NCT04613089 · RECRUITING
Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database
This is an observational registry study tracking how Neuronal Ceroid Lipofuscinosis (NCL), also called Batten Disease, progresses over time. Researchers collect medical records, questionnaires, and clinical exam data to understand how motor skills, language, cognition, seizures, vision, and behavior change. No new treatment is given. Because no phase is listed, this is a data-gathering effort, not a drug or device trial.
Eligibility criteria
Inclusion Criteria: - Patients with a confirmed molecular diagnosis of a form of NCL Disease Additional inclusion criteria for Group/Cohort: "CLN2 Disease - ERT (Brineura) Treated": * Documented diagnosis of TPP1 deficiency * Previous or current treatment with intracerebroventricular ERT with cerliponase alpha * Patients that are currently participating in post-marketing studies will be allowed to participate. Exclusion Criteria: - Patients with no confirmed molecular diagnosis of a form of NCL Disease
The sponsor's own eligibility wording, lightly reformatted. The study team makes the final eligibility decision — worth discussing with your doctor.
Eligibility criteria as of 2021-10-29