Using stem cells to treat moderate-to-severe asthma in young adults

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 testing if two types of stem cells can be safely used to help young adults with moderate-to-severe asthma feel better.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 18 Years to 30 Years
• Sex: All
• Sponsor: Emory University (Academic / other)
• Drugs / interventions: immunotherapy
• Locations: 1 site (Atlanta, Georgia)
• Trial ID: NCT05035862 on ClinicalTrials.gov

What this trial studies

This Phase 1 clinical trial investigates the safety and maximum tolerated dose of two types of mesenchymal stromal cells (MSCs) in young adults with moderate-to-severe persistent asthma. Participants will receive either allogenic cord tissue-derived MSCs or interferon gamma-primed bone marrow MSCs through an intravenous infusion. The study aims to enroll up to 24 participants, with a focus on assessing the safety and toxicity of these treatments over a year-long period. Participants will undergo multiple visits and may receive compensation for their involvement.

Who should consider this trial

Good fit: Ideal candidates are young adults aged 18 to 30 with a physician diagnosis of moderate-to-severe persistent asthma and evidence of atopy.

Not a fit: Patients with positive Panel Reactive Antibodies (PRA) tests against the MSC product or those using corticosteroids shortly before the study may not benefit.

Why it matters

Potential benefit: If successful, this treatment could provide a novel therapeutic option for patients suffering from poorly controlled asthma.

How similar studies have performed: While the use of MSCs in asthma treatment is an emerging field, this specific approach with interferon gamma-primed MSCs is novel and has not been extensively tested in prior studies.

Eligibility criteria

Inclusion Criteria:

* 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):

* 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.

Where this trial is running

Atlanta, Georgia

• Children's Healthcare of Atlanta — Atlanta, Georgia, United States (Recruiting)

Study contacts

• Principal investigator: Edwin Horwitz, MD — Emory University
• Study coordinator: Anne Fitzpatrick, PhD

Conditions Asthma