The purpose of this study is to evaluate the pharmacodynamics, safety, tolerability, pharmacokinetics, and efficacy of RO7204239, a humanized monoclonal antibody that binds to human latent myostatin, in ambulant adult participants with facioscapulohumeral muscular dystrophy (FSHD).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
51
University of Irvine Medical Center (UCIMC)
Orange, California, United States
Regents of the University of Colorado
Aurora, Colorado, United States
University of Kansas Medical Center
Fairway, Kansas, United States
Percent change from baseline in contractile muscle volume (CMV) of quadriceps femoris muscles as assessed by magnetic resonance imaging (MRI) bilaterally
Time frame: Week 52
Percentage of participants with adverse events (AEs)
Time frame: Up to 2.5 years
Change from baseline in serum concentration of total latent myostatin
Time frame: Through 2 years
Change from baseline in serum concentration of free latent myostatin
Time frame: Through 2 years
Change from baseline in serum concentration of mature myostatin
Time frame: Through 2 years
Percent change from baseline in CMV of 36 muscles based on whole body MRI
Time frame: Weeks 28 and 52
Change from baseline in fat fraction of 36 muscles based on whole body MRI
Time frame: Weeks 28 and 52
Percent change from baseline in CMV of quadriceps femoris muscles as assessed by MRI bilaterally
Time frame: Week 28
Change from baseline in fat fraction of quadriceps femoris muscles as assessed by MRI bilaterally
Time frame: Weeks 28 and 52
Percent change from baseline in CMV of tibialis anterior muscles as assessed by MRI bilaterally
Time frame: Weeks 28 and 52
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Kennedy Krieger Institute
Baltimore, Maryland, United States
Virginia Commonwealth University Medical Center
Richmond, Virginia, United States
Rigshospitalet
København Ø, Denmark
Policlinico Universitario Agostino Gemelli
Rome, Lazio, Italy
Asst Grande Ospedale Metropolitano Niguarda
Milan, Lombardy, Italy
National Hospital for Neurology and Neurosurgery,
London, United Kingdom
Royal Victoria Infirmary
Newcastle upon Tyne, United Kingdom
Change from baseline in fat fraction of tibialis anterior muscles as assessed by MRI bilaterally
Time frame: Weeks 28 and 52
Percent change from baseline in CMV of biceps brachii muscles as assessed by MRI bilaterally
Time frame: Weeks 28 and 52
Change from baseline in fat fraction of biceps brachii muscles as assessed by MRI bilaterally
Time frame: Weeks 28 and 52
Percent change from baseline in contractile cross-sectional area (CSA) of skeletal muscle in the proximal lower limb muscles as assessed by MRI bilaterally
Time frame: Weeks 28 and 52
Change from baseline in fat fraction of proximal lower limb muscles as assessed at a single mid-femur slice bilaterally by MRI
Time frame: Weeks 28 and 52
Serum concentration of RO7204239
Time frame: Through 2 years
Maximum serum concentration (Cmax) of RO7204239
Time frame: Through 2 years
Area under the concentration-time curve (AUC) of RO7204239
Time frame: Through 2 years
Trough concentration (Ctrough) of RO7204239
Time frame: Through 2 years
Percentage of participants with anti-drug antibodies (ADAs)
Time frame: Baseline up to approximately 2 years