Please note - Enrolment targets have been met for the Phase III Study of Dexpramipexole. Recruitment for this trial is now closed.

A Phase III study sponsored by Biogen Idec to evaluate the efficacy, safety and pharmacokinetics of dexpramipexole in patients with ALS will be starting soon in Canada. The Canadian ALS Research Network (CALS), a consortium composed of all the academic ALS clinics across Canada, has reviewed the protocol and supports conducting the study in Canada. The six CALS centres chosen for the study include the ALS clinic at the University of Montreal (Clinique SLA, Centre hospitalier de l'Université de Montréal [CHUM] Hôpital Notre-Dame), McGill University (Montreal Neurological Hospital), University of Toronto (Sunnybrook Health Sciences Centre), University of Western Ontario (Motor Neuron Disease Clinic, London), University of Calgary (University of Calgary Medical Clinic) and University of British Columbia (GF Strong Rehabilitation Centre, Vancouver). The multi-national trial will also be conducted in the United States, Europe and Australia.

The Phase III trial will be a randomized, double-blind, placebo-controlled, parallel-group study for people with familial ALS (FALS) or sporadic ALS (SALS). Approximately 804 patients will be randomly assigned to one of two groups, the first group receiving a 150 mg twice-daily dose of dexpramipexole and the second receiving a placebo. Patients will be followed for a period of between 12 and 18 months.

“We are very excited that this study will soon be underway across Canada, and we are hopeful of a positive result for our patients with ALS,” says Lorne Zinman, MD, chair of CALS and medical director of the ALS/Neuromuscular Clinic at Sunnybrook Health Sciences Centre. He adds that this trial “is aimed at patients with ALS in the earliest phase of the disease.”

The study will enrol men and women between the ages of 18 and 80 with a diagnosis of FALS or SALS and with the onset of the first ALS symptoms having occurred within 24 months of dosing. Patients taking or not taking Riluzole are eligible; patients must be able to swallow tablets at the time of study entry. They must also have upright slow vital capacity (SVC) of 65 per cent or more at screening. Exclusion criteria include other medically significant illness or clinically significant abnormal laboratory values. Pregnant or breastfeeding women are excluded, as are those patients who are currently taking pramipexole or other dopamine agonists or who have had prior exposure to dexpramipexole. Other eligibility criteria may apply. Detailed eligibility evaluations must be conducted by clinical personnel at participating clinical centres.

Dexpramipexole is a novel, orally administered compound under development by Biogen Idec for the treatment of ALS. It has demonstrated neuroprotective properties in multiple in vitro and in vivo studies and may work by increasing the efficiency of mitochondria, the energy producing portion of the body’s cells. In a Phase II study, dexpramipexole achieved its primary objective evaluating safety and tolerability. The compound also showed a trend toward dose-related slowing of functional decline and a trend toward extending survival at the highest dose of 150 mg twice daily.

For more information about this trial, click here.