CLN3 disease – ‘Batten-1’ clinical research update
10 May 2023
The Beyond Batten Disease Foundation together with industry partner Theranexus recently met with the FDA following the end of Phase 1/2 (a safety study) for the Batten-1 clinical trial. The investigational drug 'Batten-1' is a novel and proprietary drug containing the active ingredient miglustat.
In the FDA meeting, plans to commence the Phase 3 of the clinical trial in paediatric patients (age 4-16 yrs) were discussed. The FDA have now given clear guidance to BBDF and Theranexus to finalize the Phase 3 protocol and to begin determining research sites. A contract research organisation (CRO) will now commence feasibility studies at sites around the world to determine which sites will be best suited for the trial. BBDF/Theranexus anticipate this study to be open for enrolment by the end of the year. Read more from the press release here.
IMPORTANT NOTE TO FAMILIES
If you are interested in participating in the Batten-1 clinical study, and your child has been diagnosed with CLN3 disease and is currently aged 4-16 years (or turning 4 in 2023-24), we strongly recommend you fill in [ this online form ] to register your interest, as soon as possible.
If you have any questions, please feel free to reach out to Dr Ineka Whiteman, Head of Research and Medical Affairs any time at research@bdsraaustralia.org.