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	NCT02147886 (ClinicalTrials.gov)	July 2014	21/5/2014	Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph Disease	A Single-Center, Randomized, Double-Blind, Parallel-Group, Dose-Controlled Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Patients With Machado-Joseph Disease	Machado-Joseph Disease / Spinocerebellar Ataxia 3	Drug: Cabaletta for IV infusion once weekly during 24 weeks	Bioblast Pharma Ltd.	NULL	Completed	18 Years	75 Years	Both	15	Phase 2	Israel

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

NCT02147886
(ClinicalTrials.gov)

July 2014

21/5/2014

Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph Disease

A Single-Center, Randomized, Double-Blind, Parallel-Group, Dose-Controlled Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Patients With Machado-Joseph Disease

Machado-Joseph Disease / Spinocerebellar Ataxia 3

Drug: Cabaletta for IV infusion once weekly during 24 weeks

Bioblast Pharma Ltd.

NULL

Completed

18 Years

75 Years

Both

Phase 2

Israel