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

NCT02745184 | Completed Phase 1

• 1 other identifier: 201602101
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

Idiopathic pulmonary fibrosis (IPF) is a chronic and ultimately fatal disease characterized by a progressive damage of lung structure and decline in lung function.This study intends to carry out an open, single-center, non-randomized, self control phase I clinical trial. During the treatment, lung stem cells will be isolated from patients' own bronchi and expanded in vitro. Cultured cells will be injected directly into the lesion by fiberoptic bronchoscopy after lavage. After 24-week observation, the investigators will evaluate the safety and efficacy of the treatment by measuring the key clinical indicators.

Conditions

Idiopathic Pulmonary Fibrosis

Outcome Measures

Primary Outcomes (1)

• The primary outcome of the study was the incidence and severity of the cell therapy-related AEs

  To evaluate the safety of the drugs

  24 weeks

Secondary Outcomes (8)

• Incidence of complication related to bronchoscopy

  1 week

• Evaluation of cell therapy efficacy through DLCO-SB test

  24 weeks

• exercise tolerance test (6MWD)

  24 weeks

• Life quality: assessed by St. George respiratory questionnaire (SGRQ)

  24 weeks

• Change from baseline in forced vital capacity (FVC)

  24 weeks

Study Arms (1)

lung stem cells

EXPERIMENTAL

Patients will receive 0.5-5x10\^6 (0.5-5 million)/Kg/person cells of clinical grade lung stem cells (LSCs)injected via fiberoptic bronchoscopy after fully lavage of the localized lesions.

Biological: Lung stem cells

Interventions

Lung stem cells BIOLOGICAL

Patients will receive 0.5-5x10\^6 (0.5-5 million) /Kg/person cells of clinical grade lung stem cells (LSCs) injected via fiberoptic bronchoscopy after fully lavage of the localized lesions.

lung stem cells

Eligibility Criteria

• Age: 50 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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) 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.

You may not qualify if:

• 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 serious significant pulmonary infection and needing anti-infection treatment;
• 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;

Sponsors & Collaborators

• Shanghai East Hospital: lead
• Regend Therapeutics: collaborator

Study Sites (1)

Shanghai East Hospital

Shanghai, Shanghai Municipality, 200123, China

Location

Related Publications (1)

• Zuo W, Zhang T, Wu DZ, Guan SP, Liew AA, Yamamoto Y, Wang X, Lim SJ, Vincent M, Lessard M, Crum CP, Xian W, McKeon F. p63(+)Krt5(+) distal airway stem cells are essential for lung regeneration. Nature. 2015 Jan 29;517(7536):616-20. doi: 10.1038/nature13903. Epub 2014 Nov 12.

  PMID: 25383540 BACKGROUND

MeSH Terms

Conditions

Idiopathic Pulmonary Fibrosis

Condition Hierarchy (Ancestors)

Pulmonary Fibrosis; Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: April 15, 2016
• First Posted: April 20, 2016
• Study Start: March 30, 2017
• Primary Completion: August 31, 2020
• Study Completion: August 31, 2020
• Last Updated: August 31, 2023

Record last verified: 2023-08

Locations

CN (1)