Clinical Trial: Study With Batten-1 (Miglustat Liquid Formulation) to Treat Ceroid Lipofuscinosis Neuronal 3 (CLN3) Disease

U.S., Oct. 21 -- ClinicalTrials.gov registry received information related to the study (NCT07882290) titled 'Study With Batten-1 (Miglustat Liquid Formulation) to Treat Ceroid Lipofuscinosis Neuronal 3 (CLN3) Disease' on July 21.

Brief Summary: The goal of this open-label, single-arm, multicenter study is to assess the efficacy and safety of Batten-1 (oral liquid formulation of miglustat) (15 mg/kg up to 600 mg/day) in approximately 27 pediatric subjects with CLN3 disease over a period of 52 weeks, using visual acuity as primary efficacy endpoint, The main questions it aims to answer are:

* Does Batten-1 slow down or stabilize the visual acuity decline in pediatric CLN3 patients ?

* What medical problems do participants have when taking ...