Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

Overview

  • Study type

    Interventional
  • Study phase

    II
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Rochester, Minnesota: 16-007541
    NCT ID: NCT02592941
    Sponsor Protocol Number: MP-104-CL-037

About this study

The expanded access program will provide access to treatment with deflazacort in children, adolescent, and adult patients with DMD in the U.S. who are ineligible, unable, or otherwise unwilling to enroll in a clinical study examining the efficacy of deflazacort while a new drug application is under preparation and review. Enrollment is open to all eligible patients.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Main Inclusion Criteria:

  • Confirmed diagnosis of Duchenne muscular dystrophy
  • The patient is ≥ 5 years old
  • Current on all childhood vaccinations including the chicken pox vaccine

Main Exclusion Criteria:

  • History or current medication condition that could affect safety or poses an additional risk
  • Hypersensitivity or allergic reaction to steroids or their formulations

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status

Rochester, Minn.

Mayo Clinic principal investigator

Duygu Selcen, M.D.

Closed for enrollment

Additional contact information

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Phone: 800-664-4542 (toll-free)

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