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
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
1EUCTR2010-020048-36-GB
(EUCTR)
31/01/201117/06/2010A safety, tolerability and preliminary efficacy study of idursulfase-IT in patients with Hunter syndrome associated with learning disabilityA Phase I/II, Randomized, Safety and Ascending Dose Ranging Study of Intrathecal Idursulfase-IT administered in conjunction with intravenous Elaprase in Pediatric Patients with Hunter Syndrome and Cognitive Impairment Treatment of Hunter syndrome and cognitive impairment
MedDRA version: 14.1;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Name: Idursulfase (I2S)-IT
Product Code: I2S-IT
INN or Proposed INN: NA
Other descriptive name: Idursulfase-IT
Shire HGT, IncNULLNot Recruiting Female: no
Male: yes
20 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;United Kingdom
2EUCTR2011-000212-25-Outside-EU/EEA
(EUCTR)
19/02/2014An extension to the safety, tolerability and preliminary efficacy study of Idursulfase-IT in patients with Hunter syndrome associated with learning disabilityAn Open-Label Extension of Study HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Intrathecal Idursulfase-IT Administered in Conjunction with Intravenous Elaprase® in Pediatric Patients with Hunter Syndrome and Cognitive Impairment Treatment of Hunter syndrome and cognitive impairment
MedDRA version: 16.1;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Name: Idursulfase(I2S)-IT
Product Code: I2S-IT
INN or Proposed INN: IDURSULFASE
Other descriptive name: idursulfase-IT
Shire HGT IncNULLNAFemale: no
Male: yes
15Canada;United States