Status:
TERMINATED
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
Lead Sponsor:
PTC Therapeutics
Conditions:
Limb-Girdle Muscular Dystrophy
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief 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 ...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
October 31 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2021
Estimated Enrollment :
11 Patients enrolled
Trial Details
Trial ID
NCT03783923
Start Date
October 31 2019
End Date
January 1 2021
Last Update
June 27 2022
Active Locations (18)
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1
Rare Disease Research, LLC
Atlanta, Georgia, United States, 30324
2
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
3
The University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
4
Hugo W Moser Research Institute at Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205