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

May 20, 2020 updated by: Marilyn Glassberg

A Phase I, Trial to Evaluate the Safety, Tolerability, and Potential Efficacy of Allogeneic Human Mesenchymal Stem Cell Infusion in Patients With Mild Asthma

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

3

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Florida
      • Miami, Florida, United States, 33125
        • University of Miami Miller School of Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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 Names:
  • 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 Names:
  • allogeneic mesenchymal stem cell

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Week 4 post infusion

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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)
Participants will be followed from 1 week to an expected average of 48 weeks following infusion
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
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
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)
Participants will be followed from 1 week to an expected average of 48 weeks following infusion
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
Participants will be followed from 1 week to an expected average of 48 weeks following infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Marilyn Glassberg

Collaborators

The Marcus Foundation

Investigators

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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 22, 2017

Primary Completion (Actual)

March 3, 2020

Study Completion (Actual)

April 29, 2020

Study Registration Dates

First Submitted

April 24, 2017

First Submitted That Met QC Criteria

April 29, 2017

First Posted (Actual)

May 2, 2017

Study Record Updates

Last Update Posted (Actual)

May 21, 2020

Last Update Submitted That Met QC Criteria

May 20, 2020

Last Verified

May 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 20160350

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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