MediciNova has received approval from the US Food and Drug Administration (FDA) to start a clinical trial of MN-166 (ibudilast) to treat patients with amyotrophic lateral sclerosis (ALS).
The randomised, double-blind, placebo-controlled trial includes a six-month treatment period followed by a six-month open-label extension.
The principal investigator of the trial is Carolinas HealthCare System Neurosciences Institute in Charlotte director of Carolinas Neuromuscular/ALS-MDA Center, Dr Benjamin Rix Brooks.
The trial is designed to assess several efficacy endpoints, including functional activity (ALSFRS-R), respiratory function, muscle strength, and non-invasive ventilation (NIV) utilisation of MN-166.
The trial includes two treatment arms, MN-166 and matching placebo, while randomisation will occur in a 2:1 ratio (MN-166: placebo).
In addition, the trial will also monitor the safety and tolerability of MN-166 60mg/day versus placebo when administered in combination with riluzole in ALS patients.
Brooks said: “We are excited to initiate this study of ibudilast which targets a disease with limited treatment options.
“Ibudilast has demonstrated attenuating effects on activated glia cells, which are considered to play a key role in disease progression in ALS patients.”
MediciNova president and chief executive officer Yuichi Iwaki said: “We are very pleased to have successfully completed the FDA review period and look forward to initiating patient enrolment this fall.
“The safety and efficacy data from this trial will be important to our overall development efforts and should be complementary to efforts underway for proof-of-concept trials of MN-166 in progressive MS and drug addiction in the US.
“While MN-166 development is addressing unmet medical needs in all of its neurological indications, ALS may represent the largest unmet medical need.”
In order to participate in the trial, subjects must have a diagnosis of sporadic or familial ALS with onset of less than three years from first clinical weakness prior to screening. Around 60 male and female patients aged 18 to 80 years will be enrolled.
The subjects must also be on a stable dose of riluzole for at least one month prior to trial drug treatment.
The company said that 40 subjects will take part in the MN-166 group, while the remaining 20 in the placebo group.
Following the completion of the double-blind phase, patients randomised to the placebo arm will continue for another six months as well as receive open-label MN-166.
The trial is funded by both Carolinas HealthCare System and MediciNova.