Ignite Creation Date:
2024-10-26 @ 3:36 PM
Last Modification Date:
2024-10-26 @ 3:36 PM
Study NCT ID:
NCT06529146
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
None
First Post:
2024-07-26
Brief Title:
Real-World Data Study of Troriluzole-Treated Patients With Spinocerebellar Ataxia SCA Compared to a Matched Natural History Control
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Real-World Data Study Assessing the Efficacy of Troriluzole-Treated Subjects With Spinocerebellar Ataxia SCA Compared to a Matched External SCA Control Using Natural History Cohort Data
Status:
ACTIVE_NOT_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:
None
Brief Summary:
The purpose of this study is to leverage two sources of real-world data RWD to assess the effectiveness of troriluzole after three years of treatment in patients with SCA by comparison to an external control of untreated patients who were followed in a natural history cohort
Real world evidence of effectiveness will be assessed from the RWD sources to examine the treatment effects of toriluzole in SCA out to 3 years Progression rates of SCA differ by genotype and long-term follow-up is needed to assess for potential efficacy in this rare disease
Real world evidence of effectiveness will be assessed from the RWD sources to examine the treatment effects of toriluzole in SCA out to 3 years Progression rates of SCA differ by genotype and long-term follow-up is needed to assess for potential efficacy in this rare disease
Detailed Description:
This study comprises multiple sources of RWD including 1 the Clinical Research Consortium for the Study of Cerebellar Ataxia CRC-SCAUS SCA Natural History cohort 2 the European Integrated Project on Spinocerebellar Ataxias EUROSCAEuropean SCA Natural History Cohort and 3 the 3 year OLE data from troriluzole treated subjects in Study BHV4157-206 NCT03701399 Each participant of the study will have their efficacy andor safety data collected as pre-specified in the original protocols from the RWD sources
The effectiveness of troriluzole in SCA after 3 years of treatment from the long-term open-label extension from Study BHV4157-206 will be compared to external control subjects collected from CRC-SCA US SCA Natural History cohort and EUROSCA European SCA Natural History Cohort A propensity score matching PSM analysis will be utilized to create equipoise across groups being examined in the analysis
The primary outcome will be change from baseline in the modified functional Scale for the Assessment and Rating of Ataxia f-SARA Another endpoint examined will be a newly developed and validated composite endpoint for SCA the Spinocerebellar Ataxia Composite Score SCACOMS
The effectiveness of troriluzole in SCA after 3 years of treatment from the long-term open-label extension from Study BHV4157-206 will be compared to external control subjects collected from CRC-SCA US SCA Natural History cohort and EUROSCA European SCA Natural History Cohort A propensity score matching PSM analysis will be utilized to create equipoise across groups being examined in the analysis
The primary outcome will be change from baseline in the modified functional Scale for the Assessment and Rating of Ataxia f-SARA Another endpoint examined will be a newly developed and validated composite endpoint for SCA the Spinocerebellar Ataxia Composite Score SCACOMS
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