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Last update 08 May 2025

Spinocerebellar Ataxia 8

Last update 08 May 2025

Basic Info

Synonyms

SCA8, SCA8 Spinocerebellar ataxia 8, SPINOCEREBELLAR ATAXIA 8

+ [6]

Introduction

Main features described as cerebellar ataxia and cognitive dysfunction in almost three quarters of patients and pyramidal and sensory signs in approximately a third of patients. Other features include dysexecutive disorders and commonly psychiatric disorders.

Drugs associated with Spinocerebellar Ataxia 8

Trigriluzole

Target

EAAT2 x NMDA receptor x Nav1.5 x VGSCs

Mechanism

EAAT2 modulators [+3]

Active Org.

Biohaven Pharmaceuticals, Inc. [+3]

Originator Org.

Yale University

Active Indication

Machado-Joseph Disease [+7]

Inactive Indication

Alzheimer Disease [+2]

Drug Highest PhaseNDA/BLA

First Approval Ctry. / Loc.-

First Approval Date20 Jan 1800

CAD-1883

Target

Potassium channel

Mechanism

Potassium channel agonists

Active Org.

Saniona AB

Originator Org.

Cadent Therapeutics, Inc.

Active Indication

Essential Tremor [+1]

Inactive Indication

Machado-Joseph Disease [+4]

Drug Highest PhasePhase 1

First Approval Ctry. / Loc.-

First Approval Date20 Jan 1800

ENZ-001

Target

DMPK

Mechanism

DMPK gene modulators

Active Org.

Enzerna Biosciences, Inc.

Originator Org.

University of North Carolina System

Active Indication

Fuchs' Endothelial Dystrophy [+2]

Inactive Indication-

Drug Highest PhasePreclinical

First Approval Ctry. / Loc.-

First Approval Date20 Jan 1800

Clinical Trials associated with Spinocerebellar Ataxia 8

NCT04301284

/ WithdrawnPhase 2

Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, and Efficacy of CAD-1883 in Adults With Spinocerebellar Ataxia (Synchrony-1)

This is a randomized, double-blind, placebo-controlled Phase 2 study evaluating oral administration of CAD-1883 in the treatment of adults with a genotypic diagnosis of Spinocerebellar Ataxia (SCA). This study offers the opportunity to understand the safety, tolerability, and efficacy of CAD-1883 in the SCA patient population.

Start Date01 Jun 2021

Sponsor / Collaborator

Cadent Therapeutics, Inc.

NCT03701399

/ Active, not recruitingPhase 3

A Phase III, Long-Term, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Participants With Spinocerebellar Ataxia.

The purpose of this study is to compare the efficacy of Troriluzole (200 mg once daily) versus placebo after 48 weeks of treatment in subjects with spinocerebellar ataxia (SCA).

Start Date08 Mar 2019

Sponsor / Collaborator

Biohaven Pharmaceuticals, Inc.

NCT02960893

/ CompletedPhase 2/3

A Phase IIb/III, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Subjects With Spinocerebellar Ataxia

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).

Start Date15 Dec 2016

Sponsor / Collaborator

Biohaven Pharmaceuticals, Inc.

100 Clinical Results associated with Spinocerebellar Ataxia 8

100 Translational Medicine associated with Spinocerebellar Ataxia 8

0 Patents (Medical) associated with Spinocerebellar Ataxia 8

189

Literatures (Medical) associated with Spinocerebellar Ataxia 8

01 Jun 2025·Parkinsonism & Related Disorders

Expert Commentary for “A case of spinocerebellar ataxia type 8 with clinical manifestations as Parkinson's disease”

Article

Author: Cen, Zhidong ; Luo, Wei ; Zheng, Xiaosheng

01 Mar 2025·Neurobiology of Disease

Cerebral cortical functional hyperconnectivity in a mouse model of spinocerebellar ataxia type 8 (SCA8)

Article

Author: Manikonda, Keerthi ; Popa, Laurentiu S ; Gerhart, Morgan L ; Ranum, Laura P W ; Ebner, Timothy J ; Nietz, Angela K ; Carter, Russell E

Spinocerebellar Ataxia Type 8 (SCA8) is an inherited neurodegenerative disease caused by a bidirectionally expressed CTG•CAG expansion mutation in the ATXN8 and ATXN8OS genes. While SCA8 patients have motor abnormalities, patients may also exhibit psychiatric symptoms and cognitive dysfunction. It is difficult to elucidate how the disease alters brain function in areas with little or no degeneration producing both motor and cognitive symptoms. Using transparent polymer skulls and CNS-wide GCaMP6f expression, we studied neocortical networks throughout SCA8 progression using wide-field Ca²⁺ imaging in a transgenic mouse model of SCA8. Compared to wild-type controls, neocortical networks in SCA8+ mice were hyperconnected globally, which leads to network configurations with increased global efficiency and centrality. At the regional level, significant network changes occurred in nearly all cortical regions, however mainly involved sensory and association cortices. Changes in functional connectivity in anterior motor regions worsened later in the disease. Near perfect decoding of animal genotype was obtained using a generalized linear model based on canonical correlation strengths between activity in cortical regions. The major contributors to decoding were concentrated in the somatosensory, higher visual and retrosplenial cortices and occasionally extended into the motor regions, demonstrating that the areas with the largest network changes are predictive of disease state.

01 Feb 2025·Parkinsonism & Related Disorders

A case of spinocerebellar ataxia type 8 with clinical manifestations as Parkinson's disease

Article

Author: Ran, Ran ; Guo, Yi ; Wang, Yi ; Mima, Dunzhu ; Wang, Lin

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