We have undertaken three clinical trials in ALS to date:
- A single-arm, open label phase 1/2 study – completed.
- A single-arm, open label, dose-escalation phase 2a study – completed.
- A randomized, double-blind, placebo-controlled phase 2 study – completed.
In all three trials, the primary endpoints were the safety and tolerability of a single intramuscular, intrathecal or simultaneous intramuscular and intrathecal administration. Key secondary efficacy endpoints included the rate of change in the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) (and in forced vital capacity (FVC) during the post-treatment period compared to a three month pre-treatment run-in period.