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 | NCT04182763 (ClinicalTrials.gov) | December 4, 2019 | 24/11/2019 | CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN) | A Phase 2 Study of a Vitamin Metabolite for PKAN | Pantothenate Kinase-Associated Neurodegeneration | Other: CoA-Z;Other: Placebo | Oregon Health and Science University | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);Washington State University;Oregon State University;Spoonbill Foundation | Recruiting | 3 Months | 89 Years | All | 51 | Phase 2 | United States |
| 3 | NCT03726996 (ClinicalTrials.gov) | January 14, 2019 | 29/10/2018 | Desipramine 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 Dystrophy | Drug: Desipramine | Duke University | NULL | Terminated | 3 Years | 17 Years | All | 4 | Phase 4 | United States |
| 4 | NCT03570931 (ClinicalTrials.gov) | November 5, 2018 | 11/6/2018 | A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy | A Prospective Open-label Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy | Infantile Neuroaxonal Dystrophy | Drug: RT001 | Retrotope, Inc. | NULL | Active, not recruiting | 18 Months | 10 Years | All | 19 | Phase 2;Phase 3 | United States |
| 5 | ChiCTR1900021076 | 2018-09-04 | 2019-01-27 | A study for efficacy of pantethine in the treatment of pantothenate kinase-associated neurodegeneration | A study for efficacy of pantethine in the treatment of pantothenate kinase-associated neurodegeneration | pantothenate kinase-associated neurodegeneration | Experimental group:Standardized basal treatment + pantethine;Control group:Standardized basal treatment; | Department of Pediatrics, Peking University First Hospital | NULL | Recruiting | 0 | 16 | Both | Experimental group:12;Control group:6; | China | |
| 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 | EUCTR2016-001955-29-FR (EUCTR) | 29/01/2018 | 04/06/2018 | Efficacy, 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. | NULL | Not Recruiting | Female: yes Male: yes | 82 | Phase 3 | United States;France;Czech Republic;Canada;Poland;Spain;Norway;Germany;Italy;United Kingdom | ||
| 7 | EUCTR2016-001955-29-IT (EUCTR) | 10/11/2017 | 05/11/2020 | Efficacy, 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, INC | NULL | Not Recruiting | Female: yes Male: yes | 82 | Phase 3 | United States;France;Czechia;Czech Republic;Canada;Spain;Poland;Norway;Germany;United Kingdom;Italy | ||
| 8 | EUCTR2016-001955-29-ES (EUCTR) | 16/10/2017 | 30/03/2017 | Efficacy, 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. | NULL | Not Recruiting | Female: yes Male: yes | 82 | Phase 3 | France;United States;Czech Republic;Canada;Poland;Spain;Norway;Germany;Italy;United Kingdom | |||
| 9 | EUCTR2016-001955-29-CZ (EUCTR) | 06/10/2017 | 20/04/2017 | Efficacy, 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. | NULL | Not Recruiting | Female: yes Male: yes | 82 | Phase 3 | France;United States;Czech Republic;Canada;Poland;Spain;Norway;Germany;Italy;United Kingdom | ||
| 10 | NCT03041116 (ClinicalTrials.gov) | July 17, 2017 | 2/12/2016 | Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Patients | Efficacy, 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 Extension | Pantothenate Kinase-Associated Neurodegeneration | Drug: fosmetpantotenate (RE-024);Drug: Placebo | Travere Therapeutics, Inc. | NULL | Active, not recruiting | 6 Years | 65 Years | All | 82 | Phase 3 | United States;Canada;Czechia;France;Germany;Italy;Norway;Poland;Spain |
| 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 |
| 11 | EUCTR2016-001955-29-GB (EUCTR) | 02/06/2017 | 11/04/2017 | Efficacy, 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. | NULL | Not Recruiting | Female: yes Male: yes | 82 | Phase 3 | United States;Serbia;France;Czech Republic;Canada;Spain;Poland;Norway;Germany;Italy;United Kingdom | ||
| 12 | NCT03019458 (ClinicalTrials.gov) | February 10, 2017 | 9/1/2017 | MINGO Supplemental Trial in X-linked Dystonia-Parkinsonism Patients | MINGO Supplemental Trial in X-linked Dystonia Parkinsonism Patients: A Prospective Randomized, Open-labeled, Parallel Group Trial | X-Linked Dystonia Parkinsonism | Dietary Supplement: MINGO | Sunshine Care Foundation | Jose R. Reyes Memorial Medical Center;Massachusetts General Hospital | Completed | 18 Years | 90 Years | All | 50 | N/A | Philippines |
| 13 | 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 | ||
| 14 | 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 | ||
| 15 | 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 | ||
| 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 |
| 16 | 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 |
| 17 | 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 | |||
| 18 | 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 | ||
| 19 | 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 |
| 20 | 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 | ||
| 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 |
| 21 | NCT03333200 (ClinicalTrials.gov) | January 11, 2012 | 24/4/2017 | Longitudinal Study of Neurodegenerative Disorders | Longitudinal Study of Neurodegenerative Disorders | MLD;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 Disease | Other: Palliative Care;Biological: Hematopoetic Stem Cell Transplantation | University of Pittsburgh | NULL | Recruiting | N/A | N/A | All | 1500 | United States | |
| 22 | 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 | |||
| 23 | EUCTR2016-001955-29-PL (EUCTR) | 02/08/2018 | Efficacy, 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. | NULL | Not Recruiting | Female: yes Male: yes | 82 | Phase 3 | United States;France;Czech Republic;Canada;Spain;Poland;Norway;Germany;United Kingdom;Italy | |||
| 24 | EUCTR2016-001955-29-DE (EUCTR) | 23/03/2017 | Efficacy, 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. | NULL | Not Recruiting | Female: yes Male: yes | 82 | Phase 3 | United States;France;Czech Republic;Canada;Spain;Poland;Norway;Germany;United Kingdom;Italy | |||
| 25 | EUCTR2016-001955-29-NO (EUCTR) | 20/04/2017 | Efficacy, 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. | NULL | Not Recruiting | Female: yes Male: yes | 82 | Phase 3 | France;United States;Czech Republic;Canada;Poland;Spain;Germany;Norway;Italy;United Kingdom |