A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (NCT05126758)

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

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 if ≥ 18 years of age or assent with parental or guardian informed consent if \< 18 years of age. If a third-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 years of age) as confirmed by the Investigator.
3. Confirmatory genetic testing performed to have reached a diagnosis of DMD at any time in the past or currently performed at a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory or equivalent.
4. Performance of the Upper Limb test (PUL) entry item scores 2-6 and total PUL score less than or equal to 40. For Cohort A only: 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 (approximately 6 patients with these characteristics).
5. 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).
6. If non-ambulatory, loss of independent ambulation between 10th and 18th year birthday (standing unassisted or ability to take, at most, several steps independently is not considered ambulation). Subjects who are considered non-ambulatory between the ages of 9 and10 may be enrolled with prior approval from the sponsor.
7. Receiving standard of care therapy at an experienced, multidisciplinary DMD center as evidenced by regular cardiac and pulmonary monitoring, systemic glucocorticoid treatment, and at-home range of motion exercises.
8. Treatment with systemic glucocorticoids for at least 12 months and at a stable dose at least 6 months prior to study participation, except for either weight-based dose adjustment or a decrease in steroid dose of ≤ 10% for toxicity. For patients on chronic deflazacort, treatment with an equivalent dose of prednisone or prednisolone for a period of ≤ 30 days to bridge lack of availability of deflazacort during the 6 months prior to randomization is acceptable.
9. Current and up-to-date immunizations according to children and adolescent Centers for Disease Control and Prevention immunization schedule at the discretion of the Investigator.
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 of the trial.
12. Sexually active subjects and their partners who are fertile must agree to use effective method(s) of contraception.

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (20 Sites)

• Phoenix Children's Hospital — Phoenix, Arizona
• Arkansas Children's Hospital — Little Rock, Arkansas
• UCSD Altman Clinical and Translational Research Institute — La Jolla, California
• Children's Hospital of Los Angeles, Division of Neurology — Los Angeles, California
• University of California, Davis — Sacramento, California
• Children's Hospital Colorado — Aurora, Colorado
• Rare Disease Research, LLC — Atlanta, Georgia
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois
• University of Iowa Hospitals and Clinics — Iowa City, Iowa
• Boston Children's Hospital — Boston, Massachusetts
• University of Missouri Health Care — Columbia, Missouri
• Saint Louis Children's Hospital — St Louis, Missouri
• Rare Disease Research NC LLC — Hillsborough, North Carolina
• Akron Children's Hospital — Akron, Ohio
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio
• Children's Health Specialty Care Pavilion — Dallas, Texas
• University of Utah Hospital — Salt Lake City, Utah
• University of Virginia Children's Hospital — Charlottesville, Virginia
• Seattle Children's — Seattle, Washington
• Children's Wisconsin — Milwaukee, Wisconsin

💰 Cost, Insurance & Patient Rights

• 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
• Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
• Voluntary Participation: You may withdraw at any time without affecting your standard medical care.