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BronchiectasisPhase 1/2NCT03655808

Autologous Transplantation of Bronchial Basal Cells for Treatment of Bronchiectasis

A Phase 1/2 study testing Bronchial basal cells (BBCs) and Bronchoscopic airway clearance treatment （B-ACT） in people with Bronchiectasis, run by Ruijin Hospital at 1 site in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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 randomized, single-blinded, controlled pilot clinical trial at 1/2 phase. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Bronchiectasis
What is being tested?
Bronchial basal cells (BBCs) and Bronchoscopic airway clearance treatment （B-ACT）
Type of treatment
Surgery or procedure
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
37 participants
Where
1 siteChina
Study dates
21 Jun 2020to 17 May 2023
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.
Show all 14Show fewer
  • 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 locations and contacts

1 site in 1 country.

China1 site

  • Ruijin Hospital Shanghai JiaoTong University School of Medicine

    Shanghai, Shanghai Municipality, 200025

Other trials in China

About this study

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.

Conditions
Bronchiectasis
Being tested
Bronchial basal cells (BBCs), Bronchoscopic airway clearance treatment （B-ACT）
Keywords
Bronchiectasis, Bronchial basal cells, Autologous transplantation
Medical subject headings
Bronchiectasis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Bronchial basal cells (BBCs)Biological

    Also called P63+ lung stem/progenitor cells, Airway basal cells, Lung stem/progenitor cells

    Airway epithelial cells were collected in patients of the cell treatment group by a disposable 2-mm brush in order to obtain BBCs. The obtained tissue were washed and enzymatically digested to form a single-cell suspension, which was then cultured under R-Clone system, a patented technique of Regend Therapeutics, Ltd.. For patients in the cell treatment group, on the basis of B-ACT therapy, they were also received autologous BBCs transplantation. Cell suspension was diluted with normal saline. Fibrotic bronchoscopy was guided into the lobular or segmental airways and cells were instilled into lobes.

  • Bronchoscopic airway clearance treatment （B-ACT）Procedure or surgery

    The bronchoscopy was performed by board-certified respiratory physicians of Ruijin Hospital using a flexible fiber-optic bronchoscope. B-ACT therapy was performed on all patients in both groups according to the protocol. In brief, continuous suction was performed with the sputum aspirator from the trachea to the subsegmental during the entering of bronchoscope to remove the visible secretions from entire respiratory tract, and then operators used normal saline to collect lavage fluid (the volume various depending on the operator's judgement).

Study groups in NCT03655808
Study groupRoleWhat they receive
Cell treatment groupExperimentalPatients receive both autologous BBCs transplantation and B-ACT therapy.
Receives: Biological: Bronchial basal cells (BBCs); Procedure: Bronchoscopic airway clearance treatment （B-ACT）
Control groupSham comparatorPatients only receive B-ACT therapy.
Receives: Procedure: Bronchoscopic airway clearance treatment （B-ACT）

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Diffusing capacity of the lung for carbon monoxide (DLCO)

    An indicator of pulmonary function

    Measured: Week 4, 12, and 24 after treatment

Secondary outcomes (11)

  1. Forced expiratory volume measured at the first second (FEV1)

    An indicator for pulmonary function test to assess airway obstruction

    Measured: Week 4, 12, and 24 after treatment

  2. 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

    Measured: Week 4, 12, and 24 after treatment

  3. 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

    Measured: Week 4, 12, and 24 after treatment

  4. Maximum Mid Expiratory Flow (MMEF)

    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

    Measured: Week 4, 12, and 24 after treatment

  5. 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

    Measured: Week 4, 12, and 24 after treatment

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

    An indicator for heart and pulmonary function

    Measured: Week 4, 12, and 24 after treatment

  7. St. George's Respiratory Questionnaire (SGRQ)

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

    Measured: Week 4, 12, and 24 after treatment

  8. 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".

    Measured: Week 4, 12, and 24 after treatment

  9. Bronchiectasis Severity Index (BSI)

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

    Measured: Week 4, 12, and 24 after treatment

  10. 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

    Measured: Week 4, 12, and 24 after treatment

  11. Safety outcomes: adverse events

    Any unexpected and unfavorable medical occurrence related to any medical intervention in the study

    Measured: Through study completion, an average of 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Aug 2018
  2. Study start21 Jun 2020
  3. Primary completion17 May 2023
  4. Study completion17 May 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Eligibility patients were randomly assigned in a 1:1 ratio to receive either autologous cell transplantation therapy (cell treatment group) or bronchoscopic airway clearance treatment (B-ACT therapy, control group).
Masking
Triple blind
Who is masked
Participant, Care provider, Outcomes assessor
Enrolment
37 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03655808
Sponsor's protocol ID
201810
Status last verified
Nov 2023
First submitted
22 Aug 2018
Last updated
5 Dec 2023
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • 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.

    PubMed 25383540

  • 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.

    PubMed 29344809

  • Yan J, Zhang W, Feng Y, Liu X, Niu L, Guo Y, Zhou L, Shi M, Di C, Zhang Q, Wang X, Zhou J, Dai R, Ni L, Bao Z, Yan T, Hu Y, Wang P, Zhang T, Zhou M, Zuo W, Qu J. Autologous transplantation of P63+ lung progenitor cells in patients with bronchiectasis: A randomized, single-blind, controlled trial. Cell Rep Med. 2024 Nov 19;5(11):101819. doi: 10.1016/j.xcrm.2024.101819.

    PubMed 39566467

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Bronchial basal cells (BBCs) and Bronchoscopic airway clearance treatment （B-ACT） in people with Bronchiectasis, run by Ruijin Hospital at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Diffusing capacity of the lung for carbon monoxide (DLCO) (time frame: Week 4, 12, and 24 after treatment). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03655808, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Dec 2023.

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