Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database

ConditionsNeuronal Ceroid Lipofuscinosis Batten Disease CLN1 Disease CLN2 Disease CLN3 Disease CLN4 Disease CLN5 Disease CLN6 Disease CLN7 Disease CLN8 Disease CLN10 Disease CLN11 Disease CLN12 Disease CLN13 Disease CLN14 Disease

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

AgeNot listed

SponsorUniversitätsklinikum Hamburg-Eppendorf

About this trial

This is an observational study that aims at assessing the natural history of NCL diseases as part of the international DEM-CHILD Database.

1. Patient data are collected from medical records, patient questionnaires and routine follow up clinical examinations with focus on assessing progression in key areas of disease such as motor, language, cognition, seizures, vision, and behavior. 2. A local biorepository of samples from genetically defined NCL patients will be established as well as a virtual biorepository within the DEM-CHILD DB to be able to easily localize international availability of patient samples.

Eligibility criteria

Qualifiers

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.

Disqualifiers

None

Trial design

Treatments tested in this trial

Natural History

Treatment groups

500 Participants

are divided into 14 treatment groups

Treatment groups

See each treatment group below.

Sponsors and collaborators

Universitätsklinikum Hamburg-Eppendorf

Lead sponsor

Source ClinicalTrials.gov