Intrathecal Trial Update – Trial structure

I failed to update on something important in my last post.

Caveat: this is my understanding and if you are considering involvement in the clinical trial, you absolutely should confirm and ask independent questions of the trial coordinators. The reason I post this update is that (1) sometimes it is difficult to process new and very important information unless you’ve thought about it for awhile and talked to those important to you, so it might be difficult for someone hearing this for the first time after they’ve walked in the door or gotten on the phone; some need more time to think before being expected to ask more questions of those involved in the trial; (2) some make preliminary decisions about whether to try to be involved in the trial and I would hate for someone to not have information that might be important in whether they even decide to head for that door.

With that being said, an important change occurred last month in the structure of the clinical trial. You see, when we entered the trial, the structure involved three cohorts or groups of dosing – 10mg, 30mg, and 100mg. They were to be assigned in sequential order as opposed to the boys being randomized to a group because of safety. Although the drug had been dosed in mice and monkeys, my guess is that no one wanted to take the chance of giving a high dose immediately to a child.

Well, the lowest dose appeared quite effective in Case’s situation, for example, so it does make one consider whether a lower dose would be just as effective. My understanding is that the FDA posed the same question and required a new cohort of 4 boys at 1mg to be done after the 30mg cohort.

Also, although the 100mg dosing cohort is set to go after the 1mg, in clinical trials, things are always subject to change, so there could end up being changes there as well depending on the ongoing results of the 10mg, 30mg, and now 1mg cohorts.

Again, I stress that this is just my understanding and you should ask many questions about the trial structure and other issues before participating or considering participation in the trial. I’ve just found that sometimes families want to have a better understanding of the big picture before they walk in the door with the doctors. I know I did.

About Melissa

Proud mom of Case (7, MPS II), Tyson (9), and Brock (8). Advocate. Speaker. Writer. Inventor. House cleaner. Babysitter. Taxi Driver.