clinical trial · NCT05174039
An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease
Beyond Batten Disease Foundation·phase2·completed·n = 6
Batten DiseaseMiglustat 100 milligrams (mg) Oral Capsule
brief summary
This is an open label study in approximately 6 subjects in 2 centers to assess the safety, PK, and efficacy of the maximum tolerable dose (MTD) of oral miglustat (100 mg once daily \[QD\] to 200 mg 3 times daily \[TID\]) in subjects ≥ 17 years of age with CLN3 disease over a period of 104 weeks.
started
Mar 10, 2022
primary completion
May 30, 2024
completion
May 30, 2024
last updated
Sep 9, 2025
official title
An Open-label Safety, Pharmacokinetic, and Efficacy Study of the Combination of Miglustat for the Treatment of CLN3 Disease in Patients 17 Years of Age and Older
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol