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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT02960893
Other study ID # BHV4157-201
Secondary ID
Status Active, not recruiting
Phase Phase 2/Phase 3
First received
Last updated
Start date December 15, 2016
Est. completion date September 23, 2024

Study information

Verified date April 2024
Source Biohaven Pharmaceuticals, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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).


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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 141
Est. completion date September 23, 2024
Est. primary completion date August 18, 2017
Accepts healthy volunteers No
Gender All
Age group 18 Years to 75 Years
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Troriluzole
Randomization Phase: Neat (i.e., drug substance without excipients); loose filled capsule.
Placebo
Drug: Placebo Randomization Phase: Matching placebo loose filled capsule.
Troriluzole
Extension phase: Neat capsule or formulated capsule (i.e., drug substance with excipients).

Locations

Country Name City State
United States University of Michigan Ann Arbor Michigan
United States Emory University Atlanta Georgia
United States Johns Hopkins University Baltimore Maryland
United States Harvard University (Beth Israel Deaconess Medical Center) Boston Massachusetts
United States Harvard University (Massachusetts General Hospital) Boston Massachusetts
United States Northwestern University Chicago Illinois
United States University of Chicago Chicago Illinois
United States University of Texas Southwestern Dallas Texas
United States University of Colorado Denver Denver Colorado
United States University of Florida Gainesville Florida
United States Houston Methodist Research Center Houston Texas
United States CNS Trial Long Beach California
United States University of California, Los Angeles Los Angeles California
United States Columbia University New York New York
United States St. Joseph's Hospital and Medical Center Phoenix Arizona
United States University of Rochester Medical Center Rochester New York
United States University of California, San Francisco San Francisco California
United States University of South Florida Tampa Florida

Sponsors (1)

Lead Sponsor Collaborator
Biohaven Pharmaceuticals, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Other Change From Randomization Baseline in Total Score on the Scale for the Assessment and Rating of Ataxia (SARA) at Extension Phase Week 48 The severity of ataxia was assessed with the SARA, an 8-item clinical rating scale from 0 (no ataxia) to 40 (most severe ataxia). The total score was derived as the sum of the individual items, which included gait (0-8), stance (0-6), sitting (0-4), speech disturbance (0-6), finger chase (0-4), nose-finger test (0-4), fast alternating hand movements (0-4), and heel-shin slide (0-4). Since the finger chase, nose-finger test, fast alternating hand movements, and heel-shin slide were repeated on both the right and left side, the average of the right and left side assessments was used to derive the total score. A negative change in score shows improvement. Randomization Baseline, Extension Phase Week 48
Primary Change From Baseline in Total Score on the Scale for the Assessment and Rating of Ataxia (SARA) at Randomization Phase Week 8 The severity of ataxia was assessed with the SARA, an 8-item clinical rating scale from 0 (no ataxia) to 40 (most severe ataxia). The total score was derived as the sum of the individual items, which included gait (0-8), stance (0-6), sitting (0-4), speech disturbance (0-6), finger chase (0-4), nose-finger test (0-4), fast alternating hand movements (0-4), and heel-shin slide (0-4). Since the finger chase, nose-finger test, fast alternating hand movements, and heel-shin slide were repeated on both the right and left side, the average of the right and left side assessments was used to derive the total score. A negative change in score shows improvement. Baseline, Randomization Phase Week 8
Secondary Number of Participants With Deaths, Serious Adverse Events (SAEs), Adverse Events (AEs) Leading to Discontinuation, and Treatment-Emergent AEs (TEAEs) During the Randomization Phase An AE was defined as any new untoward medical occurrence or worsening of a pre-existing medical condition, unfavorable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not related to study drug. An SAE was defined as an event which was fatal or life threatening, required or prolonged hospitalization, was significantly or permanently disabling or incapacitating, constituted a congenital anomaly or a birth defect, or suspected transmission of an infectious agent, or encompassed any other clinically significant event that could jeopardize the subject or require medical or surgical intervention to prevent one of the aforementioned outcomes. TEAEs were defined as those AEs that developed, worsened, or became serious after the first dose of study drug. AEs from first dose of study drug to 2 weeks after the last dose (up to 10 weeks). SAEs from signing of informed consent form (ICF) to the start of the Extension (Ext) Phase or 30 days after the last dose (up to 12 weeks).
Secondary Number of Participants With Deaths, Serious Adverse Events (SAEs), Adverse Events (AEs) Leading to Discontinuation, and Treatment Emergent AEs (TEAEs) During the Extension Phase An AE was defined as any new untoward medical occurrence or worsening of a pre-existing medical condition, unfavorable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not related to study drug. An SAE was defined as an event which was fatal or life threatening, required or prolonged hospitalization, was significantly or permanently disabling or incapacitating, constituted a congenital anomaly or a birth defect, or suspected transmission of an infectious agent, or encompassed any other clinically significant event that could jeopardize the subject or require medical or surgical intervention to prevent one of the aforementioned outcomes. TEAEs were defined as those AEs that developed, worsened, or became serious after the first dose of study drug. AEs from first dose Ext Phase troriluzole to 2 weeks after last dose (up to 50 weeks after last subject enrolled in Ext Phase). SAEs from signing of ICF to 30 days after last dose (up to 52 weeks after last subject enrolled in Ext Phase).
Secondary Number of Participants Who Received at Least One Dose of Troriluzole in the Randomization Phase or Extension Phase With Deaths, Serious Adverse Events (SAEs), Adverse Events (AEs) Leading to Discontinuation, and Treatment Emergent AEs (TEAEs) An AE was defined as any new untoward medical occurrence or worsening of a pre-existing medical condition, unfavorable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not related to study drug. An SAE was defined as an event which was fatal or life threatening, required or prolonged hospitalization, was significantly or permanently disabling or incapacitating, constituted a congenital anomaly or a birth defect, or suspected transmission of an infectious agent, or encompassed any other clinically significant event that could jeopardize the subject or require medical or surgical intervention to prevent one of the aforementioned outcomes. TEAEs were defined as those AEs that developed, worsened, or became serious after the first dose of study drug. AEs from first dose of troriluzole to 2 weeks after last dose (up to 50 weeks after last Ext subject enrolled or up to 58 weeks after last Randomization [Rand] subject enrolled). SAEs from signing of ICF to 30 days after the last dose of troriluzole.
Secondary Number of Participants With Impression of Benefit Via Use of the Patient Global Impression of Change (PGI-C) Index Scale During Randomization Phase PGI-C is a patient self-reported global index scale that was used to rate the response of a condition to therapy. Participants rated their impression of benefit based on the following 7 categories: No change (or condition has gotten worse); Almost the same, hardly any change at all; A little better, but no noticeable change; Somewhat better, but the change has not made any real difference; Moderately better, and a slight but noticeable change; Better and a definitive improvement that has made a real and worthwhile difference; A great deal better and a considerable improvement that has made all the difference. Randomization Phase Week 8
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