Actively Recruiting
Italian NCL Registry: a Registry for NCL as an Integration Tool for Future Therapeutic Strategies
Led by IRCCS Fondazione Stella Maris · Updated on 2026-03-27
50
Participants Needed
1
Research Sites
156 weeks
Total Duration
On this page
Sponsors
I
IRCCS Fondazione Stella Maris
Lead Sponsor
F
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Collaborating Sponsor
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
Italian NCL Registry: a Registry for NCL as an Integration Tool for Future Therapeutic Strategies
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Genetically confirmed diagnosis of neuronal ceroid lipofuscinosis
- Participants or their parents/legal guardians must provide informed consent for enrollment and data privacy
You will not qualify if you...
- Diagnosis of other neurodegenerative diseases
- Lack of informed consent
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
IRCCS Fondazione Stella Maris
Pisa, Italy, 56128
Actively Recruiting
Research Team
F
Filippo M Santorelli, Dr.
CONTACT
S
Stefania Della Vecchia, Dr.
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
0
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here