Spastic Paraplegia - Centers of Excellence Research Network
Spastic Paraplegia - Centers of Excellence Research Network (SP-CERN) - Natural History Study Pilot
Boston Children's Hospital
100 participants
Jun 4, 2024
OBSERVATIONAL
Conditions
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
Inclusion Criteria1
- 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 Criteria1
- Not having such a diagnosis and/or not being related to such individual.
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Locations(11)
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NCT06553976