120. Hereditary dystonia Clinical trials / Disease details
Clinical trials : 26 / Drugs : 19 - (DrugBank : 3) / Drug target genes : 2 - Drug target pathways : 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 | 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 | Canada;Czech Republic;Norway;United States;Poland;Italy;United Kingdom;France;Germany;Spain | ||
| 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] | 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 | ||
| 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 | Canada;Czech Republic;Norway;United States;Poland;Italy;United Kingdom;France;Germany;Spain | ||
| 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 |
| 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-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 | |||
| 8 | 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 | |||
| 9 | 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 |