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_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1EUCTR2005-001651-37-HU
(EUCTR)
10/12/200704/10/2007Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease.Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease. Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 18 of the protocol:1BACKGROUND AND RATIONALE1.1Disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Trade Name: Zavesca
Product Name: Miglustat
Product Code: OGT918
INN or Proposed INN: miglustat
Actelion Pharmaceuticals LtdNULLNot RecruitingFemale: yes
Male: yes
50Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy
2EUCTR2005-001651-37-CZ
(EUCTR)
02/10/200617/08/2006Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease. - MAINTENANCEOpen-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease. - MAINTENANCE Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol:1BACKGROUND AND RATIONALE1.1Disease
MedDRA version: 8.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Trade Name: Zavesca
Product Name: Miglustat
Product Code: OGT918
INN or Proposed INN: miglustat
Actelion Pharmaceuticals LtdNULLNot RecruitingFemale: yes
Male: yes
50Hungary;Germany;United Kingdom;Czech Republic;Spain;Italy
3EUCTR2005-001651-37-ES
(EUCTR)
12/04/200625/01/2006A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher DiseaseA phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol:1BACKGROUND AND RATIONALE1.1Disease Trade Name: Zavesca
Product Name: Miglustat
Product Code: OGT918
INN or Proposed INN: miglustat
Actelion Pharmaceuticals LtdNULLNot RecruitingFemale: yes
Male: yes
50Phase 4Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy
4EUCTR2005-001651-37-DE
(EUCTR)
28/02/200616/05/2006Open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance therapy after a switch from Enzyme Replacement Therapy in adult patients with stable Type 1 Gaucher disease - MAINTENANCEOpen-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance therapy after a switch from Enzyme Replacement Therapy in adult patients with stable Type 1 Gaucher disease - MAINTENANCE Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 18 of the protocol:1BACKGROUND AND RATIONALE1.1Disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Trade Name: ZAVESCA
Product Name: Miglustat
Product Code: OGT918
INN or Proposed INN: miglustat
Actelion Pharmaceuticals LtdNULLNot RecruitingFemale: yes
Male: yes
50Hungary;Czech Republic;Spain;Germany;Italy;United Kingdom
5EUCTR2005-001651-37-GB
(EUCTR)
27/10/200503/10/2005A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher DiseaseA phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol: 1BACKGROUND AND RATIONALE 1.1DiseaseTrade Name: Zavesca
Product Name: Miglustat
Product Code: OGT918
INN or Proposed INN: miglustat
Actelion Pharmaceuticals LtdNULLNot Recruiting Female: yes
Male: yes
50 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): yesHungary;Czech Republic;Spain;Germany;Italy;United Kingdom
6NCT00316498
(ClinicalTrials.gov)
October 17, 200219/4/2006Saccadic Eye Movements in Patients With Niemann-Pick Type C DiseaseA Phase I/II Randomized, Controlled Study of OGT 918 in Adult and Juvenile Patients With Niemann Pick C DiseaseNiemann Pick DiseasesDrug: OGT918National Eye Institute (NEI)NULLCompleted12 YearsN/AAll30Phase 1United States;United Kingdom