Real-World Data Study of Troriluzole-Treated Patients With Spinocerebellar Ataxia (SCA) Compared to a Matched Natural History Control
NCT Number: NCT06529146
Status: ENROLLING_BY_INVITATION
Study Type: OBSERVATIONAL
Conditions: Spinocerebellar Ataxias
Interventions: DRUG: BHV-4157
Sponsor: Biohaven Therapeutics Ltd.
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 1000
Study Design: Observational Model: |Time Perspective: p
Start Date: 2019-03-18
Completion Date: 2026-01
Locations: Biohaven, New Haven, Connecticut, 06510, United States

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.

Primary Outcome Measures: Change from Baseline in the total score of the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA) at Year 3 in troriluzole-treated subjects is compared to natural history subjects from CRC-SCA, To compare the effectiveness of troriluzole in treating SCA, as measure by the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA), in subjects randomized to treatment with troriluzole relative to natural history controls from the natural history dataset after 3 years of treatment. The f-SARA is a scale with a range of 0 to 16, where an increase in the total score indicates a worsening of symptoms., Up to 3 years of treatment
Secondary Outcome Measures: Change from baseline in the f-SARA at Year 2 in troriluzole-treated subjects is compared to the change from baseline in the mapped f-SARA at Year 2 in natural history subjects from CRC-SCA, The f-SARA is a scale with a range of 0 to 16, where an increase in the total score indicates a worsening of symptoms., Up to 2 years of treatment|Change from baseline in the f-SARA at Year 1 in troriluzole-treated subjects is compared to the change from baseline in the mapped f-SARA at Year 1 in natural history subjects from CRC-SCA, The f-SARA is a scale with a range of 0 to 16, where an increase in the total score indicates a worsening of symptoms., Up to 1 year of treatment|Change from baseline in the f-SARA at Year 3 in troriluzole-treated subjects is compared to the change from baseline in the mapped f-SARA at Year 3 in natural history subjects from EUROSCA, The f-SARA is a scale with a range of 0 to 16, where an increase in the total score indicates a worsening of symptoms., Up to 3 years of treatment|Change from baseline in the f-SARA at Year 2 in troriluzole-treated subjects is compared to the change from baseline in the mapped f-SARA at Year 2 in natural history subjects from EUROSCA, The f-SARA is a scale with a range of 0 to 16, where an increase in the total score indicates a worsening of symptoms., Up to 2 years of treatment|Change from baseline in the f-SARA at Year 1 in troriluzole-treated subjects is compared to the change from baseline in the mapped f-SARA at Year 1 in natural history subjects from EUROSCA, The f-SARA is a scale with a range of 0 to 16, where an increase in the total score indicates a worsening of symptoms., Up to 1 year of treatment|Change from baseline in the f-SARA at Year 3 in troriluzole-treated subjects is compared to the change from baseline in the mapped f-SARA at Year 3 in pooled (CRC-SCA and EUROSCA) natural history subjects, The f-SARA is a scale with a range of 0 to 16, where an increase in the total score indicates a worsening of symptoms., Up to 3 years of treatment|Change from baseline in the f-SARA at Year 2 in troriluzole-treated subjects is compared to the change from baseline in the mapped f-SARA at Year 2 in in pooled (CRC-SCA and EUROSCA) natural history subjects, The f-SARA is a scale with a range of 0 to 16, where an increase in the total score indicates a worsening of symptoms., Up to 2 years of treatment|Change from baseline in the f-SARA at Year 1 in troriluzole-treated subjects is compared to the change from baseline in the mapped f-SARA at Year 1 in pooled (CRC-SCA and EUROSCA) natural history subjects, The f-SARA is a scale with a range of 0 to 16, where an increase in the total score indicates a worsening of symptoms., Up to 1 year of treatment|Change from baseline in Spinocerebellar Ataxia Composite Score (SCACOMS) at Year 3 in troriluzole-treated subjects is compared to that of in CRC-SCA natural history subjects, The SCACOMS (SCA Composite Scale) is a newly developed and validated endpoint for SCA, derived from an analysis of two SCA natural history studies (CRC-SCA and EUROSCA). The score range of SCACOMS is 0-50, where an increase in the total score indicates a worsening of symptoms., Up to 3 years of treatment|Change from baseline in SCACOMS at Year 2 in troriluzole-treated subjects is compared to that of in CRC-SCA natural history subjects, The SCACOMS (SCA Composite Scale) is a newly developed and validated endpoint for SCA, derived from an analysis of two SCA natural history studies (CRC-SCA and EUROSCA). The score range of SCACOMS is 0-50, where an increase in the total score indicates a worsening of symptoms., Up to 2 years of treatment|Change from baseline in SCACOMS at Year 1 in troriluzole-treated subjects is compared to that of in CRC-SCA natural history subjects, The SCACOMS (SCA Composite Scale) is a newly developed and validated endpoint for SCA, derived from an analysis of two SCA natural history studies (CRC-SCA and EUROSCA). The score range of SCACOMS is 0-50, where an increase in the total score indicates a worsening of symptoms., Up to 1 year of treatment