19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

  
2 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2015-003105-41-GB
(EUCTR)
13/10/201508/09/2015Treatment for Nonsense Mutation Mucopolysaccharidosis Type ICNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Ataluren (COMPASS) - COMPASS Nonsense mutation Mucopolysaccharidosis Type I
MedDRA version: 18.1;Level: PT;Classification code 10056886;Term: Mucopolysaccharidosis I;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
PTC Therapeutics Inc.NULLNot Recruiting Female: yes
Male: yes
5 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited Kingdom
2EUCTR2014-002596-28-GB
(EUCTR)
28/01/201512/06/2015Treatment for Nonsense Mutation Mucopolysaccharidosis Type IA Phase 2 Study of Ataluren (PTC124) as Treatment for Nonsense Mutation Mucopolysaccharidosis Type I Nonsense mutation Mucopolysaccharidosis Type I
MedDRA version: 20.1;Level: PT;Classification code 10056886;Term: Mucopolysaccharidosis I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
PTC Therapeutics Inc.NULLNot RecruitingFemale: yes
Male: yes
18Phase 2Germany;United Kingdom