Hereditary Spastic Paraplegia Clinical Trial
— HSPOfficial title:
Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP)
NCT number | NCT04712812 |
Other study ID # | P00033016 |
Secondary ID | |
Status | Recruiting |
Phase | |
First received | |
Last updated | |
Start date | April 27, 2020 |
Est. completion date | April 26, 2025 |
The Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP) is focused on gathering longitudinal clinical data as well as biological samples (skin and/or blood and/or saliva) from male or female patients who exhibited onset of HSP symptoms at 18 years old or younger with (1) a clinical diagnosis of hereditary spastic paraplegia and/or (2) the presence of variants in HSP related genes and/or be a relative of a person with such a diagnosis. Currently, the treatment for this disorder is generally symptomatic and available therapies improve quality of life, but are grossly inefficient in slowing the disease progression. Access to the registry information will be limited to the study staff who are responsible for recruitment and maintenance of the registry. We hope that recruitment into registry for studies will advance knowledge of the causes, clinical course, diagnosis and treatment of these conditions.
Status | Recruiting |
Enrollment | 400 |
Est. completion date | April 26, 2025 |
Est. primary completion date | April 26, 2024 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | All |
Age group | 30 Days and older |
Eligibility | Inclusion Criteria: - Onset of hereditary spastic paraplegia symptoms before the age of 18 years - The presence of variants in HSP related genes and/or a relative of a person with such a diagnosis Exclusion Criteria: - Not having such a diagnosis and/or not being related to such individual |
Country | Name | City | State |
---|---|---|---|
United States | Boston Children's Hospital | Boston | Massachusetts |
Lead Sponsor | Collaborator |
---|---|
Boston Children's Hospital | CureAP4 Foundation |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Establishment of data repository | Create a registry to perform an initial cross sectional analysis of clinical, imaging and molecular data to establish the disease spectrum. | Through study completion, an average of 1 year | |
Primary | Establishment of bio-repository | Create a repository of biological samples and collection of longitudinal clinical data that helps establish the natural history of early onset HSP. | Through study completion, an average of 1 year | |
Primary | Registry for recontact | Create a registry that allows for re-identification and re-contact of participants by appropriate investigators. | Through study completion, an average of 1 year |
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