan
      • Contact: Yi Wei; Phone Number: +86 18872984075; Email: yiwei4075@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Aged ≥18 years and <80 years, male or female.
2. Definite diagnosis of acute respiratory distress syndrome (ARDS) according to the Berlin Definition.
3. Patients with PaO₂/FiO₂ < 200 mmHg and receiving invasive mechanical ventilation via endotracheal intubation.
4. Diagnosis of ARDS confirmed no more than 72 hours prior to enrollment.
5. No plan for parenthood within 1 year and agree to take effective contraceptive measures during the study period. Female participants of childbearing potential must have a negative serum pregnancy test.

6. The subject fully understands the purpose of the study, as well as the nature, methods, and potential reactions of the investigational drug. The subject voluntarily signs the informed consent form to participate in the study and agrees to comply with the requirements of the study protocol. If the subject is unable to provide consent or has limited capacity to consent, consent must be obtained from the subject's legal guardian.

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Exclusion Criteria:

1. Use of inhaled pulmonary vasodilators (e.g., nitric oxide or prostaglandins).
2. Current receipt or planned receipt of extracorporeal membrane oxygenation (ECMO) during the study period.
3. Expected survival < 3 months due to causes other than respiratory failure.
4. Cerebrovascular or cardiovascular events within 3 months prior to study drug administration, including unstable angina, congestive heart failure, myocardial infarction within the past 12 months, hemodynamic instability, known left ventricular ejection fraction (LVEF) < 40%, or clinically significant arrhythmia or conduction abnormality.
5. Inability to tolerate single-use bronchoscopic imaging catheter examination, including but not limited to the following: active massive hemoptysis; severe hypertension and arrhythmia; myocardial infarction or unstable angina within 4-6 weeks prior to screening; severe cardiac dysfunction; uncorrectable bleeding tendency (platelet count < 60 × 10⁹/L), such as severe coagulation disorders, uremia, or severe pulmonary hypertension; severe superior vena cava syndrome; suspected aortic aneurysm; multiple pulmonary bullae.
6. History of severe allergic reaction, or known allergy or hypersensitivity to any component of the investigational product.
7. Breastfeeding or pregnant women.
8. Participation in any drug clinical trial within 3 months prior to enrollment.
9. Any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in this study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

6

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 5 mg rhKGF-2	Drug: Recombinant Human Keratinocyte Growth Factor-2 for Inhalation (Lyophilized Powder); The investigational drug will be reconstituted with water for injection to a concentration of 1 mg/mL, and then administered via a single-use bronchoscopic imaging catheter.
No Intervention: Blank Control for 5 mg Cohort
Experimental: 10 mg rhKGF-2	Drug: Recombinant Human Keratinocyte Growth Factor-2 for Inhalation (Lyophilized Powder); The investigational drug will be reconstituted with water for injection to a concentration of 1 mg/mL, and then administered via a single-use bronchoscopic imaging catheter.
No Intervention: Blank Control for 10 mg Cohort
Experimental: 15 mg rhKGF-2	Drug: Recombinant Human Keratinocyte Growth Factor-2 for Inhalation (Lyophilized Powder); The investigational drug will be reconstituted with water for injection to a concentration of 1 mg/mL, and then administered via a single-use bronchoscopic imaging catheter.
No Intervention: Blank Control for 15 mg Cohort

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of adverse events (AE) and incidence of adverse drug reactions (ADR);		At Baseline, Day 1, Day 2, Day 3, Day 5, Day 7, Day 14 and Day 28.
Incidence of serious adverse events (SAE)		At Baseline, Day 1, Day 2, Day 3, Day 5, Day 7, Day 14 and Day 28.
Various laboratory test parameters：White blood cell (WBC) count	Unit of measure: 3.5 - 9.5 × 10^9/L	At Baseline, Day 1, Day 2, Day 3, Day 5, Day 7, Day 14 and Day 28.
Various Laboratory test parameters: Serum creatinine (Cr) level	Unit of measure: 44 -115 μmol/L	At Baseline, Day 1, Day 2, Day 3, Day 5, Day 7, Day 14 and Day 28.
Various laboratory test parameters: Total Bilirubin	Unit of measure: 3.4-20.4 umol /L	At Baseline, Day 1, Day 2, Day 3, Day 5, Day 7, Day 14 and Day 28.
Incidence of airway spasm (increased airway resistance) caused by local drug stimulation		At Baseline, Day 1, Day 2, Day 3, Day 5, Day 7, Day 14 and Day 28.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Improvement rate of oxygenation indexPaO₂/FiO₂ ratio (PFR)		at Day 3 and Day 7
PaO₂/FiO₂ ratio (PFR) and its change from baseline		at Day 3, Day 5, and Day 7
Number of days from baseline to first PaO₂/FiO₂ ratio (PFR) > 300 mmHg or liberation from mechanical ventilation for at least 12 consecutive hours		From baseline to first PFR > 300 mmHg or liberation from mechanical ventilation for at least 12 consecutive hours
Change from baseline in the Murray Lung Injury Score(LIS).	Score (scale range: 0-4; higher scores indicate more severe lung injury)	at Day 7
Change from baseline in Radiographic Assessment of Lung Edema (RALE) score;	Score (scale range: 0-48; higher scores indicate a worse outcome);	at Day 3, Day 7, Day 14, and Day 28
Proportion of patients with improvement in ARDS severity grade		at Day 7 and Day 14
Proportion of patients with progression in ARDS severity grade		at Day 7 and Day 14
All-cause mortality		at Day 28
Systemic evaluation measures: Ventilator-free days at Day 28	Ventilator-free days at Day 28 (unit: days; higher scores indicate a better outcome).	at Day 28
Systemic evaluation measures: Non-ICU hospital stay days at Day 28	Non-ICU hospital stay days at Day 28 (unit: days; higher scores indicate a better outcome as applicable).	at Day 28.
Systemic evaluation measures: Non-hospitalization days at Day 28	Non-hospitalization days at Day 28 (unit: days; higher scores indicate a better outcome).	at Day 28

Collaborators and Investigators

• Sponsor: Shanghai Zhongshan Hospital
• Investigators: Study Director: Yuanlin Song, Shanghai Zhongshan Hospital; Principal Investigator: Jing Bi, Shanghai Zhongshan Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): April 10, 2026
• Primary Completion (Estimated): November 30, 2026
• Study Completion (Estimated): December 30, 2026

Study Registration Dates

• First Submitted: April 12, 2026
• First Submitted That Met QC Criteria: April 26, 2026
• First Posted (Actual): April 30, 2026

Study Record Updates

• Last Update Posted (Actual): April 30, 2026
• Last Update Submitted That Met QC Criteria: April 26, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: ARDS (Acute Respiratory Distress Syndrome); rh-KGF2
• Additional Relevant MeSH Terms: Neoplasms; Immune System Diseases; Respiratory Tract Diseases; Neoplasms by Histologic Type; Lung Diseases; Lymphatic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Lymphoma, Non-Hodgkin; Lymphoma; Lung Injury; Hemic and Lymphatic Diseases; Lymphoma, Follicular; Acute Lung Injury; Circulatory and Respiratory Physiological Phenomena; Respiratory Mechanics; Respiration; Respiratory Physiological Phenomena; Inhalation
• Other Study ID Numbers: B2026-113R

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: All IPD that underlie results in a publication.
• IPD Sharing Time Frame: Sharing will begin within 6 months after article publication.
• IPD Sharing Access Criteria: IPD and supporting information will be avaible to researchers upon reasonable request (e.g. with a practical and meaningful research proposal).
• IPD Sharing Supporting Information Type: ICF

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No