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Treatment of Idiopathic Pulmonary Fibrosis(IPF) by REGEND001

Primary Purpose

Idiopathic Pulmonary Fibrosis

Status
Completed
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
REGEND001 Autologous Therapy Product
Sponsored by
Regend Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Idiopathic Pulmonary Fibrosis

Eligibility Criteria

50 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Male or female, aged between 50 to 75; Subjects diagnosed with IPF according to guidelines for the diagnosis of idiopathic pulmonary fibrosis 2018 edition; Subjects with 30%~79% of the predicted value in diffusing capacity for carbon monoxide (DLCO) and more than 50% of the predicted value in forced vital capacity (FVC) in pulmonary function tests 3 months before screening; Subjects with typical High-resolution computed tomography (HR-CT) imaging findings of idiopathic pulmonary fibrosis in the past 12 months; Subjects tolerant to bronchofiberscope; Subjects fully informed of the purpose, method and possible discomfort of the trial, agreeing to participate in the test, and voluntarily signing the informed consent; Subjects with good adherence, willingness to take medication and regular follow-up examinations as required by the protocol ; Subjects able to understand and cooperate with the completion of pulmonary function tests. Exclusion Criteria: Subjects who cannot tolerate cell therapy Pregnant or lactating women; Subjects with syphilis or any of human immunodeficiency virus (HIV), hepatitis B virus (HBV), hepatitis C virus (HCV) positive antibody; Of which stable HBV carriers after drug treatment (DNA titer ≤500 IU/mL or copy number <1000 copies/mL) and cured hepatitis C patients (HCV RNA is negative) can be enrolled; Subjects with malignant tumors or a history of malignant tumors; Subjects with taking drugs which caused lung fibroblast such as amiodarone in a long term before screening; Subjects with infections in lung or other site, including bacterial and viral infections, requiring intravenous treatment before cell transplantation; Subjects with a history of invasive or noninvasive mechanical ventilation within 4 weeks; Subjects with any of the following lung diseases: asthma, active tuberculosis, pulmonary embolism, pneumothorax, pulmonary hypertension, pneumoconiosis, etc.; lung cancer, bronchiolitis obliterans or other active lung disease; Pneumonia currently or within the last 4 weeks; Pneumonectomy Previously; Subjects needing oxygen therapy currently (oxygen therapy time> 15h/d); Subjects suffering from serious other systemic diseases, such as myocardial infarction, unstable angina, liver cirrhosis, acute glomerulonephritis, connective tissue disease, etc.; Subjects with following results : leukopenia (leukopenia < 4×10^9/L) or agranulocytosis (leukocyte < 1.5×10^9/L or neutrophils < 0.5×10^9/L) of any cause; Blood creatinine > 2.5 times the upper limit of normal; Alanine transaminase (ALT) and Aspartate transaminase (AST) > 2.5 times the upper limit of normal values in the laboratory tests. Subjects with a history of mental illness or suicide risk, epilepsy or other central nervous system disorders Subjects with severe arrhythmias (such as ventricular tachycardia, frequent supraventricular tachycardia, atrial fibrillation, atrial flutter, etc.) or atrioventricular block of degree II or above, shown by 12-lead Electrocardiogram (ECG); Subjects with a history of abusing alcohol and illicit drug; Subjects who are allergic to cattle products; Subjects who participated in other clinical trials in the past 3 months; Subjects with poor compliance and difficult to complete the investigation; Investigators, employees of research centers or family members of them (none of whom are suitable to participate in the trial to ensure the objectivity of the research); Subjects who had an acute exacerbation of IPF or hospitalized for other respiratory diseases 3 or more times in the past 1 year; Subjects who take nintedanib for medication within 1 month, or plan to continue taking nintedanib for medication; Subjects with other acquired or congenital immunodeficiency disorders, or with a history of organ transplantation or cell transplant therapy; Subjects whose expected survival may be less than one year judged by the investigator; Male participants of childbearing potential and female participants within childbearing age were reluctant to use effective contraception from the time of signing the informed consent to 6 months after cell therapy; Subjects assessed as inappropriate to participate in this clinical trial by investigator.

Sites / Locations

  • Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
  • The First Affiliated Hospital of Guangzhou Medical University
  • Ruijin Hospital, Shanghai Jiao Tong University School of Medicine

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

REGEND001 autologous bronchial basal cells

Arm Description

Transplantation of autologous bronchial basal cells

Outcomes

Primary Outcome Measures

Incidence and severity of the cell therapy-related adverse events (AEs)
Dose escalation is based on incidence of cell therapy-related AEs. Severity of cell therapy-related adverse events (AEs) is evaluated according to Common Terminology Criteria for Adverse Events (CTCAE).

Secondary Outcome Measures

Incidence of complication related to bronchoscopy
REGEND001 Autologous Therapy Product is given to subjects by bronchoscopy. Thus, incidence of complication related to bronchoscopy is adapted to assess the safety of the products.
Change of lung diffusing capacity for single-breath carbon monoxide (DLCO-sb) from baseline
DLCO-sb is measured by the single-breath method. It is considered a measure of the conductance of CO across the alveolar-capillary membrane and its binding with hemoglobin.
Change of forced vital capacity (FVC) from baseline
FVC indicates the volume of air that can forcibly be blown out after full inspiration.
Change of the ratio of diffusing capacity for carbon monoxide/ the alveolar volume (DLCO/VA) from baseline
The DLCO test refers to the diffusing capacity for carbon monoxide in the lungs. It's a type of pulmonary function test that helps to assess how well gas is exchanged between the lungs and the bloodstream.Since DLCO is affected by the amount of inhaled gas and lung volume, the subject's alveolar ventilation (VA) should be considered when evaluating diffusion function to exclude the effect of lung volume on diffusion volume.
Change of 6-minute-walk test (6MWT) from baseline
The 6MWT is a commonly used test for the objective assessment of functional exercise capacity by testing the distance patients can walk at the fastest speed within 6 minutes.
Change of St. George's respiratory questionnaire (SGRQ) scale from baseline
Quality of life was assessed by St. George's respiratory questionnaire (SGRQ) scale. Total score, ranged from 0 to 100, is the sum of points from all items. A higher value represents a worse outcome.
Change of imaging of lung by high resolution computed tomography (HR-CT)
Images of lung will be analyzed to indicate the newly-derived pulmonary structure.
Idiopathic pulmonary fibrosis (IPF) exacerbation events
Frequency and severity of IPF exacerbation events will be evaluated.
Blood routine
Number of cases of participants with abnormal laboratory test results.
Urine routine
Number of cases of participants with abnormal laboratory test results.
Blood biochemistry
Number of cases of participants with abnormal laboratory test results.
12-lead Electrocardiogram (ECG)
Number of cases of participants with abnormal 12-lead Electrocardiogram (ECG)
Carcinoembryonic antigen (CEA)
CEA is a tumor marker used for early diagnosis of lung cancer.
Neuron-specific enolase (NSE)
NSE is a tumor marker significantly elevated in small cell lung cancer.
Cytokeratin-19-fragment (CYFRA21-1)
CYFRA21-1 is a tumor marker which has important value for the pathological classification and prognosis evaluation of lung cancer.
Squamous cell carcinoma antigen (SCC)
SCC is a specific marker for lung squamous cell carcinoma.Tumor markers are monitored to assess the safety.

Full Information

First Posted
November 15, 2022
Last Updated
October 10, 2023
Sponsor
Regend Therapeutics
Collaborators
Peking Union Medical College Hospital, Ruijin Hospital, The First Affiliated Hospital of Guangzhou Medical University, Regend Therapeutics XLotus (Jiangxi) Co, Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT05657184
Brief Title
Treatment of Idiopathic Pulmonary Fibrosis(IPF) by REGEND001
Official Title
An Open-labelled Clinical Study to Explore the Safety, Tolerability and Preliminary Efficacy of REGEND001 Autologous Therapy Product for Treatment of Idiopathic Pulmonary Fibrosis (IPF).
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Completed
Study Start Date
July 19, 2021 (Actual)
Primary Completion Date
June 9, 2023 (Actual)
Study Completion Date
June 9, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Regend Therapeutics
Collaborators
Peking Union Medical College Hospital, Ruijin Hospital, The First Affiliated Hospital of Guangzhou Medical University, Regend Therapeutics XLotus (Jiangxi) Co, Ltd.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
Idiopathic pulmonary fibrosis (IPF) is a serious chronic (long term) disease with injury of lung tissues. REGEND001 Autologous Therapy Product, made from bronchial basal cells with ability to regenerate lung tissue, is promising to IPF treatment. In this study, a single-armed clinical trial is ongoing to assess the safety and tolerability of REGEND001 Autologous Therapy Product in treatment of IPF. Different doses of REGEND001 Autologous Therapy Product is evaluated to establish a dose-response relationship and to recommend appropriate dose for subsequent clinical trials.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Idiopathic Pulmonary Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Actual)

8. Arms, Groups, and Interventions

Arm Title
REGEND001 autologous bronchial basal cells
Arm Type
Experimental
Arm Description
Transplantation of autologous bronchial basal cells
Intervention Type
Drug
Intervention Name(s)
REGEND001 Autologous Therapy Product
Intervention Description
Transplantation of REGEND001 Autologous Therapy Product for dosage re-escalation.
Primary Outcome Measure Information:
Title
Incidence and severity of the cell therapy-related adverse events (AEs)
Description
Dose escalation is based on incidence of cell therapy-related AEs. Severity of cell therapy-related adverse events (AEs) is evaluated according to Common Terminology Criteria for Adverse Events (CTCAE).
Time Frame
Within 24 weeks after treatment
Secondary Outcome Measure Information:
Title
Incidence of complication related to bronchoscopy
Description
REGEND001 Autologous Therapy Product is given to subjects by bronchoscopy. Thus, incidence of complication related to bronchoscopy is adapted to assess the safety of the products.
Time Frame
Within 24 weeks after treatment
Title
Change of lung diffusing capacity for single-breath carbon monoxide (DLCO-sb) from baseline
Description
DLCO-sb is measured by the single-breath method. It is considered a measure of the conductance of CO across the alveolar-capillary membrane and its binding with hemoglobin.
Time Frame
4, 12, and 24 weeks after treatment
Title
Change of forced vital capacity (FVC) from baseline
Description
FVC indicates the volume of air that can forcibly be blown out after full inspiration.
Time Frame
4 weeks after treatment, 12 weeks after treatment and 24 weeks after treatment
Title
Change of the ratio of diffusing capacity for carbon monoxide/ the alveolar volume (DLCO/VA) from baseline
Description
The DLCO test refers to the diffusing capacity for carbon monoxide in the lungs. It's a type of pulmonary function test that helps to assess how well gas is exchanged between the lungs and the bloodstream.Since DLCO is affected by the amount of inhaled gas and lung volume, the subject's alveolar ventilation (VA) should be considered when evaluating diffusion function to exclude the effect of lung volume on diffusion volume.
Time Frame
4 weeks after treatment, 12 weeks after treatment and 24 weeks after treatment
Title
Change of 6-minute-walk test (6MWT) from baseline
Description
The 6MWT is a commonly used test for the objective assessment of functional exercise capacity by testing the distance patients can walk at the fastest speed within 6 minutes.
Time Frame
4 weeks after treatment, 12 weeks after treatment, and 24 weeks after treatment
Title
Change of St. George's respiratory questionnaire (SGRQ) scale from baseline
Description
Quality of life was assessed by St. George's respiratory questionnaire (SGRQ) scale. Total score, ranged from 0 to 100, is the sum of points from all items. A higher value represents a worse outcome.
Time Frame
4 weeks after treatment, 12 weeks after treatment, and 24 weeks after treatment
Title
Change of imaging of lung by high resolution computed tomography (HR-CT)
Description
Images of lung will be analyzed to indicate the newly-derived pulmonary structure.
Time Frame
24 weeks after treatment
Title
Idiopathic pulmonary fibrosis (IPF) exacerbation events
Description
Frequency and severity of IPF exacerbation events will be evaluated.
Time Frame
Within 24 weeks after treatment
Title
Blood routine
Description
Number of cases of participants with abnormal laboratory test results.
Time Frame
Within 24 weeks after treatment.
Title
Urine routine
Description
Number of cases of participants with abnormal laboratory test results.
Time Frame
Within 24 weeks after treatment.
Title
Blood biochemistry
Description
Number of cases of participants with abnormal laboratory test results.
Time Frame
Within 24 weeks after treatment
Title
12-lead Electrocardiogram (ECG)
Description
Number of cases of participants with abnormal 12-lead Electrocardiogram (ECG)
Time Frame
Within 24 weeks after treatment
Title
Carcinoembryonic antigen (CEA)
Description
CEA is a tumor marker used for early diagnosis of lung cancer.
Time Frame
Baseline, 12 weeks after treatment, 24 weeks after treatment
Title
Neuron-specific enolase (NSE)
Description
NSE is a tumor marker significantly elevated in small cell lung cancer.
Time Frame
Baseline, 12 weeks after treatment, 24 weeks after treatment
Title
Cytokeratin-19-fragment (CYFRA21-1)
Description
CYFRA21-1 is a tumor marker which has important value for the pathological classification and prognosis evaluation of lung cancer.
Time Frame
Baseline, 12 weeks after treatment, 24 weeks after treatment
Title
Squamous cell carcinoma antigen (SCC)
Description
SCC is a specific marker for lung squamous cell carcinoma.Tumor markers are monitored to assess the safety.
Time Frame
Baseline, 12 weeks after treatment, 24 weeks after treatment

10. Eligibility

Sex
All
Minimum Age & Unit of Time
50 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or female, aged between 50 to 75; Subjects diagnosed with IPF according to guidelines for the diagnosis of idiopathic pulmonary fibrosis 2018 edition; Subjects with 30%~79% of the predicted value in diffusing capacity for carbon monoxide (DLCO) and more than 50% of the predicted value in forced vital capacity (FVC) in pulmonary function tests 3 months before screening; Subjects with typical High-resolution computed tomography (HR-CT) imaging findings of idiopathic pulmonary fibrosis in the past 12 months; Subjects tolerant to bronchofiberscope; Subjects fully informed of the purpose, method and possible discomfort of the trial, agreeing to participate in the test, and voluntarily signing the informed consent; Subjects with good adherence, willingness to take medication and regular follow-up examinations as required by the protocol ; Subjects able to understand and cooperate with the completion of pulmonary function tests. Exclusion Criteria: Subjects who cannot tolerate cell therapy Pregnant or lactating women; Subjects with syphilis or any of human immunodeficiency virus (HIV), hepatitis B virus (HBV), hepatitis C virus (HCV) positive antibody; Of which stable HBV carriers after drug treatment (DNA titer ≤500 IU/mL or copy number <1000 copies/mL) and cured hepatitis C patients (HCV RNA is negative) can be enrolled; Subjects with malignant tumors or a history of malignant tumors; Subjects with taking drugs which caused lung fibroblast such as amiodarone in a long term before screening; Subjects with infections in lung or other site, including bacterial and viral infections, requiring intravenous treatment before cell transplantation; Subjects with a history of invasive or noninvasive mechanical ventilation within 4 weeks; Subjects with any of the following lung diseases: asthma, active tuberculosis, pulmonary embolism, pneumothorax, pulmonary hypertension, pneumoconiosis, etc.; lung cancer, bronchiolitis obliterans or other active lung disease; Pneumonia currently or within the last 4 weeks; Pneumonectomy Previously; Subjects needing oxygen therapy currently (oxygen therapy time> 15h/d); Subjects suffering from serious other systemic diseases, such as myocardial infarction, unstable angina, liver cirrhosis, acute glomerulonephritis, connective tissue disease, etc.; Subjects with following results : leukopenia (leukopenia < 4×10^9/L) or agranulocytosis (leukocyte < 1.5×10^9/L or neutrophils < 0.5×10^9/L) of any cause; Blood creatinine > 2.5 times the upper limit of normal; Alanine transaminase (ALT) and Aspartate transaminase (AST) > 2.5 times the upper limit of normal values in the laboratory tests. Subjects with a history of mental illness or suicide risk, epilepsy or other central nervous system disorders Subjects with severe arrhythmias (such as ventricular tachycardia, frequent supraventricular tachycardia, atrial fibrillation, atrial flutter, etc.) or atrioventricular block of degree II or above, shown by 12-lead Electrocardiogram (ECG); Subjects with a history of abusing alcohol and illicit drug; Subjects who are allergic to cattle products; Subjects who participated in other clinical trials in the past 3 months; Subjects with poor compliance and difficult to complete the investigation; Investigators, employees of research centers or family members of them (none of whom are suitable to participate in the trial to ensure the objectivity of the research); Subjects who had an acute exacerbation of IPF or hospitalized for other respiratory diseases 3 or more times in the past 1 year; Subjects who take nintedanib for medication within 1 month, or plan to continue taking nintedanib for medication; Subjects with other acquired or congenital immunodeficiency disorders, or with a history of organ transplantation or cell transplant therapy; Subjects whose expected survival may be less than one year judged by the investigator; Male participants of childbearing potential and female participants within childbearing age were reluctant to use effective contraception from the time of signing the informed consent to 6 months after cell therapy; Subjects assessed as inappropriate to participate in this clinical trial by investigator.
Facility Information:
Facility Name
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
City
Beijing
State/Province
Beijing
Country
China
Facility Name
The First Affiliated Hospital of Guangzhou Medical University
City
Guangzhou
State/Province
Guangdong
Country
China
Facility Name
Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
City
Shanghai
State/Province
Shanghai
Country
China

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Treatment of Idiopathic Pulmonary Fibrosis(IPF) by REGEND001

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