A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

Exclusion Criteria

• •1. Participants with Left Ventricular Ejection Fraction (LVEF) \< 35%
• •2. Participants with elbow-flexion contractures \> 30° in both extremities
• •3. Participants with Body Mass Index (BMI) \> 45
• •4. Participants with documentation of exon 44 skip-amenable mutation(s) in the dystrophin gene
• •5. Participants with documentation of dystrophin deletion mutation(s) encompassing and limited to exons 3-7
• •6. Participants with percent predicted FVC (FVC%p) \< 35%
• •7. Participants with inability to perform consistent FVC measurement within ±15% during paired testing at screening
• •8. Participants with risk of near-term respiratory decompensation in the judgment of the investigator, or the need for initiation of non-invasive ventilator support as defined by serum bicarbonate ≥ 29 mmol/L at screening
• •9. Participants with history of non DMD-related chronic respiratory disease requiring ongoing or intermittent treatment, including, but not limited to, asthma, bronchitis, and tuberculosis
• •10. Participants with acute respiratory illness within 30 days prior to screening
• •11. Participants with initiation of non-invasive ventilation within 30 days prior to screening, or the anticipated need to initiate non-invasive ventilation within the 12 months following screening
• •12. Participants with planned or anticipated thoracic or spinal surgery within the 12 months following randomization
• •13. Participants with planned or anticipated lower extremity surgery within the 12 months following randomization, if ambulatory
• •14. Participants with known hypersensitivity to Dimethyl Sulfoxide (DMSO) or bovine products
• •15. Participants with initiation of treatment with metformin or insulin within 3 months prior to randomization
• •16. Participants with initiation of treatment with an FDA-approved exon skipping therapy for the treatment of DMD within 24 months prior to randomization or dose adjustments to the therapy within 12 months prior to randomization with the exception of weight-based dose adjustments.
• •17. Participants who received treatment with Human Growth Hormone (HGH) within 3 months prior to randomization, unless on a stable dose (as determined by the site PI) for at least 24 months prior to randomization
• •18. Participants who received Treatment with idebenone within 3 months prior to randomization
• •19. Participants who received treatment with a cell therapy product within 12 months prior to randomization
• •20. Participants who received treatment with an investigational product within 6 months prior to randomization
• •21. Participants with history, or current use, of drugs or alcohol that could impair their ability to comply with participation in the trial
• •22. Participants with inability to comply with the investigational plan and follow-up visit schedule for any reason, in the judgment of the investigator