This site became the new ClinicalTrials.gov on June 19th. Learn more.
Show more
ClinicalTrials.gov Menu IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu IMPORTANT: Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu
Give us feedback
Trial record 1 of 1 for:    NCT02592941
Previous Study | Return to List | Next Study

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

Expanded access is currently available for this treatment.
Verified January 2017 by Marathon Pharmaceuticals, LLC
Sponsor:
Collaborators:
Parexel
Dohmen Life Science Services
Information provided by (Responsible Party):
Marathon Pharmaceuticals, LLC
ClinicalTrials.gov Identifier:
NCT02592941
First received: October 29, 2015
Last updated: January 30, 2017
Last verified: January 2017
  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 Marathon Pharmaceuticals, LLC:

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

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02592941

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

  Show 37 Study Locations
Sponsors and Collaborators
Marathon Pharmaceuticals, LLC
Parexel
Dohmen Life Science Services
  More Information

Additional Information:
Responsible Party: Marathon Pharmaceuticals, LLC
ClinicalTrials.gov Identifier: NCT02592941     History of Changes
Other Study ID Numbers: MP-104-CL-037
Study First Received: October 29, 2015
Last Updated: January 30, 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

ClinicalTrials.gov processed this record on August 16, 2017