One step closer

We returned to Texas Neurology today where José completed his regimen screening, bringing him one step closer to being fully enrolled in the Healey ALS Platform Trial. What does that mean?

The trial is currently testing four different drugs at the same time. Once a participant clears the initial screening for the trial, they are randomly assigned (by a computer) to one of four treatments:

1. Regimen A – Zilucoplan, by UCB
2. Regimen B – Verdiperstat, by Biohaven Pharmaceutecals
3. Regimen C – CNM-Au8, by Clene Nanomedicine, Inc.
4. Regimen D – Pridopidine, by Prilenia Therapeutics

José has been randomly assigned to Regimen A which requires a meningitis vaccine (two shots!), which he received today.

After being assigned to a regimen, José was randomized again to determine whether he’ll receive the active drug or a placebo. Most clinical trials have a 1:1 ratio of drug to placebo assignments (each patient has a 50% chance of receiving the study drug. The Healey trail has a 3:1 ratio of active drug to placebo assignments (each patient has a 75% chance of receiving the study drug). Neither José, nor any of the clinic staff, will know whether he is receiving the active drug or placebo throughout the duration of the six-month placebo-controlled portion of the study. At the end of six months, he’ll have the option to receive the study drug for up to one year through what is called Open Label Extension.

What happens next?

We’ll return to Dallas in two weeks for the baseline visit which is when José will officially start the trial and learn how to administer the daily shots (yep, daily) for the next six months.

More about Zilucoplan

Zilucoplan is a type of drug known as a complement inhibitor. Zilucoplan is designed to work by preventing inflammation and subsequent tissue injury caused by a component of the body’s immune system called the complement system.

Half way to our goal!

Today’s step forward also coincides with hitting the mid-point of our crowdfunding campaign. A BIG MAHALO (thank you) to everyone who has helped us reach 50% of our fundraising goal! If you are able to chip-in or share the link with your networks, you’ll find it here on GoFundMe.