I was in the Vitality ALS (tirasemptive) trial that was held back in 2016-2017, the first ALS trial in years (before the Ice Bucket funds kicked off interest in potential treatments). It was a 48 week double blind trial, and a 48 week open label extension that was shutdown half way through the year after the endpoints were deemed unsuccessful. The trial started with almost 750 participants across 17 countries, and ended with about 350 participants when they shut down the open label extension trial. I had to return the unused drugs when they ended the trial. I have asked for, and have not received, any of my quantified trial data (monthly ALSFRS, FVC, SVC, strength tests, and blood tests), and the trial data was maintained separate from my ALS Clinic chart records.
I still have no confirmation that I was, or was not, in the placebo group. When I titrated up to the maximum tested dose in the open label extension I noticed some of the side effects (jitters), but the blind trial tested three dosage levels and the placebo, so I could have been a lower dose participant in the trial. The treatment goal was to stimulate fast twitch muscle to preserve function, not to preserve neurons. I was told the efficacy endpoints looked good, but only at the maximum dose and the side effects at that dosage were deemed too severe for the benefit.
I was told I appeared to be a slow progression PALS three months after the trials ended. During the trial I was told to stop my prior exercise habits to avoid tainting the results, a lack of movement that I believe accelerated my progression in small muscle groups that may have been preserved better with ROM and light effort exercise (what I slowly returned to with great difficulty, after the trial, with some restoration of function).
I advise anyone in a trial to document a personal log of any test results that can be copied down as the testing is performed. The official test data is the property of the trial sponsor (drug company), and you do not have the rights to official copies of the trial data collected. If you want to document your progression or improvement, you have to do it yourself.