Recruitment for Phase I/II trial in CLN3 disease now underway
4 February 2022
The Beyond Batten Disease Foundation (BBDF) and Theranexus are now actively recruiting patients for its Phase I/II clinical trial. Six patients 17-year-old or older will receive BBDF-101 in escalating doses during the first 4.5 months to establish tolerability and pharmacokinetics of trehalose, miglustat and a combination of trehalose/miglustat.
Read more here: https://beyondbatten.org/news/bbdf-101-phase-i-ii-trial-underway/