HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)

Approximately 24, and not more than 30, subjects will be randomized into the study, in two
sequential enrollment groups. Safety data from Group 1 will undergo a Data Safety Monitoring
Board (DSMB) review prior to initiation of enrollment for Group 2.

The first 6-8 randomized subjects will comprise Group 1, and will include a minimum of 3
subjects completing intracoronary infusion with CAP-1002. The DSMB will conduct a review of
interim safety data through 72 hours post-Day 0 for at least 3 infused subjects and for at
least 6 subjects overall.

Enrollment of Group 2 will begin per DSMB recommendations following their review of the 72
hour safety data from Group 1. Group 2 will include approximately 18 subjects. Screening and
randomization will continue until at total of 12 subjects are infused with CAP 1002 or 30
subjects are randomized into the study, whichever comes first.

All subjects assigned to the active treatment arm will receive an intended total dose of up
to 75 million (M) CAP-1002 cells infused as 25M cells into each of the three left ventricle
cardiac territories (anterior, lateral, inferior/posterior).

Subjects randomized to receive usual care will continue to be cared for and treated in
whatever manner the investigator deems most appropriate for the subject on an ongoing basis,
and will receive no infusion.

Randomization will take place within 30 days of the first screening procedure. After
completion of the screening procedures, eligible subjects randomized to active treatment arm
will receive CAP-1002 administered via intracoronary infusion on Day 0. Day 0 for eligible
subjects randomized to the usual care arm will occur 7 days after the date of randomization.
All randomized subjects will have a follow-up telephone call on Study Day 3, and study visits
at Weeks 2 and 6, and at Months 3, 6 and 12 post Day 0.

Inclusion Criteria:

1. Male subjects 18 years of age or older must be able to provide informed consent and
follow up with protocol procedures. Male subjects at least 12 years of age but younger
than 18 years of age must be able to provide assent with parent or guardian providing
permission for study participation. Only male subjects will be randomized into this
study.

2. Documented diagnosis of Duchenne Muscular Dystrophy by genetic mutation analysis.

3. Cardiomyopathy with left ventricular scar by LGE in at least 4 segments as assessed by
contrast-enhanced MRI and EF >35% at the time of screening.

4. Use of evidence based medical-therapy in accordance with the "DMD Care Considerations
Working Group" guidelines for the management of DMD, for at least three months prior
to signing the consent form (or, providing assent) or documented contraindication or
intolerance or patient preference.

5. Subjects must be taking systemic glucocorticoids for at least six months prior to
screening.

6. Subjects must be 12 years of age or older at time of screening

7. Subjects must be appropriate candidates for cardiac catheterization and intracoronary
infusion of CAP-1002, in the judgement of the site's interventional cardiologist.

Exclusion Criteria:

1. Therapy with intravenous inotropic or vasoactive medications at the time of screening.

2. Inability to undergo cardiac catheterization and/or MRI without general anesthesia.

3. Immunologic incompatibility with all available Master Cell Banks (MCBs) by
single-antigen bead (SAB) serum antibody profiling.

4. Planned or likely major surgery in the next 12 months after planned randomization.

5. Left Ventricular Assist Devices (LVAD) or those subjects actively in the process of
acquiring a LVAD.

6. Contraindication to cardiac MRI.

7. Known hypersensitivity to contrast agents.

8. Estimated glomerular filtration rate (GFR) <60 mL/min, as calculated by the CKD-EPI
cystatin C equation (Inker, Schmid et al. 2012).

9. Active infection not responsive to treatment.

10. Active systemic allergic reaction(s), connective tissue disease or autoimmune
disorder(s).

11. History of cardiac tumor or cardiac tumor demonstrated on screening MRI.

12. History of previous stem cell therapy.

13. History of use of medications listed in Appendix 3 within 3 months prior to signing
the ICF / Assent through completion of the study infusion.

14. Known moderate-to-severe aortic stenosis/insufficiency or severe mitral
stenosis/regurgitation.

15. Current active alcohol or drug abuse.

16. Known history of Human Immunodeficiency Virus (HIV) infection.

17. Known history of chronic viral hepatitis.

18. Abnormal liver function (ALT/AST >10 times the upper reference range) and/or abnormal
hematology (hematocrit <25%, WBC <3000 μl, platelets <100,000 μl) studies without a
reversible, identifiable cause.

19. Known hypersensitivity to bovine products.

20. Known hypersensitivity to dimethyl sulfoxide (DMSO).

21. Uncontrolled diabetes (HbA1c >9.0).

22. Inability to comply with protocol-related procedures, including required study visits.

23. Any condition or other reason that, in the opinion of the Investigator or Medical
Monitor, would render the subject unsuitable for the study.

24. Currently receiving investigational treatment on another clinical study or expanded
access protocol, including any of the following:

- Received investigational intervention within 30 days prior to randomization

- Treatment and/or an incomplete follow-up to treatment with any investigational
cell based therapy within 6 months prior to randomization

- Active participation in other research therapy for cardiovascular
repair/regeneration