This multicenter, randomized, double-blind, 12-week, placebo-controlled multiple dose study will investigate the safety and tolerability of RO6885247 in adult and pediatric patients with spinal muscular atrophy (SMA).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
9
Unnamed facility
Stanford, California, United States
Safety: Incidence of adverse events (AEs)
Time frame: Up to 20 weeks
Pharmacokinetics: RO6885247 plasma concentrations
Time frame: Up to 16 weeks
Pharmacokinetics: RO6885247 exposure, area under the concentration-time curve (AUC-tau, over the 24-hour dosing interval)
Time frame: Up to 12 weeks
Pharmacodynamics: SMN protein levels in blood
Time frame: Up to 20 weeks
Effect of RO6885247 on muscle electrophysiology, as assessed by Compound Muscle Action Potential (CMAP)
Time frame: Up to 20 weeks
Effect of RO6885247 on Electrical Impedance Myography
Time frame: Up to 20 weeks
Pharmacodynamics: In vivo splicing modification of SMN2 mRNA in blood
Time frame: Up to 20 weeks
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Unnamed facility
Orlando, Florida, United States
Unnamed facility
Chicago, Illinois, United States
Unnamed facility
Boston, Massachusetts, United States
Unnamed facility
St Louis, Missouri, United States
Unnamed facility
New York, New York, United States
Unnamed facility
Toronto, Ontario, Canada
Unnamed facility
Montreal, Quebec, Canada
Ch Pitie Salpetriere; Institut de Myologie
Paris, France
Unnamed facility
Paris, France
...and 16 more locations