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Clinical Trials in Italy / NCT06844877
Recruiting Observational

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

NCT06844877 · tracked via the Priya Life Science Italy tracker
Sponsor
IRCCS Fondazione Stella Maris
Phase
Observational
Started
2024-07-19
Last updated
2026-03-27

Condition(s) studied

Neuronal Ceroid Lipofuscinosis

Study 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).

Eligibility

Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
Inclusion Criteria: * genetically confirmed diagnosis of neuronal ceroid lipofuscinosis * participants/parents/legal guardians will have to give informed consent for enrollment in the registry and privacy data management Exclusion Criteria: * subjects affected by other forms of neurodegenerative diseases. * lack of informed consent

Primary outcome measure(s)

  • Establishment of the NCL Italian Registry to systematically document the clinical presentation and natural history of patients affected by NCLs — 12 months
    We will record information related to NCL natural history in 10 patients

Trial sites (1)

FacilityCityRegionStatus
IRCCS Fondazione Stella Maris Pisa Italy Recruiting

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06844877 on ClinicalTrials.gov ↗ ← All trials in Italy