The trial is expected to start enrolling patients soon across several sites in the U.S. “We are very pleased to achieve this milestone and look forward to initiating patient enrollment shortly,” Yuichi Iwaki, MD, PhD, president and CEO of MediciNova, said in a press release.
It will recruit approximately 150 participants who have had ALS for no more than 18 months with mild disability, determined by a score of at least 35 on the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) at screening.
Participants will initiate treatment with Rilutek (riluzole) for at least 30 days, after which they will start taking either 100 mg per day of ibudilast or a placebo for a period of nine months.
During the trial, researchers will evaluate the impact of ibudilast in ALS progression and patients’ functional disability by determining changes in ALSFRS-R score at the end of the treatment period. In addition to this primary goal, they will also assess changes in patients’ muscle strength, quality of life, and respiratory function, as well as the safety and tolerability profile of ibudilast.
Ibudilast is a small molecule that was designed to inhibit the activity of PDE4 and PDE10 enzymes and of MIF protein. This broad approach is thought to reduce the activity of immune cells in the brain and increase the production of neurotrophic factors that support the survival and growth of nerve cells that control movement.
This investigational therapy has been granted fast track status and orphan drug designation by the U.S. Food and Drug Administration for the treatment of ALS. These designations are intended to expedite and support its clinical development, review, and potential approval.
Ibudilast’s benefits in ALS have been explored in a previous Phase 2 trial (NCT02238626) in combination with Rilutek.
Results showed that add-on treatment with ibudilast could significantly improve functional activity and quality of life in ALS patients compared with Rilutek alone. The combo therapy could also delay disease worsening and improve survival rates.