COPD (Chronic Obstructive Pulmonary Disease)
Conditions
Keywords
airway basal stem cells, regenerative medicine, COPD
Brief summary
Chronic obstructive pulmonary disease (COPD) is a common, progressive, and heterogeneous respiratory disorder. It is associated with substantial morbidity and mortality worldwide, leading to progressive decline in lung function, impaired quality of life, and a significant healthcare burden. REGEND001, made from airway basal stem cells with ability to regenerate lung tissue, is promising to COPD treatment. In this study, a multicenter, randomized, blinded, placebo-parallel-controlled trial is performed to verify the efficacy and safety of REGEND001 in treatment of COPD patients with emphysema.
Interventions
BIOLOGICALPlacebo
Participants enrolled will have a bronchoscopy with brushing for cell collection, followed by a single adiministratoin of placebo via bronchoscopy and 1 year of follow-up.
BIOLOGICALREGEND001
Participants enrolled will have a bronchoscopy with brushing for cell collection, followed by a single adiministratoin of REGEND001 via bronchoscopy and 1 year of follow-up.
Sponsors
Regend Therapeutics
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
40 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Male or female, aged 40-75 years； * Diagnosed with COPD; * Post-bronchodilator FEV1/FVC \<0.7 * Emphysematous lesions demonstrated by HRCT; * Hemoglobin-corrected DLCO%predicted ≥20% and \<70%; * Post-bronchodilator FEV1%predicted ≤80%; * COPD Assessment Test (CAT) score ≥3;
Exclusion criteria
* History of ≥2 COPD exacerbations leading to hospitalization within 1 year prior to screening, or a COPD exacerbation leading to hospitalization within 2 months prior to screening; * Current or previous history of malignancy; * An assessed life expectancy of \<1 year; * Respiratory tract infection or systemic infection requiring treatment, or severe localized infection within 4 weeks prior to screening; * History of invasive or non-invasive mechanical ventilation within 4 weeks prior to screening; * Diagnosis of pneumonia within 3 months prior to screening; * Abnormal coagulation function affecting the safety of fiberoptic bronchoscopy procedures at screening; * Subjects requiring long-term maintenance anticoagulant therapy or antiplatelet therapy, who are unable to discontinue such medications for at least 1 week prior to cell collection and cell infusion;
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in the measured Diffusing Capacity of the Lung for Carbon Monoxide (DLCO） | Week 52 after treatment | The change from baseline in measured DLCO is defined as the difference between the absolute measured DLCO value at baseline and that measured at the assessment time point after treatment. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in COPD Assessment Test (CAT) score | Week 12, 24 and 52 after treatment | The change from baseline in CAT score is defined as the difference between the CAT score at baseline and the CAT score measured at the specified post-treatment assessment time point. |
| Proportion of subjects with a ≥2-point improvement or worsening from baseline in CAT score | Week 12, 24 and 52 after treatment | Minimal clinically important difference for CAT is recognized as 2 points. |
| Change from baseline in DLCO | Week 12, 24 and 52 after treatment | DLCO is assessed by absolute measured value, hemoglobin (Hb)-corrected measured/predicted ratios and corresponding area under curve (AUC). |
| Proportion of subjects with a ≥11% relative improvement or worsening from baseline in absolute measured DLCO | Week 12, 24 and 52 after treatment | Minimal clinically important difference (MCID) of DLCO is recognized as 11%. |
| Change from baseline in alveolar volume (VA) | Week 12, 24 and 52 after treatment | VA is assessed by measured values, measured/predicted ratio and corresponding area under curve (AUC). |
| Change from baseline in forced expiratory volume in the first second (FEV1) post-bronchodilator | Week 12, 24 and 52 after treatment | FEV1 is assessed by measured value, measured/predicted ratio and corresponding area under curve (AUC). |
| Change from baseline in 6-minute walk distance (6MWD) | Week 12, 24 and 52 after treatment | 6MWD is assessed by the measured value and corresponding area under curve |
| Proportion of subjects with a ≥30-meter change from baseline in 6MWD | Week 12, 24 and 52 after treatment | Minimal clinically important difference (MCID) of 6MWD is recognized as 30 meters. |
| Change from baseline in St. George's Respiratory Questionnaire for COPD (SGRQ-C) score | Week 12, 24 and 52 after treatment | The St. George's Respiratory Questionnaire for COPD (SGRQ-C) is a validated disease-specific patient-reported outcome measure used to assess the impact of COPD on patients' health status and quality of life. Total score, ranged from 0 to 100, is the sum of points from all items. A higher value represents a worse outcome. |
| Proportion of subjects with a ≥4-point improvement or worsening from baseline in SGRQ-C score | Week 12, 24 and 52 after treatment | Minimal clinically important difference for SGRQ is recognized as 4 points. |
| Annualized rate of COPD exacerbations (AECOPD), adjusted for prior exacerbation history and baseline characteristics | Week 52 after treatment | The annualized rate of COPD exacerbations (AECOPD), adjusted for prior exacerbation history and baseline characteristics, represents the adjusted frequency of acute exacerbations of COPD over the assessment period. |
| Change from baseline in arterial blood gas parameters | Week 52 after treatment | The change from baseline in arterial blood gas parameters reflects changes in gas exchange function and respiratory status over the assessment period. |
| Change from baseline in HRCT images | Week 52 after treatment | HRCT images are quantitatively analyzed using dedicated software to measure emphysema volume (mL) and functional lung volume (mL). |
| Adverse Event | Hour 24, Week 12, 24 and 52 after treatment | An adverse event (AE) is any untoward medical occurrence in a subject who has received a study intervention, regardless of whether or not it is considered related to the intervention. |
Countries
China
Contacts
CONTACTShiyue Li, Professor and Chief Physician
Outcome results
None listed