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Allogeneic Human Cells (hMSC) Via Intravenous Delivery in Patients With Mild Asthma (ASTEC)

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ClinicalTrials.gov Identifier: NCT03137199
Recruitment Status : Recruiting
First Posted : May 2, 2017
Last Update Posted : May 20, 2019
Sponsor:
Information provided by (Responsible Party):
Marilyn Glassberg, University of Miami

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

Condition or disease Intervention/treatment Phase
Asthma Biological: hMSCs Phase 1

Detailed Description:

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

Group 1: 3 subjects will receive a single administration of allogeneic hMSCs: 20 million cells delivered via peripheral intravenous infusion Group 2: 3 subjects will receive a single administration of allogeneic hMSCs: 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, diffusing capacity (DLCO), 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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description: a first group of 3 patients will receive a lower dose of 20 million hMSCs, followed by a second group of 3 patients to receive a dose of 100 million hMSCs
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I, Trial to Evaluate the Safety, Tolerability, and Potential Efficacy of Allogeneic Human Mesenchymal Stem Cell Infusion in Patients With Mild Asthma
Actual Study Start Date : June 22, 2017
Estimated Primary Completion Date : November 2020
Estimated Study Completion Date : May 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Asthma

Arm Intervention/treatment
Experimental: Group receiving 20 million hMSCs
3 patients will receive a single administration of allogeneic hMSCs: 20 x106 (20 million) cells delivered via peripheral intravenous infusion
Biological: hMSCs
intravenous infusion of bone marrow-derived allogeneic stem cells
Other Name: allogeneic mesenchymal stem cell

Experimental: Group receiving 100 million hMSCs
3 patients will receive a single administration of allogeneic hMSCs: 1 x108 (100 million) cells delivered via peripheral intravenous infusion
Biological: hMSCs
intravenous infusion of bone marrow-derived allogeneic stem cells
Other Name: allogeneic mesenchymal stem cell




Primary Outcome Measures :
  1. Number of Participant with treatment emergent serious adverse events [ Time Frame: Week 4 post infusion ]
    as defined as the incidence of any treatment-emergent serious adverse events; these are a composite of death, non-fatal pulmonary embolism, stroke, hospitalization for worsening dyspnea and clinically significant laboratory test abnormalities


Secondary Outcome Measures :
  1. Difference in lung function [ Time Frame: Participants will be followed from 1 week to an expected average of 48 weeks following infusion ]

    Difference in FEV1 Variability in morning peak expiratory flow measurements

    Difference in frequency of acute exacerbations defined as:

    hospitalizations, missed work days, and/or oral steroids for more than 3 days Decrease in fractional excretion of inhaled NO (FENO; less than 50 ppb)


  2. Decrease in peripheral eosinophilia [ Time Frame: Participants will be followed from 1 week to an expected average of 48 weeks following infusion ]
    Decrease in number of peripheral eosinophils

  3. Difference in subject reported dyspnea and quality of life assessments [ Time Frame: Participants will be followed from 1 week to an expected average of 48 weeks following infusion ]

    Difference in subject reported dyspnea and quality of life assessments:

    Asthma Control Test (ACT) and Asthma Control Questionnaire (ACQ)


  4. Death from any cause [ Time Frame: Participants will be followed from 1 week to an expected average of 48 weeks following infusion ]
    Death from any cause



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Provide written informed consent
  • be between 18 and 65 years at the time of signing the Informed Consent
  • have a clinical diagnosis of asthma prior to screening in accordance with the guidelines of the American Thoracic Society/European Respiratory Society
  • ACQ over 1.25
  • have a smoking history of less than 10 pack‐years total and have not been smoking for at least the last 12 months
  • Perform a positive methacholine challenge at screening and repeat positive methacholine challenge at baseline visit (14 days later)
  • Have normal or mild obstructive spirometry
  • Have normal right heart function as documented by Doppler echo or right heart catheterization
  • If female, be surgically sterile, post‐menopausal (more than 1 year), or practice double barrier methods of birth control
  • Subjects may receive non‐drug therapies including oxygen supplementation no greater than 2L/minute, and pulmonary rehabilitation
  • Subjects may be on standard of care asthma medications including inhaled corticosteroids‐long acting beta agonist at a dose not greater than 1 mg of a fluticasone equivalent

Exclusion Criteria:

  • Have any active infection that is not treated
  • Be unable to perform any of the assessments required for endpoint analysis.
  • currently receive (or have received within four weeks of screening) experimental agents for the treatment of asthma
  • be actively listed (or expecting to be listed in the near future) for transplant of any organ
  • Have clinically important abnormal screening laboratory values : blood screening tests (Hematology, Chemistry, CBC including Eosinophil count) results that are not within normal limits (according to UMHC Laboratory Reference Ranges) 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 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
  • Have a non‐pulmonary condition that limits lifespan to less than a year.
  • Have a history of drug or alcohol abuse within the past 24 months.
  • Be serum positive for HIV, hepatitis BsAg or Viremia hepatitis C
  • Be currently participating (or have participated within the previous 30 days) in an investigational therapeutic or device trial.
  • Have hypersensitivity to dimethyl sulfoxide (DMSO)
  • Have a resting oxygen saturation (SpO2) on room air of more than 93% at sea level or more than 88% at an altitude above 5,000 feet above sea level (1524 meters)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03137199


Contacts
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Contact: Emmanuelle Simonet, MA 305-243-3728 esimonet@med.miami.edu
Contact: Jennifer Parra, ARNP 305-243-3728 j.hernandez4@med.miami.edu

Locations
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United States, Florida
University of Miami Miller School of Medicine Recruiting
Miami, Florida, United States, 33125
Contact: Emmanuelle Simonet, M.A.    305-243-3728    esimonet@med.miami.edu   
Contact: Eliana Mendes    305-243-2568    emendes@med.miami.edu   
Principal Investigator: Marilyn K Glassberg, M.D.         
Sub-Investigator: Rafael Calderon, M.D.         
Sponsors and Collaborators
Marilyn Glassberg
Investigators
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Principal Investigator: Marilyn K Glassberg, MD University of Miami

Additional Information:
Publications:

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Responsible Party: Marilyn Glassberg, Professor of Medicine, Surgery, and Pediatrics, University of Miami
ClinicalTrials.gov Identifier: NCT03137199     History of Changes
Other Study ID Numbers: 20160350
First Posted: May 2, 2017    Key Record Dates
Last Update Posted: May 20, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Marilyn Glassberg, University of Miami:
stem cell
mesenchymal stem cell
lung
asthma

Additional relevant MeSH terms:
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Asthma
Bronchial Diseases
Respiratory Tract Diseases
Lung Diseases, Obstructive
Lung Diseases
Respiratory Hypersensitivity
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases