APL2-ALS-206 Trial

Pegcetacoplan

Pegcetacoplan (also known as APL-2) has not previously been tested in people with ALS, but has been tested in previous trials in healthy volunteers and people with blood, kidney and eye diseases. Researchers believe that the immune system is changed in people with ALS and that this contributes to nerve cell loss. Pegcetacoplan is designed to shut down the functioning of part of your immune system. Therefore, it is now being investigated whether pegcetacoplan can possibly prevent the loss of nerve cells.

Study design

This phase 2 study was commissioned by Apellis Pharmaceuticals and is a double-blind, randomised, placebo-controlled study. This means we’re comparing the effects of pegcetacoplan with the effects of a placebo. A placebo is a drug without an active ingredient, a ‘fake drug’.

Both pegcetacoplan and the placebo are to be administered by injection twice a week. Participants are randomly assigned either the placebo treatment or the pegcetacoplan treatment. Neither the participant nor the research team will know which treatment the participant receives.

The entire study will last 116 weeks, which is approximately 2 years. During this period, the participant will visit the hospital 11 times for examinations. Participants will receive treatment with pegcetacoplan or the placebo for 52 weeks. After this phase, all participants will enter the ‘open label’ phase, during which everyone will receive pegcetacoplan.

Entry criteria

The main criteria for participation in this study are as follows:

• The participant is at least 18 years old
• The duration of their illness is no more than 72 weeks from when symptoms first appeared
• The participant’s lung function is at least 60%, without the use of a ventilator
• Participants must not get pregnant or be breastfeeding throughout the duration of the study
• Participants have been vaccinated within the last 5 years, or are willing to be vaccinated, against pneumococcus (Streptococcus pneumoniae), meningococcus (Neisseria meningitidis; types A, C, W, Y and B), and HiB (Haemophilus influenzae type B).
• If the participant has a diaphragm pacing or considers undertaking the procedure during the study, participation is not possible
• When using Riluzole: a stable dose of ≥30 days prior to participation
• When using Edaravone: a stable dose of ≥60 days prior to participation

This study requires 228 participants from different countries. Several TRICALS centres are participating in this study. The study is currently ongoing in the following European countries: The Netherlands, Belgium, France, Ireland, Italy, and Spain.