Sustained-release oral fampridine in multiple sclerosis: a randomised, double-blind, controlled trial

Area of Science:

• Neurology
• Clinical Pharmacology
• Rehabilitation Medicine

Background:

• Clinical studies indicate fampridine (4-aminopyridine) enhances motor function in multiple sclerosis (MS).
• This phase III study investigated sustained-release fampridine for MS-related ambulatory deficits.

Purpose of the Study:

• To assess the efficacy and safety of oral, sustained-release fampridine in individuals with multiple sclerosis and walking impairments.
• To determine the proportion of patients who responded to fampridine treatment based on timed walking tests.

Main Methods:

• A randomized, multicenter, double-blind, placebo-controlled phase III trial.
• 301 patients with multiple sclerosis were randomized to fampridine (10 mg twice daily) or placebo for 14 weeks.
• Response defined by consistent improvement on a timed 25-foot walk; primary outcome was the proportion of responders.

Main Results:

• A significantly higher proportion of fampridine-treated patients were timed walk responders (35%) compared to placebo (8%; p<0.0001).
• Fampridine responders demonstrated a 25.2% improvement in walking speed, maintained throughout treatment.
• Responders showed significantly greater improvement on the 12-item Multiple Sclerosis Walking Scale compared to non-responders.

Conclusions:

• Oral, sustained-release fampridine effectively improves walking ability in some individuals with multiple sclerosis.
• The observed improvement in walking is linked to a reduction in patient-reported ambulatory disability, representing a clinically meaningful therapeutic benefit.
• Safety profile of fampridine was consistent with prior investigations.