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

  
1 trial found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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agemin
Inclusion_
agemax
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PhaseCountries
1EUCTR2005-004384-33-GB
(EUCTR)
19/12/200517/10/2005A Phase 2, Open-Label, Single Dose Level, 24-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients with Fabry Disease - Phase 2 study of AT1001 in Fabry DiseaseA Phase 2, Open-Label, Single Dose Level, 24-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients with Fabry Disease - Phase 2 study of AT1001 in Fabry Disease Fabry Disease
MedDRA version: 8.1;Level: LLT;Classification code 10016016
Product Name: 1- Deoxygalactonojirimycin
Product Code: AT1001
INN or Proposed INN: N/A
Other descriptive name: Migalastat hydrochloride
Amicus TherapeuticsNULLNot Recruiting Female: no
Male: yes
8 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited Kingdom