My clinical Trial experience

[Ulf]

Hello Everyone, I wanted to write about my trial experience. I was part of the cytokinetics phase 2 trial. The drug helped me to retain my muscles and increased my strength for the 22 weeks. Sadly afterwards I lost some muscles during flush out period. I'm slow progressing. I'm now on the arimoclomol study. I believe I'm getting the real drug as when I take it my fasciculation die down to like 5 percent. They are involuntary so I don't think placebo effect can cause this. I also feel better when I take it and haven't lost anymore muscles so far.

I also heard from the chief investigator that she talked to the people from Nurown and the FDA wants them to complete the trial.

 

[suchahoward]

I also heard from the chief investigator that she talked to the people from Nurown and the FDA wants them to complete the trial.

I just completed the trial, got the placebo, so this is great news, but I haven't seen this anywhere and I follow this closely.

 

[lgelb]

It's not news. Trials are completed as a matter of course, unless unexpected toxicity arises in humans or animals, the interim results are so compelling that everyone should just jump on the treatment, the interim results clearly show insufficient efficacy, and/or the company runs out of money.

So the FDA wouldn't normally call anyone and say, "You should complete this trial." An independent review committee, not affiliated with the FDA, weighs in on whether it's OK to continue, once interim data come in. There was an interim review last year, which went well. And at AAN this year, the company reported possible biomarkers of treatment response.

Later, the final data are presented to (1) internal FDA reviewers, who write a report of their findings, and then (2) a panel that includes outside experts, that reviews the FDA report and holds its own discussion and vote. The FDA does not have to follow this vote, but usually does.

 

[suchahoward]