Autologous Transplantation of Bronchial Basal Cells for Treatment of Bronchiectasis

Shanghai Jiao Tong University

Status and phase: Completed; Phase 2; Phase 1

Conditions: Bronchiectasis

Treatments: Biological: Bronchial basal cells (BBCs); Procedure: Bronchoscopic airway clearance treatment （B-ACT）

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT03655808

201810

Details and patient eligibility

About

Bronchiectasis is a disease resulted from progressive destruction of bronchi with no effective drug for its treatment. In this study, we intends to carry out a randomized, single-blinded, controlled pilot clinical trial at 1/2 phase. During the process, autologous bronchial basal cells (BBCs) will be dissected from trial tissue via bronchoscopic brushing. Then the BBCs will be expanded and detected by quality control. In the following, qualified BBCs will be injected directly into the lesion by fiberoptic bronchoscopy after airway clearance. The investigators will evaluate the safety and effectiveness of the treatment by measuring a serial of indicators, including occurrence of adverse events, pulmonary function, 6 minute walk distance (6MWD), the distance-saturation product (DSP), St. George's Respiratory Questionnaire (SGRQ), FACED scoring, bronchiectasis severity index (BSI) , and high-resolution computed tomography (HRCT) at week 4, week 12, and week 24 after cell transplantation.

Enrollment: 37 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Subjects diagnosed as bronchiectasis.
• Subjects with a DLCO < 80% predicted value.
• Subjects with stable condition for more than 2 weeks.
• Subjects can do pulmonary function tests.
• Subjects can tolerate bronchoscopy.
• Subjects signed informed consent.

Exclusion criteria

• Women of childbearing age at the stage of pregnancy or lactation, or those without taking effective contraceptive measures.
• Subjects with positive serological tests for hepatitis B virus (HBV), hepatitis C virus (HCV), human immunodeficiency virus (HIV) or syphilis (HBV carriers and patients with stable chronic hepatitis B could be accepted if titers of HBV DNA < 500 IU/mL or copies < 1000 copies/mL; patients with curative hepatitis C were eligible if HCV RNA tests were negative).
• Subjects with any malignancy.
• Subjects with any of the following pulmonary diseases: active tuberculosis, pulmonary embolism, pneumothorax, multiple huge bullae, uncontrolled asthma, acute exacerbation of chronic bronchitis or extremely severe COPD.
• Subjects with other serious diseases, such as poorly controlled diabetes, myocardial infarction, unstable angina, cirrhosis, and acute glomerulonephritis.
• Subjects with leukopenia (WBC less than 4x10^9 / L) or agranulocytosis (WBC less than 1.5x10^9 / L or neutrophils less than 0.5x10^9 / L) caused by any reason.
• Subjects with severe renal impairment, serum creatinine> 1.5 times of the upper limit of normal.
• Subjects with liver disease or liver damage: ALT, AST, total bilirubin> 2 times of the upper limit of normal.
• Subjects with a history of mental illness or suicide risk, with a history of epilepsy or other central nervous system disorders.
• Subjects with severe arrhythmias (such as ventricular tachycardia, frequent superventricular tachycardia, atrial fibrillation, and atrial flutter, etc.) or cardiac degree II or above conduction abnormalities displayed via 12-lead ECG.
• Subjects with a history of alcohol or illicit drug abuse.
• Subjects accepted by any other clinical trials within 3 months before the enrollment.
• Subjects with poor compliance, difficult to complete the study.
• Any other conditions that might increase the risk of subjects or interfere with the clinical trial.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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37 participants in 2 patient groups

Cell treatment group

Experimental group

Description:

Patients receive both autologous BBCs transplantation and B-ACT therapy.

Treatment:

Procedure: Bronchoscopic airway clearance treatment （B-ACT）

Biological: Bronchial basal cells (BBCs)

Control group

Sham Comparator group

Description:

Patients only receive B-ACT therapy.

Treatment:

Procedure: Bronchoscopic airway clearance treatment （B-ACT）