DDrare: Database of Drug Development for Rare Diseases

18. 脊髄小脳変性症（多系統萎縮症を除く。）
［臨床試験数：59，薬物数：78（DrugBank：28），標的遺伝子数：44，標的パスウェイ数：59］

Searched query = "Spinocerebellar degeneration", "SCD", "Spinocerebellar ataxia type I", "SCA1", "Spinocerebellar ataxia type II", "SCA2", "Spinocerebellar ataxia type III", "SCA3", "Machado-Joseph disease", "Spinocerebellar ataxia type VI", "SCA6", "Spinocerebellar ataxia type VII", "SCA7", "Spinocerebellar ataxia type X", "SCA10", "Spinocerebellar ataxia type XII", "SCA12", "Dentatorubural pallidoluysian atrophy", "Dentatorubropallidoluysial atrophy", "DRPLA", "Naito-Koyanagi disease", "Friedreich ataxia", "FRDA", "Ataxia with vitamin E deficiency", "AVED", "Early-onset ataxia with ocular motor ataxia and hypoalbuminemia", "EOAH"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = BINOCRIT*1SIR 40000UI/1ML; Baclofen; Bupropion; Bupropion & Citalopram; Butylphthalide; C-Trelin OD Tab(5mg Taltirelin Hydrate); CTI-1601; Cabaletta; Cabaletta for IV infusion once weekly during 24 weeks; Carbonate; Citalopram; Coenzyme Q10; Deferiprone; EGB 761 (r) 120 mg; EGb 761 120 mg; EPOETIN ALFA; EPREX*1FL 40000UI/ML 1ML; EPREX*1SIR 10000UI 1ML; EPREX*1SIR 40000UI/ML 1ML; EU1/97/031-032; EU1/97/031/031-032; Epoeitin beta; Epoetin alfa; Erythropoietin; Etravirine; Etravirine Tablets; Flumazenil; Gamma interferon; Ginkgo; Ginkgo biloba; Granocyte; H-116PI-DE.pdf; High-dose intravenous immunoglobulin (IVIG); IMUKIN*SC 6F 0,5ML 100MCG; INTERFERON GAMMA-1B; Idebenone; Interferon Gamma; Interferon Gamma-1b; Interferon gamma-1b; Iron; Iron chelating intervention; KPS-0373; KPS-0373, High dose; KPS-0373, Low dose; Lenograstim; Lithium Carbonate; Lithium carbonate; Lu AA24493; MIN-102; Methylprednisolone; NICOTINAMIDE; Neo-Recormon; Neorecormon; Nicotinamide; Nicotinamide Riboside; Nicotinamide riboside; Omaveloxolone; Omaveloxolone Capsules, 10 mg; Omaveloxolone Capsules, 150 mg; Omaveloxolone Capsules, 160 mg; Omaveloxolone Capsules, 2.5 mg; Omaveloxolone Capsules, 20 mg; Omaveloxolone Capsules, 300 mg; Omaveloxolone Capsules, 40 mg; Omaveloxolone Capsules, 5 mg; Omaveloxolone Capsules, 80 mg; Other: Exercise Intervention; RHuG-CSF; Resveratrol; Riluzole; Riluzole PMCS; Rokan (r) Novo 120 mg; Rosuvastatin; SUB09246MIG; Standardized Ginkgo Biloba Extract (EGb 761 (r); TAK-831; Varenicline; Vatiquinone

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT04577352 (ClinicalTrials.gov)	November 17, 2020	30/9/2020	A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia	A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study With Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich Ataxia (MOVE-FA)	Friedreich Ataxia	Drug: Vatiquinone;Drug: Placebo	PTC Therapeutics	NULL	Not yet recruiting	7 Years	N/A	All	126	Phase 2;Phase 3	United States;Australia;Canada;Italy

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

NCT04577352
(ClinicalTrials.gov)

November 17, 2020

30/9/2020

A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study With Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich Ataxia (MOVE-FA)

Friedreich Ataxia

Drug: Vatiquinone;Drug: Placebo

PTC Therapeutics

NULL

Not yet recruiting

7 Years

N/A

All

126

Phase 2;Phase 3

United States;Australia;Canada;Italy