Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
PTC Therapeutics
Age
18–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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).
You may not be if
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.
Clareo Health | A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
