Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
Condition: Duchenne Muscular Dystrophy
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne
Intervention: Pamrevlumab
Intervention description: Pamrevlumab, 10 milligrams (mg)/milliliter (mL), single dose vials
Intervention MeSH terms: Mitogens
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 21.0
Sex: Male
Minimum age: 12 Years
Healthy volunteers: No
Keywords: Duchenne; muscular; dystrophy; DMD; non-ambulatory

Summary:

      This is a Phase 2, open-label, single arm trial of pamrevlumab (FG-3019) to estimate
      pamrevlumab's safety and efficacy in non-ambulatory participants with DMD.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Written consent/assent by participant and/or legal guardian as per regional and/or
             institutional review board (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 ppFVC 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

          -  Participants currently receiving heart failure cardiac medications (for example,
             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/liter (L)

          -  Adequate hematological function

               1. Platelets >100,000/microliter (μL)

               2. Hemoglobin >12 grams (g)/deciliter (dL)

               3. Absolute neutrophil count >1500/μL

          -  Adequate hepatic function

               1. No history or evidence of liver disease

               2. Gamma glutamyl transferase (GGT) ≤3 x upper limit of normal (ULN)

               3. Total bilirubin ≤1.5 x ULN

          -  If sexually active, will use medically accepted contraceptives during participation in
             the study and for 3 months after the last dose of study drug

        Exclusion Criteria:

          -  Requires ≥16 hours continuous ventilation

          -  Prior or ongoing medical condition that, in the investigator's opinion, could
             adversely affect the safety of the participant, 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, including any of the following:

               1. Need for intravenous diuretics or inotropic support within 3 months prior to
                  screening

               2. 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 human immunodeficiency virus
             (HIV)

          -  Body mass index (BMI) ≥40 kilograms (kg)/square meter (m^2) or weight >117 kg

          -  Exposure to another investigational drug or another approved product for DMD (for
             example, eteplirsen or golodirsen) 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.