Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Safety and Potential Efficacy of Human Mesenchymal Stem Cells in Non‐Cystic Fibrosis Bronchiectasis (CELEB)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02625246
Recruitment Status : Completed
First Posted : December 9, 2015
Last Update Posted : August 27, 2019
Sponsor:
Information provided by (Responsible Party):
Marilyn Glassberg, University of Miami

Tracking Information
First Submitted Date  ICMJE December 4, 2015
First Posted Date  ICMJE December 9, 2015
Last Update Posted Date August 27, 2019
Actual Study Start Date  ICMJE February 4, 2016
Actual Primary Completion Date May 15, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 4, 2015)
Number of Participant with treatment emergent serious adverse events [ Time Frame: Week 4 post infusion ]
incidence of any treatment-emergent serious adverse events defined as the composite of death, non-fatal pulmonary embolism, stroke, hospitalization for worsening dyspnea and clinically significant laboratory test abnormalities
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02625246 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: December 5, 2018)
  • Difference in Colony Forming Units (CFUs) in semiquantitative culture of sputum [ Time Frame: Participants will be followed from 1 week to an expected average of 24 weeks following infusion. ]
    Difference in CFUs in semiquantitative culture of sputum
  • rate of decline of lung function [ Time Frame: Participants will be followed from 12 weeks to an expected average of 24 weeks following infusion. ]
    difference in absolute decline of forced expiratory volume at one second (FEV1) percent predicted
  • frequency of acute exacerbations [ Time Frame: Participants will be followed from 12 weeks to an expected average of 48 weeks following infusion. ]
    increased cough and sputum production, fever, new or worsened dyspnea in less than 30 days, new or worsened hypoxemia in the absence of other identifiable causes
  • reported dyspnea and quality of life assessment [ Time Frame: Participants will be followed from 4 weeks to an expected average of 48 weeks following infusion. ]
    using quality of life tool questionnaire QOL-B version 2
  • death from any cause [ Time Frame: Participants will be followed for the duration of the trial, which is an expected average of 48 weeks. ]
    death from any cause
Original Secondary Outcome Measures  ICMJE
 (submitted: December 4, 2015)
  • Difference in Colony Forming Units (CFUs) in semiquantitative culture of sputum [ Time Frame: Participants will be followed from 1 week to an expected average of 24 weeks following infusion. ]
    Difference in CFUs in semiquantitative culture of sputum
  • rate of decline of lung function [ Time Frame: Participants will be followed from 12 weeks to an expected average of 24 weeks following infusion. ]
    difference in absolute decline of FEV1 percent predicted
  • frequency of acute exacerbations [ Time Frame: Participants will be followed from 12 weeks to an expected average of 48 weeks following infusion. ]
    increased cough and sputum production, fever, new or worsened dyspnea in less than 30 days, new or worsened hypoxemia in the absence of other identifiable causes
  • reported dyspnea and quality of life assessment [ Time Frame: Participants will be followed from 4 weeks to an expected average of 48 weeks following infusion. ]
    using quality of life tool questionnaire QOL-B version 2
  • death from any cause [ Time Frame: Participants will be followed for the duration of the trial, which is an expected average of 48 weeks. ]
    death from any cause
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Potential Efficacy of Human Mesenchymal Stem Cells in Non‐Cystic Fibrosis Bronchiectasis
Official Title  ICMJE A Phase I, Trial to Evaluate the Safety, Tolerability, and Potential Efficacy of Allogeneic Human Mesenchymal Stem Cell (hMSC) Infusion in Patients With Non-Cystic Fibrosis Bronchiectasis
Brief Summary To demonstrate the safety of bone marrow-derived allogeneic human Mesenchymal Stem Cells (hMSCs) in patients with bronchiectasis receiving standard of care therapy, and to explore treatment efficacy
Detailed Description

A Phase 1 investigation will be performed to test the safety of two doses of bone-marrow derived hMSCs (20,000,000 and 100,000,000) administered via peripheral intravenous infusion.

Group 1: 3 subjects will receive a single administration of allogeneic hMSCs: 20 x106 (20 million) cells delivered via peripheral intravenous infusion Group 2: 3 subjects will receive a single administration of allogeneic hMSCs: 1 x108 (100 million) cells delivered via peripheral intravenous infusion Interim safety analysis will be performed four weeks after the 1st subject is enrolled in each cohort. Continued safety and tolerability with review of adverse events (AEs) will be assessed at each visit. Efficacy parameters (pulmonary function tests, lung diffusion capacity, lung volumes, 6-Minute Walk Test (6MWT), and dyspnea/Quality of Life (QOL) questionnaires) will be assessed every 12 weeks until study completion. Clinical laboratory tests to assess safety will be performed at every visit.

High Resolution Computed Tomography (HRCT) scan will be performed at the baseline visit (if not done within three months prior to enrollment) and then at week 24.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Bronchiectasis
Intervention  ICMJE Biological: hMSCs
intravenous infusion of bone marrow-derived allogeneic stem cells
Other Name: allogeneic mesenchymal stem cell
Study Arms  ICMJE
  • Experimental: Group 1
    3 patients will receive a single administration of allogeneic hMSCs: 20 x106 (20 million) cells delivered via peripheral intravenous infusion
    Intervention: Biological: hMSCs
  • Experimental: Group 2
    3 patients will receive a single administration of allogeneic hMSCs: 1 x108 (100 million) cells delivered via peripheral intravenous infusion
    Intervention: Biological: hMSCs
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 4, 2015)
6
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE May 15, 2019
Actual Primary Completion Date May 15, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Provide written informed consent,
  • be between 30 and 87 years old at the time of signing the Informed Consent,
  • weight over 45 and under 150 kg,
  • have a clinical diagnosis of non-CF bronchiectasis prior to screening,
  • Have had at least 2 exacerbations in the past year as documented by physician office or hospital visits (Use of antibiotics of at least one time in the last year),
  • Show a baseline FEV1 between 25% and 85% predicted and over or equal to 1 L and a baseline diffusion capacity of lung for carbon monoxide (DLCO) over or equal to 30% (corrected for hemoglobin but not alveolar volume),
  • Have a normal Right Ventricular function, as documented by Doppler echo or right heart catheterization,
  • if a female of childbearing potential, agree to abide by contraception rules defined below.
  • Subjects may receive nondrug therapies including oxygen supplementation not greater than 4 Liters per minute and pulmonary rehabilitation.
  • Subjects may be on chronic macrolide or inhaled antibiotic treatment bronchiectasis

Exclusion Criteria:

  • Have HRCT and or surgical lung biopsy results inconsistent with the diagnosis of non-CF bronchiectasis. (Exclusion of emphysema and or diffuse parenchymal disease)
  • be unable to perform any of the assessments required for endpoint analysis (report safety or tolerability concerns, perform Pulmonary Function Tests (PFT) or HRCT, undergo blood draws, read and respond to questionnaire
  • If a female of childbearing potential, have a follicle stimulating hormone (FSH) under 25.8 IU/L
  • be actively treated for an acute infectious exacerbation of bronchiectasis
  • Have an active infection that is not treated
  • Have had active infections occurring within a minimum of 4 weeks of study treatment
  • Be currently on treatment for NTM infections
  • Have had positive sputum cultures for nontuberculous mycobacterial (NTM) within the past 6 months
  • Have a history of drug or alcohol abuse within the past 24 months.
  • Be currently receiving (or have received within four weeks of screening) experimental agents for the treatment of bronchiectasis or have been enrolled in clinical trials within the previous 30 days
  • Be actively listed (or expect future listing) for transplant of any organ.
  • Have clinically important abnormal screening laboratory values.
  • Have a serious comorbid illness that, in the opinion of the investigator, may compromise the safety or compliance of the patient or preclude successful completion of the study.
  • Have any other condition that, in the opinion of the investigator, may compromise the safety or compliance of the patient or preclude successful completion of the study.
  • Have known allergies to penicillin or streptomycin.
  • Be an organ transplant recipient.
  • Have a clinical history of malignancy within 5 years (i.e., patients with prior malignancy must be disease free for 5 years), except curatively-treated basal cell carcinoma of skin, squamous cell carcinoma of skin, or cervical carcinoma.
  • Have a non-pulmonary condition that limits lifespan to less than 1 year.
  • Be serum positive for HIV, hepatitis BsAg (surface agent reactive) or Viremic hepatitis C.
  • Have hypersensitivity to dimethyl sulfoxide (DMSO)
  • Be unable to maintain saturated oxygen (SpO2) of more than 93% on room air at sea level at rest) or an SpO2 of more than 88% on room air over 5,000 feet (1524 meters) above sea level at rest.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 30 Years to 87 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02625246
Other Study ID Numbers  ICMJE 20150627
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Marilyn Glassberg, University of Miami
Study Sponsor  ICMJE Marilyn Glassberg
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Glassberg K Marilyn, MD University of Miami
PRS Account University of Miami
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP