Clinical Trial

Italian NCL Registry: a Registry for NCL as an Integration Tool for Future Therapeutic Strategies

Recruiting
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Record status
This record was last updated March 27, 2026 (before its estimated July 19, 2026 completion). Its status may not reflect the trial's current state.
Summary
The goal is to create a solid and harmonious disease registry of patient affected by neuronal ceroid lipofuscinosis (NCLs) that facilitates the collection and management of patients' data over time encouraging the research and the development of future clinical trials. In-depth clinical phenotyping will develop significant clinical outcome measures that can be used in clinical trials and will allow the phenotypic complexity of the disease to be captured with the use of validated clinical scales, biomarkers and so-called patient reported outcomes (PROs).
Protocol Amendment History 1 amendment
This ClinicalTrials.gov record has been amended once since 2025-02-19.
Trial Details
NCT Number NCT06844877
Lead Sponsor IRCCS Fondazione Stella Maris
Collaborators: Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Bambino Gesù Hospital and Research Institute
Conditions Neuronal Ceroid Lipofuscinosis
Enrollment 50 participants
Start Date 2024-07-19
Primary Completion 2026-07-19 (estimated)
Study Completion 2027-07-19 (estimated)
Updated on ClinicalTrials.gov 2026-03-27