Clinical Trial: A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)
Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional
Official Title: A Multicenter, Randomized, Double Blind, Placebo Controlled, Multiple Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO6885247 Following 12 Weeks of Treat
Brief Summary: 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).
Detailed Summary:
Sponsor: Hoffmann-La Roche
Current Primary Outcome: Safety: Incidence of adverse events (AEs) [ Time Frame: Up to 20 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- 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 ]
Original Secondary Outcome: Same as current
Information By: Hoffmann-La Roche
Dates:
Date Received: September 11, 2014
Date Started: November 2014
Date Completion:
Last Updated: December 20, 2016
Last Verified: December 2016
