An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
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|ClinicalTrials.gov Identifier: NCT02295748|
Recruitment Status : Completed
First Posted : November 20, 2014
Last Update Posted : December 8, 2017
|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Drug: Deflazacort||Phase 1|
This is an open label, long-term extension study in approximately 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) who participated in the MP-104-CL-005 PK study.
Subjects may have received up to 8 (+2 days) doses of deflazacort during the MP-104-CL-005 PK study, at which point treatment will be continued at the same dose and frequency until such time that deflazacort becomes commercially available or the study is terminated. Concomitant corticosteroid therapy will be prohibited during the study while the subject is taking deflazacort.
Subjects will be given medication to take at home for once-daily, morning dosing following the 8 hour PK sample on Day 8 (+2 days) of the MP-104-CL-005 PK study. Subjects will begin dosing at home the day following Day 8 (+2 days) of the MP-104-CL-005 PK study.
Safety will be monitored throughout the study by repeated clinical and laboratory evaluations every 3 months for the first year, and then every 3 or 6 months, depending on subjects' disease status.
Subjects will be contacted via telephone between study visits to determine if any adverse events (AE) have occurred since the last study visit. Subjects who terminate the study early will be contacted if the Principal Investigator (PI) deems necessary.
Treatment will be continued for a maximum of three years or until such time that deflazacort becomes commercially available or the study is terminated.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||24 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label, Multi-Center, Long-Term Extension Study to Evaluate the Safety and Tolerability of Orally Administrated Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy|
|Actual Study Start Date :||December 2014|
|Actual Primary Completion Date :||August 24, 2017|
|Actual Study Completion Date :||August 24, 2017|
0.9 mg/kg oral deflazacort (between 2 to 12 x 6mg tablets based on body weight) will be administered daily.
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 rapidly to the active drug 21-desacetyldeflazacort.
Other Name: DFZ
- Number, frequency, and severity of adverse events [ Time Frame: 3 years ]Long-term safety and tolerability will be characterized by the number, frequency, and severity of adverse events from Day 1 through End of Study or Early Termination.
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): NCT02295748
|United States, California|
|Los Angeles, California, United States, 90095|
|United States, Illinois|
|Lurie Children's Hospital|
|Chicago, Illinois, United States, 60611|
|United States, New York|
|University of Rochester|
|Rochester, New York, United States, 14642|
|United States, Utah|
|University of Utah|
|Salt Lake City, Utah, United States, 84112|
|Study Director:||Marathon Bioscience Center||Marathon Pharmaceuticals|