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.
| 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 |
| 2 | NCT04273165 (ClinicalTrials.gov) | September 17, 2020 | 14/2/2020 | Safety and Efficacy of Etravirine in Friedreich Ataxia Patients | A Phase 2 Clinical Trial to Test the Safety and Efficacy of Etravirine in Friedreich Ataxia Patients | Friedreich Ataxia | Drug: Etravirine Tablets | IRCCS Eugenio Medea | University of Rome Tor Vergata | Recruiting | 10 Years | 40 Years | All | 30 | Phase 2 | Italy |
| 3 | NCT04192136 (ClinicalTrials.gov) | September 3, 2020 | 6/12/2019 | NAD+ and Exercise in FA | NAD+ Precursor Supplementation With Exercise Training to Increase Aerobic Capacity in Friedreich's Ataxia | Friedreich Ataxia 1 | Dietary Supplement: Nicotinamide Riboside;Dietary Supplement: Placebo;Other: Exercise Intervention | Children's Hospital of Philadelphia | National Institutes of Health (NIH) | Recruiting | 10 Years | 40 Years | All | 72 | N/A | United States |
| 4 | NCT04519567 (ClinicalTrials.gov) | July 31, 2020 | 17/8/2020 | Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia | A 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 Ataxia | Friedreich Ataxia | Biological: CTI-1601;Biological: Placebo | Larimar Therapeutics, Inc. | Veristat, Inc.;Metrum Research Group, LLC | Recruiting | 18 Years | N/A | All | 30 | Phase 1 | United States |
| 5 | EUCTR2017-002163-17-ES (EUCTR) | 08/04/2020 | 23/01/2020 | 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 Faculty | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 225 | Phase 2;Phase 3 | Spain;Austria;Germany;United Kingdom | ||
| 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 |
| 6 | NCT03761511 (ClinicalTrials.gov) | March 2020 | 27/11/2018 | Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia | A Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicentre Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia | Friedreich Ataxia | Drug: Nicotinamide;Drug: Placebo | RWTH Aachen University | Assistance Publique - Hôpitaux de Paris | Not yet recruiting | 18 Years | N/A | All | 225 | Phase 2 | Austria;France;Germany;Italy;Spain;United Kingdom |
| 7 | EUCTR2017-002163-17-GB (EUCTR) | 21/01/2020 | 31/10/2019 | Use 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) | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 225 | Phase 2;Phase 3 | Spain;Austria;Germany;United Kingdom | ||
| 8 | NCT04176991 (ClinicalTrials.gov) | December 11, 2019 | 14/11/2019 | Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia | A 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 Ataxia | Friedreich Ataxia | Biological: CTI-1601;Biological: Placebo | Larimar Therapeutics, Inc. | Veristat, Inc.;Metrum Research Group, LLC | Completed | 18 Years | N/A | All | 28 | Phase 1 | United States |
| 9 | EUCTR2017-002163-17-AT (EUCTR) | 04/09/2019 | 01/08/2019 | A 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 Faculty | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 225 | Phase 2;Phase 3 | Austria;Germany | ||
| 10 | NCT03933163 (ClinicalTrials.gov) | May 23, 2019 | 16/4/2019 | Micronised Resveratrol as a Treatment for Friedreich Ataxia | A Randomised Placebo-controlled Crossover Trial of Micronised Resveratrol as a Treatment for Friedreich Ataxia | Friedreich Ataxia | Drug: Resveratrol | Murdoch Childrens Research Institute | NULL | Recruiting | 16 Years | N/A | All | 40 | Phase 2 | Australia |
| 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 |
| 11 | NCT03917225 (ClinicalTrials.gov) | March 26, 2019 | 10/4/2019 | A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients | A Double-Blind, Placebo-controlled Study on the Effects of MIN-102 on Biochemical, Imaging, Neurophisyiological, and Clinical Markers in Patients With Friedreich's Ataxia | Friedreich Ataxia | Drug: MIN-102;Drug: Placebo | Minoryx Therapeutics, S.L. | NULL | Completed | 12 Years | 60 Years | All | 36 | Phase 2 | Belgium;France;Germany;Spain |
| 12 | NCT04107740 (ClinicalTrials.gov) | February 20, 2019 | 10/9/2019 | C-Trelin Orally Disintegrated(OD) Tablet 5mg in Ataxia Due to Spinocerebellar Degeneration | Multicenter, 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 Degeneration | Spinocerebellar Degeneration | Drug: C-Trelin OD Tab(5mg Taltirelin Hydrate);Drug: Placebo | MediForum Pharm | NULL | Recruiting | 20 Years | N/A | All | 166 | Phase 4 | Korea, Republic of |
| 13 | NCT03660917 (ClinicalTrials.gov) | January 1, 2019 | 15/5/2018 | Riluzole in Patients With Spinocerebellar Ataxia Type 7 | Riluzole in Patients With Spinocerebellar Ataxia Type 7: a Randomized , Double-blind, Placebo-controlled Pilot Trial With a Lead in Phase | SCA7 | Drug: Riluzole;Drug: Placebo | S. Andrea Hospital | NULL | Not yet recruiting | 7 Years | N/A | All | 34 | Phase 2;Phase 3 | Italy |
| 14 | EUCTR2017-002163-17-DE (EUCTR) | 25/10/2018 | 20/11/2017 | A 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) | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 225 | Phase 2;Phase 3 | Spain;Austria;Germany;United Kingdom | ||
| 15 | JPRN-UMIN000031788 | 2018/06/01 | 01/04/2018 | coenzymeQ10 for Autosomal recessive spinocerebellar ataxia type II | Autosomal recessive spinocerebellar ataxia type II | high dose of coenzyme Q10 | Department of Neurology, Tokushima University, Japan | NULL | Complete: follow-up complete | 50years-old | 100years-old | Male and Female | 1 | Not selected | Japan | |
| 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 |
| 16 | EUCTR2017-003084-34-GB (EUCTR) | 22/01/2018 | 28/11/2017 | Testing granulocyte-colony stimulating factor (GCSF) in patients with Friedreich Ataxia | The 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 Bristol | NULL | Not Recruiting | Female: Male: | 7 | Phase 2 | United Kingdom | |||
| 17 | NCT03214588 (ClinicalTrials.gov) | November 8, 2017 | 10/7/2017 | Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia | A 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 Ataxia | Friedreich Ataxia | Drug: TAK-831;Drug: TAK-831 Placebo | Takeda | NULL | Completed | 18 Years | 55 Years | All | 67 | Phase 2 | United States |
| 18 | ChiCTR-IIR-17012717 | 2017-09-20 | 2017-09-18 | Treatment of SCA3/MJD patients with oral DL-3-n-butylphthalide: A randomized, double blind, placebo controlled trial | Treatment of SCA3/MJD patients with oral DL-3-n-butylphthalide: A randomized, double blind, placebo controlled trial | SCA3/MJD | Test group:DL-3-n-butylphthalide;Control group:Placebo; | Xiangya Hospital of Central South University | NULL | Recruiting | 10 | 60 | Both | Test group:60;Control group:60; | 2 (Phase 2 study) | China |
| 19 | EUCTR2017-001481-23-FR (EUCTR) | 12/09/2017 | 30/10/2017 | NA | MULTICENTER, 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) | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | France | ||
| 20 | NCT02889302 (ClinicalTrials.gov) | November 15, 2016 | 31/8/2016 | An 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 Degeneration | Drug: KPS-0373;Drug: Placebo | Kissei Pharmaceutical Co., Ltd. | NULL | Completed | 20 Years | N/A | All | 203 | Phase 3 | Japan |
| 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 |
| 21 | NCT03888664 (ClinicalTrials.gov) | June 26, 2016 | 10/3/2019 | Open Trail of ?IFN for Friedreich Ataxia | Safety and Efficacy of ?IFN Treatment in Friedreich Ataxia | Friedreich Ataxia | Drug: gamma interferon | IRCCS Eugenio Medea | NULL | Completed | 10 Years | 40 Years | All | 12 | Phase 2 | NULL |
| 22 | NCT02705547 (ClinicalTrials.gov) | May 2016 | 5/3/2016 | Rosuvastatin (Crestor) in Friedreich Ataxia | Open-label Biomarker Study of Rosuvastatin (Crestor) for the Treatment of Patients With Friedreich Ataxia | Friedreich Ataxia | Drug: Rosuvastatin | Children's Hospital of Philadelphia | Friedreich's Ataxia Research Alliance | Completed | 18 Years | 65 Years | All | 12 | Early Phase 1 | United States |
| 23 | NCT02424435 (ClinicalTrials.gov) | June 2015 | 16/4/2015 | Methylprednisolone Treatment of Friedreich Ataxia | Open-label Pilot Study of Methylprednisolone for the Treatment of Patients With Friedreich Ataxia (FRDA) | Friedreich Ataxia | Drug: Methylprednisolone | Children's Hospital of Philadelphia | Friedreich's Ataxia Research Alliance | Completed | 5 Years | N/A | All | 11 | Early Phase 1 | United States |
| 24 | NCT02255435 (ClinicalTrials.gov) | January 2015 | 30/9/2014 | RTA 408 Capsules in Patients With Friedreich's Ataxia - MOXIe | A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia (MOXIe) | Friedreich Ataxia | Drug: 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 mg | Reata Pharmaceuticals, Inc. | AbbVie;Friedreich's Ataxia Research Alliance | Active, not recruiting | 16 Years | 40 Years | All | 172 | Phase 2 | United States;Australia;Austria;Italy;United Kingdom;Brazil |
| 25 | 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 |
| 26 | NCT01970098 (ClinicalTrials.gov) | October 9, 2013 | 22/10/2013 | A 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 Degeneration | Drug: KPS-0373, High dose;Drug: KPS-0373, Low dose;Drug: Placebo | Kissei Pharmaceutical Co., Ltd. | NULL | Completed | 20 Years | N/A | All | 374 | Phase 3 | Japan |
| 27 | NCT01970137 (ClinicalTrials.gov) | October 2013 | 22/10/2013 | A 24-week Open-label Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD) | Spinocerebellar Degeneration | Drug: KPS-0373, High dose;Drug: KPS-0373, Low dose | Kissei Pharmaceutical Co., Ltd. | NULL | Completed | 20 Years | N/A | Both | Phase 3 | Japan | ||
| 28 | NCT01970124 (ClinicalTrials.gov) | October 2013 | 22/10/2013 | A Long-Term Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD) | Spinocerebellar Degeneration | Drug: KPS-0373, High dose;Drug: KPS-0373, Low dose | Kissei Pharmaceutical Co., Ltd. | NULL | Completed | 20 Years | N/A | Both | Phase 3 | Japan | ||
| 29 | NCT01970111 (ClinicalTrials.gov) | October 2013 | 22/10/2013 | An Extension Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD) | Spinocerebellar Degeneration | Drug: KPS-0373, High dose;Drug: KPS-0373, Low dose | Kissei Pharmaceutical Co., Ltd. | NULL | Completed | 20 Years | N/A | Both | Phase 3 | Japan | ||
| 30 | JPRN-UMIN000011560 | 2013/08/30 | 26/08/2013 | Trial 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 University | NULL | Recruiting | 20years-old | Not applicable | Male and Female | 40 | Phase 1;Phase 2 | Japan | |
| 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 |
| 31 | 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 |
| 32 | JPRN-UMIN000011111 | 2013/07/16 | 15/07/2013 | Spinal blood flow and metabolism in neurological diseases | motor neuron disease including ALS, multiple sclerosis, stroke, Parkinson disease, spinocerebellar degeneration, multiple system atrophy | PET 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 Sciences | NULL | Recruiting | 20years-old | Not applicable | Male and Female | 70 | Not applicable | Japan | |
| 33 | 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 |
| 34 | 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 | |||
| 35 | NCT01493973 (ClinicalTrials.gov) | January 2013 | 15/12/2011 | Efficacy Study of Epoetin Alfa in Friedreich Ataxia | A Double-blind, Randomized, Placebo-controlled, Clinical Trial to Test the Efficacy of Epoetin Alfa on Physical Performance of Friedreich Ataxia Patients. | Friedreich Ataxia | Drug: Epoetin alfa;Drug: Placebo | Federico II University | Friedreich's Ataxia Research Alliance;Associazione Italiana per la lotta alle Sindromi Atassiche (AISA) | Completed | 12 Years | N/A | Both | 56 | 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 |
| 36 | NCT01716221 (ClinicalTrials.gov) | October 2012 | 25/10/2012 | An Objective Double-blind Evaluation of Bupropion and Citalopram in an Individual With Friedreich Ataxia | An Objective Double-blind Evaluation of Bupropion and Citalopram in an Individual With Friedreich Ataxia | Friedreich Ataxia | Drug: bupropion & Citalopram;Drug: Bupropion & Placebo;Drug: Placebo & Citalopram;Drug: Placebo & Placebo | University of Colorado, Denver | NULL | Completed | N/A | N/A | Female | 1 | Phase 4 | NULL |
| 37 | EUCTR2011-006156-37-IT (EUCTR) | 10/07/2012 | 26/07/2012 | Erythropoieitn in Friedreich Ataxia | A 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 II | NULL | Not Recruiting | Female: yes Male: yes | Phase 2 | Italy | |||
| 38 | NCT01384435 (ClinicalTrials.gov) | June 2011 | 23/6/2011 | A 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 SCD | SCD | Drug: KPS-0373;Drug: Placebo | Kissei Pharmaceutical Co., Ltd. | NULL | Completed | 20 Years | N/A | Both | 200 | Phase 2 | Japan |
| 39 | NCT01096082 (ClinicalTrials.gov) | May 2011 | 29/3/2010 | Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3 | Randomized Clinical Trial to Assess the Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3 | Spinocerebellar Ataxia Type 3;Machado Joseph Disease | Drug: Lithium Carbonate;Drug: Placebo | Hospital de Clinicas de Porto Alegre | NULL | Completed | 16 Years | 80 Years | Both | 62 | Phase 2;Phase 3 | Brazil |
| 40 | NCT01339884 (ClinicalTrials.gov) | April 2011 | 14/4/2011 | A Study of Resveratrol as Treatment for Friedreich Ataxia | An Open Label Clinical Pilot Study of Resveratrol as Treatment for Friedreich Ataxia | Friedreich Ataxia | Drug: Resveratrol | Murdoch Childrens Research Institute | Friedreich's Ataxia Research Alliance | Completed | 18 Years | N/A | Both | 27 | Phase 1;Phase 2 | Australia |
| 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 |
| 41 | EUCTR2008-003662-25-IT (EUCTR) | 21/10/2009 | 01/10/2009 | Studio 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 | Studio 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/S | NULL | Not Recruiting | Female: yes Male: yes | 36 | Germany;Italy;Austria | |||
| 42 | EUCTR2008-003662-25-AT (EUCTR) | 02/10/2009 | 14/08/2009 | Randomised, double blind, placebo controlled study of Lu AA24493 in patients with Friedreich's Ataxia to evaluate safety and tolerability and to explore efficacy | Randomised, 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/S | NULL | Not Recruiting | Female: yes Male: yes | 36 | Germany;Italy;Austria | |||
| 43 | NCT01004016 (ClinicalTrials.gov) | October 2009 | 27/10/2009 | A 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 SCD | Spinocerebellar Degeneration | Drug: KPS-0373;Drug: Placebo | Kissei Pharmaceutical Co., Ltd. | NULL | Completed | 20 Years | N/A | Both | 20 | Phase 2 | Japan |
| 44 | EUCTR2009-010865-22-IT (EUCTR) | 06/05/2009 | 16/04/2009 | An open-label, single treatment, safety and efficacy, long-term study of deferiprone in subjects with Friedreich?s ataxia - ND | An open-label, single treatment, safety and efficacy, long-term study of deferiprone in subjects with Friedreich?s ataxia - ND | patients affected by Friedreich Ataxia | INN or Proposed INN: Deferiprone | APOPHARMA INC., TORONTO, ONTARIO | NULL | Not Recruiting | Female: yes Male: yes | 74 | Belgium;France;Spain;Italy | |||
| 45 | EUCTR2008-000040-13-AT (EUCTR) | 11/02/2009 | 27/01/2009 | Effects of recombinant human Erythropoietin on circulating and intramuscular endothelial progenitor cells, neovascularisation and oxidative metabolism of skeletal muscle in Friedreich’s Ataxia | Effects 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 Neurologie | NULL | Not Recruiting | Female: yes Male: yes | 7 | Phase 2 | Austria | ||
| 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 |
| 46 | EUCTR2007-003331-23-IT (EUCTR) | 16/07/2008 | 10/07/2008 | A study investigating the safety and tolerability of deferiprone in patients with Friedreich's Ataxia - ND | A 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, ONTARIO | NULL | Not Recruiting | Female: yes Male: yes | 80 | United Kingdom;Belgium;France;Spain;Italy | |||
| 47 | NCT00824512 (ClinicalTrials.gov) | June 2008 | 15/1/2009 | Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia | Efficacy 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 Ataxia | Drug: EGb 761 120 mg;Drug: Placebo | Ipsen | NULL | Completed | 12 Years | 22 Years | All | 22 | Phase 2 | France |
| 48 | NCT00683943 (ClinicalTrials.gov) | May 18, 2008 | 22/5/2008 | Lithium Treatment for Patients With Spinocerebellar Ataxia Type I | Pilot Study of Tolerability of Lithium Therapy in Patients With Spinocerebellar Ataxia Type I (SCA1) | Spinocerebellar Ataxia Type I | Drug: Lithium Carbonate | National Institute of Neurological Disorders and Stroke (NINDS) | NULL | Completed | 18 Years | 65 Years | All | 13 | Phase 1 | United States |
| 49 | EUCTR2007-005371-34-FR (EUCTR) | 18/03/2008 | 20/12/2007 | EFFICACY 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 | EFFICACY 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 years | Trade 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 PHARMA | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Phase 2 | France | |||
| 50 | EUCTR2007-003357-85-IT (EUCTR) | 10/10/2007 | 09/11/2007 | Randomized placebo-controlled double-blind trial to assess safety and efficacy of erythropoietin in adult patients with Friedreich's ataxia (a pilot study) - ND | Randomized 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 BESTA | NULL | Not Recruiting | Female: yes Male: yes | 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 |
| 51 | EUCTR2005-005938-12-AT (EUCTR) | 08/06/2006 | 05/04/2006 | Eine 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 Boesch | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 2 | Austria | ||
| 52 | NCT00229632 (ClinicalTrials.gov) | September 27, 2005 | 29/9/2005 | Idebenone to Treat Friedreich's Ataxia | A Six Month Double-Blind, Placebo-Controlled Phase 2 Clinical Trial to Determine the Safety and Efficacy of Idebenone Administered to Patients With Friedreich's Ataxia | Friedreich Ataxia | Drug: Idebenone | National Institute of Neurological Disorders and Stroke (NINDS) | NULL | Completed | 9 Years | 17 Years | All | 51 | Phase 2 | United States |
| 53 | NCT00224640 (ClinicalTrials.gov) | March 2005 | 16/9/2005 | Iron-Chelating Therapy and Friedreich Ataxia | Effect of Iron-Chelating Therapy in Friedreich Ataxia. Study Phase I/II | Friedreich Ataxia | Drug: Iron chelating intervention | Assistance Publique - Hôpitaux de Paris | NULL | Completed | 13 Years | N/A | Both | 15 | Phase 1;Phase 2 | France |
| 54 | NCT00863538 (ClinicalTrials.gov) | November 2004 | 16/3/2009 | Phase II Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD) | An Open-label, Phase II Study of KPS-0373 in Patients With SCD | Spinocerebellar Degeneration | Drug: KPS-0373 | Kissei Pharmaceutical Co., Ltd. | NULL | Completed | 20 Years | N/A | Both | 40 | Phase 2 | Japan |
| 55 | NCT00078481 (ClinicalTrials.gov) | February 2004 | 27/2/2004 | Phase 1 Trial of Idebenone to Treat Patients With Friedreich's Ataxia | Phase 1B Clinical Trial to Establish the Safety and Tolerability of a Multiple-Dose Regimen of Idebenone Administered to Patients With Friedreich's Ataxia | Friedreich Ataxia | Drug: Idebenone | National Institute of Neurological Disorders and Stroke (NINDS) | NULL | Completed | N/A | N/A | Both | 16 | Phase 1 | United States |
| 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 |
| 56 | NCT00034242 (ClinicalTrials.gov) | April 2002 | 24/4/2002 | High-Dose Intravenous Immunoglobulin to Treat Cerebellar Degeneration | The Efficacy of High-Dose Intravenous Immunoglobulin Therapy In Patients With Cerebellar Degeneration: A Double Blind, Placebo Controlled Trial | Spinocerebellar Degenerations | Drug: high-dose intravenous immunoglobulin (IVIG) | National Institute of Neurological Disorders and Stroke (NINDS) | NULL | Completed | N/A | N/A | Both | 20 | Phase 2 | United States |
| 57 | NCT00015808 (ClinicalTrials.gov) | May 2001 | 6/5/2001 | Safety Study of Idebenone to Treat Friedreich's Ataxia | Phase I Clinical Trial to Establish the Maximum Tolerated Dose of Idebenone in Children, Adolescents, and Adults With Friedreich's Ataxia | Friedreich Ataxia | Drug: Idebenone | National Institute of Neurological Disorders and Stroke (NINDS) | NULL | Completed | N/A | N/A | Both | 100 | Phase 1 | United States |
| 58 | EUCTR2008-003662-25-DE (EUCTR) | 21/08/2009 | Randomised, double blind, placebo controlled study of Lu AA24493 in patients with Friedreich's Ataxia to evaluate safety and tolerability and to explore efficacy | Randomised, 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/S | NULL | Not Recruiting | Female: yes Male: yes | 36 | Austria;Germany;Italy | ||||
| 59 | JPRN-JapicCTI-050031 | 06/09/2005 | Intrathecal 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 injury | Intervention name : Baclofen Dosage And administration of the intervention : Intertheacal injection | DAIICHI SANKYO COMPANY, LIMITED | NULL | BOTH | Phase 4 | NULL |