120. 遺伝性ジストニア Hereditary dystonia Clinical trials / Disease details
臨床試験数 : 25 / 薬物数 : 21 - (DrugBank : 4) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 2
| 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 | |
| 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] | Product Name: Fosmetpantotenate Product Code: RE-024 INN or Proposed INN: Fosmetpantotenate | Retrophin, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 82 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | 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 |
| 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) | February 10, 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 | ||
| 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] | Trade Name: Ferriprox Product Name: Deferiprone 80 mg/mL oral solution (Ferriprox) INN or Proposed INN: Deferiprone Other descriptive name: Deferiprone | ApoPharma Inc | NULL | Not Recruiting | Female: yes Male: yes | 90 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | 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 | ||
| 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-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 | |||
| 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-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 |