MERIDIAN is a Phase 2 clinical trial evaluating the effectiveness and safety of the investigational medication, pegcetacoplan, in adults with sporadic MND.
Pegcetacoplan, also known as APL-2, is the drug being evaluated in this study. It has been designed to target C3, a central protein in the complement cascade, part of the immune system which controls neuroinflammation. Increased levels of C3 have been found at the motor end plate (also called the neuromuscular junction) where nerves and muscles meet. It is thought that reducing the activation of C3 may result in less C3 depositing at the motor end plate, which may reduce inflammation. This may slow the progression of MND.
Participation in MERIDIAN is expected to last for approximately 2 years and may be followed by a Long-Term Extension Study.
The drug is given as a subcutaneous (under the skin) infusion twice a week. Study participants and/or their care partner(s) will be taught how to administer the study drug at home.
Year 1: Study participants will be randomly assigned (randomised) to either receive pegcetacoplan or placebo for the first year of the study. There is a 67% (2 in 3) chance of receiving pegcetacoplan, and a 33% (1 in 3) chance of receiving placebo during the first year of the study. Neither the participants nor their study team will know the study treatment group assignment.
Year 2: All study participants will receive pegcetacoplan during Year 2 of the study.
There are up to 8 in-clinic visits in Year 1 (including screening) and 3 in-clinic visits in Year 2. Additional assessments will be done at home and via the phone.
Pegcetacoplan is an investigational medication not currently approved for use in patients with ALS in any country worldwide. A possible treatment resulting from this study is likely years away.
The trial’s main goal is to assess a combined score of physical function and survival after one year of treatment against placebo. Secondary measures of safety include side effects and suicidal thoughts throughout the trial’s two years; changes in daily life abilities, lung function, and muscle strength are other secondary goals.
The Australian Centres running this trial are;
New South Wales
• Brain and Mind Centre, Camperdown, New South Wales, Australia, 2050
• Central Coast Neurosciences Research, Erina, New South Wales, Australia, 2250
• Royal Brisbane and Women's Hospital, Herston, Queensland, Australia, 4029
• Gold Coast University Hospital, Southport, Queensland, Australia, 4215
• Neuro-Immunology Clinical Research Education and Support Service (N-CRESS), Austin Health, Heidelberg, Victoria, Australia, 3084
Further information is available here