Troriluzole for Spinocerebellar Ataxia

Recruiting at 22 trial locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Biohaven Pharmaceuticals, Inc.

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This trial is testing whether a medication called Troriluzole can help people with spinocerebellar ataxia by balancing a brain chemical to prevent damage. Troriluzole is related to riluzole, which has been shown to prolong survival and slow functional deterioration in patients with ALS.

Eligibility Criteria

Adults with spinocerebellar ataxia (SCA) types SCA1, SCA2, SCA3, SCA7, and SCA10 who can walk 8 meters unaided (devices okay), have a specific score on the f-SARA gait test, and are medically stable. Those with severe cognitive impairment or conditions that could affect their ataxia severity aren't eligible.

Inclusion Criteria

Your f-SARA total score is 3 or higher during screening.

I have or might have a specific type of hereditary ataxia (SCA1, SCA2, SCA3, SCA7, or SCA10).

I have been diagnosed with a specific type of hereditary ataxia (SCA1, SCA2, SCA3, SCA7, or SCA10).

Exclusion Criteria

You have severe muscle stiffness or uncontrollable muscle movements that could make it difficult for the SARA instrument to measure the severity of your ataxia.

I have liver disease or a history of bad reactions to medications due to my liver.

My memory and thinking skills test score is below 24.

Treatment Details

Interventions

• Placebos (Drug)
• Troriluzole (Glutamate Modulator)

Trial Overview The trial is testing Troriluzole (200mg daily) against a placebo over 48 weeks to see if it's more effective in treating symptoms of spinocerebellar ataxia. Participants will be randomly assigned to either the drug or placebo group.

Participant Groups

Group I: Troriluzole Experimental Treatment 1 Intervention

Randomization phase (Randomization through Week 48): Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48-week duration of the double-blind randomization phase. Open-label extension (OLE) phase (OLE Week 1 up to Week 192): Participants who were eligible and agreed to enroll in an OLE phase, will receive troriluzole 200 mg capsules orally once daily for 4 weeks in a blinded manner. After OLE Week 4, all participants will then receive open label troriluzole 200 mg up to Week 192.

Group II: Placebo Placebo Group 2 Interventions

Randomization phase (Randomization through Week 48): Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase. OLE phase (Week 1 up to Week 192): Participants who were eligible and agreed to enroll in an OLE phase, will receive troriluzole 140 mg capsules orally once daily for the first 4 weeks in a blinded manner. After OLE Week 4, all participants will then receive open label troriluzole 200 mg up to Week 192.

Who Is Running the Clinical Trial?

Biohaven Pharmaceuticals, Inc.

Lead Sponsor

Trials

49

Recruited

30,100+