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AsthmaPhase 1ASTECNCT03137199

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

A Phase 1 study testing hMSCs in people with Asthma, run by Marilyn Glassberg at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
Asthma
What is being tested?
hMSCs
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
3 participants
Where
1 siteUnited States
Study dates
22 Jun 2017to 29 Apr 2020
Phase
Phase 1
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 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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)
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  • 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

Who cannot join

  • 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
Show all 14Show fewer
  • 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)

Trial locations and contacts

1 site in 1 country.

United States1 site
  • University of Miami Miller School of Medicine

    Miami, Florida, 33125

Other trials in United States

About this study

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

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.

Read the 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.

Conditions
Asthma
Being tested
hMSCs
Keywords
stem cell, mesenchymal stem cell, lung, asthma
Medical subject headings
Asthma

What participants receive

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

  • hMSCsBiological

    Also called allogeneic mesenchymal stem cell

    intravenous infusion of bone marrow-derived allogeneic stem cells

Study groups in NCT03137199
Study groupRoleWhat they receive
Group receiving 20 million hMSCsExperimental3 patients will receive a single administration of allogeneic hMSCs: 20 x106 (20 million) cells delivered via peripheral intravenous infusion
Receives: Biological: hMSCs
Group receiving 100 million hMSCsExperimental3 patients will receive a single administration of allogeneic hMSCs: 1 x108 (100 million) cells delivered via peripheral intravenous infusion
Receives: Biological: hMSCs

What is being measured

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

  1. Number of Participant with treatment emergent serious adverse events

    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

    Measured: Week 4 post infusion

Secondary outcomes (4)
  1. Difference in lung function

    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)

    Measured: Participants will be followed from 1 week to an expected average of 48 weeks following infusion

  2. Decrease in peripheral eosinophilia

    Decrease in number of peripheral eosinophils

    Measured: Participants will be followed from 1 week to an expected average of 48 weeks following infusion

  3. Difference in subject reported dyspnea and quality of life assessments

    Difference in subject reported dyspnea and quality of life assessments:

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

    Measured: Participants will be followed from 1 week to an expected average of 48 weeks following infusion

  4. Death from any cause

    Death from any cause

    Measured: Participants will be followed from 1 week to an expected average of 48 weeks following infusion

Timeline

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

  1. First posted2 May 2017
  2. Study start22 Jun 2017
  3. Primary completion3 Mar 2020
  4. Study completion29 Apr 2020

Why it stopped: First cohort completed. Per sponsor decision.

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
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
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)
Enrolment
3 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03137199
Sponsor's protocol ID
20160350
Acronym
ASTEC
Status last verified
May 2020
First submitted
24 Apr 2017
Last updated
21 May 2020
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Szefler SJ, Wenzel S, Brown R, Erzurum SC, Fahy JV, Hamilton RG, Hunt JF, Kita H, Liu AH, Panettieri RA Jr, Schleimer RP, Minnicozzi M. Asthma outcomes: biomarkers. J Allergy Clin Immunol. 2012 Mar;129(3 Suppl):S9-23. doi: 10.1016/j.jaci.2011.12.979.

    PubMed 22386512
  • Malmstrom K, Rodriguez-Gomez G, Guerra J, Villaran C, Pineiro A, Wei LX, Seidenberg BC, Reiss TF. Oral montelukast, inhaled beclomethasone, and placebo for chronic asthma. A randomized, controlled trial. Montelukast/Beclomethasone Study Group. Ann Intern Med. 1999 Mar 16;130(6):487-95. doi: 10.7326/0003-4819-130-6-199903160-00005.

    PubMed 10075616
  • Damera G, Panettieri RA. Irreversible airway obstruction in asthma: what we lose, we lose early. Allergy Asthma Proc. 2014 Mar-Apr;35(2):111-8. doi: 10.2500/aap.2013.34.3724.

    PubMed 24717787
  • Bousquet J, Mantzouranis E, Cruz AA, Ait-Khaled N, Baena-Cagnani CE, Bleecker ER, Brightling CE, Burney P, Bush A, Busse WW, Casale TB, Chan-Yeung M, Chen R, Chowdhury B, Chung KF, Dahl R, Drazen JM, Fabbri LM, Holgate ST, Kauffmann F, Haahtela T, Khaltaev N, Kiley JP, Masjedi MR, Mohammad Y, O'Byrne P, Partridge MR, Rabe KF, Togias A, van Weel C, Wenzel S, Zhong N, Zuberbier T. Uniform definition of asthma severity, control, and exacerbations: document presented for the World Health Organization Consultation on Severe Asthma. J Allergy Clin Immunol. 2010 Nov;126(5):926-38. doi: 10.1016/j.jaci.2010.07.019.

    PubMed 20926125
  • Pelaia G, Vatrella A, Maselli R. The potential of biologics for the treatment of asthma. Nat Rev Drug Discov. 2012 Dec;11(12):958-72. doi: 10.1038/nrd3792.

    PubMed 23197041
  • Pavord ID, Korn S, Howarth P, Bleecker ER, Buhl R, Keene ON, Ortega H, Chanez P. Mepolizumab for severe eosinophilic asthma (DREAM): a multicentre, double-blind, placebo-controlled trial. Lancet. 2012 Aug 18;380(9842):651-9. doi: 10.1016/S0140-6736(12)60988-X.

    PubMed 22901886
  • Jia G, Erickson RW, Choy DF, Mosesova S, Wu LC, Solberg OD, Shikotra A, Carter R, Audusseau S, Hamid Q, Bradding P, Fahy JV, Woodruff PG, Harris JM, Arron JR; Bronchoscopic Exploratory Research Study of Biomarkers in Corticosteroid-refractory Asthma (BOBCAT) Study Group. Periostin is a systemic biomarker of eosinophilic airway inflammation in asthmatic patients. J Allergy Clin Immunol. 2012 Sep;130(3):647-654.e10. doi: 10.1016/j.jaci.2012.06.025. Epub 2012 Aug 1.

    PubMed 22857879
  • Lai T, Wang S, Xu Z, Zhang C, Zhao Y, Hu Y, Cao C, Ying S, Chen Z, Li W, Wu B, Shen H. Long-term efficacy and safety of omalizumab in patients with persistent uncontrolled allergic asthma: a systematic review and meta-analysis. Sci Rep. 2015 Feb 3;5:8191. doi: 10.1038/srep08191.

    PubMed 25645133
  • Peirsman EJ, Carvelli TJ, Hage PY, Hanssens LS, Pattyn L, Raes MM, Sauer KA, Vermeulen F, Desager KN. Exhaled nitric oxide in childhood allergic asthma management: a randomised controlled trial. Pediatr Pulmonol. 2014 Jul;49(7):624-31. doi: 10.1002/ppul.22873. Epub 2013 Sep 4.

    PubMed 24039119
  • Anderson WJ, Short PM, Williamson PA, Lipworth BJ. Inhaled corticosteroid dose response using domiciliary exhaled nitric oxide in persistent asthma: the FENOtype trial. Chest. 2012 Dec;142(6):1553-1561. doi: 10.1378/chest.12-1310.

    PubMed 23364390
  • Gupta N, Su X, Popov B, Lee JW, Serikov V, Matthay MA. Intrapulmonary delivery of bone marrow-derived mesenchymal stem cells improves survival and attenuates endotoxin-induced acute lung injury in mice. J Immunol. 2007 Aug 1;179(3):1855-63. doi: 10.4049/jimmunol.179.3.1855.

    PubMed 17641052
  • Lee JW, Fang X, Gupta N, Serikov V, Matthay MA. Allogeneic human mesenchymal stem cells for treatment of E. coli endotoxin-induced acute lung injury in the ex vivo perfused human lung. Proc Natl Acad Sci U S A. 2009 Sep 22;106(38):16357-62. doi: 10.1073/pnas.0907996106. Epub 2009 Aug 31.

    PubMed 19721001
  • Fang X, Neyrinck AP, Matthay MA, Lee JW. Allogeneic human mesenchymal stem cells restore epithelial protein permeability in cultured human alveolar type II cells by secretion of angiopoietin-1. J Biol Chem. 2010 Aug 20;285(34):26211-22. doi: 10.1074/jbc.M110.119917. Epub 2010 Jun 16.

    PubMed 20554518
  • Gupta N, Krasnodembskaya A, Kapetanaki M, Mouded M, Tan X, Serikov V, Matthay MA. Mesenchymal stem cells enhance survival and bacterial clearance in murine Escherichia coli pneumonia. Thorax. 2012 Jun;67(6):533-9. doi: 10.1136/thoraxjnl-2011-201176. Epub 2012 Jan 16.

    PubMed 22250097
  • Krasnodembskaya A, Samarani G, Song Y, Zhuo H, Su X, Lee JW, Gupta N, Petrini M, Matthay MA. Human mesenchymal stem cells reduce mortality and bacteremia in gram-negative sepsis in mice in part by enhancing the phagocytic activity of blood monocytes. Am J Physiol Lung Cell Mol Physiol. 2012 May 15;302(10):L1003-13. doi: 10.1152/ajplung.00180.2011. Epub 2012 Mar 16.

    PubMed 22427530

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing hMSCs in people with Asthma, run by Marilyn Glassberg at 1 site in United States.

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 11 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: stopped early, as of the sponsor's last update on 21 May 2020. 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 United States. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this 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.

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 was stopped early (reason given: First cohort completed. Per sponsor decision.). Results are not available in the source record.

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?

Number of Participant with treatment emergent serious adverse events (time frame: Week 4 post infusion). 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 NCT03137199, which also lists 72 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 21 May 2020.

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