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Glutamate Modulator

Troriluzole for Spinocerebellar Ataxia

Phase 3
Waitlist Available
Research Sponsored by Biohaven Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Subjects with a known or suspected diagnosis of specific hereditary ataxias: SCA1, SCA2, SCA3, SCA7, and SCA10
Subjects with a known or suspected diagnosis of the following specific hereditary ataxias: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8 and SCA10; currently only enrolling SCA 1, SCA2, SCA3, SCA7, and SCA10 (the cap has been met for SCA6 and SCA8 (on May 31, 2019));
Must not have
MMSE score <24
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to week 48
Awards & highlights
Pivotal Trial

Summary

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.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
While not specified here, potential side effects for Troriluzole may include nausea, dizziness, fatigue or other common drug-related reactions. The exact side effects will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have or might have a specific type of hereditary ataxia (SCA1, SCA2, SCA3, SCA7, or SCA10).
Select...
I have been diagnosed with a specific type of hereditary ataxia (SCA1, SCA2, SCA3, SCA7, or SCA10).
Select...
My condition has been genetically confirmed by a certified lab, or I have a family member with a confirmed diagnosis and I am willing to undergo genetic testing.
Select...
I can walk 8 meters by myself, but I can use a cane or other aid if needed.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
My memory and thinking skills test score is below 24.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to week 48
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to week 48 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change from Baseline in the total score of the Modified Functional Scale for the Assessment and Rating of Ataxia (f-SARA) after 48 weeks of treatment.
Secondary study objectives
1. Change from baseline in Patient Impression of Function and Activities of Daily Living Scale (PIFAS) score at Randomization Phase Week 48
Change from baseline in Activities of Daily Living Scale from the Friedreich's Ataxia Rating Scale (FARS-ADL) at Randomization Week 48.
Ataxia

Side effects data

From 2021 Phase 2 trial • 350 Patients • NCT03605667
11%
Fall
9%
Weight decreased
9%
Decreased appetite
8%
Diarrhoea
8%
Urinary tract infection
6%
Dizziness
5%
Oedema peripheral
5%
Anxiety
3%
Liver function test increased
3%
Headache
2%
Pulmonary embolism
1%
Facial bones fracture
1%
Lacunar infarction
1%
Fatigue
1%
Atrial fibrillation
1%
Cardiac arrest
1%
Myocardial infarction
1%
Vertigo
1%
Pancreatitis
1%
Small intestinal obstruction
1%
Upper gastrointestinal haemorrhage
1%
Hypothermia
1%
Pelvic mass
1%
Hepatic lesion
1%
Clostridium difficile colitis
1%
Colonic abscess
1%
Diverticulitis
1%
Pneumonia
1%
Sepsis
1%
Frostbite
1%
Hip fracture
1%
Back pain
1%
Breast cancer
1%
Non-small cell lung cancer
1%
Neuropsychiatric symptoms
1%
Aggression
1%
Major depression
1%
Paranoia
1%
Somnambulism
1%
Respiratory failure
1%
Deep vein thrombosis
1%
Thrombosis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Troriluzole - Randomization Phase
Placebo - Randomization Phase/ Troriluzole - OLE Phase
Troriluzole - Randomization Phase/ Troriluzole - OLE Phase
Placebo - Randomization Phase

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: Arm 1: BHV-4157Experimental Treatment1 Intervention
Troriluzole 200mg by mouth
Group II: Arm 2: PlaceboPlacebo Group1 Intervention
Placebo 200mg by mouth
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
troriluzole
2018
Completed Phase 2
~350

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Spinocerebellar Ataxias (SCA) often focus on modulating glutamate activity, as excessive glutamate can lead to neurotoxicity and neuronal damage. Troriluzole, for example, is designed to reduce glutamate release and enhance its reuptake, thereby protecting neurons from excitotoxicity. This mechanism is crucial for SCA patients because it aims to slow down the progression of neurodegeneration and alleviate symptoms associated with the disease. By stabilizing glutamate levels, these treatments help maintain neuronal function and potentially improve motor coordination and quality of life for SCA patients.
Glutamate stimulates [3H]phorbol 12,13-dibutyrate binding in cultured Bergmann glia cells.Blockade of pilocarpine-induced cerebellar phosphoinositide hydrolysis with metabotropic glutamate antagonists: evidence for an indirect control of granule cell glutamate release by muscarinic agonists.

Find a Location

Who is running the clinical trial?

Biohaven Pharmaceuticals, Inc.Lead Sponsor
48 Previous Clinical Trials
37,383 Total Patients Enrolled
2 Trials studying Spinocerebellar Ataxias
141 Patients Enrolled for Spinocerebellar Ataxias

Media Library

Troriluzole (Glutamate Modulator) Clinical Trial Eligibility Overview. Trial Name: NCT03701399 — Phase 3
Spinocerebellar Ataxias Research Study Groups: Arm 2: Placebo, Arm 1: BHV-4157
Spinocerebellar Ataxias Clinical Trial 2023: Troriluzole Highlights & Side Effects. Trial Name: NCT03701399 — Phase 3
Troriluzole (Glutamate Modulator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03701399 — Phase 3
Spinocerebellar Ataxias Patient Testimony for trial: Trial Name: NCT03701399 — Phase 3
~32 spots leftby Dec 2025