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NCT Number: NCT06529146

Real-World Data Study of Troriluzole-Treated Patients With Spinocerebellar Ataxia (SCA) Compared to a Matched Natural History Control

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.

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Key information

Conditions

Spinocerebellar Ataxias Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Ataxia Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Nervous System Diseases Neurodegenerative Diseases Neurologic Manifestations Spinal Cord Diseases Spinocerebellar Degenerations

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Biohaven

New Haven, Connecticut, 06510, United States

About this study

This study comprises multiple sources of RWD including: 1) the Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA/US SCA Natural History cohort; 2) the European Integrated Project on Spinocerebellar Ataxias (EUROSCA/European 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 and/or 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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria for troriluzole-treated participants (BHV4157-206):

  • Between the ages of 18-75
  • Genetic confirmation of the following specific hereditary ataxias: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8, and SCA10
  • Screening f-SARA total score of ≥3 and score of ≥1 on gait item of the f-SARA.
  • Ability to ambulate 8 meters without human assistance (canes and other devices were allowed)
  • Subjects initially randomized to troriluzole

Key Inclusion Criteria for participants selected from the natural history studies:

  • Between ages of 18-75
  • CRC-SCA: either a genetic confirmation or a diagnosis of SCA 1, 2, 3, 6, 7, 8, and 10 in themselves or a family member; EUROSCA: genetic confirmation of SCA genotypes 1, 2, 3 and 6.

Key Exclusion Criteria for troriluzole-treated participants (BHV4157-206 study):

  • Screening f-SARA score of 4 on any item of the f-SARA
  • Any other medical condition that could predominantly explain or contribute significantly to the subjects' symptoms of ataxia or that could confound assessment of ataxia symptoms

Key Exclusion Criteria for participants selected from the natural history studies:

  • Treatment with troriluzole

Treatment and study plan

BHV-4157

Drug

BHV-4157 (troriluzole) 200 mg QD

Primary outcomes

  1. 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

    Time frame: Up to 3 years of treatment

    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.

Secondary outcomes

  1. 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

    Time frame: Up to 2 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.

  2. 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

    Time frame: Up to 1 year 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.

  3. 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

    Time frame: Up to 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.

  4. 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

    Time frame: Up to 2 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.

  5. 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

    Time frame: Up to 1 year 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.

  6. 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

    Time frame: Up to 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.

  7. 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

    Time frame: Up to 2 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.

  8. 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

    Time frame: Up to 1 year 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.

  9. 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

    Time frame: Up to 3 years of treatment

    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.

  10. Change from baseline in SCACOMS at Year 2 in troriluzole-treated subjects is compared to that of in CRC-SCA natural history subjects

    Time frame: Up to 2 years of treatment

    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.

  11. Change from baseline in SCACOMS at Year 1 in troriluzole-treated subjects is compared to that of in CRC-SCA natural history subjects

    Time frame: Up to 1 year of treatment

    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.

Sponsors and collaborators

Lead sponsor

Biohaven Therapeutics Ltd.

Industry

Registry information

Official study 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

Important dates

Study start
2019
Primary completion
2024
Study completion
2026
First posted
Jul 31, 2024
Registry last updated
Jun 9, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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