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A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD) (PTCEMF)
study id #: NCT03642145
condition: Duchenne Muscular Dystrophy
The primary objective of this study is to evaluate the safety of a 0.9 milligrams per kilogram (mg/kg) and 0.45 mg/kg daily dose of deflazacort with a comparable natural history control group after 52 weeks of treatment in males with DMD aged greater than or equal to (>=) 2 to lesser than (<) 5 years.
The study will comprise of 2 periods (Period 1: 52-week safety and pharmacokinetics [PK], and Period 2: 52-week extension). Participants will be randomized in a 1:1 ratio to one of 2 treatment arms: 0.9 mg/kg deflazacort, and 0.45 mg/kg of deflazacort. A historic control group (which should match the study population as closely as possible) will be used as a comparator to characterize the safety and tolerability of deflazacort.
mechanism of action: Glucocorticoid to delay decline in muscle strength
last updated: January 21, 2019
start date: October 31, 2018
estimated completion: July 31, 2021
phase of development: Phase 3
- Treatment Emergent Adverse Events (TEAEs) [ Time Frame: 52 weeks ]
The primary endpoint for this study is a descriptive safety assessment of 0.45 mg/kg and 0.9 mg/kg of Deflazacort compared to a comparable natural history cohort after 52 weeks of treatment in males with DMD ages >=2 to <5 . Safety parameters that will be assessed include number of participants with incidence of Treatment of Emergent Adverse Events (TEAEs).
- Mean change from baseline in Height [ Time Frame: 52 weeks ]
The primary endpoint for this study is a descriptive safety assessment of 0.45 mg/kg and 0.9 mg/kg of Deflazacort compared to a comparable natural history cohort after 52 weeks of treatment in males with DMD ages >=2 to <5 . Safety parameters that will be assessed also include changes from baseline in Height.
- Mean change from baseline in Body Weight [ Time Frame: 52 Weeks ]
The primary endpoint for this study is a descriptive safety assessment of 0.45 mg/kg and 0.9 mg/kg of Deflazacort compared to a comparable natural history cohort after 52 weeks of treatment in males with DMD ages >=2 to <5 . Safety parameters that will be assessed also include changes from baseline in Weight.
- Peak Plasma Concentration [ Time Frame: 52 Weeks ]
Pharmacokinetic Parameters (Cmax) based on blood sampling for PK (Drug concentrations) on Visit 1 and Visit 2 of Deflazacort treatment.
- Time of Maximum Concentration [ Time Frame: 52 weeks ]
Pharmacokinetic Parameters (Tmax) based on blood sampling for PK (Drug concentrations) on Visit 1 and Visit 2 of Deflazacort treatment.
• In the opinion of the Investigator, the participant and parent(s)/caregiver are capable of complying with protocol requirements.
• The participant's legally acceptable representative signs and dates a written informed consent form and any required privacy authorization prior to the initiation of any study procedures.
• The participant must have a diagnosis of Duchene muscular dystrophy (DMD) defined by genetic or biopsy confirmation of DMD or have documented, increased serum creatine kinase more than 40 times the upper limit of normal and (ULN) and shown phenotypic signs of DMD.
• The participant is male and aged >=2 to <5 years at screening visit.
• The participant weighs between 11 kg and 50 kg at the study at screening visit.
• Ability to comply with scheduled visits, oral drug administration, and study procedures.
• The participant is current on childhood vaccinations according to the Center for Disease Control (CDC) recommended immunizations for children from birth through 6 years old. Note: The investigator should discuss timing of receipt of the varicella vaccine with the caregiver prior to initiation of chronic steroid treatment. Administration of live or live attenuated vaccines is not recommended in patients receiving immunosuppressive doses of corticosteroids. Participants whose caregivers decline vaccinations as a matter of personal belief are may be included.
• Baseline health is judged to be stable based on medical history, physical examination, laboratory profiles, and vital signs at screening, as deemed by the Investigator.
• The participant is able to ingest the oral tablets either whole or crushed.
• The participant has received 4 weeks or more of continuous corticosteroid therapy within 3 months of study screening visit.
• The participant has, in the judgment of the Investigator, clinically significant abnormal clinical laboratory parameters at screening or baseline that may affect safety.
• The participant has, in the judgment of the Investigator, a history or current medical condition that could affect safety including, but not limited to:
o Major renal or hepatic impairment
o Immunosuppression or other contraindications for corticosteroid treatment
o History of chronic systemic fungal or viral infections
o Diabetes mellitus or significant glucose intolerance
o Idiopathic hypercalciuria
o Symptomatic cardiomyopathy Note: Elective surgeries can be discussed with medical monitor.
• The participant has a history of hypersensitivity or allergic reaction to steroids or their formulations including, but not limited to lactose, sucrose, etc.
• The participant has received any drug, including prescription and non-prescription medications, and herbal remedies known to be significant inhibitors and/or inducers of CYP3A4 enzymes and/or P glycoprotein (P-gp) 14 days prior to the first dose of study drug.
• The participant has an indication that requires long-term use of strong CYP3A4 inhibitors and/or inducers that would interfere with the pharmacokinetics of Deflazacort.
• The participant 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
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