A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) (NCT03783923) | Clinical Trial Compass
TerminatedPhase 3
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
Stopped: The study was terminated early due to low enrollment and missing efficacy assessment data due to missed visits related to COVID-19.
United States11 participantsStarted 2019-10-31
Plain-language 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).
Who can participate
Age range18 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
* Partici…
What they're measuring
1
Change From Baseline in Time to Climb 4 Stairs After 26 Weeks of Treatment With Deflazacort