AsthmaPhase 1NCT05035862
Mechanisms of Interferon Gamma-primed Mesenchymal Stromal Cells (MSCs) for Moderate-to-severe Persistent Asthma
A Phase 1 study testing Albuterol Sulfate, Interferon gamma-primed mesenchymal stromal cells (MSCs) and Prednisone in people with Asthma, run by Emory University at 1 site in United States.
Trial summary
This study is a two strata, dose escalation Phase I clinical trial designed to assess the safety and determine the maximal tolerated dose (MTD) of allogenic cord tissue derived MSCs (cMSCs, stratum 1) and allogeneic, interferon-γ primed bone marrow MSCs (γMSCs, stratum 2). Read more about this study
- Who is it for?
- Ages 18 to 30 years
- Conditions the study is for
- Asthma
- What is being tested?
- Albuterol Sulfate, Interferon gamma-primed mesenchymal stromal cells (MSCs) and Prednisone
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 16 Mar 2022to 16 Dec 2022
- 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 30 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age 18 through 30 years at the screening visit
- Physician diagnosis of asthma
- Onset of asthma during childhood
- Evidence of atopy, evidenced by allergic rhinitis, aeroallergen sensitization, elevated total immunoglobulin E (IgE) level based on age-dependent reference values, or blood eosinophil counts > or = 150 cells/microliter
- Moderate-to-severe persistent asthma as defined by the National Asthma Education and Prevention Program Expert Panel Report-4
- Exclusion criteria at the screening visit include any of the following (may be re-enrolled):
Show all 28Show fewer
- A Panel Reactive Antibodies (PRA) test is positive for human leukocyte antigens (HLA) antibodies against the γMSC product
- Oral or injectable corticosteroid use within the two-week period prior to the screening visit.* Nasal corticosteroids may be used at any time during this trial at the discretion of the study's Medical Principal Investigator.
- Use of medications known to significantly interact with corticosteroid disposition within the two-week period prior to the screening visit, including but not limited to carbamazepine, erythromycin or other macrolide antibiotics, phenobarbital, phenytoin, rifampin, and ketoconazole*
- Presence of chronic or active lung disease other than asthma, including disorders of the airways or chest wall
- Current smoking or vaping
- History of premature birth before 35 weeks gestation
- Significant medical illness other than asthma, including thyroid disease, diabetes mellitus, sickle cell disease, Cushing's disease, Addison's disease, hepatic disease, immune deficiency, or concurrent medical problems that could require oral corticosteroids during the study or that would place the subject at increased risk of participating in the study
- A history of cataracts, glaucoma, or any other medical disorder associated with an adverse effect to corticosteroids
- History of adverse reactions to corticosteroids or short-acting bronchodilators or any of their ingredients
- Receiving allergen immunotherapy other than an established maintenance regimen (continuous regimen for ≥ 3 months)*
- Pregnancy or lactation
- If the participant is a female, failure to practice abstinence or use of an acceptable birth control method
- Inability to perform study procedures
- Current participation in another investigational drug trial
- Evidence that the participant may be unreliable or nonadherent, or may move from the clinical center area before trial completion
- Exclusion criteria at the randomization/infusion visit include any of the following:
- Clinically significant deterioration in the level of asthma control, evidenced by:
- Decrease in post-bronchodilator forced expiratory volume in one second (FEV1) of 15% (absolute change) compared to the post-bronchodilator FEV1 value obtained at the baseline visit, or
- An asthma exacerbation
- Clinically significant thrombocytopenia, anemia, neutropenia or elevations in the white blood cell count, assessed at the screening visit
- Positive pregnancy test
- The investigators will also ask participants to refrain from receiving new asthma therapies such as biologics until the final safety determination is made 7 days after the γMSC infusion (at study Day 14). They will also ask participants to refrain from participating in other interventional drug studies for the duration of their participation.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
About this study
Official title: A Phase 1 Study to Evaluate Safety, Toxicity, and Potential Mechanisms of Interferon Gamma-primed Mesenchymal Stromal Cells (MSCs) for Moderate-to-severe Persistent Asthma
This study is a two strata, dose escalation Phase I clinical trial designed to assess the safety and determine the maximal tolerated dose (MTD) of allogenic cord tissue derived MSCs (cMSCs, stratum 1) and allogeneic, interferon-γ primed bone marrow MSCs (γMSCs, stratum 2). Each stratum is designed to independently accrue 3 children at a dose level 1 of 2x106 cells/kg and 6 children at dose level 2 of 10x106 cells/kg, resulting in 9 children in each stratum. The primary objectives are to determine the safety and toxicity of allogeneic cord tissue derived MSCs and allogeneic interferon-γ primed bone marrow derived MSCs.
Read the detailed description
Asthma affects one out of every 10 patients in the United States. Many of these patients have poor asthma symptom control. For example, patients with moderate-to-severe persistent asthma have ongoing symptoms and airway inflammation despite aggressive treatment with asthma medications. These patients are at increased risk for medication-related side effects and potentially life-threatening exacerbations. Novel therapies are critically needed for this population.
Mesenchymal stem cells (MSCs) are cells that reside in the bone marrow. MSCs are anti-inflammatory and also promote body tissue repair. This study will determine whether one form of MSCs called "interferon gamma-primed MSCs or γMSCs" are safe for patients with moderate-to-severe asthma.
Patients will receive a single intravenous infusion of γMSCs at either 2x10^6 cells/kg or 5x10^6 cells/kg. Up to 12 young adults will be enrolled. The total sample size will not exceed 24 participants.
The study will take place at Children's Healthcare of Atlanta (for patient activities) and at Emory University (for laboratory research activities). Participants will be identified from the asthma clinics at Children's Healthcare of Atlanta. Participants will complete up to 12 visits over 1 year and will be compensated for their time and travel. At the completion of the study, any samples remaining after experimentation will be de-identified and made available for future research.
While some study participants may receive no direct benefit from participating in this study, others may benefit from the close monitoring of their respiratory health, specialized asthma education, and general evaluation of their condition, including lung function tests. Some participants also achieve psychological benefit from participating in an important research study and from interaction with the study staff. It is also possible that the knowledge obtained from this study, such as identification of biomarkers, may assist in the creation of novel asthma therapies in the future.
- Conditions
- Asthma
- Being tested
- Albuterol Sulfate, Interferon gamma-primed mesenchymal stromal cells (MSCs), Prednisone
- Keywords
- Asthma
- Medical subject headings
- Asthma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Albuterol SulfateDrug
Participants who experience symptoms of cough, dyspnea, chest tightness or wheezing will initiate use of albuterol (2 inhalations, 90 mcg/actuation) by metered dose inhaler (MDI) every 20 minutes for up to 1 hour and then every 4 hours if necessary.
Interferon gamma-primed mesenchymal stromal cells (MSCs)Drug
Also called Interferon gamma (IFNγ)-primed human bone marrow-derived mesenchymal stromal cells
IFNγ-primed bone marrow MSCs at a dose level of 2x106 cells/kg and a dose level of 5x106 cells/kg
PrednisoneDrug
Prednisone is recommended if the participant uses more than 12 inhalations of albuterol in 24 hours (excluding preventive use before exercise), or if the patient has ongoing symptoms for 48 hours or longer. The recommended prednisone dose for acute exacerbations is 2 mg/kg/day (maximum 60 mg) as a single dose for two days followed by 1 mg/kg/day (maximum 30 mg) as a single dose for two days. All administered doses will be rounded down to the nearest 10 mg.
| Study group | Role | What they receive |
|---|---|---|
| Infusion of γMSCs | Experimental | Escalating doses Dose escalation design with two dose levels. The low dose level involves a single intravenous infusion of γMSCs at 2x106 cells/kg. The high dose level involves a single intravenous infusion of γMSCs at 5x106 cells/kg. Receives: Drug: Albuterol Sulfate; Drug: Interferon gamma-primed mesenchymal stromal cells (MSCs); Drug: Prednisone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in number of adverse events and severe adverse events post-intervention
This outcome will measure safety of allogeneic cord tissue derived MSCs and allogeneic interferon-γ primed bone marrow derived MSCs. Assessments will be made by physical examination and further investigation as indicated. Events will be classified according to the NIH Clinical Toxicity Criteria for Adverse Events (CTCAE), version 5. All recorded adverse events and serious adverse events will be documented and recorded. Their attribution to the γMSC product will be determined. Adverse events that may be attributable to the study product include dyspnea, cough, wheezing, respiratory failure, allergic reaction, anaphylaxis, and infusion-related reaction.
Measured: During the infusion, during the observation interval after the infusion (2 hours postinfusion), one day after the infusion, and at 7 to 30 days after the infusion (study day 14 to 37)
Number of grade ≥3 adverse reaction attributable to the γMSC product
This outcome will measure toxicity. Toxicity is defined as any grade ≥3 adverse reaction and attributable to the γMSC product (attribution listed as at least probable), occurring from MSC infusion (at study day 7) through 7 days post-infusion (study day 14). Toxicity is considered unacceptable.
Measured: 7 to 30 days post-infusion (study day 14 to 37)
Secondary outcomes (5)
Change in lung function test
Change in lung function test and asthma characterization will determine the clinical impact of MSC therapy
Measured: Baseline, 7 to 30 days post-infusion (study day 14 to 37)
In- vivo trafficking of MSCs after intravenous infusion
In vivo trafficking of MSCs after intravenous infusion
Measured: 7 to 30 days post-infusion (study day 14 to 37)
Upper airway inflammation of MSC treatment
Upper airway inflammation will be determined by analysis of small molecule inflammatory constituents in exhaled breath condensate.
Measured: 7 to 30 days post-infusion (study day 14 to 37)
Circulating inflammatory cells of MSC treatment
Circulating cell inflammation will be determined by flow cytometric analysis of peripheral blood cells and AbSeq analysis of peripheral blood cells.
Measured: 7 to 30 days post-infusion (study day 14 to 37)
Biophysical characteristics of the cell products and correlation with clinical outcome
Characteristics of the γMSCs will be determined by comprehensive analysis at the Marcus Center for Therapeutic Cell Characterization and Manufacturing (MC3M)
Measured: 7 to 30 days post-infusion (study day 14 to 37)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Sep 2021
- Study start16 Mar 2022
- Primary completion16 Dec 2022
- Study completion16 Dec 2022
Why it stopped: The study terminated due to funding ending.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AsthmaStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- The Marcus Foundation, Ossium Health, Inc.
- Responsible party
- Principal investigator, Edwin Horwitz, Emory University
- Investigators
- Edwin Horwitz, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05035862
- Sponsor's protocol ID
- STUDY00002655
- Other IDs
- 2709
- Status last verified
- May 2025
- First submitted
- 3 Sep 2021
- Last updated
- 3 Jun 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual de-identified participant data will be shared and will be made available
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study to Evaluate Safety, Toxicity, and Potential Mechanisms of Interferon Gamma-primed Mesenchymal Stromal Cells (MSCs) for Moderate-to-severe Persistent Asthma
- ClinicalTrials.gov ID
- NCT05035862
- Acronym / sponsor ID
- STUDY00002655
- Conditions
- Asthma
- Keywords
- Asthma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study terminated due to funding ending.
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- The Marcus Foundation, Ossium Health, Inc.
- Study start
- 16 Mar 2022 (actual)
- Primary completion
- 16 Dec 2022 (actual)
- Study completion
- 16 Dec 2022 (actual)
- First posted
- 5 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is a two strata, dose escalation Phase I clinical trial designed to assess the safety and determine the maximal tolerated dose (MTD) of allogenic cord tissue derived MSCs (cMSCs, stratum 1) and allogeneic, interferon-γ primed bone marrow MSCs (γMSCs, stratum 2). Each stratum is designed to independently accrue 3 children at a dose level 1 of 2x106 cells/kg and 6 children at dose level 2 of 10x106 cells/kg, resulting in 9 children in each stratum. The primary objectives are to determine the safety and toxicity of allogeneic cord tissue derived MSCs and allogeneic interferon-γ primed bone marrow derived MSCs.
Detailed description
Asthma affects one out of every 10 patients in the United States. Many of these patients have poor asthma symptom control. For example, patients with moderate-to-severe persistent asthma have ongoing symptoms and airway inflammation despite aggressive treatment with asthma medications. These patients are at increased risk for medication-related side effects and potentially life-threatening exacerbations. Novel therapies are critically needed for this population.
Mesenchymal stem cells (MSCs) are cells that reside in the bone marrow. MSCs are anti-inflammatory and also promote body tissue repair. This study will determine whether one form of MSCs called "interferon gamma-primed MSCs or γMSCs" are safe for patients with moderate-to-severe asthma.
Patients will receive a single intravenous infusion of γMSCs at either 2x10^6 cells/kg or 5x10^6 cells/kg. Up to 12 young adults will be enrolled. The total sample size will not exceed 24 participants.
The study will take place at Children's Healthcare of Atlanta (for patient activities) and at Emory University (for laboratory research activities). Participants will be identified from the asthma clinics at Children's Healthcare of Atlanta. Participants will complete up to 12 visits over 1 year and will be compensated for their time and travel. At the completion of the study, any samples remaining after experimentation will be de-identified and made available for future research.
While some study participants may receive no direct benefit from participating in this study, others may benefit from the close monitoring of their respiratory health, specialized asthma education, and general evaluation of their condition, including lung function tests. Some participants also achieve psychological benefit from participating in an important research study and from interaction with the study staff. It is also possible that the knowledge obtained from this study, such as identification of biomarkers, may assist in the creation of novel asthma therapies in the future.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Infusion of γMSCs | Experimental | Drug: Albuterol Sulfate, Drug: Interferon gamma-primed mesenchymal stromal cells (MSCs), Drug: PrednisoneEscalating doses Dose escalation design with two dose levels. The low dose level involves a single intravenous infusion of γMSCs at 2x106 cells/kg. The high dose level involves a single intravenous infusion of γMSCs at 5x106 cells/kg. |
Who may qualify
- Ages
- 18 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (28 listed)
- Age 18 through 30 years at the screening visit
- Physician diagnosis of asthma
- Onset of asthma during childhood
- Evidence of atopy, evidenced by allergic rhinitis, aeroallergen sensitization, elevated total immunoglobulin E (IgE) level based on age-dependent reference values, or blood eosinophil counts > or = 150 cells/microliter
- Moderate-to-severe persistent asthma as defined by the National Asthma Education and Prevention Program Expert Panel Report-4
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Albuterol SulfateParticipants who experience symptoms of cough, dyspnea, chest tightness or wheezing will initiate use of albuterol (2 inhalations, 90 mcg/actuation) by metered dose inhaler (MDI) every 20 minutes for up to 1 hour and then every 4 hours if necessary. | Drug | Infusion of γMSCs |
| Interferon gamma-primed mesenchymal stromal cells (MSCs)Also: Interferon gamma (IFNγ)-primed human bone marrow-derived mesenchymal stromal cellsIFNγ-primed bone marrow MSCs at a dose level of 2x106 cells/kg and a dose level of 5x106 cells/kg | Drug | Infusion of γMSCs |
| PrednisonePrednisone is recommended if the participant uses more than 12 inhalations of albuterol in 24 hours (excluding preventive use before exercise), or if the patient has ongoing symptoms for 48 hours or longer. The recommended prednisone dose for acute exacerbations is 2 mg/kg/day (maximum 60 mg) as a single dose for two days followed by 1 mg/kg/day (maximum 30 mg) as a single dose for two days. All administered doses will be rounded down to the nearest 10 mg. | Drug | Infusion of γMSCs |
Primary outcome
Change in number of adverse events and severe adverse events post-intervention
Time frame: During the infusion, during the observation interval after the infusion (2 hours postinfusion), one day after the infusion, and at 7 to 30 days after the infusion (study day 14 to 37)
This outcome will measure safety of allogeneic cord tissue derived MSCs and allogeneic interferon-γ primed bone marrow derived MSCs. Assessments will be made by physical examination and further investigation as indicated. Events will be classified according to the NIH Clinical Toxicity Criteria for Adverse Events (CTCAE), version 5. All recorded adverse events and serious adverse events will be documented and recorded. Their attribution to the γMSC product will be determined. Adverse events that may be attributable to the study product include dyspnea, cough, wheezing, respiratory failure, allergic reaction, anaphylaxis, and infusion-related reaction.
Number of grade ≥3 adverse reaction attributable to the γMSC product
Time frame: 7 to 30 days post-infusion (study day 14 to 37)
This outcome will measure toxicity. Toxicity is defined as any grade ≥3 adverse reaction and attributable to the γMSC product (attribution listed as at least probable), occurring from MSC infusion (at study day 7) through 7 days post-infusion (study day 14). Toxicity is considered unacceptable.
5 secondary outcomes
Change in lung function test
Time frame: Baseline, 7 to 30 days post-infusion (study day 14 to 37)
In- vivo trafficking of MSCs after intravenous infusion
Time frame: 7 to 30 days post-infusion (study day 14 to 37)
Upper airway inflammation of MSC treatment
Time frame: 7 to 30 days post-infusion (study day 14 to 37)
Circulating inflammatory cells of MSC treatment
Time frame: 7 to 30 days post-infusion (study day 14 to 37)
Biophysical characteristics of the cell products and correlation with clinical outcome
Time frame: 7 to 30 days post-infusion (study day 14 to 37)
Results and safety
The study was stopped early (reason given: The study terminated due to funding ending.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2025.
- Individual participant dataSharing planned: Yes. Individual de-identified participant data will be shared and will be made available
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Edwin Horwitz, MD principal investigator
Emory University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Albuterol Sulfate, Interferon gamma-primed mesenchymal stromal cells (MSCs) and Prednisone in people with Asthma, run by Emory University 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 28 inclusion and 0 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 3 Jun 2025. 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: The study terminated due to funding ending.). 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?
Change in number of adverse events and severe adverse events post-intervention (time frame: During the infusion, during the observation interval after the infusion (2 hours postinfusion), one day after the infusion, and at 7 to 30 days after the infusion (study day 14 to 37)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 3 Jun 2025.