Clinical trial NCT03642145 (phase 3, status: withdrawn)
Conditions: duchenne muscular dystrophy
ICD codes: G71.01
Drugs: deflazacort
SMILES: [H][C@@]12C[C@@]3([H])[C@]4([H])CCC5=CC(=O)C=C[C@]5(C)[C@@]4([H])[C@@]([H])(O)C[C@]3(C)[C@@]1(N=C(C)O2)C(=O)COC(C)=O
Reason stopped: 
    study is no longer necessary given the safety and efficacy of emflaza in this age range has
    already been established after review of already available data.
  
Eligibility criteria:

        Inclusion Criteria:

          -  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 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 (ULN) and shown phenotypic signs of DMD.

          -  The participant weighs between 11 kilograms (kg) and 50 kg 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 participants
             receiving immunosuppressive doses of corticosteroids. Participants whose caregivers
             decline vaccinations as a matter of personal belief 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.

        Exclusion Criteria:

          -  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:

               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 or significant glucose intolerance

               5. Idiopathic hypercalciuria

               6. 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
             cytochrome P3A4 (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 30 days prior to study treatment with the exception of
             observational cohort studies or non-interventional studies.