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Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)
study id #: NCT02606136
condition: Duchenne Muscular Dystrophy
status: active, not recruitingpurpose:
This is a Phase 2, open-label, single arm trial of pamrevlumab (FG-3019) to estimate pamrevlumab’s safety and efficacy in non-ambulatory subjects with DMD.
intervention: pamrevlumab (FG-3019)
mechanism of action: Fibrosis preventor to support muscle
start date: November 2015
estimated completion: April 2020
phase of development: Phase 2
size / enrollment: 22
Each subject will receive pamrevlumab (35 mg/kg) every two weeks by intravenous infusion for up to 156 weeks. After at least 10 to 12 subjects complete one year of treatment interim analysis may increase total subjects enrolled to approximately 32. All subjects will be closely monitored for safety. Efficacy assessments will be performed routinely over the course of the study.
- Annual change in percent predicted annual forced vital capacity (FVC) during treatment with pamrevlumab. [ Time Frame: From baseline to 104 weeks ]
- Change in forced expiratory volume (FEV1) [ Time Frame: From baseline to 104 weeks ]
- Change in maximum inspiratory pressure (MIP) [ Time Frame: From baseline to 104 weeks ]
- Change in maximum expiratory pressure (MEP) [ Time Frame: From baseline to 104 weeks ]
- Change in peak expiratory flow (PEF) [ Time Frame: From baseline to 104 weeks ]
- Change in peak cough flow [ Time Frame: From baseline to 104 weeks ]
- Change in left ventricular ejection fraction (LVEF) [ Time Frame: From baseline to 104 weeks ]
- Change in Performance of Upper Limb (PUL) Score [ Time Frame: From baseline to 104 weeks ]
- Change in grip strength [ Time Frame: From baseline to 104 weeks ]
- Change in pinch strength [ Time Frame: From baseline to 104 weeks ]
- Change in Brooke scale for upper extremity [ Time Frame: From baseline to 104 weeks ]
- Change in cardiac fibrosis score assessed by MRI [ Time Frame: From baseline to 104 weeks ]
- Change in upper arm (bicep) muscle fat and fibrosis assessed by MRI [ Time Frame: From baseline to 104 weeks ]
• At least 12 years of age
• Written consent/assent by patient and/or legal guardian as per regional and/or IRB requirements
• 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
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
• Requires >=16 hours continuous ventilation
• Prior or ongoing medical condition that, in the investigator's opinion, could adversely affect the safety of the subject, makes it unlikely that the course of 156 weeks of treatment and follow-up would be completed, or could impair the assessment of study results
• Anticipated spine surgery within 156 weeks
• Severe uncontrolled heart disease
Need for intravenous diuretics or inotropic support within 3 months prior to screening
Hospitalization for a heart failure exacerbation or arrhythmia in last 3 months
• Arrhythmia requiring anti-arrhythmic therapy
• Hospitalization due to respiratory failure in the last 6 weeks
• Poorly controlled asthma or underlying lung disease such as bronchopulmonary dysplasia
• Known or suspected active hepatitis B or C or history of HIV
• BMI >=40 kg/m^2 or weight >117 kg
• Exposure to another investigational drug within 28 days prior to start of study treatment
• Exposure to another investigational drug or another approved product for DMD (e.g. eteplirsen) within 28 days prior to start of study treatment (or 5 half-lives of the product whichever is longer) prior to first screening visit with the exception of deflazacort. Use of deflazacort if regarded by the principal investigator as standard of care is allowed.
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