TXA127 in Non-Ambulant Patients With DMD Cardiomyopathy

Inclusion Criteria:

1. Male subjects 16 years of age or older who provide informed consent and can follow upwith protocol procedures. Parental or guardian consent is required for subjects atleast 16 years of age but younger than 18 years of age.
2. Documented diagnosis of Duchenne muscular dystrophy by genetic mutation analysis.
3. Documented cardiomyopathy, as assessed by echocardiogram with:
4. For a patient ≤ 20 years of age, EF > 35% and < 55% and fractional shortening of ≤ 28% at the time of screening.
5. For a patient > 20 years of age, EF > 20% and fractional shortening ≤ 28% at thetime of screening.
6. Reproducible (+/- 10%) difference between screening and baseline of percent predictedFVC , using the best out of 3 efforts at each visit:
7. For a patient ≤ 20 years of age, FVC between 45% and 85%, inclusive. Patientshould not utilize non-invasive ventilation such as CPAP or BiPAP.
8. For a patient >20 years of age, all of the following should exist: FVC > 20%, EF > 20% in baseline ECHO and ability to be off non-invasive ventilation, such asCPAP and BiPAP, for at least 4 consecutive hours a day (24 hours period).
9. Subjects must be taking systemic glucocorticoids for at least six months prior toscreening.
10. Subjects taking mineralocorticoid receptor antagonists, must be taking the drug for atleast three months prior to screening
11. Non-ambulant and cared for by a trained caregiver

Exclusion Criteria:

1. Therapy with intravenous inotropic or vasoactive medications at the time of screening
2. Planned or likelihood of major surgery in the 6 months after planned enrollment.
3. Patient is using a left ventricular assist device (LVAD) or actively in the process ofacquiring a LVAD.
4. Estimated glomerular filtration rate (GFR) <50 mL/min, as calculated by the CKD-EPICreatinine equation 2021 (https://www.kidney.org/professionals/kdoqi/gfr_calculator)
5. Patient is suffering from unstable systemic allergic reaction(s), connective tissuedisease or autoimmune disorder(s), requiring active intervention
6. History of cardiac tumor or current cardiac tumor
7. Known moderate-to-severe aortic stenosis/insufficiency or severe mitralstenosis/regurgitation
8. Current alcohol or drug abuse
9. Known history of chronic viral hepatitis unless considered cured based on hepatitis CRNA negative results
10. Hepatic dysfunction upon screening evidenced by bilirubin levels or gamma-GT levelsabove normal, deemed as clinically significant by the PI/Sub-I, and/or abnormalhematology (hematocrit <25%, WBC <3000/μl, platelets <100,000/μl), without areversible, identifiable cause. Total bilirubin elevations > 2 times the upperreference range, consistent with Gilbert's Syndrome, may be enrolled if there is noother evidence of liver dysfunction
11. Uncontrolled diabetes (HbA1c >9.0 percent)
12. Inability to comply with protocol-related procedures, including required study visits
13. Any condition or other reason that, in the opinion of the investigator or MedicalMonitor, would render the subject unsuitable for the study
14. Currently receiving or received within 90 days of enrollment (Day 1) aninvestigational treatment on another clinical study or expanded access protocol. Thiswill include patients currently being treated or who have not completed follow-up totreatment with an investigational cell-based therapy within 6 months prior toenrollment and patients actively receiving an investigational therapy forcardiovascular repair/regeneration.