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 120. 遺伝性ジストニア [臨床試験数:15,薬物数:17(DrugBank:6),標的遺伝子数:2,標的パスウェイ数:2] 

Searched query = "Hereditary dystonia", "DYT1 dystonia", "DYT2 dystonia", "DYT3 dystonia", "X-linked dystonia-parkinsonism", "Lubag", "DYT4 dystonia", "DYT5 dystonia", "Segawa syndrome", "Dopa-responsive dystonia", "DYT6 dystonia", "DYT7 dystonia", "DYT8 dystonia", "Paroxysmal nonkinesigenic dyskinesia 1", "PNKD1", "DYT9 dystonia", "Paroxysmal dystonic choreathetosis with episodic ataxia and spasticity", "Paroxysmal choreoathetosis and episodic ataxia and spasticity", "DYT10 dystonia", "Episodic kinesigenic dyskinesia 1", "EKD1", "DYT11 dystonia", "Myoclonus-dystonia syndrome", "DYT12 dystonia", "Rapid-onset dystonia-parkinsonism", "Alternating hemiplegia of childhood", "Cerebellar ataxia, areflexia, pes cavus, optic atropy, and sensorineural hearing loss", "CAPOS", "DYT13 dystonia", "DYT14 dystonia", "DYT15 dystonia", "DYT16 dystonia", "DYT17 dystonia", "DYT18 dystonia", "Paroxysmal execise-induced dyskinesia", "DYT19 dystonia", "Episodic kinesigenic dyskinesia 2", "DYT20 dystonia", "Paroxysmal nonkinesigenic dyskinesia 2", "PNKD2", "Neurodegeneration with Brain Iron Accumulation 1", "Pantothenate kinase-associated neurodegeneration", "PKAN", "NBIA1", "Hallervorden-Spatz syndrome", "Hypoprebetalipoproteinemia, acanthocytosis, retinitis pigmentosa, pallidal degeneration", "Neurodegeneration with Brain Iron Accumulation 2", "Infantile neuroaxonal dystrophy", "INAD", "NBIA2", "Karak syndrome", "Neurodegeneration with Brain Iron Accumulation 3", "Neuroferritinopathy", "NBIA3", "Neurodegeneration with Brain Iron Accumulation 4", "Aceruloplasminemia", "Hereditary ceruloplasmin deficiency", "NBIA4", "Neurodegeneration with Brain Iron Accumulation 5", "NBIA5", "Beta-propeller protein-associated neurodegeneration", "BPAN", "Fatty Acid Hydroxylase-associated neurodegeneration", "Dysmyelinating leukodystrophy and spastic paraparasis with or without dystonia, spastic paraplegia 35"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03726996January 30, 20194 November 2019Desipramine 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 UniversityNot recruiting3 Years17 YearsAll4Phase 4United States
2NCT03570931November 5, 201822 July 2019A 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.Not recruiting18 Months10 YearsAll19Phase 2/Phase 3United States
3ChiCTR19000210762018-09-0428 January 2019A 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 neurodegenerationpantothenate kinase-associated neurodegenerationExperimental group:Standardized basal treatment + pantethine;Control group:Standardized basal treatment;Department of Pediatrics, Peking University First HospitalRecruiting016BothExperimental group:12;Control group:6;OtherChina
4EUCTR2016-001955-29-ES16/10/201723 October 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 ExtensionPantothenate 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
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: Fosmetpantotenate
CAS Number: 1858268-66-2
Current Sponsor code: RE-024
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 75-
Pharmaceutical form of the placebo: Powder for oral suspension
Route of administration of the placebo: Oral use
Retrophin, Inc.AuthorisedFemale: yes
Male: yes
82Phase 3France;United States;Czech Republic;Canada;Spain;Norway;Germany;Italy;United Kingdom
5NCT03041116July 17, 201714 January 2019Efficacy 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: Placebo;Drug: fosmetpantotenate (RE-024)Retrophin, Inc.Not recruiting6 Years65 YearsAll82Phase 3United States;Canada;Czechia;France;Germany;Italy;Norway;Poland;Spain
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT03019458February 10, 201716 December 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 HospitalNot recruiting18 Years90 YearsAll50N/APhilippines
7EUCTR2014-001427-79-GB30/12/201528 May 2018An 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-EXTPantothenate 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
Pharmaceutical Form: Oral solution
INN or Proposed INN: Deferiprone
CAS Number: 30652-11-0
Other descriptive name: Deferiprone
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 80-
ApoPharma IncNot RecruitingFemale: yes
Male: yes
89Phase 3United States;Germany;Italy;United Kingdom
8NCT02408354March 201516 December 2017Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of ChildhoodEtude Pilote, Comparative, Monocentrique, randomisée, en Cross Over, en Double Aveugle, Contre Placebo, Testant l'efficacité de l'Huile triheptanoïne Dans Les Hémiplégies Alternantes de l'Enfant HEMIHEPAlternating Hemiplegia of ChildhoodDrug: Triheptanoin;Drug: PlaceboInstitut National de la Santé Et de la Recherche Médicale, FranceNot recruiting15 YearsN/AAll10Phase 2France
9EUCTR2012-000845-11-GB24/10/201330 April 2019An 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) - TIRCONPantothenate 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)
Pharmaceutical Form: Oral solution
INN or Proposed INN: Deferiprone
CAS Number: 30652-11-0
Current Sponsor code: V03AC02
Other descriptive name: Deferiprone
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 80-
Pharmaceutical form of the placebo: Oral solution
Route of administration of the placebo: Oral use
ApoPharma IncNot Recruiting Female: yes
Male: yes
90Phase 3United States;Poland;Germany;Italy;United Kingdom
10NCT01741532December 13, 201215 July 2019Efficacy 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)Not recruiting4 YearsN/AAll89Phase 3United States;Germany;Italy;United Kingdom;Poland
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2012-000845-11-DE16/07/201221 November 2016An 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) - TIRCONPantothenate kinase-associated neurodegeneration (PKAN);Therapeutic area: Diseases [C] - Nervous System Diseases [C10]Product Name: Deferiprone 80 mg/mL oral solution
Pharmaceutical Form: Oral solution
INN or Proposed INN: Deferiprone
CAS Number: 30652-11-0
Current Sponsor code: V03AC02
Other descriptive name: Deferiprone
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 80-
Pharmaceutical form of the placebo: Oral solution
Route of administration of the placebo: Oral use
ApoPharma Inc.Not RecruitingFemale: yes
Male: yes
90Phase 3United States;Poland;Germany;United Kingdom;Italy
12NCT03333200January 11, 201211 November 2019Longitudinal 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 PittsburghRecruitingN/AN/AAll1500Phase 1United States
13NCT02635841October 200916 December 2017Compassionate Use of Deferiprone in Patients With PKANThe Compassionate Use of Deferiprone in Patients With Pantothenate Kinase-Associated NeurodegenerationPantothenate Kinase-Associated NeurodegenerationDrug: DeferiproneApoPharmaNot recruiting7 YearsN/AAllN/A
14NCT00931164August 200919 October 2017Sodium Oxybate in Patients With Alternating Hemiplegia of Childhood (AHC-SO)Single-center Phase I/II Trial of Sodium Oxybate in Patients With Alternating Hemiplegia of Childhood (AHC-SO Trial)Alternating Hemiplegia of ChildhoodDrug: Sodium OxybateUniversity of UtahAlternating Hemiplegia of Childhood Foundation;Jazz PharmaceuticalsNot recruiting6 Months25 YearsAll6Phase 1/Phase 2United States
15EUCTR2008-003059-56-IT29/07/200819 March 2012Phase 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 - NDPKAN
MedDRA version: 9.1 Level: HLT Classification code 10042259 Term: Structural brain disorders NEC
Trade Name: FERRIPROX*100CPR RIV 500MG
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Deferiprone
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 25-
ISTITUTO NEUROLOGICO CARLO BESTANot RecruitingFemale: yes
Male: yes
Phase 2Italy

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