NCT04613089 Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database
| NCT ID | NCT04613089 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Universitätsklinikum Hamburg-Eppendorf |
| Condition | Neuronal Ceroid Lipofuscinosis |
| Study Type | OBSERVATIONAL |
| Enrollment | 500 participants |
| Start Date | 2020-04-08 |
| Primary Completion | 2050-04-08 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 500 participants in total. It began in 2020-04-08 with a primary completion date of 2050-04-08.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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
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
Contact & Investigator
Angela Schulz, MD, PhD
PRINCIPAL INVESTIGATOR
Head of NCL-Specialty Clinic
Frequently Asked Questions
Who can join the NCT04613089 clinical trial?
This trial is open to participants of all sexes, studying Neuronal Ceroid Lipofuscinosis. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT04613089 currently recruiting?
Yes, NCT04613089 is actively recruiting participants. Contact the research team at m.nickel@uke.de for enrollment information.
Where is the NCT04613089 trial being conducted?
This trial is being conducted at Hamburg, Germany.
Who is sponsoring the NCT04613089 clinical trial?
NCT04613089 is sponsored by Universitätsklinikum Hamburg-Eppendorf. The principal investigator is Angela Schulz, MD, PhD at Head of NCL-Specialty Clinic. The trial plans to enroll 500 participants.