Although dalfampridine extended-release treatment was found safe and well-tolerated in a pilot study in adults with cerebral palsy (CP), the drug failed to show any impact on walking ability.
Although the trial, which focused on adult CP patients with walking difficulties, was small, the lack of benefits prompted researchers to drop the idea of further testing the drug in CP patients.
The study, “Safety, Tolerability, and Sensorimotor Effects of Extended-Release Dalfampridine in Adults With Cerebral Palsy: A Pilot Study,” was published in the journal Clinical Therapeutics.
Dalfampridine extended-release is known as Ampyra when prescribed for patients with multiple sclerosis (MS) to improve their walking ability. The treatment, developed by Acorda Therapeutics, was seen to have similar effects in patients with walking difficulties after a stroke.
This made researchers at the Mellen Center for MS Treatment and Research at the Cleveland Clinic propose that dalfampridine may also help people with CP.
The Phase 1 trial (NCT01468350) consisted of two parts. Both used a so-called cross-over design, in which patients first receive the active treatment and then switch to placebo, or vice versa.
In the first phase, 11 CP patients were randomized to receive a single dose of dalfampridine or placebo and wait for two days before switching groups for another single dose.
The second part, in which 24 patients participated, was made up of twice-daily dosing for a week, and a washout period of one week before switching groups.
There were no serious adverse events during the study, and no one stopped the treatment because of side effects. Analyses showed that headache, fatigue, and insomnia were more common after dalfampridine treatment. In addition, patients experienced diarrhea and nausea to an equal extent after both dalfampridine and placebo.
Researchers did not note any new side effects, besides those reported in earlier studies.
Although the trial was small, researchers examined if the treatment produced any notable benefits. Among the 24 patients in the second part, the majority mostly had problems with hand movements, while only 38 percent having walking difficulties as the dominant feature.
Initial analyses suggested that patients may have improved their walking speed after dalfampridine treatment, but a comprehensive analysis failed to show any differences between the dalfampridine and placebo groups.
Based on these observations, the research team concluded that it is not worth to pursuing further trials of dalfampridine in CP patients.