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

Open to males ages 10 years and up

1. Male subjects at least 10 years of age at time of consent who are willing and able to provide informed consent to participate in the trial and diagnosed with DMD as confirmed by the Investigator
2. Genetically confirmed DMD
3. Performance of the Upper Limb test (PUL) entry item scores 2-6 and total PUL score less than or equal to 40. Enrollment of patients with PUL entry score 6, Exon 44 skipping amenable, and/or Exon 3 through 7 deletions will be capped at no more than 10% of the total study population.
4. Reduced ability to walk/run (if ambulatory): subjects must take more than 10 seconds for the 10-meter walk/run (i.e., velocity < 1 meter/second)
5. If non-ambulatory, loss of independent ambulation between 10th and 18th year birthday
6. Treatment with systemic glucocorticoids for at least 12 months and at a stable dose at least 6 months prior to study participation, except for weight-based or toxicity-related adjustments
7. Current and up-to-date immunizations
8. Adequate venous access for parenteral IP infusions and routine blood collection

9. Assessed by the Investigator as willing and able to comply with the requirements of the trial

   10. Sexually active subjects and their partners who are fertile must agree to use

   effective method(s) of contraception

1. Left ventricular ejection fraction (LVEF) less than or equal to 35% prior to randomization
2. Elbow-flexion contractures > 30° in both extremities
3. Body mass index (BMI) > 45
4. Percent predicted forced vital capacity (FVC%) < 35% within 6 months prior to randomization
5. Inability to perform consistent PUL 2.0 measurement within ± 2 points without shoulder domain or within ± 3 points with shoulder domain during paired testing at screening
6. Risk of near-term respiratory decompensation in the judgment of the Investigator, or the need for initiation of day and night non-invasive ventilator support as defined by serum bicarbonate ≥ 29 mmol/L at screening
7. History of non DMD-related chronic respiratory disease requiring ongoing or intermittent treatment, including, but not limited to, asthma, bronchitis, and tuberculosis
8. Acute respiratory illness within 30 days prior to screening and during screening

9. Initiation of nocturnal non-invasive ventilation within 30 days prior to screening

   10. Planned or anticipated thoracic or spinal surgery within the 6 months following

   randomization

   11. Planned or anticipated lower extremity surgery within the 6 months following

   randomization, if ambulatory

   12. Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products 13. Initiation of treatment with metformin or insulin within 3 months prior to

   randomization

   14. Initiation of treatment with an FDA-approved exon skipping therapy for the treatment

   of DMD and/or non-weight based adjustments within 12 months prior to randomization

   15. Treatment with human growth hormone within 3 months prior to randomization, unless on

   a stable dose allowing for weight-based dose adjustments (as determined by the site Investigator) for at least 24 months prior to randomization

   16. Treatment with a cell therapy product within 12 months prior to randomization; any

   prior exposure to CAP-1002 will be excluded

   17. Treatment with an investigational product within 6 months prior to randomization 18. History, or current use, of drugs or alcohol that could impair the ability to comply

   with participation in the trial

   19. Inability to comply with the investigational plan and follow-up visit schedule for any

   reason, in the judgment of the investigator

   20. Inability to undergo a cardiac MRI