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Trial record 1 of 1 for:    NCT02592941
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Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

Expanded access is currently available for this treatment.
Verified August 2017 by PTC Therapeutics
Sponsor:
ClinicalTrials.gov Identifier:
NCT02592941
First Posted: October 30, 2015
Last Update Posted: August 18, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborators:
Parexel
Dohmen Life Science Services
Information provided by (Responsible Party):
PTC Therapeutics
  Purpose
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.

Condition Intervention
Duchenne Muscular Dystrophy Drug: Deflazacort

Study Type: Expanded Access     What is Expanded Access?
Official Title: An Open Label, Expanded Access Protocol Intended to Provide Treatment With MP-104 (Deflazacort) to U.S. Children, Adolescents, and/or Adults With Duchenne Muscular Dystrophy

Resource links provided by NLM:


Further study details as provided by PTC Therapeutics:

Intervention Details:
    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.
    Other Name: DFZ
Detailed Description:
Deflazacort will be supplied free of charge and shipped directly to patients or caregivers, as appropriate. Treating physicians will continue to follow their standard of care activities and procedures for management of DMD. Treating physicians participating in the expanded access program are required to collect/document any patient or caregiver reported safety events and report to the sponsor..
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02592941


Contacts
Contact: Call Center 1-844-800-4DMD (4363)

  Show 37 Study Locations
Sponsors and Collaborators
PTC Therapeutics
Parexel
Dohmen Life Science Services
  More Information

Additional Information:
Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT02592941     History of Changes
Other Study ID Numbers: MP-104-CL-037
First Submitted: October 29, 2015
First Posted: October 30, 2015
Last Update Posted: August 18, 2017
Last Verified: August 2017

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Deflazacort
Anti-Inflammatory Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs