Clinical Efficacy and Safety of Autologous Lung Stem Cell Transplantation in Patients With Idiopathic Pulmonary Fibrosis

• Register: ClinicalTrials.gov
• Last refreshed on: 11 September 2023
• Main ID: NCT02745184
• Date of registration: 15/04/2016
• Prospective Registration: Yes
• Primary sponsor: Shanghai East Hospital
• Public title: Clinical Efficacy and Safety of Autologous Lung Stem Cell Transplantation in Patients With Idiopathic Pulmonary Fibrosis
• Scientific title: Clinical Efficacy and Safety of Autologous Lung Stem Cell Transplantation in Patients With Idiopathic Pulmonary Fibrosis
• Date of first enrolment: March 30, 2017
• Target sample size: 10
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT02745184
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

China

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Male or female, aged between 50 to 75;

2. Subjects diagnosed with IPF according to guidelines for the diagnosis of idiopathic
pulmonary fibrosis 2018 edition;

3. Subjects with 30%~79% of the predicted value in diffusing capacity for carbon monoxide
(DLCO) in pulmonary function tests 3 months before screening;

4. Subjects with typical High-resolution computed tomography (HR-CT) imaging findings of
idiopathic pulmonary fibrosis in the past 12 months;

5. Subjects tolerant to bronchofiberscope;

6. 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;

7. Subjects with good adherence, willingness to take medication and regular follow-up
examinations as required by the protocol;

8. Subjects able to understand and cooperate with the completion of pulmonary function
tests.

Exclusion Criteria:

1. Subjects who cannot tolerate cell therapy;

2. Pregnant or lactating women;

3. 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;

4. Subjects with malignant tumors or a history of malignant tumors;

5. Subjects with serious significant pulmonary infection and needing anti-infection
treatment;

6. Subjects with taking drugs which caused lung fibroblast such as amiodarone in a long
term before screening;

7. Subjects with infections in lung or other site, including bacterial and viral
infections, requiring intravenous treatment before cell transplantation;

8. Subjects with a history of invasive or noninvasive mechanical ventilation within 4
weeks;

9. 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;

10. Subjects needing oxygen therapy currently (oxygen therapy time> 15h/d);

11. Subjects suffering from serious other systemic diseases, such as myocardial
infarction, unstable angina, liver cirrhosis, acute glomerulonephritis, connective
tissue disease, etc.;

12. 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;

13. Subjects with a history of mental illness or suicide risk, epilepsy or other central
nervous system disorders;

14. 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);

15. Subjects with a history of abusing alcohol and illicit drug;

16. Subjects with serious heart disease [New York Heart Association (NYHA) class III-IV];

17. Subjects who are allergic to cattle products;

18. Subjects who participated in other clinical trials in the past 3 months;

19. Subjects with poor compliance and difficult to complete the investigation;

20. 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);

21. Subjects who had an acute exacerbation of IPF or hospitalized for other respiratory
diseases 3 or more times in the past 1 year;

22. Subjects who take nintedanib for medication within 1 month, or plan to continue taking
nintedanib for medication;

23. Subjects with other acquired or congenital immunodeficiency disorders, or with a
history of organ transplantation or cell transplant therapy;

24. Subjects whose expected survival may be less than one year judged by the investigator;

25. 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;

26. Subjects assessed as inappropriate to participate in this clinical trial by
investigator.



Age minimum: 50 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Idiopathic Pulmonary Fibrosis

Intervention(s)

Biological: Lung stem cells

Primary Outcome(s)

The primary outcome of the study was the incidence and severity of the cell therapy-related AEs [Time Frame: 24 weeks]

Secondary Outcome(s)

Life quality: assessed by St. George respiratory questionnaire (SGRQ) [Time Frame: 24 weeks]

Change from baseline in forced vital capacity (FVC) [Time Frame: 24 weeks]

exercise tolerance test (6MWD) [Time Frame: 24 weeks]

Evaluation of cell therapy efficacy through DLCO-SB test [Time Frame: 24 weeks]

Change from baseline in imaging of lung by high resolution computed tomography (HR-CT) [Time Frame: 24 weeks]

Incidence of complication related to bronchoscopy [Time Frame: 1 week]

Change from baseline in forced expiratory volume in one second (FEV1) [Time Frame: 24 weeks]

IPF exacerbation events [Time Frame: 24 weeks]

Secondary ID(s)

201602101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Regend Therapeutics