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.
| 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 | 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 | ||
| 2 | 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 | ||
| 3 | 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 | |||
| 4 | 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 | ||
| 5 | 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 |
| 6 | 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 | ||
| 7 | 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 | |||
| 8 | 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 | |||
| 9 | 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 |