Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database
Recruiting
Observational Study
Neuronal 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
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 500 (estimated)
- Sponsor
- Universitätsklinikum Hamburg-Eppendorf · Other
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
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 bi…
Trial Locations
Loading…
Loading trial locations…
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 ce…
Contacts