Registry for Hereditary Spastic Paraplegia to Support Future Treatments
STOP-HSP.Net: a Registry for Hereditary Spastic Paraplegia as an Integration Tool for Future Therapeutic Strategies
This study is creating a database to track people with hereditary spastic paraplegia over time to help understand the condition better and support future treatments.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 500 (estimated) |
| Sex | All |
| Sponsor | IRCCS Fondazione Stella Maris Academic / other |
| Locations | 1 site (Pisa) |
| Trial ID | NCT06572046 on ClinicalTrials.gov |
What this trial studies
This registry aims to create a comprehensive database of patients affected by hereditary spastic paraplegia (HSP) to facilitate the collection and management of patient data over time. It will involve five clinical centers where participants will be assessed annually, allowing for the monitoring of disease progression and the development of clinical outcome measures. The registry will utilize validated clinical scales, biomarkers, and patient-reported outcomes to capture the phenotypic complexity of HSP. Biological samples will also be collected for future research and analysis.
Who should consider this trial
Good fit: Ideal candidates include individuals with a clinical diagnosis of pure or complex hereditary spastic paraplegia, regardless of genetic diagnosis.
Not a fit: Patients with secondary forms of hereditary spastic paraplegia or significant comorbidities that affect their clinical picture may not benefit from this registry.
Why it matters
Potential benefit: If successful, this registry could enhance the understanding of hereditary spastic paraplegia and pave the way for the development of targeted therapies.
How similar studies have performed: Other registries for rare diseases have shown success in facilitating research and improving patient outcomes, suggesting a positive outlook for this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * clinical diagnosis of pure or complex HSP/spastic ataxia, even in the absence of a known genetic diagnosis * participants/parents/legal guardians will have to give informed consent for enrollment in the registry and privacy data management Exclusion Criteria: * subjects affected by secondary forms of HSP * presenting comorbidities that affect the general clinical picture according to clinical judgment * lack of informed consent
Where this trial is running
Pisa
- IRCCS Fondazione Stella Maris — Pisa, Italy (Recruiting)
Study contacts
- Principal investigator: Filippo M Santorelli, Dr. — IRCCS Fondazione Stella Maris
- Study coordinator: Filippo M Santorelli, Dr.
- Email: filippo.santorelli@fsm.unipi.it
- Phone: +39 050886275
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.