The below email was sent to A-T parents in the United States notifying them of a new clinical trial that is currently recruiting.
Dear A-T Families,
IntraBio, a drug development company, is now conducting a clinical trial to test a product called N-Acetyl-L-Leucine in children and adults with A-T, aiming to treat the movement challenges they experience. Also known by its brand name Aqneursa, the product has drawn attention for its potential neuroprotective and symptom-relieving effects, particularly in neurological and neurodegenerative diseases. Since the FDA recently approved it for a different neurological disorder (Niemann-Pick Type C), a successful trial in A-T could potentially speed up the approval process for our community.
N-Acetyl-L-Leucine is classified as a nutraceutical – a chemically modified form of the essential amino acid L-leucine. This modification improves its absorption and may enhance its ability to cross cell membranes, including the blood-brain barrier.
Here are some key details about the trial:
- Eligible age: 4 years and older
- Duration: 6.5 months
- Commitment: 6 visits to the study site
- Enrollment: Each site may recruit up to 10 participants
- Recruitment period: Now through June 2025
Two sites are currently enrolling, and both are prioritizing families who live within reasonable driving distance. Families should reach out directly to the sites for more information:
- Massachusetts General Hospital (Boston, MA)
Franziska Hoche, MD – FHOCHE@mgh.harvard.edu - University of Texas Medical Center (Houston, TX)
Maria Poblete – Maria.poblete@uth.tmc.edu
Janaei Phillips – Janaei.Phillips@uth.tmc.edu
An additional site at UCLA in Los Angeles may open before recruitment ends—we’ll keep you posted if and when that’s confirmed.
You can find more information about the trial here: https://clinicaltrials.gov/study/NCT06673056
Although this study is only for individuals with A-T who are age 4 and up, we’re sharing this update with all U.S. A-T families so that everyone stays informed about ongoing research opportunities.
All the best,
Jennifer
Jennifer Thornton, Executive Director