A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
Condition: Limb-Girdle Muscular Dystrophy
MeSH terms: Muscular Dystrophies; Muscular Dystrophies, Limb-Girdle
Intervention: Deflazacort
Intervention description: Deflazacort tablet will be administered as per the dose and schedule specified in the arm.
Intervention MeSH terms: Deflazacort
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 11.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      This study is designed to evaluate the safety and efficacy of deflazacort in participants
      with LGMD2I. Most participants enrolled will have a screening visit and 3 additional visits
      (after 1, 13, and 26 weeks of treatment).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Genetic diagnosis of LGMD2I (confirmed mutation in the fukutin-related protein [FKRP]
             gene).

          -  Ability to ascend 4 stairs greater than or equal to (≥) 2.5 seconds and be able to
             complete the ascent and descent both at screening and baseline.

          -  Ability to understand the nature of the study and the consent form and to comply with
             study related procedures.

          -  Must weigh between 35 to 112.5 kilograms (kg).

        Exclusion Criteria:

          -  Received ≥4 weeks of continuous, systemic corticosteroid therapy within 3 months of
             study screening visit.

          -  Presence of significant cardiomyopathy as defined by echocardiogram (left ventricular
             ejection fraction less than (<) 30 percent [%]) at screening.

          -  Requires fulltime ventilator support.

          -  History of chronic systemic fungal or viral infections.

          -  History of recent bacterial infection (including tuberculosis) per discretion of the
             Investigator.

          -  Diagnosis of diabetes mellitus (controlled and/or uncontrolled) defined as glycated
             hemoglobin (HbA1c) ≥6.5% (based on historical or present diagnosis).

          -  History of immunosuppression or other contraindications to glucocorticosteroid
             therapy.

          -  Requires concomitant use or greater than (>) 1 week of drugs or substances that are
             moderate to strong cytochrome P3A4 (CYP3A4) inhibitors (for example, clarithromycin,
             fluconazole, diltiazem, verapamil, grapefruit juice) or moderate or strong CYP3A4
             inducers (that is, rifampin, efavirenz, carbamazepine, phenytoin) at baseline.

          -  Participated in an interventional clinical trial within the last 3 months prior the
             baseline visit.

          -  Unable or unwilling to comply with the contraceptive requirements of the protocol.

          -  Female participants who are pregnant and/or breastfeeding.

          -  Evidence or history of clinically significant hematological, renal, endocrine,
             pulmonary, gastrointestinal, cardiovascular, hepatic, neurologic, psychiatric, or
             allergic disease.