120. Hereditary dystonia
25 clinical trials,   22 drugs   (DrugBank: 4 drugs),   2 drug target genes,   2 drug target pathways

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"
10 trials found
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
2NCT02635841
(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
3EUCTR2014-001427-79-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
4EUCTR2014-001427-79-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
5NCT02174848
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2012-000845-11-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
7EUCTR2012-000845-11-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
8NCT01741532
(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
9EUCTR2012-000845-11-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
10EUCTR2008-003059-56-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