US-based Alexion Pharmaceuticals has started a single, multinational, placebo-controlled trial designed to assess the safety and efficacy of eculizumab (Soliris) in patients with relapsing neuromyelitis optica (NMO), a life-threatening, ultra-rare neurologic disorder.
Primary objective of the multinational, double-blind, placebo-controlled trial is to evaluate the efficacy of eculizumab compared to placebo in patients with relapsing NMO, based on the time to first relapse and relapse risk reduction.
The trial's secondary endpoints include safety and tolerability, as well as additional efficacy outcome measures.
Patient enrolment and dosing have started in the trial and recruitment is open to adults with a diagnosis of NMO or NMO spectrum disorder with relapsing disease.
In NMO patients, chronic, uncontrolled complement activation results in severe damage to the central nervous system (CNS), mainly impacting the optic nerve and spinal cord.
At present, there are no approved treatments for NMO, which is characterised by severe weakness, paralysis, respiratory failure, loss of bowel and bladder function, blindness and premature death.
The company has also started a single, multinational, placebo-controlled trial in patients with refractory generalised myasthenia gravis (MG), another rare and debilitating neurologic disorder caused by uncontrolled complement activation.
In this multinational, double-blind, parallel-group, placebo-controlled trial, the primary objective is to evaluate the efficacy of eculizumab compared to placebo on patients' motor function, as measured by the improvement in MG- Activities of Daily Living (MG-ADL) score at 26 weeks.
Secondary endpoints in this trial include safety and tolerability, as well as additional efficacy measures.
According to the company, patients with MG, uncontrolled complement activation due to antibodies directed at the neuromuscular junction can eventually lead to profound and debilitating weakness of several groups throughout the body.
Alexion executive vice-president and global head of R&D Martin Mackay said it may have the potential to help patients living with rare and devastating disorders.
"We look forward to enrolling patients in these placebo-controlled studies to confirm the clinical benefits of eculizumab in the treatment of NMO and MG, which would be an important development for these underserved patient populations," Mackay said.
Soliris currently is approved in the US, EU, Japan and other countries for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).
Both PNH and aHUS are debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation.
The company said that Soliris is not approved in any country for the treatment of NMO or MG.