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

  
1 trial found
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agemin
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1EUCTR2016-001955-29-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