Troriluzole for Spinocerebellar Ataxia

Not currently recruiting at 22 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Biohaven Pharmaceuticals, Inc.

Disqualifiers: Spasticity, Dystonia, Liver disease, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 48 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a potential new treatment, Troriluzole, for individuals with spinocerebellar ataxia (SCA), a genetic condition affecting coordination and balance. The goal is to determine if Troriluzole is more effective than a placebo (a non-active substance) over 48 weeks. Participants will receive either Troriluzole or a placebo during this period and may continue with the study for further observation. It suits individuals with a specific type of hereditary ataxia who can walk with or without assistance and are medically stable. As a Phase 3 trial, this study represents the final step before potential FDA approval, offering participants a chance to contribute to a treatment nearing widespread availability.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. Please consult with the trial team for guidance.

Is there any evidence suggesting that Troriluzole is likely to be safe for humans?

Research has shown that Troriluzole is generally safe and well-tolerated by patients. Studies from six different trials indicate that it usually does not cause liver problems, which supports its overall safety. These studies reported no major adverse events or serious side effects. This evidence suggests that, based on current research, Troriluzole is safe for use in humans. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for spinocerebellar ataxia?

Troriluzole is unique because it targets glutamate regulation, a new approach in treating spinocerebellar ataxia. Unlike current treatments that mainly focus on symptom management, troriluzole aims to address the underlying neurological issues by modulating glutamate, potentially offering a more comprehensive benefit. Researchers are excited about troriluzole because this novel mechanism could slow disease progression, offering hope for improved quality of life for patients.

What evidence suggests that Troriluzole might be an effective treatment for spinocerebellar ataxia?

Research has shown that Troriluzole, which participants in this trial may receive, might slow the progression of spinocerebellar ataxia (SCA). Studies have found that it can delay the disease by 1.5 to 2.2 years compared to no treatment. In one important study, Troriluzole met its primary goal by improving patients' ability to perform daily activities. This suggests that Troriluzole might help people with SCA maintain or even enhance their daily functioning. Overall, these findings provide promising evidence that Troriluzole could be a helpful treatment for managing SCA. ^{6 7 8 9 10}

Are You a Good Fit for This Trial?

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.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Troriluzole or placebo for 48 weeks in a double-blind randomization phase

48 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants may opt into continuation of treatment with open-label Troriluzole up to Week 192

192 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Placebos
• Troriluzole

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

In a case series of 13 patients with degenerative cerebellar ataxia, treatment with acetyl-DL-leucine (5 g/day) for one week led to significant improvements in motor function, as measured by the Scale for the Rating and Assessment of Ataxia (SARA), with a notable decrease in scores from 16.1 to 12.8.

Patients also reported enhanced quality of life during treatment, and importantly, no side effects were observed, suggesting a favorable risk-benefit profile for acetyl-DL-leucine in managing ataxic symptoms.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of acetyl-DL-leucine in patients with cerebellar ataxia: a case series. Strupp, M., Teufel, J., Habs, M., et al.[2021]

In a study of 153 ALS patients, riluzole was found to have an acceptable safety profile, with 50.3% experiencing adverse effects, primarily gastrointestinal issues, hepatotoxicity, and fatigue.

Riluzole's mechanism of action suggests it may also be beneficial for other neurodegenerative diseases related to glutamate excitotoxicity, warranting further investigation and monitoring for its use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Adverse efects of riluzole (Rilutek) in the treatment of amyotrophic lateral sclerosis]. Roch-Torreilles, I., Camu, W., Hillaire-Buys, D.[2013]

Citations

1. biohaven.com biohaven.com/wp-content/uploads/AAN-2025-poster_troriluzole-SCA.pdf

Comparative Effectiveness of Troriluzole versus Untreated ... To examine the treatment benefits of troriluzole over 3 years in patients with SCA by conducting a matched comparison of troriluzole-treated ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT02960893

Trial in Adult Participants With Spinocerebellar Ataxia (SCA) The primary purpose of this study was to compare the efficacy of BHV-4157 ... Trial of Troriluzole in Adult Participants With Spinocerebellar Ataxia.

3. neurology.org neurology.org/doi/10.1212/WNL.0000000000209018

Comparative Effectiveness of Troriluzole Versus Untreated ... These results correspond to 50–70% slowing of disease progression (ie, 1.5 to 2.2 years delay) for troriluzole-treated subjects, compared to the untreated ...

4. prnewswire.com prnewswire.com/news-releases/biohaven-achieves-positive-topline-results-in-pivotal-study-of-troriluzole-in-spinocerebellar-ataxia-sca-302255056.html

Biohaven Achieves Positive Topline Results in Pivotal ... Troriluzole 200 mg dosed orally, once daily, in patients with SCA met the study's primary endpoint on the change from baseline in the modified functional Scale.

5. neurologyadvisor.com neurologyadvisor.com/news/troriluzole-priority-review-granted-for-spinocerebellar-ataxia/

Troriluzole Priority Review Granted for Spinocerebellar ... The BH4157-206-RWE study evaluated the effectiveness of troriluzole after 3 years of treatment in patients with SCA. Troriluzole data from the 3 ...

6. ir.biohaven.com ir.biohaven.com/news-releases/news-release-details/biohaven-achieves-positive-topline-results-pivotal-study

Biohaven Achieves Positive Topline Results in Pivotal ... Troriluzole is the very first treatment to show a delay in disease progression that can give patients additional years of independence.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT03701399

Troriluzole in Adult Participants With Spinocerebellar Ataxia The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure.

8. biohaven.com biohaven.com/wp-content/uploads/BioH-TRO-HepaticSafety-POS-AAN-Final_4-12-24.pdf

Troriluzole Exhibits Favorable Hepatic Safety Profile ... For integration purposes, safety data were pooled by indication and overall for the 6 studies with unblinded and/or open-label data: BHV4157-201.

9. ema.europa.eu ema.europa.eu/en/medicines/human/EPAR/dazluma

Dazluma | European Medicines Agency (EMA) Scores range from 0 to 16 with higher scores indicating more severe ataxia. During the assessment, the company also presented real-world data ...

10. clinicaltrials.gov clinicaltrials.gov/study/NCT02960893

Trial in Adult Participants With Spinocerebellar Ataxia (SCA) The primary purpose of this study was to compare the efficacy of BHV-4157 (Troriluzole) 140 milligrams (mg) once daily versus placebo after 8 weeks of treatment ...