A Clinical Study to Evaluate the Efficacy and Safety of REGEND001 Cell Therapy on Idiopathic Pulmonary Fibrosis (IPF)
Study Details
Study Description
Brief Summary
Idiopathic pulmonary fibrosis (IPF) is a serious chronic (long term) disease with injury of lung tissues. REGEND001 is a cell therapy product, made from bronchial basal cells with ability to regenerate lung tissue, is promising to IPF treatment. This is a multi-center, randomized, double-blinded, parallel and placebo-controlled phase II clinical study to evaluate the efficacy and safety of REGEND001 in IPF patients.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: REGEND001
|
Biological: REGEND001
REGEND001: 1-1.5×10^6 bronchial basal cells/kg administrated by bronchoscopy.
|
| Placebo Comparator: Placebo
|
Biological: Placebo
Placebo: Sodium chloride injection administrated by bronchoscopy.
|
Outcome Measures
Primary Outcome Measures
- The ratio of subjects with improvement of lung carbon oxide diffusion function (DLCO) [12 and 24 weeks after treatment]
DLCO is measured by the single-breath method. Improvement is defined as an increase in DLCO from baseline.
Secondary Outcome Measures
- Change from baseline in lung diffusing capacity [12 and 24 weeks after treatment]
DLCO will be used to evaluate the lung diffusing capacity. 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.
Other Outcome Measures
- Change from baseline in lung ventilatory capacity [12 and 24 weeks after treatment]
Forced vital capacity (FVC) and forced expiratory volume in one second (FEV1) will be used to evaluate the lung ventilatory capacity. FVC indicates the volume of air that can forcibly be blown out after full inspiration. FEV1 is the volume of breath exhaled with effort in one second.
- Progression-free survival (PFS) [Within 24 weeks after treatment]
PFS refers to the time from randomization or initiation of treatment to the occurrence of disease progression or death.
- Change from baseline in St. George's respiratory questionnaire (SGRQ) scale [12 and 24 weeks after treatment]
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 from baseline in 6-minute-walk test (6MWT) [12 and 24 weeks after treatment]
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 from enrollment to all-cause death [up to 24 weeks(first period), 5 years (second period).]
Time from enrollment to all-cause death is used to evaluate the overall survival of the population.
- Blood oxygen saturation [12 and 24 weeks after treatment]
Blood oxygen saturation is the measure of how much oxygen is traveling through body in red blood cells.
- Change from baseline in images of lung by high resolution computed tomography (HR-CT) [12 and 24 weeks after treatment]
HR-CT images of lung will be analyzed to indicate the change of pulmonary structure.
- Change from baseline in C-reactive protein (CRP) [12 and 24 weeks after treatment]
The level of CRP increases when there's inflammation in the body.
- Time to acute exacerbations of idiopathic pulmonary fibrosis (AE-IPF) [Within 24 weeks after treatment]
AE-IPF is an often deadly complication of IPF, which is defined as an acute, clinically significant respiratory deterioration characterized by evidence of new widespread alveolar abnormality.
- Temperature [Within 24 weeks after treatment]
Number of cases with abnormal body temperature.
- Breathing [Within 24 weeks after treatment]
Number of cases with abnormal breathing.
- Pulse [Within 24 weeks after treatment]
Number of cases with abnormal pulse.
- Blood pressure [Within 24 weeks after treatment]
Number of cases with abnormal blood pressure.
- Symptoms, physical examination [Within 24 weeks after treatment]
Number of cases with abnormal physical examination
- 12-lead ECG [Within 24 weeks after treatment]
Number of cases with abnormal 12-lead Electrocardiogram (ECG).
- Blood routine [Within 24 weeks after treatment]
Number of cases with abnormal laboratory test results
- Liver & Kidney function check [Within 24 weeks after treatment]
Number of cases with abnormal results in Liver & Kidney function check
- Blood sugar [Within 24 weeks after treatment]
Number of cases with abnormal results
- Function of blood clotting [Within 24 weeks after treatment]
Number of cases with abnormal function of blood clotting.
- Antibody testing for autoimmune diseases [Within 24 weeks after treatment]
Antibodies related to autoimmune diseases are tested for safety assessment
- Carcinoembryonic antigen (CEA) [Within 24 weeks after treatment]
CEA is a tumor marker used for early diagnosis of lung cancer.Clinically significant changes of this markers will be assessed.
- Neuron-specific enolase (NSE) [Within 24 weeks after treatment]
NSE is a tumor marker significantly elevated in small cell lung cancer. Clinically significant changes of this marker will be assessed
- Cytokeratin-19-fragment (CYFRA21-1) [Within 24 weeks after treatment]
CYFRA21-1 is a tumor marker which is valuable for the pathological classification and prognosis evaluation of lung cancer. Clinically significant changes of this marker will be assessed
- Squamous cell carcinoma antigen (SCC) [Within 24 weeks after treatment]
SCC is a specific marker for lung squamous cell carcinoma. Clinically significant changes of this marker will be assessed
- Other cases of adverse effects [Within 24 weeks after treatment]
Other cases of adverse effects will be recorded and compared.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Male or female, aged between 40 to 75;
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Subjects diagnosed with IPF according to guidelines for the diagnosis of idiopathic pulmonary fibrosis 2022 edition;
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Subjects tolerant to bronchofiberscope;
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Subjects tolerant to test of lung function;
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Subjects able to voluntarily sign the informed consent and cooperate with the completion of pulmonary function tests;
Exclusion Criteria:
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Female subject who is pregnant, nursing, or planning to be pregnant in half a year after using this product (or male subjects planning to have a pregnant spouse);
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At the time of screening, subject who is positive in each of treponema pallidum antibody (TP-Ab), human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody test, except the following: (1) Hepatitis B virus carriers (only HBsAg positive, no hepatitis symptoms and signs, all liver function tests are normal); (2) Cured hepatitis C patients with negative result in HCV ribonucleic acid (RNA) test.
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Subject with malignant tumors or a history of malignant tumors;
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Subject with severe anemia, poorly controlled agranulocytosis and thrombocytopenia at screening;
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Subject at risk of suicide or has a history of mental illness or epilepsy at the time of screening;
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Subject with severe arrhythmias or atrioventricular block of degree II or above, shown by 12-lead Electrocardiogram (ECG);
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Subject who participated in other interventional clinical trials in the past 3 months;
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Subject assessed as inappropriate to participate in this clinical trial by investigators.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Peking Union Medical College Hospital, Chinese Academy of Medical Sciences | Beijing | Beijing | China | |
| 2 | Ruijin Hospital, Shanghai Jiaotong University School Of Medicine | Shanghai | Shanghai | China | 200025 |
| 3 | Renji Hospital, Shanghai Jiaotong University School Of Medicine | Shanghai | Shanghai | China | 200127 |
Sponsors and Collaborators
- Regend Therapeutics
- Peking Union Medical College Hospital
- RenJi Hospital
- Ruijin Hospital
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- REGEND001-IPF-231-V1.1