On February 16, 2021, the A-T Children’s Project sent the below email to A-T families in the United States announcing that the pharmaceutical company IntraBio has expanded their clinical trial to test N-Acetyl-L-Leucine in A-T patients, and we will be helping with recruitment. 

Dear A-T Parents,

Last year, we let you know that the A-T Children’s Project was helping to recruit participants age 18 and over for a clinical trial at the University of California Los Angeles (UCLA), sponsored by a company called IntraBio, that is testing N-Acetyl-L-Leucine to treat the movement disorder seen in A-T.

In the United States, the FDA first limited the study to adults with A-T to make sure that the drug was safe. Now, the FDA has granted permission for IntraBio to expand the study, and people with A-T age 6 and over will be considered for enrollment.

The study will require six trips to UCLA. Beyond obtaining approval to treat patients with the drug during the trial, IntraBio has permission from the FDA to extend treatment afterward, which means that study participants will be able to continue to take the drug after the study is completed if they so choose, with on-going visits at UCLA every six months.

As part of the study, researchers will need to evaluate how participants move. Therefore, participants will be asked to perform the 9 Hole Peg Test (which you can read more about here) or the 8 Meter Walk Test.

All study costs, including travel, will be paid by IntraBio. We understand that most families are continuing to quarantine because of the pandemic, but we are bringing this opportunity to you for those who feel comfortable with air travel or live within driving distance of UCLA.

If you and your child with A-T are interested in participating, please email info@atcp.org the following information:

  • Parent name
  • Email
  • Phone
  • Child’s name
  • DOB
  • City and State

We will be passing your information along to the study coordinators at UCLA who will then reach out to you directly.

While this study is for people with A-T age 6 and over, we are sending it to all A-T parents in the United States so that you can stay current on what’s happening.

All the best,

Jennifer Thornton, Executive Director

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