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

Sponsor
IRCCS Fondazione Stella Maris
Study ID
NCT06844877
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Study Details

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).

Key Dates

Start date
Jul 19, 2024
Status verified
Mar 2026
Primary completion
Jul 19, 2026
Completion
Jul 19, 2027

Study Design

Enrollment
50 participants (estimated)

Primary Outcome Measure

Establishment of the NCL Italian Registry to systematically document the clinical presentation and natural history of patients affected by NCLs [ Time Frame: 12 months ]

Central Contacts

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