The Phase 3 REFALS trial exploring the impact of oral levosimendan (ODM-109) in patients with amyotrophic lateral sclerosis (ALS) has completed its target enrollment, the therapy’s manufacturer, Orion, announced.
A total of 496 participants were recruited across 104 sites in the United States, Canada, Europe, and Australia. They will take 1-2 mg of levosimendan per day for up to 48 weeks.
“The completion of patient enrollment is an important clinical milestone that takes us a step closer toward a potential new treatment option for patients with ALS,“ Christer Nordstedt, MD, PhD, senior vice president of research and development at Orion, said in a press release.
The REFALS trial (NCT03505021) was designed to evaluate the effectiveness of the investigational therapy to improve ALS patients’ respiratory muscle function, help them maintain their respiratory capacity, and preserve their overall functionality.
Researchers will primarily assess the ability of levosimendan to change slow vital capacity (pulmonary assessment) in the supine position (lying down with the face and torso facing up) at 12 weeks. They will also evaluate changes in ALS functional rating scale and respiratory events throughout the study.
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“We would like to thank all the patients living with ALS, their caregivers and dedicated researchers who made it possible to complete this phase of the study so rapidly,” Nordstedt said. “We expect the study to be completed in just over a year.”
ALS patients who complete the REFALS trial will also have the opportunity to continue treatment with levosimendan for as long as needed in the recently launched REFALS-ES (NCT03948178) extension study. It will evaluate the long-term safety and efficacy of the investigational therapy.
Levosimendan is a calcium sensitizer and potassium channel opener originally developed for the treatment of acute heart failure. It is approved in approximately 60 countries — although not in the U.S. — under the trade name Simdax as an intraveneous medicine for this indication.
As levosimendan can also improve the activity of skeletal muscle cells, such as those responsible for controlling breathing, it may hold potential to help preserve respiratory function in ALS patients.
Results from a previous Phase 2 trial, called LEVALS (NCT02487407), failed to effectively demonstrate levosimendan’s ability to promote changes in slow vital capacity when sitting. Still, the data supported a beneficial effect of the oral therapy on improving slow vital capacity in the supine position.