Ignite Creation Date:
2024-10-26 @ 3:37 PM
Last Modification Date:
2024-10-26 @ 3:37 PM
Study NCT ID:
NCT06553976
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-08-12
Brief Title:
Spastic Paraplegia - Centers of Excellence Research Network
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Spastic Paraplegia - Centers of Excellence Research Network SP-CERN - Natural History Study Pilot
Status:
RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
SP-CERN
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 HSPs 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
Detailed Description:
The hereditary spastic paraplegias HSPs include over 80 rare neurogenetic disorders collectively representing the most prevalent cause of inherited spasticity and related disabilities globally In all forms of HSP there is a progressive deterioration of the long axonal tracts resulting in substantial motor dysfunction and various other symptoms Primary lateral sclerosis PLS is a related degenerative neurological disorder characterized by the progressive deterioration of upper motor neurons Both conditions result in muscle weakness and spasticity with significant morbidity and impact on quality of life
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 SP-CERN provides a registry and natural history study across the whole age span a biobank and a genome archive This will set the stage for a multitude of opportunities for improved diagnosis and trial readiness A second objective is to harmonize this effort with similar consortia especially in Europe in addition to Asia South America and Africa to help accelerate basic and clinical research on HSP and PLS on a global level In summary SP-CERN will support critical research infrastructure for collaborative high-quality research on HSP and PLS in North America and beyond
General aims include
A Establish a shared clinical database a repository of biospecimen samples and a central database for the storage of all genetic data in SP-CERN
B Synchronize and harmonize collaborations between institutions clinical sites and international collaborators through the development of a central research protocol in order to standardize outcome measures and maximize the quality of research and data to ensure clinical trial readiness by regulatory standards
C Build comprehensive programs for advancements in diagnosis provide more opportunities for innovative treatments and increase access to high-quality healthcare for HSP and PLS patients
Specific aims for this first pilot study are
1a Enrollment of 100 individuals with genetically-confirmed hereditary spastic paraplegia type 4 SPG4 or hereditary spastic paraplegia type 5A SPG5A in the shared clinical database
1b Biobanking of blood samples from 100 individuals with SPG4 or SPG5A in a shared biobank
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 SP-CERN provides a registry and natural history study across the whole age span a biobank and a genome archive This will set the stage for a multitude of opportunities for improved diagnosis and trial readiness A second objective is to harmonize this effort with similar consortia especially in Europe in addition to Asia South America and Africa to help accelerate basic and clinical research on HSP and PLS on a global level In summary SP-CERN will support critical research infrastructure for collaborative high-quality research on HSP and PLS in North America and beyond
General aims include
A Establish a shared clinical database a repository of biospecimen samples and a central database for the storage of all genetic data in SP-CERN
B Synchronize and harmonize collaborations between institutions clinical sites and international collaborators through the development of a central research protocol in order to standardize outcome measures and maximize the quality of research and data to ensure clinical trial readiness by regulatory standards
C Build comprehensive programs for advancements in diagnosis provide more opportunities for innovative treatments and increase access to high-quality healthcare for HSP and PLS patients
Specific aims for this first pilot study are
1a Enrollment of 100 individuals with genetically-confirmed hereditary spastic paraplegia type 4 SPG4 or hereditary spastic paraplegia type 5A SPG5A in the shared clinical database
1b Biobanking of blood samples from 100 individuals with SPG4 or SPG5A in a shared biobank
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None