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

You may not qualify if:

• Subjects with non-syndromic variants reported to be associated with a vision-only phenotype or subjects above 8 years with clinical presentation at baseline of vision-only phenotype.
• Have been evaluated at baseline visit with a LogMAR score \> 1.9 for best corrected visual acuity.
• Have any ocular conditions that in the opinion of the investigator might affect visual assessments (e.g. cataract, glaucoma, diabetes, amblyopia, etc…).
• Have received gene therapy or stem cell therapy intended to modify the course of CLN3 disease.
• Have participated in another interventional study during the last three months before screening.
• Have been exposed to miglustat within 1 week prior to baseline, or is scheduled to receive an investigational device or drug (other than test product) during the study.
• Have a medical condition that, in the opinion of the investigator, would interfere with the efficacy or safety assessments or increase the subject's risk of adverse events (AEs), including any active malignancy of any type except non-melanoma skin cancer.
• Have a clinically significant abnormality in their clinical laboratory values (hematology, chemistry) at screening that would preclude their participation in the study.
• Have a cardiac impairment including repolarization disturbances (prolonged QTc), ventricular hypertrophy and sinus node dysfunction with severe bradycardia and other conduction issues.
• Have a known allergy or hypersensitivity to miglustat, or any component of the Investigational Medicinal Product (IMP) formulation.
• Have a severe renal disease (estimated Glomerular Filtration Rate (eGFR) \< 30 mL/min).
• Have a medical history of HIV, hepatitis B, hepatitis C, or positive results at screening.