Back to results

Autologous Transplantation of Bronchial Basal Cells for Treatment of Bronchiectasis

Primary Purpose

Bronchiectasis

Status

Unknown status

Phase

Phase 1

Locations

Study Type

Interventional

Intervention

Bronchial basal cells

About this trial

This is an interventional treatment trial for Bronchiectasis focused on measuring Bronchiectasis, Bronchial basal cells, Autologous transplantation

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept 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 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 syphilis or any of HIV, HBV, HCV positive antibody.
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 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.

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

BBCs transplantation

Arm Description

Autologous Bronchial basal cells transplantation

Outcomes

Primary Outcome Measures

Diffusing capacity of the lung for carbon monoxide (DLCO)

An indicator of pulmonary function

Secondary Outcome Measures

Forced expiratory volume measured at the first second (FEV1)

An indicator for pulmonary function test to assess airway obstruction

Forced vital capacity (FVC)

An indicator for pulmonary function test to indicate the maximum amount of air a person can expel from the lungs after a maximum inhalation

The ratio of forced expiratory volume in the first one second to the forced vital capacity (FEV1/FVC)

An indicator in pulmonary function test to represent the proportion of a person's vital capacity that they are able to expire in the first second of forced expiration to the full vital capacity

Maximum Mid Expiratory Flow (MMF)

An indicator in pulmonary function test to stand for maximal (mid-)expiratory flow and is the peak of expiratory flow as taken from the flow-volume curve and measured in liters per second

Maximum Voluntary Ventilation (MVV)

An indicator in pulmonary function test to measure the maximum amount of air that can be inhaled and exhaled within one minute

6 minute walk distance (6MWD) and distance saturation product (DSP)

An indicator for heart and pulmonary function

High resolution computed tomography (HR-CT) imaging of lung

An indicator for analysis of dilated bronchi, HR-CT images of lung will be analyzed to indicate the pulmonary structure

St. George's Respiratory Questionnaire (SGRQ)

An indicator to measure health status (quality of life) in patients with diseases of airways obstruction

FACED scoring

An assessment of severity tool, validated for people with non-cystic fibrosis bronchiectasis. F means "FEV1", A means "Age", C means "Chronic colonization", E means "Extension", D means "Dyspnoea".

Bronchiectasis Severity Index (BSI)

A combination of clinical, radiological and microbiological features to predict morbidity and mortality.

Full Information

NCT ID

NCT03655808

First Posted

August 22, 2018

Last Updated

November 4, 2018

Sponsor

Ruijin Hospital

Collaborators

Regend Therapeutics

1. Study Identification

Unique Protocol Identification Number

NCT03655808

Brief Title

Autologous Transplantation of Bronchial Basal Cells for Treatment of Bronchiectasis

Official Title

Autologous Transplantation of Bronchial Basal Cells for Treatment of Bronchiectasis

Study Type

Interventional

2. Study Status

Record Verification Date

November 2018

Overall Recruitment Status

Unknown status

Study Start Date

November 2018 (Anticipated)

Primary Completion Date

September 2020 (Anticipated)

Study Completion Date

March 2021 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Ruijin Hospital

Collaborators

Regend Therapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

5. Study Description

Brief Summary

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 single-centered, non-randomized and self-controlled clinical trial at an early phase. During the process, autologous bronchial basal cells (BBCs) will be dissected from trial tissue from 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 lavage. After six-month observation, the investigators will evaluate the safety and effectiveness of the treatment by measuring a serial of indicators, including occurrence of adverse events, the CT imaging of dilated bronchi, pulmonary function, 6 minute walk distance (6MWD), the distance-saturation product (DSP), St. George's Respiratory Questionnaire (SGRQ), FACED scoring and bronchiectasis severity index (BSI) evaluation.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Bronchiectasis

Keywords

Bronchiectasis, Bronchial basal cells, Autologous transplantation

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Model Description

self-control

Masking

None (Open Label)

Allocation

N/A

Enrollment

20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

BBCs transplantation

Arm Type

Experimental

Arm Description

Autologous Bronchial basal cells transplantation

Intervention Type

Biological

Intervention Name(s)

Bronchial basal cells

Intervention Description

Bronchial basal cells transplantation

Primary Outcome Measure Information:

Title

Diffusing capacity of the lung for carbon monoxide (DLCO)

Description

An indicator of pulmonary function

Time Frame

6 months

Secondary Outcome Measure Information:

Title

Forced expiratory volume measured at the first second (FEV1)

Description

An indicator for pulmonary function test to assess airway obstruction

Time Frame

6 months

Title

Forced vital capacity (FVC)

Description

An indicator for pulmonary function test to indicate the maximum amount of air a person can expel from the lungs after a maximum inhalation

Time Frame

6 months

Title

The ratio of forced expiratory volume in the first one second to the forced vital capacity (FEV1/FVC)

Description

An indicator in pulmonary function test to represent the proportion of a person's vital capacity that they are able to expire in the first second of forced expiration to the full vital capacity

Time Frame

6 months

Title

Maximum Mid Expiratory Flow (MMF)

Description

An indicator in pulmonary function test to stand for maximal (mid-)expiratory flow and is the peak of expiratory flow as taken from the flow-volume curve and measured in liters per second

Time Frame

6 months

Title

Maximum Voluntary Ventilation (MVV)

Description

An indicator in pulmonary function test to measure the maximum amount of air that can be inhaled and exhaled within one minute

Time Frame

6 months

Title

6 minute walk distance (6MWD) and distance saturation product (DSP)

Description

An indicator for heart and pulmonary function

Time Frame

6 months

Title

High resolution computed tomography (HR-CT) imaging of lung

Description

An indicator for analysis of dilated bronchi, HR-CT images of lung will be analyzed to indicate the pulmonary structure

Time Frame

6 months

Title

St. George's Respiratory Questionnaire (SGRQ)

Description

An indicator to measure health status (quality of life) in patients with diseases of airways obstruction

Time Frame

6 months

Title

FACED scoring

Description

An assessment of severity tool, validated for people with non-cystic fibrosis bronchiectasis. F means "FEV1", A means "Age", C means "Chronic colonization", E means "Extension", D means "Dyspnoea".

Time Frame

6 months

Title

Bronchiectasis Severity Index (BSI)

Description

A combination of clinical, radiological and microbiological features to predict morbidity and mortality.

Time Frame

6 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

75 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Subjects diagnosed as bronchiectasis. Subjects with a DLCO < 80% predicted value. Subjects with stable condition for more than 2 weeks. 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 syphilis or any of HIV, HBV, HCV positive antibody. 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 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.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Jieming Qu, M.D., Ph.D

Phone

+86-021-64370045

jmqu0906@163.com

First Name & Middle Initial & Last Name or Official Title & Degree

Wei Zuo, Ph. D

Phone

+86-21-65985082

zuow@regend.cn

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Yun Feng, M.D., Ph.D

Organizational Affiliation

Ruijin Hospital

Official's Role

Principal Investigator

12. IPD Sharing Statement

Plan to Share IPD

Citations:

PubMed Identifier

25383540

Citation

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.

Results Reference

background

PubMed Identifier

29344809

Citation

Ma Q, Ma Y, Dai X, Ren T, Fu Y, Liu W, Han Y, Wu Y, Cheng Y, Zhang T, Zuo W. Regeneration of functional alveoli by adult human SOX9+ airway basal cell transplantation. Protein Cell. 2018 Mar;9(3):267-282. doi: 10.1007/s13238-018-0506-y. Epub 2018 Jan 17.

Results Reference

background

Learn more about this trial

Autologous Transplantation of Bronchial Basal Cells for Treatment of Bronchiectasis

We'll reach out to this number within 24 hrs