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

Plain-language summary

This observational study follows the natural course of neuronal ceroid lipofuscinosis (NCL, Batten disease) over time through the international DEM-CHILD database. It enrolls patients with a molecularly confirmed NCL diagnosis, including a subgroup of patients with TPP1 deficiency (CLN2 disease). The study is a non-phase study and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Recruiting

Sponsor

Universitätsklinikum Hamburg-Eppendorf

Intervention

Natural History

Start date

4/8/2020

Estimated completion

4/8/2050

Countries

Germany

Related genes

Locations

• Hamburg, Germany

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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