18. Spinocerebellar degeneration
59 clinical trials,   78 drugs   (DrugBank: 28 drugs),   44 drug target genes,   59 drug target pathways

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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
1NCT04577352
(ClinicalTrials.gov)
November 17, 202030/9/2020A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich AtaxiaA 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 AtaxiaDrug: Vatiquinone;Drug: PlaceboPTC TherapeuticsNULLNot yet recruiting7 YearsN/AAll126Phase 2;Phase 3United States;Australia;Canada;Italy
2NCT04273165
(ClinicalTrials.gov)
September 17, 202014/2/2020Safety and Efficacy of Etravirine in Friedreich Ataxia PatientsA Phase 2 Clinical Trial to Test the Safety and Efficacy of Etravirine in Friedreich Ataxia PatientsFriedreich AtaxiaDrug: Etravirine TabletsIRCCS Eugenio MedeaUniversity of Rome Tor VergataRecruiting10 Years40 YearsAll30Phase 2Italy
3NCT04192136
(ClinicalTrials.gov)
September 3, 20206/12/2019NAD+ and Exercise in FANAD+ Precursor Supplementation With Exercise Training to Increase Aerobic Capacity in Friedreich's AtaxiaFriedreich Ataxia 1Dietary Supplement: Nicotinamide Riboside;Dietary Supplement: Placebo;Other: Exercise InterventionChildren's Hospital of PhiladelphiaNational Institutes of Health (NIH)Recruiting10 Years40 YearsAll72N/AUnited States
4NCT04519567
(ClinicalTrials.gov)
July 31, 202017/8/2020Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's AtaxiaA Phase 1 Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CTI-1601 Versus Placebo in Subjects With Friedreich's AtaxiaFriedreich AtaxiaBiological: CTI-1601;Biological: PlaceboLarimar Therapeutics, Inc.Veristat, Inc.;Metrum Research Group, LLCRecruiting18 YearsN/AAll30Phase 1United States
5EUCTR2017-002163-17-ES
(EUCTR)
08/04/202023/01/2020MULTICENTRE STUDY OF THE EFFICACY AND SAFETY OF NICOTINAMIDE IN PATIENTS WITH FRIEDREICHS ATAXIA.A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTICENTRE STUDY OF THE EFFICACY AND SAFETY OF NICOTINAMIDE IN PATIENTS WITH FRIEDREICHS ATAXIA. - NICOFA Friedreich Ataxia;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: Nicotinamide
INN or Proposed INN: NICOTINAMIDE
RWTH Aachen University represented by the Rector himself, represented by the Dean of the Medical FacultyNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
225Phase 2;Phase 3Spain;Austria;Germany;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
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Inclusion_
agemin
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agemax
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PhaseCountries
6NCT03761511
(ClinicalTrials.gov)
March 202027/11/2018Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich AtaxiaA Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicentre Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich AtaxiaFriedreich AtaxiaDrug: Nicotinamide;Drug: PlaceboRWTH Aachen UniversityAssistance Publique - Hôpitaux de ParisNot yet recruiting18 YearsN/AAll225Phase 2Austria;France;Germany;Italy;Spain;United Kingdom
7EUCTR2017-002163-17-GB
(EUCTR)
21/01/202031/10/2019Use of Nicotinamide for the Treatment of Patients with Friedreich's Ataxia.A RANDOMISED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTICENTRE STUDY OF THE EFFICACY AND SAFETY OF NICOTINAMIDE IN PATIENTS WITH FRIEDREICH´S ATAXIA (NICOFA) - NICOFA Friedreich's ataxia.Friedreich ataxia is the most frequent early-onset autosomal recessive hereditary ataxia. It is caused by a pathological expansion of a GAA repeat in the first intron of the frataxin gene (FXN) and results in decreased levels of FXN protein. FXN deficiency results in a relentlessly progressive neurodegenerative condition which frequently presents around puberty. Patients gradually lose coordination, become dysarthric and are frequently wheelchair-bound as adolescents.
MedDRA version: 20.0;Level: PT;Classification code 10017374;Term: Friedreich's ataxia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: Nicotinamide
Product Name: Nicotinamide
INN or Proposed INN: Nicotinamide
Other descriptive name: SUB09246MIG
RWTH Aachen University, Center for Translational & Clinical Research Aachen (CTC-A)NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
225Phase 2;Phase 3Spain;Austria;Germany;United Kingdom
8NCT04176991
(ClinicalTrials.gov)
December 11, 201914/11/2019Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's AtaxiaA Phase 1 Single Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CTI-1601 Versus Placebo in Subjects With Friedreich's AtaxiaFriedreich AtaxiaBiological: CTI-1601;Biological: PlaceboLarimar Therapeutics, Inc.Veristat, Inc.;Metrum Research Group, LLCCompleted18 YearsN/AAll28Phase 1United States
9EUCTR2017-002163-17-AT
(EUCTR)
04/09/201901/08/2019A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTICENTRE STUDY OF THE EFFICACY AND SAFETY OF NICOTINAMIDE IN PATIENTS WITH FRIEDREICHS ATAXIA.A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTICENTRE STUDY OF THE EFFICACY AND SAFETY OF NICOTINAMIDE IN PATIENTS WITH FRIEDREICHS ATAXIA. - NICOFA Friedreich Ataxia;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: Nicotinamide
INN or Proposed INN: NICOTINAMIDE
RWTH Aachen University represented by the Rector himself, represented by the Dean of the Medical FacultyNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
225Phase 2;Phase 3Austria;Germany
10NCT03933163
(ClinicalTrials.gov)
May 23, 201916/4/2019Micronised Resveratrol as a Treatment for Friedreich AtaxiaA Randomised Placebo-controlled Crossover Trial of Micronised Resveratrol as a Treatment for Friedreich AtaxiaFriedreich AtaxiaDrug: ResveratrolMurdoch Childrens Research InstituteNULLRecruiting16 YearsN/AAll40Phase 2Australia
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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agemin
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agemax
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gender
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size
PhaseCountries
11NCT03917225
(ClinicalTrials.gov)
March 26, 201910/4/2019A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female PatientsA Double-Blind, Placebo-controlled Study on the Effects of MIN-102 on Biochemical, Imaging, Neurophisyiological, and Clinical Markers in Patients With Friedreich's AtaxiaFriedreich AtaxiaDrug: MIN-102;Drug: PlaceboMinoryx Therapeutics, S.L.NULLCompleted12 Years60 YearsAll36Phase 2Belgium;France;Germany;Spain
12NCT04107740
(ClinicalTrials.gov)
February 20, 201910/9/2019C-Trelin Orally Disintegrated(OD) Tablet 5mg in Ataxia Due to Spinocerebellar DegenerationMulticenter, Randomized, Double-blind, Placebo-controlled, Phase IV Clinical Trial to Evaluate and Compare the Safety and Efficacy of C-Trelin OD Tab 5mg(Taltirelin Hydrate) in Patients With Ataxia Induced by Spinocerebellar DegenerationSpinocerebellar DegenerationDrug: C-Trelin OD Tab(5mg Taltirelin Hydrate);Drug: PlaceboMediForum PharmNULLRecruiting20 YearsN/AAll166Phase 4Korea, Republic of
13NCT03660917
(ClinicalTrials.gov)
January 1, 201915/5/2018Riluzole in Patients With Spinocerebellar Ataxia Type 7Riluzole in Patients With Spinocerebellar Ataxia Type 7: a Randomized , Double-blind, Placebo-controlled Pilot Trial With a Lead in PhaseSCA7Drug: Riluzole;Drug: PlaceboS. Andrea HospitalNULLNot yet recruiting7 YearsN/AAll34Phase 2;Phase 3Italy
14EUCTR2017-002163-17-DE
(EUCTR)
25/10/201820/11/2017A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTICENTRE STUDY OF THE EFFICACY AND SAFETY OF NICOTINAMIDE IN PATIENTS WITH FRIEDREICHS ATAXIA.A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP, MULTICENTRE STUDY OF THE EFFICACY AND SAFETY OF NICOTINAMIDE IN PATIENTS WITH FRIEDREICHS ATAXIA. - NICOFA Friedreich Ataxia;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: Nicotinamide
INN or Proposed INN: NICOTINAMIDE
RWTH Aachen University represented by the Center for Translational & Clinical Research Aachen (CTC-A)NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
225Phase 2;Phase 3Spain;Austria;Germany;United Kingdom
15JPRN-UMIN000031788
2018/06/0101/04/2018coenzymeQ10 for Autosomal recessive spinocerebellar ataxia type II Autosomal recessive spinocerebellar ataxia type IIhigh dose of coenzyme Q10Department of Neurology, Tokushima University, JapanNULLComplete: follow-up complete50years-old100years-oldMale and Female1Not selectedJapan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
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gender
Target_
size
PhaseCountries
16EUCTR2017-003084-34-GB
(EUCTR)
22/01/201828/11/2017Testing granulocyte-colony stimulating factor (GCSF) in patients with Friedreich AtaxiaThe pharmacological effects of granulocyte-colony stimulating factor (GCSF) on frataxin expression in patients with Friedreich Ataxia - GCSF for Friedreich Ataxia Friedreich Ataxia
MedDRA version: 20.0;Level: LLT;Classification code 10003592;Term: Ataxia cerebellar;System Organ Class: 100000004852
MedDRA version: 20.0;Level: PT;Classification code 10017374;Term: Friedreich's ataxia;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
University of BristolNULLNot Recruiting Female:
Male:
7Phase 2United Kingdom
17NCT03214588
(ClinicalTrials.gov)
November 8, 201710/7/2017Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich AtaxiaA Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Arm Study to Evaluate Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adult Subjects With Friedreich AtaxiaFriedreich AtaxiaDrug: TAK-831;Drug: TAK-831 PlaceboTakedaNULLCompleted18 Years55 YearsAll67Phase 2United States
18ChiCTR-IIR-17012717
2017-09-202017-09-18Treatment of SCA3/MJD patients with oral DL-3-n-butylphthalide: A randomized, double blind, placebo controlled trialTreatment of SCA3/MJD patients with oral DL-3-n-butylphthalide: A randomized, double blind, placebo controlled trial SCA3/MJDTest group:DL-3-n-butylphthalide;Control group:Placebo;Xiangya Hospital of Central South UniversityNULLRecruiting1060BothTest group:60;Control group:60;2 (Phase 2 study)China
19EUCTR2017-001481-23-FR
(EUCTR)
12/09/201730/10/2017NAMULTICENTER, RANDOMIZED, DOUBLE BLIND, PLACEBO CONTROLLED CLINICAL TRIAL WITH RILUZOLE IN SPINOCEREBELLAR ATAXIA TYPE 2 - ATRIL SCA2 patients, both gender, at least 18 years of age
MedDRA version: 20.0;Level: PT;Classification code 10057660;Term: Spinocerebellar ataxia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: Riluzole PMCS
Product Name: Riluzole PMCS
INN or Proposed INN: Riluzole
Other descriptive name: Riluzole
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)NULLNot RecruitingFemale: yes
Male: yes
50Phase 3France
20NCT02889302
(ClinicalTrials.gov)
November 15, 201631/8/2016An Additional Confirmatory Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)An Additional Phase III Confirmatory Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)Spinocerebellar DegenerationDrug: KPS-0373;Drug: PlaceboKissei Pharmaceutical Co., Ltd.NULLCompleted20 YearsN/AAll203Phase 3Japan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT03888664
(ClinicalTrials.gov)
June 26, 201610/3/2019Open Trail of ?IFN for Friedreich AtaxiaSafety and Efficacy of ?IFN Treatment in Friedreich AtaxiaFriedreich AtaxiaDrug: gamma interferonIRCCS Eugenio MedeaNULLCompleted10 Years40 YearsAll12Phase 2NULL
22NCT02705547
(ClinicalTrials.gov)
May 20165/3/2016Rosuvastatin (Crestor) in Friedreich AtaxiaOpen-label Biomarker Study of Rosuvastatin (Crestor) for the Treatment of Patients With Friedreich AtaxiaFriedreich AtaxiaDrug: RosuvastatinChildren's Hospital of PhiladelphiaFriedreich's Ataxia Research AllianceCompleted18 Years65 YearsAll12Early Phase 1United States
23NCT02424435
(ClinicalTrials.gov)
June 201516/4/2015Methylprednisolone Treatment of Friedreich AtaxiaOpen-label Pilot Study of Methylprednisolone for the Treatment of Patients With Friedreich Ataxia (FRDA)Friedreich AtaxiaDrug: MethylprednisoloneChildren's Hospital of PhiladelphiaFriedreich's Ataxia Research AllianceCompleted5 YearsN/AAll11Early Phase 1United States
24NCT02255435
(ClinicalTrials.gov)
January 201530/9/2014RTA 408 Capsules in Patients With Friedreich's Ataxia - MOXIeA Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia (MOXIe)Friedreich AtaxiaDrug: Omaveloxolone Capsules, 2.5 mg;Drug: Omaveloxolone Capsules, 5 mg;Drug: Omaveloxolone Capsules, 10 mg;Drug: Placebo;Drug: Omaveloxolone Capsules, 20 mg;Drug: Omaveloxolone Capsules, 40 mg;Drug: Omaveloxolone Capsules, 80 mg;Drug: Omaveloxolone Capsules, 160 mg;Drug: Omaveloxolone Capsules, 300 mg;Drug: Omaveloxolone Capsules, 150 mgReata Pharmaceuticals, Inc.AbbVie;Friedreich's Ataxia Research AllianceActive, not recruiting16 Years40 YearsAll172Phase 2United States;Australia;Austria;Italy;United Kingdom;Brazil
25NCT02147886
(ClinicalTrials.gov)
July 201421/5/2014Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph DiseaseA Single-Center, Randomized, Double-Blind, Parallel-Group, Dose-Controlled Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Patients With Machado-Joseph DiseaseMachado-Joseph Disease / Spinocerebellar Ataxia 3Drug: Cabaletta for IV infusion once weekly during 24 weeksBioblast Pharma Ltd.NULLCompleted18 Years75 YearsBoth15Phase 2Israel
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26NCT01970098
(ClinicalTrials.gov)
October 9, 201322/10/2013A Confirmatory Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)A Phase III Confirmatory Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)Spinocerebellar DegenerationDrug: KPS-0373, High dose;Drug: KPS-0373, Low dose;Drug: PlaceboKissei Pharmaceutical Co., Ltd.NULLCompleted20 YearsN/AAll374Phase 3Japan
27NCT01970111
(ClinicalTrials.gov)
October 201322/10/2013An Extension Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)Spinocerebellar DegenerationDrug: KPS-0373, High dose;Drug: KPS-0373, Low doseKissei Pharmaceutical Co., Ltd.NULLCompleted20 YearsN/ABothPhase 3Japan
28NCT01970124
(ClinicalTrials.gov)
October 201322/10/2013A Long-Term Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)Spinocerebellar DegenerationDrug: KPS-0373, High dose;Drug: KPS-0373, Low doseKissei Pharmaceutical Co., Ltd.NULLCompleted20 YearsN/ABothPhase 3Japan
29NCT01970137
(ClinicalTrials.gov)
October 201322/10/2013A 24-week Open-label Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)Spinocerebellar DegenerationDrug: KPS-0373, High dose;Drug: KPS-0373, Low doseKissei Pharmaceutical Co., Ltd.NULLCompleted20 YearsN/ABothPhase 3Japan
30JPRN-UMIN000011560
2013/08/3026/08/2013Trial of varenicline (Champix) for the treatment of spinocerebellar degeneration spinocerebellar degeneration (SCD)[High-dose Group]
Oral administration of varenicline

1-7 days 0.5 mg/day
8-14 days 1.0mg/day
15-21 days 1.5mg/day
22-56 days 2.0 mg/day
57-63 days 1.0 mg/day
64-70 days 0.5 mg/day
71-84 days Wash-out period
85-154 days 0.5 mg/day
[Low-dose Group]
Oral administration of varenicline

1-70 days 0.5 mg/day
71-84 days Wash-out period
85-91 days 0.5 mg/day
92-98 days 1.0mg/day
99-105 days 1.5mg/day
106-140 days 2.0 mg/day
141-147 days 1.0 mg/day
148-154 days 0.5 mg/day
Niigata UniversityNULLRecruiting20years-oldNot applicableMale and Female40Phase 1;Phase 2Japan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31NCT01965327
(ClinicalTrials.gov)
August 201327/8/2013Interferon Gamma-1b in Friedreich Ataxia (FRDA)Open-label Pilot Study of Interferon Gamma-1b (Actimmune™) for the Treatment of Friedreich Ataxia (FRDA)Friedreich AtaxiaDrug: Interferon Gamma-1bChildren's Hospital of PhiladelphiaFriedreich's Ataxia Research Alliance;Vidara Therapeutics Research LtdCompleted5 Years17 YearsAll12Phase 2United States
32JPRN-UMIN000011111
2013/07/1615/07/2013Spinal blood flow and metabolism in neurological diseases motor neuron disease including ALS, multiple sclerosis, stroke, Parkinson disease, spinocerebellar degeneration, multiple system atrophyPET scan study with 11C-flumazenil
PET scan study with 18F- FDG
PET scan study with 15O-H2O
Okayama University Graduate School of Medicine, Dentistry and Pharmaceutical SciencesNULLRecruiting20years-oldNot applicableMale and Female70Not applicableJapan
33NCT02035020
(ClinicalTrials.gov)
May 201312/1/2014A Phase IIa Trial to Test Safety and Efficacy Interferon Gamma Treatment in Elevating Frataxin Levels in FRDA PatientsA Phase IIa Clinical Trial to Test the Safety and Efficacy of Interferon Gamma Treatment in Elevating Frataxin Levels in Friedreich's Ataxia (FRDA) PatientsFriedreich AtaxiaDrug: gamma interferonAzienda Policlinico Umberto INULLCompleted18 Years45 YearsAll10Phase 2Italy
34EUCTR2012-001881-14-IT
(EUCTR)
01/03/201311/01/2013A phase II clinical trial to evaluate the safety and efficacy of interferon gamma in elevating frataxin levels in Friedreich ataxia patientsA 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 ROMANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 2Italy
35NCT01493973
(ClinicalTrials.gov)
January 201315/12/2011Efficacy Study of Epoetin Alfa in Friedreich AtaxiaA Double-blind, Randomized, Placebo-controlled, Clinical Trial to Test the Efficacy of Epoetin Alfa on Physical Performance of Friedreich Ataxia Patients.Friedreich AtaxiaDrug: Epoetin alfa;Drug: PlaceboFederico II UniversityFriedreich's Ataxia Research Alliance;Associazione Italiana per la lotta alle Sindromi Atassiche (AISA)Completed12 YearsN/ABoth56Phase 2Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36NCT01716221
(ClinicalTrials.gov)
October 201225/10/2012An Objective Double-blind Evaluation of Bupropion and Citalopram in an Individual With Friedreich AtaxiaAn Objective Double-blind Evaluation of Bupropion and Citalopram in an Individual With Friedreich AtaxiaFriedreich AtaxiaDrug: bupropion & Citalopram;Drug: Bupropion & Placebo;Drug: Placebo & Citalopram;Drug: Placebo & PlaceboUniversity of Colorado, DenverNULLCompletedN/AN/AFemale1Phase 4NULL
37EUCTR2011-006156-37-IT
(EUCTR)
10/07/201226/07/2012Erythropoieitn in Friedreich AtaxiaA double-blind, randomized, placebo-controlled, clinical trial to test the efficacy of Epoetin alfa on physical performance of Friedreich Ataxia patients. - FRIEMAX FRIEDREICH ATAXIA
MedDRA version: 14.1;Level: PT;Classification code 10008025;Term: Cerebellar ataxia;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: EPREX*1SIR 40000UI/ML 1ML
INN or Proposed INN: EPOETIN ALFA
Trade Name: EPREX*1SIR 10000UI 1ML
INN or Proposed INN: EPOETIN ALFA
Trade Name: BINOCRIT*1SIR 40000UI/1ML
INN or Proposed INN: EPOETIN ALFA
UNIVERSITA' DEGLI STUDI DI NAPOLI FEDERICO IINULLNot RecruitingFemale: yes
Male: yes
Phase 2Italy
38NCT01384435
(ClinicalTrials.gov)
June 201123/6/2011A Phase II Double Blind Comparative Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)A Randomized, Double Blind, Placebo-controlled Phase II Study of KPS-0373 in Patients With SCDSCDDrug: KPS-0373;Drug: PlaceboKissei Pharmaceutical Co., Ltd.NULLCompleted20 YearsN/ABoth200Phase 2Japan
39NCT01096082
(ClinicalTrials.gov)
May 201129/3/2010Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3Randomized Clinical Trial to Assess the Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3Spinocerebellar Ataxia Type 3;Machado Joseph DiseaseDrug: Lithium Carbonate;Drug: PlaceboHospital de Clinicas de Porto AlegreNULLCompleted16 Years80 YearsBoth62Phase 2;Phase 3Brazil
40NCT01339884
(ClinicalTrials.gov)
April 201114/4/2011A Study of Resveratrol as Treatment for Friedreich AtaxiaAn Open Label Clinical Pilot Study of Resveratrol as Treatment for Friedreich AtaxiaFriedreich AtaxiaDrug: ResveratrolMurdoch Childrens Research InstituteFriedreich's Ataxia Research AllianceCompleted18 YearsN/ABoth27Phase 1;Phase 2Australia
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
41EUCTR2008-003662-25-IT
(EUCTR)
21/10/200901/10/2009Studio randomizzato, in doppio cieco, controllato verso placebo, per valutare la sicurezza e la tollerabilita` e per esplorare l efficacia di Lu AA24493, in pazienti con Atassia di Friedreich - NDStudio randomizzato, in doppio cieco, controllato verso placebo, per valutare la sicurezza e la tollerabilita` e per esplorare l efficacia di Lu AA24493, in pazienti con Atassia di Friedreich - ND Friedreich`s Ataxia (FRDA)
MedDRA version: 9.1;Level: LLT;Classification code 10017374
Product Name: Lu AA24493
Product Code: Lu AA24493
H.Lundbeck A/SNULLNot RecruitingFemale: yes
Male: yes
36Germany;Italy;Austria
42EUCTR2008-003662-25-AT
(EUCTR)
02/10/200914/08/2009Randomised, double blind, placebo controlled study of Lu AA24493 in patients with Friedreich's Ataxia to evaluate safety and tolerability and to explore efficacyRandomised, double blind, placebo controlled study of Lu AA24493 in patients with Friedreich's Ataxia to evaluate safety and tolerability and to explore efficacy Friedreich's Ataxia (FRDA)
MedDRA version: 12.0;Level: LLT;Classification code 10017374;Term: Friedreich's ataxia
Product Name: Lu AA24493
Product Code: Lu AA24493
H. Lundbeck A/SNULLNot RecruitingFemale: yes
Male: yes
36Germany;Italy;Austria
43NCT01004016
(ClinicalTrials.gov)
October 200927/10/2009A Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)A Double-blind, Placebo-controlled, Crossover Study, Followed by Open-label Study of KPS-0373 in Patients With SCDSpinocerebellar DegenerationDrug: KPS-0373;Drug: PlaceboKissei Pharmaceutical Co., Ltd.NULLCompleted20 YearsN/ABoth20Phase 2Japan
44EUCTR2009-010865-22-IT
(EUCTR)
06/05/200916/04/2009An open-label, single treatment, safety and efficacy, long-term study of deferiprone in subjects with Friedreich?s ataxia - NDAn open-label, single treatment, safety and efficacy, long-term study of deferiprone in subjects with Friedreich?s ataxia - ND patients affected by Friedreich AtaxiaINN or Proposed INN: DeferiproneAPOPHARMA INC., TORONTO, ONTARIONULLNot RecruitingFemale: yes
Male: yes
74Belgium;France;Spain;Italy
45EUCTR2008-000040-13-AT
(EUCTR)
11/02/200927/01/2009Effects of recombinant human Erythropoietin on circulating and intramuscular endothelial progenitor cells, neovascularisation and oxidative metabolism of skeletal muscle in Friedreich’s AtaxiaEffects of recombinant human Erythropoietin on circulating and intramuscular endothelial progenitor cells, neovascularisation and oxidative metabolism of skeletal muscle in Friedreich’s Ataxia Friedreich's ataxia (FRDA) is the most common autosomal recessive neurodegenerativ disease (1:50 000) affecting the central and peripheral nervous system. Extraneural organs are also affected during the course of the disease as a significant proportian of patients develop cardiomyopathy or diabetes. FRDA is caused by a GAA triplet expansion in the FRDA gene on chromosome 9q13 resulting in a loss of function of the gene product Frataxin.Trade Name: Neorecormon
Product Name: Neorecormon
Product Code: EU1/97/031-032
INN or Proposed INN: H-116PI-DE.pdf
Other descriptive name: Epoeitin beta
Medizinische Universität Innsbruck, Univ.-Klinik für NeurologieNULLNot RecruitingFemale: yes
Male: yes
7Phase 2Austria
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
46EUCTR2007-003331-23-IT
(EUCTR)
16/07/200810/07/2008A study investigating the safety and tolerability of deferiprone in patients with Friedreich's Ataxia - NDA study investigating the safety and tolerability of deferiprone in patients with Friedreich's Ataxia - ND patients affected by Friedreich Ataxia
MedDRA version: 9.1;Level: LLT;Classification code 10017374;Term: Friedreich's ataxia
Product Name: Deferiprone
INN or Proposed INN: Deferiprone
APOPHARMA INC., TORONTO, ONTARIONULLNot RecruitingFemale: yes
Male: yes
80United Kingdom;Belgium;France;Spain;Italy
47NCT00824512
(ClinicalTrials.gov)
June 200815/1/2009Efficacy of EGb761 in Patients Suffering From Friedreich AtaxiaEfficacy of EGb761 120mg Bid Versus Placebo in Patients Suffering From Friedreich Ataxia. A 3 Month, Phase II, Randomised, Double Blind, Placebo Controlled, Parallel Group Clinical Study.Friedreich AtaxiaDrug: EGb 761 120 mg;Drug: PlaceboIpsenNULLCompleted12 Years22 YearsAll22Phase 2France
48NCT00683943
(ClinicalTrials.gov)
May 18, 200822/5/2008Lithium Treatment for Patients With Spinocerebellar Ataxia Type IPilot Study of Tolerability of Lithium Therapy in Patients With Spinocerebellar Ataxia Type I (SCA1)Spinocerebellar Ataxia Type IDrug: Lithium CarbonateNational Institute of Neurological Disorders and Stroke (NINDS)NULLCompleted18 Years65 YearsAll13Phase 1United States
49EUCTR2007-005371-34-FR
(EUCTR)
18/03/200820/12/2007EFFICACY OF EGb 761® 120 mg bid VERSUS PLACEBO IN PATIENTS SUFFERING FROM FRIEDREICH ATAXIAA 3 months, phase II, randomised, double blind, placebo-controlled, parallel groups, clinical study. - EGB 761 in FRIEDREICH ATAXIAEFFICACY OF EGb 761® 120 mg bid VERSUS PLACEBO IN PATIENTS SUFFERING FROM FRIEDREICH ATAXIAA 3 months, phase II, randomised, double blind, placebo-controlled, parallel groups, clinical study. - EGB 761 in FRIEDREICH ATAXIA Out patient suffering from friedreich ataxia aged from 12 to 20 yearsTrade Name: Rokan (r) Novo 120 mg
Product Name: EGB 761 (r) 120 mg
INN or Proposed INN: Standardized Ginkgo Biloba Extract (EGb 761 (r)
BEAUFOUR IPSEN PHARMANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 2France
50EUCTR2007-003357-85-IT
(EUCTR)
10/10/200709/11/2007Randomized placebo-controlled double-blind trial to assess safety and efficacy of erythropoietin in adult patients with Friedreich's ataxia (a pilot study) - NDRandomized placebo-controlled double-blind trial to assess safety and efficacy of erythropoietin in adult patients with Friedreich's ataxia (a pilot study) - ND Friedreich ataxia (FRDA) is a rare autosomal recessive neurodegenerative disorder caused a mutation in the FXN gene, which encodes a protein named frataxin. As a result of the mutation, frataxin is quantitatively reduced but qualitatively normal. Thus, any pharmacological agent able to increase frataxin intracellular levels would have a great therapeutic relevance.
MedDRA version: 6.1;Level: PT;Classification code 10003591
Trade Name: EPREX*1FL 40000UI/ML 1ML
INN or Proposed INN: Erythropoietin
Other descriptive name: Erythropoietin
ISTITUTO NEUROLOGICO CARLO BESTANULLNot RecruitingFemale: yes
Male: yes
Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
51EUCTR2005-005938-12-AT
(EUCTR)
08/06/200605/04/2006Eine offene, nicht randomisierte, einarmige Pilotstudie zur Beurteilung der Wirksamkeit von Erythropoetin bei Friedreich Ataxie.Eine offene, nicht randomisierte, einarmige Pilotstudie zur Beurteilung der Wirksamkeit von Erythropoetin bei Friedreich Ataxie. Die Friedreich Ataxie (FRDA) ist eine autosomal-rezessiv vererbte, progressive neurodegenerative Erkrankung, die mit einer Inzidenz von rund 1:50.000 auftritt. Es handelt sich dabei um die am häufigsten vorkommende Ataxie beim Menschen. Sie manifestiert sich klinisch initial als spinozerebelläre Ataxie, im weiteren Krankheitsverlauf tritt meist eine hypertrophe Kardiomyopathie hinzu, deren Komplikationen häufig für den frühen Tod der Patienten verantwortlich sind.Trade Name: Neo-Recormon
Product Name: Neo-Recormon
Product Code: EU1/97/031/031-032
Dr. Sylvia BoeschNULLNot RecruitingFemale: yes
Male: yes
12Phase 2Austria
52NCT00229632
(ClinicalTrials.gov)
September 27, 200529/9/2005Idebenone to Treat Friedreich's AtaxiaA Six Month Double-Blind, Placebo-Controlled Phase 2 Clinical Trial to Determine the Safety and Efficacy of Idebenone Administered to Patients With Friedreich's AtaxiaFriedreich AtaxiaDrug: IdebenoneNational Institute of Neurological Disorders and Stroke (NINDS)NULLCompleted9 Years17 YearsAll51Phase 2United States
53NCT00224640
(ClinicalTrials.gov)
March 200516/9/2005Iron-Chelating Therapy and Friedreich AtaxiaEffect of Iron-Chelating Therapy in Friedreich Ataxia. Study Phase I/IIFriedreich AtaxiaDrug: Iron chelating interventionAssistance Publique - Hôpitaux de ParisNULLCompleted13 YearsN/ABoth15Phase 1;Phase 2France
54NCT00863538
(ClinicalTrials.gov)
November 200416/3/2009Phase II Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)An Open-label, Phase II Study of KPS-0373 in Patients With SCDSpinocerebellar DegenerationDrug: KPS-0373Kissei Pharmaceutical Co., Ltd.NULLCompleted20 YearsN/ABoth40Phase 2Japan
55NCT00078481
(ClinicalTrials.gov)
February 200427/2/2004Phase 1 Trial of Idebenone to Treat Patients With Friedreich's AtaxiaPhase 1B Clinical Trial to Establish the Safety and Tolerability of a Multiple-Dose Regimen of Idebenone Administered to Patients With Friedreich's AtaxiaFriedreich AtaxiaDrug: IdebenoneNational Institute of Neurological Disorders and Stroke (NINDS)NULLCompletedN/AN/ABoth16Phase 1United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
56NCT00034242
(ClinicalTrials.gov)
April 200224/4/2002High-Dose Intravenous Immunoglobulin to Treat Cerebellar DegenerationThe Efficacy of High-Dose Intravenous Immunoglobulin Therapy In Patients With Cerebellar Degeneration: A Double Blind, Placebo Controlled TrialSpinocerebellar DegenerationsDrug: high-dose intravenous immunoglobulin (IVIG)National Institute of Neurological Disorders and Stroke (NINDS)NULLCompletedN/AN/ABoth20Phase 2United States
57NCT00015808
(ClinicalTrials.gov)
May 20016/5/2001Safety Study of Idebenone to Treat Friedreich's AtaxiaPhase I Clinical Trial to Establish the Maximum Tolerated Dose of Idebenone in Children, Adolescents, and Adults With Friedreich's AtaxiaFriedreich AtaxiaDrug: IdebenoneNational Institute of Neurological Disorders and Stroke (NINDS)NULLCompletedN/AN/ABoth100Phase 1United States
58JPRN-JapicCTI-050031
06/09/2005Intrathecal Baclofen therapy with the implanted pump system for Spastic Palsy (Post-marketing clinical trial extended phase 3 after NDA approval)Intrathecal Baclofen therapy with the implanted pump system for Spastic Palsy (Post-marketing clinical trial extended phase 3 after NDA approval) [Spinal cord Origin] Spinal cord Injury, Multiple sclerosis, Spinocerebellar degeneration, Circulatory disorder of the spinal, Ossification of the posterior longitudinal ligament [Cerebral Origin] Cerebral palsy, Traumatic head injuryIntervention name : Baclofen
Dosage And administration of the intervention : Intertheacal injection
DAIICHI SANKYO COMPANY, LIMITEDNULLBOTHPhase 4NULL
59EUCTR2008-003662-25-DE
(EUCTR)
21/08/2009Randomised, double blind, placebo controlled study of Lu AA24493 in patients with Friedreich's Ataxia to evaluate safety and tolerability and to explore efficacyRandomised, double blind, placebo controlled study of Lu AA24493 in patients with Friedreich's Ataxia to evaluate safety and tolerability and to explore efficacy Friedreich's Ataxia (FRDA)
MedDRA version: 12.0;Level: LLT;Classification code 10017374;Term: Friedreich's ataxia
Product Name: Lu AA24493
Product Code: Lu AA24493
H. Lundbeck A/SNULLNot RecruitingFemale: yes
Male: yes
36Austria;Germany;Italy