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	NCT01965327 (ClinicalTrials.gov)	August 2013	27/8/2013	Interferon Gamma-1b in Friedreich Ataxia (FRDA)	Open-label Pilot Study of Interferon Gamma-1b (Actimmune™) for the Treatment of Friedreich Ataxia (FRDA)	Friedreich Ataxia	Drug: Interferon Gamma -1b	Children's Hospital of Philadelphia	Friedreich's Ataxia Research Alliance;Vidara Therapeutics Research Ltd	Completed	5 Years	17 Years	All	12	Phase 2	United States
2	NCT02035020 (ClinicalTrials.gov)	May 2013	12/1/2014	A Phase IIa Trial to Test Safety and Efficacy Interferon Gamma Treatment in Elevating Frataxin Levels in FRDA Patients	A Phase IIa Clinical Trial to Test the Safety and Efficacy of Interferon Gamma Treatment in Elevating Frataxin Levels in Friedreich's Ataxia (FRDA) Patients	Friedreich Ataxia	Drug: gamma interferon	Azienda Policlinico Umberto I	NULL	Completed	18 Years	45 Years	All	10	Phase 2	Italy
3	EUCTR2012-001881-14-IT (EUCTR)	01/03/2013	11/01/2013	A phase II clinical trial to evaluate the safety and efficacy of interferon gamma in elevating frataxin levels in Friedreich ataxia patients	A phase II clinical trial to evaluate the safety and efficacy of interferon gamma in elevating frataxin levels in Friedreich ataxia patients - GIFT/1	Friedreich's Ataxia MedDRA version: 14.1;Level: SOC;Classification code 10029205;Term: Nervous system disorders;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Trade Name: IMUKIN*SC 6F 0,5ML 100MCG INN or Proposed INN: INTERFERON GAMMA -1B	AZIENDA UNIVERSITARIA POLICLINICO UMBERTO I DI ROMA	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes		Phase 2	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

NCT01965327
(ClinicalTrials.gov)

August 2013

27/8/2013

Interferon Gamma-1b in Friedreich Ataxia (FRDA)

Open-label Pilot Study of Interferon Gamma-1b (Actimmune™) for the Treatment of Friedreich Ataxia (FRDA)

Friedreich Ataxia

Drug: Interferon Gamma -1b

Children's Hospital of Philadelphia

Friedreich's Ataxia Research Alliance;Vidara Therapeutics Research Ltd

Completed

5 Years

17 Years

All

Phase 2

United States

NCT02035020
(ClinicalTrials.gov)

May 2013

12/1/2014

A Phase IIa Trial to Test Safety and Efficacy Interferon Gamma Treatment in Elevating Frataxin Levels in FRDA Patients

A Phase IIa Clinical Trial to Test the Safety and Efficacy of Interferon Gamma Treatment in Elevating Frataxin Levels in Friedreich's Ataxia (FRDA) Patients

Friedreich Ataxia

Drug: gamma interferon

Azienda Policlinico Umberto I

NULL

Completed

18 Years

45 Years

All

Phase 2

Italy

EUCTR2012-001881-14-IT
(EUCTR)

01/03/2013

11/01/2013

A phase II clinical trial to evaluate the safety and efficacy of interferon gamma in elevating frataxin levels in Friedreich ataxia patients

A phase II clinical trial to evaluate the safety and efficacy of interferon gamma in elevating frataxin levels in Friedreich ataxia patients - GIFT/1

Friedreich's Ataxia
MedDRA version: 14.1;Level: SOC;Classification code 10029205;Term: Nervous system disorders;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Trade Name: IMUKIN*SC 6F 0,5ML 100MCG
INN or Proposed INN: INTERFERON GAMMA -1B

AZIENDA UNIVERSITARIA POLICLINICO UMBERTO I DI ROMA

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

Italy