120. 遺伝性ジストニア
[臨床試験数:25,薬物数:22(DrugBank:4),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", "DYT5a dystonia", "DYT5b 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", "SS", "DRD", "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. 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
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
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]
Retrophin, Inc.NULLNot Recruiting Female: yes
Male: yes
82Phase 3France;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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
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)
January 8, 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
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]
ApoPharma IncNULLNot Recruiting Female: yes
Male: yes
90Phase 3United 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
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-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
24EUCTR2016-001955-29-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
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