Actively Recruiting
Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database
Led by Universitätsklinikum Hamburg-Eppendorf · Updated on 2021-10-29
500
Participants Needed
1
Research Sites
1565 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients with a confirmed molecular diagnosis of a form of NCL Disease
- For CLN2 Disease - ERT (Brineura) treated group: documented diagnosis of TPP1 deficiency
- Previous or current treatment with intracerebroventricular enzyme replacement therapy with cerliponase alpha
- Patients currently participating in post-marketing studies may participate
You will not qualify if you...
- Patients without a confirmed molecular diagnosis of a form of NCL Disease
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
University Medical Center Hamburg-Eppendorf
Hamburg, Germany, 20246
Actively Recruiting
Research Team
M
Miriam Nickel, MD
CONTACT
A
Angela Schulz, MD, PhD
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
14
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