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

Inclusion Criteria:

1. Male subjects at least 10 years of age at time of consent who are willing and ableto provide informed consent to participate in the trial if ≥ 18 years of age orassent with parental or guardian informed consent if < 18 years of age. If athird-party caregiver is involved, they must provide informed consent.

2. Diagnosis of DMD based on clinical and phenotypic manifestations consistent with DMD (e.g., family history of DMD, elevated creatine kinase, dystrophin muscle biopsy,calf pseudohypertrophy, history of Gowers' sign, and gait impairment before 7 yearsof age) as confirmed by the Investigator.

3. Confirmatory genetic testing performed to have reached a diagnosis of DMD at anytime in the past or currently performed at a Clinical Laboratory ImprovementAmendments (CLIA)-certified laboratory or equivalent.

4. Performance of the Upper Limb test (PUL) entry item scores 2-6 and total PUL scoreless than or equal to 40. For Cohort A only: enrollment of patients with PUL entryscore 6, Exon 44 skipping amenable, and/or Exon 3 through 7 deletions will be cappedat no more than 10% of the total study population (approximately 6 patients withthese characteristics).

5. Reduced ability to walk/run (if ambulatory): subjects must take more than 10 secondsfor the 10-meter walk/run (i.e., velocity < 1 meter/second).

6. If non-ambulatory, loss of independent ambulation between 10th and 18th yearbirthday (standing unassisted or ability to take, at most, several stepsindependently is not considered ambulation). Subjects who are considerednon-ambulatory between the ages of 9 and10 may be enrolled with prior approval fromthe sponsor.

7. Receiving standard of care therapy at an experienced, multidisciplinary DMD centeras evidenced by regular cardiac and pulmonary monitoring, systemic glucocorticoidtreatment, and at-home range of motion exercises.

8. Treatment with systemic glucocorticoids for at least 12 months and at a stable doseat least 6 months prior to study participation, except for either weight-based doseadjustment or a decrease in steroid dose of ≤ 10% for toxicity. For patients onchronic deflazacort, treatment with an equivalent dose of prednisone or prednisolonefor a period of ≤ 30 days to bridge lack of availability of deflazacort during the 6months prior to randomization is acceptable.

9. Current and up-to-date immunizations according to children and adolescent Centersfor Disease Control and Prevention immunization schedule at the discretion of theInvestigator.

10. Adequate venous access for parenteral IP infusions and routine blood collection.

11. Assessed by the Investigator as willing and able to comply with the requirements ofthe trial.

12. Sexually active subjects and their partners who are fertile must agree to useeffective method(s) of contraception.

Exclusion Criteria:

1. Left ventricular ejection fraction (LVEF) less than or equal to 35% prior torandomization.

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 torandomization.

5. Inability to perform consistent PUL 2.0 measurement within ± 2 points withoutshoulder domain or within ± 3 points with shoulder domain during paired testing atscreening.

6. Risk of near-term respiratory decompensation in the judgment of the Investigator, orthe need for initiation of day and night non-invasive ventilator support as definedby serum bicarbonate ≥ 29 mmol/L at screening.

7. History of non DMD-related chronic respiratory disease requiring ongoing orintermittent treatment, including, but not limited to, asthma, bronchitis, andtuberculosis.

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 followingrandomization.

11. Planned or anticipated lower extremity surgery within the 6 months followingrandomization, if ambulatory.

12. Known hypersensitivity to dimethyl sulfoxide (DMSO) or bovine products.

13. Initiation of treatment with metformin or insulin within 3 months prior torandomization.

14. Initiation of treatment with an FDA-approved exon skipping therapy for the treatmentof 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, unlesson a stable dose allowing for weight-based dose adjustments (as determined by thesite Investigator) for at least 24 months prior to randomization.

16. Treatment with a cell therapy product within 12 months prior to randomization; anyprior exposure to deramiocel 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 complywith participation in the trial.

19. Inability to comply with the investigational plan and follow-up visit schedule forany reason, in the judgment of the investigator.

20. Inability to undergo a cardiac MRI. For Cohort B Only - Subjects with a knownhypersensitivity to gadolinium may forgo the LGE assessment but must complete acardiac MRI without contrast. For Cohort B Only - Subjects who are unable totolerate gadolinium due to renal insufficiency as measured by an estimatedGlomerular Filtration Rate (eGFR) less than 60 mL/min/1.73 m2 may forgo the LGEassessment but must complete a cardiac MRI without contrast.

21. For Cohort B: Subjects with PUL entry score 6, Exon 44 skipping amenable, or Exon 3through 7 deletions are excluded from participation.