A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy

Exclusion Criteria

• •The subject has received any investigational compound and/or has participated in another clinical study within 90 days prior to study treatment with the exception of observational cohort studies or non-interventional studies.
• •The subject has received deflazacort within 30 days or previous discontinued deflazacort due to an intolerable reaction.
• •The subject is an immediate family member, study site employee, or is in a dependent relationship with a study site employee who is involved in the conduct of this study (e.g. spouse, parent, child, sibling) or may consent under duress.
• •Any significant finding on the Columbia suicide severity rating scale (C SSRS) for subjects (ages 12-16, inclusive), in the opinion of the PI, warrants exclusion from this study.
• •The subject has, in the judgment of the investigator, clinically significant abnormal clinical chemistry laboratory parameters that may affect safety at Screening.
• •The subject has, in the judgment of the investigator, a history or current medical condition that could affect safety including, but not limited to:
• •1. Major renal or hepatic impairment
• •2. Immunosuppression or other contraindications for corticosteroid treatment
• •3. History of chronic systemic fungal or viral infections
• •4. Diabetes mellitus
• •5. Idiopathic hypocalcuria
• •6. Symptomatic cardiomyopathy at screening
• •The subject has a history of hypersensitivity or allergic reaction to steroids or their formulations including, but not limited to lactose, sucrose, etc.
• •Inability to take tablets as assessed by site investigator.
• •Unable to refrain from or anticipates the use of:
• •* Any medications at least 4 hours before and after dosing on PK Days 1 and 8.
• •* Any vitamins, vitamins with minerals, and/or meal supplementation (e.g., Ensure, Boost, etc.) at least 4 hours before and after dosing on PK Days 1 and 8.
• •* Any drug, including prescription and non prescription medications, and herbal remedies known to be significant inhibitors of CYP 3A4 enzymes and/or P gp for 14 days prior to the first dose of study drug and throughout the study. Appropriate sources will be consulted by the PI or designee to confirm lack of pharmacokinetic/pharmacodynamic interaction with study drug. Acetaminophen may be permitted during the study (doses to be based upon appropriate age/weight ranges.
• •* Any drugs known to be significant inducers of CYP 3A4 enzymes and/or P gp for 28 days prior to the first dose of study drug and throughout the study. Appropriate sources will be consulted by the PI or designee to confirm lack of PK/pharmacodynamics interaction with study drug.
• •Subject is mentally or legally incapacitated or has significant emotional problems at the time of screening visit or expected during the conduct of the study.
• •History of any illness that, in the opinion of the PI, might confound the results of the study or poses an additional risk to the subject by their participation in the study.
• •Positive urine drug or alcohol results at screening or check in.
• •Positive urine cotinine at screening.
• •Positive results at screening for HIV, HBsAg, or HCV.
• •Hemoglobin level below the lower limit of normal at screening.
• •Donation of blood or significant blood loss within 56 days prior to the first dose of study drug.