Trial in Adult Subjects With Spinocerebellar Ataxia

Biohaven

Status and phase: Completed; Phase 3; Phase 2

Conditions: Spinocerebellar Ataxia Genotype Type 10; Spinocerebellar Ataxia Genotype Type 3; Spinocerebellar Ataxia Genotype Type 2; Spinocerebellar Ataxias; Spinocerebellar Ataxia Genotype Type 6; Spinocerebellar Ataxia Genotype Type 8; Spinocerebellar Ataxia Genotype Type 7; Spinocerebellar Ataxia Genotype Type 1

Treatments: Drug: Placebo; Drug: Troriluzole

Study type: Interventional

Funder types: Industry

Identifiers

NCT02960893

BHV4157-201

Details and patient eligibility

About

The primary purpose of this study is to compare the efficacy of BHV-4157 (Troriluzole) 140 milligrams (mg) once daily versus placebo after 8 weeks of treatment in subjects with spinocerebellar ataxia (SCA).

Full description

The study was conducted in 2 phases: Randomization Phase (8 weeks) followed by an open-label Extension Phase (48 weeks). During the Randomization Phase, participants received either Troriluzole 140 mg or matching placebo up to 8 weeks. Participants who agreed to enter the Extension Phase continued dosing of Troriluzole 140 mg for 48 weeks. The study was subsequently amended to follow participants for a total of 192 weeks in the Extension Phase.

Enrollment: 141 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Subjects with a known or suspected diagnosis of the following specific hereditary ataxias: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8 and SCA10
• Ability to ambulate 8 meters without assistance (canes and other devices allowed)
• Screening total Scale for the Assessment and Rating of Ataxia (SARA) score ≥8
• Score of ≥ 2 on the gait subsection of the SARA
• Determined by the investigator to be medically stable at baseline/randomization and must be physically able and expected to complete the trial as designed

Key Exclusion Criteria:

• Any medical condition other than one of the hereditary ataxias specified in the inclusion criteria that could predominantly explain or contribute significantly to the subjects' symptoms of ataxia
• Mini Mental State Exam (MMSE) score < 24
• SARA total score of > 30 points at screening
• Clinical history of stroke
• Active liver disease or a history of hepatic intolerance to medications that in the investigator's judgment, is medically significant

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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141 participants in 2 patient groups, including a placebo group

Troriluzole

Experimental group

Description:

Troriluzole - Randomization Phase: Participants received Troriluzole 140 mg capsules orally once daily (QD) for 8 weeks. Troriluzole/Troriluzole - Extension Phase: Participants received Troriluzole 140 mg capsules orally QD for 48 weeks.

Treatment:

Drug: Troriluzole

Drug: Troriluzole

Placebo

Placebo Comparator group

Description:

Placebo - Randomization Phase: Participants received matching placebo capsules orally QD for 8 weeks. Placebo/Troriluzole - Extension Phase: Participants who received placebo during randomization phase, received Troriluzole 140 mg capsules orally QD for 48 weeks.

Treatment:

Drug: Troriluzole

Drug: Troriluzole

Drug: Placebo