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

NCT02592941 · Status: APPROVED_FOR_MARKETING · Type: EXPANDED_ACCESS

Last updated 2018-02-28

No results posted yet for this study

Summary

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.

Conditions

Interventions

DRUG

Deflazacort

Deflazacort, a glucocorticoid with anti-inflammatory and immunosuppressive effects, is used in treating a variety of diseases. Pharmacologically it is an inactive pro-drug which is metabolized immediately to the active metabolite, 21 desacetyl-DFZ. The elimination of this metabolite is primarily via the urine in humans. Its potency is approximately 70 to 90% of prednisone and 6 mg of deflazacort has approximately the same anti-inflammatory potency as 5 mg of prednisolone or prednisone.

Sponsors & Collaborators

  • Parexel

    collaborator INDUSTRY

  • Dohmen Life Science Services

    collaborator OTHER

  • PTC Therapeutics

    lead INDUSTRY

Eligibility

Min Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02592941 on ClinicalTrials.gov