Clinical Research Directory

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

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 Batten-1? in comparison with an external group of non-treated CLN3 disease subjects from natural history studies. After a titration period of 8 weeks, participants will take Batten-1 every day up to 52 weeks and will visit the clinic every 24 weeks for checkups and tests. Participants will keep a diary for potential seizure occurrence and compliance to study drug. Interventions they'll be given include: * visual acuity assessement * motor and capability assessement * safety assessment including lab tests and electrocardiogram * assessment of the effect of the body on Batten-1 * assessment of quality of life

Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)

The purpose of this study is to evaluate the safety, tolerability and clinical activity of PLX-200 in pediatric patients with lysosomal storage disorders.

Gene Therapy for Children With CLN3 Batten Disease

This is a phase 1/2, open-label, single dose, dose-escalation clinical trial to evaluate the safety and efficacy of CLN-301 (previous NCH Code: scAAV9.P546.CLN3) delivered intrathecally into the lumbar spinal cord region of subjects with CLN3 Batten disease.