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

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Brief Title

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

Official Title

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 Phase

Phase 1

Study Type

Interventional


Primary Outcome

Number of Participant with treatment emergent serious adverse events

Secondary Outcome

 Difference in Colony Forming Units (CFUs) in semiquantitative culture of sputum

Condition

Bronchiectasis

Intervention

hMSCs

Study Arms / Comparison Groups

 Group 1
Description:  3 patients will receive a single administration of allogeneic hMSCs: 20 x106 (20 million) cells delivered via peripheral intravenous infusion

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

6

Start Date

February 4, 2016

Completion Date

May 15, 2019

Primary Completion Date

May 15, 2019

Eligibility Criteria

        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.
      

Gender

All

Ages

30 Years - 87 Years

Accepts Healthy Volunteers

No

Contacts

Glassberg K Marilyn, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02625246

Organization ID

20150627


Responsible Party

Sponsor-Investigator

Study Sponsor

Marilyn Glassberg


Study Sponsor

Glassberg K Marilyn, MD, Principal Investigator, University of Miami


Verification Date

August 2019