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.
| 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 | NCT04184453 (ClinicalTrials.gov) | December 23, 2019 | 29/11/2019 | Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia Patients | Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia Patients | Aceruloplasminemia | Drug: Deferiprone treated | First Affiliated Hospital of Fujian Medical University | NULL | Recruiting | 18 Years | 80 Years | All | 5 | Early Phase 1 | China |
| 2 | NCT02635841 (ClinicalTrials.gov) | January 8, 2016 | 17/12/2015 | Compassionate Use of Deferiprone in Patients With PKAN | The Compassionate Use of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration | Pantothenate Kinase-Associated Neurodegeneration | Drug: Deferiprone | Chiesi Canada Corp | NULL | Available | 7 Years | N/A | All | NULL | ||
| 3 | EUCTR2014-001427-79-GB (EUCTR) | 30/12/2015 | 05/11/2015 | An 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 Inc | NULL | Not Recruiting | Female: yes Male: yes | 89 | Phase 3 | United States;Germany;Italy;United Kingdom | ||
| 4 | EUCTR2014-001427-79-DE (EUCTR) | 27/03/2015 | 27/03/2015 | An 18-month extension of an international 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) - 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. | NULL | Not Recruiting | Female: yes Male: yes | 90 | Phase 3 | United States;Germany;Italy;United Kingdom | ||
| 5 | NCT02174848 (ClinicalTrials.gov) | June 2014 | 5/6/2014 | Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration | Long-term Safety and Efficacy Study of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration (PKAN) | Pantothenate Kinase-Associated Neurodegeneration | Drug: Deferiprone oral solution | ApoPharma | NULL | Completed | 5 Years | N/A | All | 68 | Phase 3 | United States;Germany;Italy;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 | EUCTR2012-000845-11-IT (EUCTR) | 24/10/2013 | 31/07/2013 | An international trial of deferiprone in patients with iron storage brain disorders | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 90 | Phase 3 | United States;Poland;Germany;United Kingdom;Italy | ||
| 7 | EUCTR2012-000845-11-GB (EUCTR) | 24/10/2013 | 19/09/2013 | An international trial of deferiprone in patients with iron storage brain disorders | A 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 Inc | NULL | Not Recruiting | Female: yes Male: yes | 90 | Phase 3 | United States;Poland;Germany;Italy;United Kingdom | |||
| 8 | NCT01741532 (ClinicalTrials.gov) | December 13, 2012 | 3/12/2012 | Efficacy 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 Neurodegeneration | Drug: Deferiprone oral solution;Drug: Placebo | ApoPharma | Food and Drug Administration (FDA) | Completed | 4 Years | N/A | All | 89 | Phase 3 | United States;Germany;Italy;United Kingdom;Poland |
| 9 | EUCTR2012-000845-11-DE (EUCTR) | 16/07/2012 | 30/03/2012 | An international trial of deferiprone in patients with iron storage brain disorders | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 90 | Phase 3 | United States;Poland;Germany;United Kingdom;Italy | ||
| 10 | EUCTR2008-003059-56-IT (EUCTR) | 29/07/2008 | 18/07/2008 | Phase II trial to assess safety and efficacy of Iron chelating agent Deferiprone in patients with Pantothenate Kinase-Associated Neurodegeneration ? ICAND ? Trial - ND | Phase 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 BESTA | NULL | Not Recruiting | Female: yes Male: yes | Phase 2 | Italy |