A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)

Stopped early · Phase 3

Conditions studied: Limb-Girdle Muscular Dystrophy

In brief

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).

Key facts

Study ID

NCT03783923

Run by

PTC Therapeutics

People needed

11

Starts

2019-10-31

Expected to finish

2021-01-01

Last updated by the study team

2022-06-27

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify 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 qualify 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.

Where it is running

• Rare Disease Research, LLC — Atlanta, Georgia, United States
• University of Iowa Hospitals and Clinics — Iowa City, Iowa, United States
• The University of Kansas Medical Center — Kansas City, Kansas, United States
• Hugo W Moser Research Institute at Kennedy Krieger Institute — Baltimore, Maryland, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Washington University School of Medicine — St Louis, Missouri, United States
• University of Pennsylvania — Philadelphia, Pennsylvania, United States
• University of Washington — Seattle, Washington, United States
• University of Alberta — Edmonton, Alberta, Canada
• Ottawa Hospital — Ottawa, Canada
• Rigshospitalet, University of Copenhagen — Copenhagen, Denmark
• CHRU de NANCY Service de Neurologie — France, France
• University Hospital La Timone — Marseille, France
• Ludwig-Maximilians University Munich, Friedrich-Baur-Institute — Munich, Germany
• Oslo University Hospital — Oslo, Norway
• Pirogov Russian National Research Medical University — Moscow, Russia
• Saint-Petersburg State Pediatric Medical University — Saint Petersburg, Russia
• Sahlgrenska University Hospital — Gothenburg, Sweden

Full record on ClinicalTrials.gov

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