120. 遺伝性ジストニア Hereditary dystonia Clinical trials / Disease details


臨床試験数 : 25 薬物数 : 21 - (DrugBank : 4) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 2

  
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1NCT04184453
(ClinicalTrials.gov)
December 23, 201929/11/2019Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia PatientsClinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia PatientsAceruloplasminemiaDrug: Deferiprone treatedFirst Affiliated Hospital of Fujian Medical UniversityNULLRecruiting18 Years80 YearsAll5Early Phase 1China
2NCT04182763
(ClinicalTrials.gov)
December 4, 201924/11/2019CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)A Phase 2 Study of a Vitamin Metabolite for PKANPantothenate Kinase-Associated NeurodegenerationOther: CoA-Z;Other: PlaceboOregon Health and Science UniversityEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Washington State University;Oregon State University;Spoonbill FoundationRecruiting3 Months89 YearsAll51Phase 2United States
3NCT03726996
(ClinicalTrials.gov)
January 14, 201929/10/2018Desipramine in Infantile Neuroaxonal Dystrophy (INAD).Novel Off-label Use of Desipramine in Infantile Neuroaxonal Dystrophy: Targeting the Sphingolipid Metabolism Pathway to Reduce Accumulation of Ceramide.Infantile Neuroaxonal DystrophyDrug: DesipramineDuke UniversityNULLTerminated3 Years17 YearsAll4Phase 4United States
4NCT03570931
(ClinicalTrials.gov)
November 5, 201811/6/2018A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal DystrophyA Prospective Open-label Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal DystrophyInfantile Neuroaxonal DystrophyDrug: RT001Retrotope, Inc.NULLActive, not recruiting18 Months10 YearsAll19Phase 2/Phase 3United States
5ChiCTR1900021076
2018-09-042019-01-27A study for efficacy of pantethine in the treatment of pantothenate kinase-associated neurodegenerationA study for efficacy of pantethine in the treatment of pantothenate kinase-associated neurodegeneration pantothenate kinase-associated neurodegenerationExperimental group:Standardized basal treatment + pantethine;Control group:Standardized basal treatment;Department of Pediatrics, Peking University First HospitalNULLRecruiting016BothExperimental group:12;Control group:6;China
6EUCTR2016-001955-29-FR
(EUCTR)
29/01/201804/06/2018Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN)Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement therapy, in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN): A Randomized, Double-blind, Placebo-Controlled Study with an Open-Label Extension Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, the most common form of Neurodegeneration with Brain Iron Accumulation (NBIA). It is a progressive, often fatal, neurodegenerative disease.
MedDRA version: 20.0;Level: PT;Classification code 10053643;Term: Neurodegenerative disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Fosmetpantotenate
Product Code: RE-024
INN or Proposed INN: Fosmetpantotenate
Other descriptive name: FOSMETPANTOTENATE DRUG SUBSTANCE
Retrophin, Inc.NULLNot RecruitingFemale: yes
Male: yes
82Phase 3United States;France;Czech Republic;Canada;Poland;Spain;Norway;Germany;Italy;United Kingdom
7EUCTR2016-001955-29-IT
(EUCTR)
10/11/201705/11/2020Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN)Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement therapy, in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN): A Randomized, Double-blind, Placebo-Controlled Study with an Open-Label Extension - - Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, the most common form of Neurodegeneration with Brain Iron Accumulation (NBIA). It is a progressive, often fatal, neurodegenerative disease.
MedDRA version: 21.1;Level: PT;Classification code 10053643;Term: Neurodegenerative disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Fosmetpantotenato
Product Code: [RE-024]
INN or Proposed INN: Fosmetpantotenato
RETROPHIN, INCNULLNot RecruitingFemale: yes
Male: yes
82Phase 3United States;France;Czechia;Czech Republic;Canada;Spain;Poland;Norway;Germany;United Kingdom;Italy
8EUCTR2016-001955-29-ES
(EUCTR)
16/10/201730/03/2017Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN)Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement therapy, in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN): A Randomized, Double-blind, Placebo-Controlled Study with an Open-Label Extension Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, the most common form of Neurodegeneration with Brain Iron Accumulation (NBIA). It is a progressive, often fatal, neurodegenerative disease.
MedDRA version: 19.1;Level: PT;Classification code 10053643;Term: Neurodegenerative disorder;System Organ Class: 10029205 - Nervous system disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Fosmetpantotenate
Product Code: RE-024
INN or Proposed INN: Fosmetpantotenate
Retrophin, Inc.NULLNot Recruiting Female: yes
Male: yes
82 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;United States;Czech Republic;Canada;Poland;Spain;Norway;Germany;Italy;United Kingdom
9EUCTR2016-001955-29-CZ
(EUCTR)
06/10/201720/04/2017Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN)Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement therapy, in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN): A Randomized, Double-blind, Placebo-Controlled Study with an Open-Label Extension Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, the most common form of Neurodegeneration with Brain Iron Accumulation (NBIA). It is a progressive, often fatal, neurodegenerative disease.
MedDRA version: 21.1;Level: PT;Classification code 10053643;Term: Neurodegenerative disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Fosmetpantotenate
Product Code: RE-024
INN or Proposed INN: Fosmetpantotenate
Other descriptive name: FOSMETPANTOTENATE DRUG SUBSTANCE
Retrophin, Inc.NULLNot RecruitingFemale: yes
Male: yes
82Phase 3France;United States;Czech Republic;Canada;Poland;Spain;Norway;Germany;Italy;United Kingdom
10NCT03041116
(ClinicalTrials.gov)
July 17, 20172/12/2016Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN PatientsEfficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), A Phosphopantothenate Replacement Therapy, in Pantothenate Kinase-Associated Neurodegeneration (PKAN) Patients: A Randomized, Double Blind, Placebo Controlled Study With an Open Label ExtensionPantothenate Kinase-Associated NeurodegenerationDrug: fosmetpantotenate (RE-024);Drug: PlaceboTravere Therapeutics, Inc.NULLActive, not recruiting6 Years65 YearsAll82Phase 3United States;Canada;Czechia;France;Germany;Italy;Norway;Poland;Spain
11EUCTR2016-001955-29-GB
(EUCTR)
02/06/201711/04/2017Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN)Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement therapy, in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN): A Randomized, Double-blind, Placebo-Controlled Study with an Open-Label Extension Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, the most common form of Neurodegeneration with Brain Iron Accumulation (NBIA). It is a progressive, often fatal, neurodegenerative disease.
MedDRA version: 20.0;Level: PT;Classification code 10053643;Term: Neurodegenerative disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Fosmetpantotenate
Product Code: RE-024
INN or Proposed INN: Fosmetpantotenate
Other descriptive name: FOSMETPANTOTENATE DRUG SUBSTANCE
Retrophin, Inc.NULLNot RecruitingFemale: yes
Male: yes
82Phase 3United States;Serbia;France;Czech Republic;Canada;Spain;Poland;Norway;Germany;Italy;United Kingdom
12NCT03019458
(ClinicalTrials.gov)
February 10, 20179/1/2017MINGO Supplemental Trial in X-linked Dystonia-Parkinsonism PatientsMINGO Supplemental Trial in X-linked Dystonia Parkinsonism Patients: A Prospective Randomized, Open-labeled, Parallel Group TrialX-Linked Dystonia ParkinsonismDietary Supplement: MINGOSunshine Care FoundationJose R. Reyes Memorial Medical Center;Massachusetts General HospitalCompleted18 Years90 YearsAll50N/APhilippines
13NCT02635841
(ClinicalTrials.gov)
February 10, 201617/12/2015Compassionate Use of Deferiprone in Patients With PKANThe Compassionate Use of Deferiprone in Patients With Pantothenate Kinase-Associated NeurodegenerationPantothenate Kinase-Associated NeurodegenerationDrug: DeferiproneChiesi Canada CorpNULLAvailable7 YearsN/AAllNULL
14EUCTR2014-001427-79-GB
(EUCTR)
30/12/201505/11/2015An international open label trial of deferiprone in patients with iron storage brain disorders - an extension to TIRCON2012V1 (a randomised controlled trial of deferiprone in patients with iron storage brain disorders).Long-term Safety and Efficacy Study of Deferiprone in Patients with Pantothenate Kinase-Associated Neurodegeneration (PKAN) - TIRCON2012V1-EXT Pantothenate Kinase-Associated Neurodegeneration (PKAN)
MedDRA version: 18.1;Level: PT;Classification code 10053643;Term: Neurodegenerative disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: Ferriprox
Product Name: Deferiprone 80 mg/mL oral solution
INN or Proposed INN: Deferiprone
Other descriptive name: Deferiprone
ApoPharma IncNULLNot RecruitingFemale: yes
Male: yes
89Phase 3United States;Germany;Italy;United Kingdom
15EUCTR2014-001427-79-DE
(EUCTR)
27/03/201527/03/2015An 18-month extension of an international trial of deferiprone in patients with iron storage brain disordersLong-term Safety and Efficacy Study of Deferiprone in Patients with Pantothenate Kinase-Associated Neurodegeneration (PKAN) - TIRCON Pantothenate kinase-associated neurodegeneration (PKAN);Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: deferiprone 80 mg/mL oral solution
INN or Proposed INN: DEFERIPRONE
ApoPharma Inc.NULLNot RecruitingFemale: yes
Male: yes
90Phase 3United States;Germany;Italy;United Kingdom
16NCT02174848
(ClinicalTrials.gov)
June 20145/6/2014Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated NeurodegenerationLong-term Safety and Efficacy Study of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration (PKAN)Pantothenate Kinase-Associated NeurodegenerationDrug: Deferiprone oral solutionApoPharmaNULLCompleted5 YearsN/AAll68Phase 3United States;Germany;Italy;United Kingdom
17EUCTR2012-000845-11-GB
(EUCTR)
24/10/201319/09/2013An international trial of deferiprone in patients with iron storage brain disordersA randomized, double-blind, placebo-controlled trial of deferiprone in patients with pantothenate kinase-associated neurodegeneration (PKAN) - TIRCON Pantothenate Kinase-Associated Neurodegeneration (PKAN)
MedDRA version: 16.0;Level: PT;Classification code 10053643;Term: Neurodegenerative disorder;System Organ Class: 10029205 - Nervous system disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: Ferriprox
Product Name: Deferiprone 80 mg/mL oral solution (Ferriprox)
INN or Proposed INN: Deferiprone
Other descriptive name: Deferiprone
ApoPharma IncNULLNot Recruiting Female: yes
Male: yes
90 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Poland;Germany;Italy;United Kingdom
18EUCTR2012-000845-11-IT
(EUCTR)
24/10/201331/07/2013An international trial of deferiprone in patients with iron storage brain disordersA randomized, double-blind, placebo-controlled trial of deferiprone in patients with pantothenate kinase-associated neurodegeneration (PKAN) - TIRCON Pantothenate kinase-associated neurodegeneration (PKAN);Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: Deferiprone 80 mg/mL oral solution
INN or Proposed INN: Deferiprone
Other descriptive name: Deferiprone
ApoPharma Inc.NULLNot RecruitingFemale: yes
Male: yes
90Phase 3United States;Poland;Germany;United Kingdom;Italy
19NCT01741532
(ClinicalTrials.gov)
December 13, 20123/12/2012Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)A Randomized, Double-blind, Placebo-controlled Trial of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)Pantothenate Kinase-Associated NeurodegenerationDrug: Deferiprone oral solution;Drug: PlaceboApoPharmaFood and Drug Administration (FDA)Completed4 YearsN/AAll89Phase 3United States;Germany;Italy;United Kingdom;Poland
20EUCTR2012-000845-11-DE
(EUCTR)
16/07/201230/03/2012An international trial of deferiprone in patients with iron storage brain disordersA randomized, double-blind, placebo-controlled trial of deferiprone in patients with pantothenate kinase-associated neurodegeneration (PKAN) - TIRCON Pantothenate kinase-associated neurodegeneration (PKAN);Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: Deferiprone 80 mg/mL oral solution
INN or Proposed INN: Deferiprone
Other descriptive name: Deferiprone
ApoPharma Inc.NULLNot RecruitingFemale: yes
Male: yes
90Phase 3United States;Poland;Germany;United Kingdom;Italy
21NCT03333200
(ClinicalTrials.gov)
January 11, 201224/4/2017Longitudinal Study of Neurodegenerative DisordersLongitudinal Study of Neurodegenerative DisordersMLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;Tay-Sachs Disease;NP Deficiency;Osteopetrosis;Alpha-Mannosidosis;Sandhoff Disease;Niemann-Pick Diseases;MPS IV;Gaucher Disease;GAN;GM1 Gangliosidoses;Morquio Disease;S-Adenosylhomocysteine Hydrolase Deficiency;Batten Disease;Pelizaeus-Merzbacher Disease;Leukodystrophy;Lysosomal Storage Diseases;Purine Nucleoside Phosphorylase Deficiency;Multiple Sulfatase Deficiency DiseaseOther: Palliative Care;Biological: Hematopoetic Stem Cell TransplantationUniversity of PittsburghNULLRecruitingN/AN/AAll1500United States
22EUCTR2008-003059-56-IT
(EUCTR)
29/07/200818/07/2008Phase II trial to assess safety and efficacy of Iron chelating agent Deferiprone in patients with Pantothenate Kinase-Associated Neurodegeneration ? ICAND ? Trial - NDPhase II trial to assess safety and efficacy of Iron chelating agent Deferiprone in patients with Pantothenate Kinase-Associated Neurodegeneration ? ICAND ? Trial - ND PKAN
MedDRA version: 9.1;Level: HLT;Classification code 10042259;Term: Structural brain disorders NEC
Trade Name: FERRIPROX*100CPR RIV 500MG
INN or Proposed INN: Deferiprone
ISTITUTO NEUROLOGICO CARLO BESTANULLNot RecruitingFemale: yes
Male: yes
Phase 2Italy
23EUCTR2016-001955-29-DE
(EUCTR)
23/03/2017Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN)Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement therapy, in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN): A Randomized, Double-blind, Placebo-Controlled Study with an Open-Label Extension Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, the most common form of Neurodegeneration with Brain Iron Accumulation (NBIA). It is a progressive, often fatal, neurodegenerative disease.
MedDRA version: 21.1;Level: PT;Classification code 10053643;Term: Neurodegenerative disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Fosmetpantotenate
Product Code: RE-024
INN or Proposed INN: Fosmetpantotenate
Other descriptive name: FOSMETPANTOTENATE DRUG SUBSTANCE
Retrophin, Inc.NULLNot RecruitingFemale: yes
Male: yes
82Phase 3United States;France;Czech Republic;Canada;Spain;Poland;Norway;Germany;United Kingdom;Italy
24EUCTR2016-001955-29-NO
(EUCTR)
20/04/2017Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN)Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement therapy, in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN): A Randomized, Double-blind, Placebo-Controlled Study with an Open-Label Extension Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, the most common form of Neurodegeneration with Brain Iron Accumulation (NBIA). It is a progressive, often fatal, neurodegenerative disease.
MedDRA version: 21.1;Level: PT;Classification code 10053643;Term: Neurodegenerative disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Fosmetpantotenate
Product Code: RE-024
INN or Proposed INN: Fosmetpantotenate
Other descriptive name: FOSMETPANTOTENATE DRUG SUBSTANCE
Retrophin, Inc.NULLNot RecruitingFemale: yes
Male: yes
82Phase 3France;United States;Czech Republic;Canada;Poland;Spain;Germany;Norway;Italy;United Kingdom
25EUCTR2016-001955-29-PL
(EUCTR)
02/08/2018Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN)Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement therapy, in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN): A Randomized, Double-blind, Placebo-Controlled Study with an Open-Label Extension Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, the most common form of Neurodegeneration with Brain Iron Accumulation (NBIA). It is a progressive, often fatal, neurodegenerative disease.
MedDRA version: 21.1;Level: PT;Classification code 10053643;Term: Neurodegenerative disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Fosmetpantotenate
Product Code: RE-024
INN or Proposed INN: Fosmetpantotenate
Other descriptive name: FOSMETPANTOTENATE DRUG SUBSTANCE
Retrophin, Inc.NULLNot RecruitingFemale: yes
Male: yes
82Phase 3United States;France;Czech Republic;Canada;Spain;Poland;Norway;Germany;United Kingdom;Italy