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

  
6 trials found
No.TrialIDDate_
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agemin
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PhaseCountries
1EUCTR2005-003512-30-FR
(EUCTR)
05/04/200726/03/2007A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme (galsulfase) in Infants with Maroteaux-Lamy Syndrome (MPS VI) - N/AA Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme (galsulfase) in Infants with Maroteaux-Lamy Syndrome (MPS VI) - N/A Mucopolysaccharidosis Type VI (MPS VI; Maroteaux-Lamy Syndrome)
MedDRA version: 6.0;Level: PT;Classification code 10056892
Trade Name: Naglazyme
Product Name: Naglazyme
INN or Proposed INN: galsulfase
Other descriptive name: recombinant human Arysulfatase B, recombinant human N-acetylgalactosamine 4-sulfatase
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
4Phase 4Portugal;France
2NCT00104234
(ClinicalTrials.gov)
February 200424/2/2005Study of rhASB in Patients With Mucopolysaccharidosis VIA Multicenter, Multinational Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With Mucopolysaccharidosis VIMucopolysaccharidosis VIDrug: N-acetylgalactosamine 4-sulfatase;Drug: Placebo/rhASBBioMarin PharmaceuticalNULLCompleted7 YearsN/AAll39Phase 3United States
3NCT00067470
(ClinicalTrials.gov)
September 200320/8/2003Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VIMucopolysaccharidosis VIDrug: Placebo;Drug: N-acetylgalactosamine 4-sulfataseBioMarin PharmaceuticalNULLCompleted7 YearsN/AAll39Phase 3United States
4NCT00048711
(ClinicalTrials.gov)
March 20026/11/2002Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VIOpen-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VIMucopolysaccharidosis VIDrug: N-acetylgalactosamine 4-sulfataseBioMarin PharmaceuticalNULLCompleted5 YearsN/ABothPhase 2United States
5NCT00048620
(ClinicalTrials.gov)
September 20004/11/2002Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VIDouble-Blind,2 Dose Group Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VIMucopolysaccharidosis VIDrug: N-acetylgalactosamine 4-sulfataseBioMarin PharmaceuticalNULLCompletedN/AN/ABothPhase 1United States
6EUCTR2004-000642-21-IE
(EUCTR)
18/06/2004A Multicenter, Multinational, Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients with Mucopolysaccharidosis VI - Aryplase Phase 3 Open-Label ExtensionA Multicenter, Multinational, Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients with Mucopolysaccharidosis VI - Aryplase Phase 3 Open-Label Extension Mucopolysaccharidosis Type VI (MPS VI; Maroteaux-Lamy Syndrome)
MedDRA version: 6.0;Level: PT;Classification code 10056892
Product Name: recombinant human N-acetylgalactosamine 4-sulfatase
Product Code: N/A
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
38 Human pharmacology (Phase 1): Therapeutic exploratory (Phase 2): Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4):Ireland