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Recruiting NCT06553976

NCT06553976 Spastic Paraplegia - Centers of Excellence Research Network

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Clinical Trial Summary
NCT ID NCT06553976
Status Recruiting
Phase
Sponsor Boston Children's Hospital
Condition Hereditary Spastic Paraplegia
Study Type OBSERVATIONAL
Enrollment 100 participants
Start Date 2024-06-04
Primary Completion 2026-06-04

Trial Parameters

Condition Hereditary Spastic Paraplegia
Sponsor Boston Children's Hospital
Study Type OBSERVATIONAL
Phase N/A
Enrollment 100
Sex ALL
Min Age N/A
Max Age N/A
Start Date 2024-06-04
Completion 2026-06-04

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Brief Summary

The Spastic Paraplegia - Centers of Excellence Research Network (SP-CERN) is a collaborative research consortium dedicated to advancing the understanding, diagnosis, and treatment of hereditary spastic paraplegia (HSP) and primary lateral sclerosis (PLS). Aims of the consortium are to a) perform natural history studies of HSP subtypes, b) discover and validate biomarkers and clinician- and patient-reported outcome measures, c) uncover HSP's molecular pathophysiology and develop rational therapeutic targets, and d) perform sufficiently powered clinical trials. The current pilot study is aimed at enrolling 100 individuals with hereditary spastic paraplegia type 4 (SPG4) or hereditary spastic paraplegia type 5A (SPG5A).

Eligibility Criteria

Inclusion Criteria: * Male or female patients of all ages with a clinical and molecular diagnosis of hereditary spastic paraplegia type 4 (SPG4, SPAST) or hereditary spastic paraplegia type 5A (SPG5A, CYP7B1). Exclusion Criteria: * Not having such a diagnosis and/or not being related to such individual.

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