120. 遺伝性ジストニア Hereditary dystonia Clinical trials / Disease details


臨床試験数 : 26 薬物数 : 19 - (DrugBank : 3) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 2

  
9 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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agemax
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PhaseCountries
1EUCTR2016-001955-29-FR
(EUCTR)
29/01/201804/06/2018Efficacy, 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.NULLNot RecruitingFemale: yes
Male: yes
82Phase 3Canada;Czech Republic;Norway;United States;Poland;Italy;United Kingdom;France;Germany;Spain
2EUCTR2016-001955-29-IT
(EUCTR)
10/11/201705/11/2020Efficacy, 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, INCNULLNot RecruitingFemale: yes
Male: yes
82Phase 3United States;France;Czechia;Czech Republic;Canada;Spain;Poland;Norway;Germany;United Kingdom;Italy
3EUCTR2016-001955-29-ES
(EUCTR)
16/10/201730/03/2017Efficacy, 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.NULLNot 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): noFrance;United States;Czech Republic;Canada;Poland;Spain;Norway;Germany;Italy;United Kingdom
4EUCTR2016-001955-29-CZ
(EUCTR)
06/10/201720/04/2017Efficacy, 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.NULLNot RecruitingFemale: yes
Male: yes
82Phase 3Canada;Czech Republic;Norway;United States;Poland;Italy;United Kingdom;France;Germany;Spain
5NCT03041116
(ClinicalTrials.gov)
July 17, 20172/12/2016Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN PatientsEfficacy, 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 ExtensionPantothenate Kinase-Associated NeurodegenerationDrug: fosmetpantotenate (RE-024);Drug: PlaceboTravere Therapeutics, Inc.NULLActive, not recruiting6 Years65 YearsAll82Phase 3United States;Canada;Czechia;France;Germany;Italy;Norway;Poland;Spain
6EUCTR2016-001955-29-GB
(EUCTR)
02/06/201711/04/2017Efficacy, 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.NULLNot RecruitingFemale: yes
Male: yes
82Phase 3United States;Serbia;France;Czech Republic;Canada;Spain;Poland;Norway;Germany;Italy;United Kingdom
7EUCTR2016-001955-29-NO
(EUCTR)
20/04/2017Efficacy, 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.NULLNot RecruitingFemale: yes
Male: yes
82Phase 3France;United States;Czech Republic;Canada;Poland;Spain;Germany;Norway;Italy;United Kingdom
8EUCTR2016-001955-29-DE
(EUCTR)
23/03/2017Efficacy, 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.NULLNot RecruitingFemale: yes
Male: yes
82Phase 3United States;France;Czech Republic;Canada;Spain;Poland;Norway;Germany;United Kingdom;Italy
9EUCTR2016-001955-29-PL
(EUCTR)
02/08/2018Efficacy, 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.NULLNot RecruitingFemale: yes
Male: yes
82Phase 3United States;France;Czech Republic;Canada;Spain;Poland;Norway;Germany;United Kingdom;Italy