Question: How do families decide which trial is best for them?
Answer: Each of the trials has different requirements for joining, so many – if not most – people will only be eligible for one trial after screening. The best way to approach this is to call the clinic contact closest to your area and discuss this with him/her. Age, weight, current medications, behavior, and IQ are all factors.
When will the trials be finished?
It all depends on enrollment. Right now, the Novartis trial and the Seaside trial are well underway and the Roche trial is just getting started. The trials need to have a certain number of people (a number determined before the trial starts) complete the trial before they can analyze the data and present results to the FDA. Right now, there is hope that the Novartis and Seaside trials could complete their enrollment by the end of the year. It all depends on how many people sign up to participate.
Is there assistance for travel?
Yes. All of the trials provide financial assistance for travel expenses. The amount depends on how far you have to travel. Please check with the coordinator at the nearest site to you for more information.
What is the open label extension and which trials offer it?
In these double-blind trials, each participant might be taking either the study drug or placebo (sugar pill). The trials of AFQ056 from Novartis and arbaclofen from Seaside Therapeutics include an open label extension, which means that participants who complete the trial are offered the choice of taking the study drug free of charge for 2 years. The trial of RO4917523 from Roche does not include an open label extension.
Does my child have to be off of all medications?
It varies in each trial. For the Novartis trial, they allow up to 2 FDA approved medications and an additional number of medications do not count against that limit (such as drugs to control seizures and minocycline). The Roche trial has no restrictions. The Seaside trial allows up to 3 medications but no SSRIs (e.g. prozac). The study site staff cannot give advice on whether or not you should stop or change medications – they can’t be seen as encouraging you to change medications to participate in the trial. You will need to make that decision with your regular doctor prior to enrolling in the trial. In some cases, such as the Seaside trial, you may resume any medications (including SSRI’s) once you are in the open-label continuation phase, if you choose to continue the study medication.
How do I determine if my child is eligible, such as having “significant behaviors” for the Novartis trial?
You can call the study coordinator at the site at which you would like to participate. They can do a phone screen with you to help determine preliminary eligibility.