A Study to Investigate Small Mobile Stem Cells (SMS) in Moderate to Severe Chronic Obstructive Pulmonary Disease

SORT-COPD II is a Phase 1b/2a, open-label, non-randomized, single-arm clinical study evaluating an investigational biologic therapy called SORT-COPD in adults with moderate to severe chronic obstructive pulmonary disease (COPD). SORT-COPD consists of Small Mobile Stem (SMS) cells suspended in Hank's Balanced Salt Solution and delivered directly to the lungs as an aerosolized mist using a vibrating mesh nebulizer. SMS cells are small adult stem cells derived from human blood. Laboratory and animal studies suggest that these cells may bind to cells involved in lung repair and influence inflammatory, vascular, extracellular matrix, and tissue-repair processes. SORT-COPD is being investigated as a potential regenerative and disease-modifying therapy for COPD. However, its safety and potential clinical effects in people with COPD have not been fully established, and participation may not provide an individual medical benefit. Up to 125 participants will be enrolled. The first 5 participants will form the Phase 1b portion of the study. These participants will receive the protocol-specified treatment regimen and will be followed through the Day 28 safety assessment. An independent Data Safety Monitoring Committee will then review the available safety and tolerability information before enrollment proceeds into Phase 2a. If the Data Safety Monitoring Committee recommends continuation, an additional 120 participants will be enrolled in the Phase 2a portion. The Phase 2a portion is intended to further characterize safety and to estimate changes from baseline following treatment. The results will be used to inform the design of subsequent controlled clinical trials. The Data Safety Monitoring Committee will continue to review cumulative safety information during the study and may recommend that enrollment or treatment continue as planned, be temporarily paused, be modified, or be stopped. All participants will receive the same treatment regimen. On each treatment day, participants will receive 2.4 billion SMS cells by oral inhalation through an Aerogen Solo vibrating mesh nebulizer used with an Aerogen Ultra device. Treatment will be administered at the study site on Days 1, 3, and 6, for a total planned dose of 7.2 billion SMS cells per participant. For each treatment, three vials containing 0.8 billion SMS cells in 1 mL will be used. Each vial will be diluted with an additional 1 mL of Hank's Balanced Salt Solution before nebulization. The three diluted portions provide 6 mL of investigational cell suspension. After the cell suspension has been administered, an additional 3 mL of Hank's Balanced Salt Solution without SMS cells will be nebulized as a washout. The total nebulized volume on each treatment day will therefore be 9 mL. Administration is expected to take approximately 30 minutes. Participants will undergo screening for up to 30 days before the first treatment. Study visits and assessments will include medical examinations, safety laboratory testing, electrocardiograms, monitoring for adverse events, pulmonary function testing, exercise assessments, respiratory questionnaires, and lung imaging. Participants will be monitored at the study site during and after each treatment. Follow-up will continue through approximately 12 months after treatment, and each participant is expected to remain in the study for up to approximately 13 months.