Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)

Open to males ages 12 years and up

• At least 12 years of age
• Written consent/assent by patient and/or legal guardian as per regional and/or IRB requirements
• Non-ambulatory
• Brooke Score for Arms and Shoulders ≤5
• Diagnosis of DMD by medical history and confirmed Duchenne mutation in available genetic testing using a validated genetic test
• Able to perform spirometry
• Able to undergo cardiac and extremity (upper arm) MRI
• Percent predicted FVC between 40 and 90, inclusive
• At least one historical FVC% predicted value within 18 months of baseline
• Left ventricular ejection fraction ≥ 45% as determined by cardiac MRI at screening or within 3 months prior to day 0
• Subjects currently receiving heart failure cardiac medications (e.g. angiotensin converting enzyme inhibitors, angiotensin-receptor blockers, and beta-blockers) must achieve a stable regimen for at least 3 months prior to screening
• On a stable dose of corticosteroids for a minimum of 6 months prior to screening with no substantial change in dosage for a minimum of 3 months (except for adjustments for changes in body weight) prior to screening and no foreseen change in corticosteroid use during the course of study participation
• Received pneumococcal vaccine and is receiving annual influenza vaccinations
• Adequate renal function: cystatin C ≤1.4 mg/L
• Adequate hematological function
• Platelets >100,000/mcL
• Hemoglobin >12 g/dL
• Absolute neutrophil count >1500/μL
• Adequate hepatic function
• No history or evidence of liver disease
• Gamma glutamyl transferase (GGT) ≤3 x upper limit of normal (ULN)
• Total bilirubin ≤1.5xULN
• If sexually active, will use medically accepted contraceptives during participation in the study and for 3 months after the last dose of study drug