19. ライソゾーム病
[臨床試験数:784,薬物数:673(DrugBank:101),標的遺伝子数:68,標的パスウェイ数:184

Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
19 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2009-015811-42-PT
(EUCTR)
04/02/201109/11/2010A study with Eliglustat Tartrate (Genz-112638) in Patients with Gaucher disease to Evaluate Once Daily Dosing versus Twice DailyA Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once Daily versus Twice Daily Dosing of Genz-112638 in Patients with Gaucher Disease Type 1 who have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638 - EDGE Gaucher Disease Type I
MedDRA version: 14.1;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Genzyme Europe B.V.NULLNot RecruitingFemale: yes
Male: yes
170Phase 3United States;Serbia;Portugal;Greece;Austria;Russian Federation;India;France;Canada;Argentina;Brazil;Romania;Croatia;Australia;Netherlands;China;Japan;Sweden
2EUCTR2009-015811-42-GR
(EUCTR)
25/01/201119/05/2011A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once Daily versus Twice Daily Dosing of Genz-112638 in Patients with Gaucher Disease Type 1 who have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638 - EDGEA Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once Daily versus Twice Daily Dosing of Genz-112638 in Patients with Gaucher Disease Type 1 who have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638 - EDGE Gaucher Disease Type I
MedDRA version: 12.1;Level: PT;Classification code 10018048;Term: Gaucher's disease
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Genzyme Europe B.V.NULLNot RecruitingFemale: yes
Male: yes
234Phase 3Portugal;Greece;Austria;Sweden
3EUCTR2009-015811-42-AT
(EUCTR)
05/01/201122/11/2010A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once Daily versus Twice Daily Dosing of Genz-112638 in Patients with Gaucher Disease Type 1 who have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638 - EDGE Gaucher Disease Type I
MedDRA version: 15.0;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Genzyme Europe B.V.NULLNot RecruitingFemale: yes
Male: yes
170Phase 3Portugal;Serbia;United States;Greece;Austria;Russian Federation;India;France;Canada;Argentina;Brazil;Romania;Croatia;Australia;Netherlands;Japan;China;Sweden
4EUCTR2009-015811-42-SE
(EUCTR)
20/12/201001/11/2010A study of Eliglustat Tartrate (Genz-112638) in Patients with Gaucher Disease to Evaluate Once Daily versus Twice Daily DosingA Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once Daily versus Twice Daily Dosing of Genz-112638 in Patients with Gaucher Disease Type 1 who have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638 - EDGE Gaucher Disease Type I
MedDRA version: 16.0;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Genzyme Europe B.V.NULLNot RecruitingFemale: yes
Male: yes
170Phase 3Portugal;Serbia;United States;Greece;Austria;Russian Federation;India;France;Canada;Argentina;Brazil;Romania;Croatia;Australia;Netherlands;Japan;China;Sweden
5NCT01074944
(ClinicalTrials.gov)
June 201023/2/2010A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Once Daily Versus Twice Daily Dosing of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638Gaucher DiseaseDrug: Eliglustat tartrateGenzyme, a Sanofi CompanyNULLCompleted18 YearsN/AAll170Phase 3United States;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2008-005223-28-ES
(EUCTR)
03/03/201017/08/2009A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have been Stabilized with CerezymeEstudio en fase 3, aleatorizado, multicéntrico, multinacional, abierto y con comparador activo para evaluar la eficacia y seguridad de Genz 112638 en pacientes con enfermedad de Gaucher de tipo 1 que se han estabilizado con Cerezyme - ENCOREA Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have been Stabilized with CerezymeEstudio en fase 3, aleatorizado, multicéntrico, multinacional, abierto y con comparador activo para evaluar la eficacia y seguridad de Genz 112638 en pacientes con enfermedad de Gaucher de tipo 1 que se han estabilizado con Cerezyme - ENCORE Enfermedad de Gaucher tipo IGaucher Disease type I
MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: INN Not available
Other descriptive name: N/A
Genzyme Europe B.V.NULLNot RecruitingFemale: yes
Male: yes
96Phase 3France;Czech Republic;Spain;Germany;Netherlands;Italy;United Kingdom
7EUCTR2008-005222-37-BG
(EUCTR)
10/02/201011/11/2009A study with Eliglustat Tartrate (Genz-112638) in Patients With GaucherDiseaseA Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 - ENGAGE Gaucher Disease type I
MedDRA version: 16.1;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Genzyme Europe B.V.NULLNot RecruitingFemale: yes
Male: yes
36Phase 3United States;Serbia;Saudi Arabia;Lebanon;Israel;Russian Federation;Colombia;United Kingdom;India;Mexico;Canada;Jordan;Bulgaria;Netherlands;Tunisia
8EUCTR2008-005223-28-IT
(EUCTR)
29/01/201003/12/2009A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have been Stabilized with Cerezyme - ENCOREA Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have been Stabilized with Cerezyme - ENCORE Gaucher Disease Type 1
MedDRA version: 12.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Product Name: Genz-112638
Product Code: Genz-112638
Trade Name: CEREZYME
INN or Proposed INN: Imiglucerase
Genzyme Europe BVNULLNot RecruitingFemale: yes
Male: yes
96Phase 3France;Czech Republic;Spain;Germany;Netherlands;United Kingdom;Italy
9EUCTR2008-005223-28-DE
(EUCTR)
21/01/201011/08/2009A Study of Eliglustat Tartrate (Genz-112638) in Patients with Gaucher Disease Who Have Reached Thereapeutic Goals with Enzyme Replacement TherapyA Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have reached therapeutic goals with enzyme replacement therapy - ENCORE Gaucher Disease type I
MedDRA version: 18.0;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Trade Name: Cerezyme
Product Name: Cerezyme
Product Code: imiglucerase
INN or Proposed INN: IMIGLUCERASE
Other descriptive name: Recombinant human derived macrophage-targeted ß-Glucocerebrosidase
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Genzyme Europe B.V.NULLNot RecruitingFemale: yes
Male: yes
150Phase 3United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Egypt;France;Czech Republic;Canada;Argentina;Brazil;Australia;Netherlands;Germany
10EUCTR2008-005223-28-GB
(EUCTR)
16/11/200906/07/2009A Study of Eliglustat Tartrate (Genz-112638) in Patients with Gaucher Disease Who Have Reached Thereapeutic Goals with Enzyme Replacement TherapyA Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic Goals with Enzyme Replacement Therapy - ENCORE Gaucher Disease type I
MedDRA version: 14.1;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Genzyme Europe B.V.NULLNot Recruiting Female: yes
Male: yes
150Phase 3United States;Spain;Turkey;Russian Federation;Italy;United Kingdom;Egypt;France;Czech Republic;Canada;Argentina;Brazil;Australia;Netherlands;Germany
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2008-005222-37-GB
(EUCTR)
06/11/200918/06/2009A study with Eliglustat Tartrate (Genz-112638) in Patients With Gaucher DiseaseA Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 - ENGAGE Gaucher Disease type I
MedDRA version: 14.1;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Genzyme Europe B.V.NULLNot Recruiting Female: yes
Male: yes
36Phase 3Serbia;United States;Saudi Arabia;Lebanon;Russian Federation;Israel;Colombia;United Kingdom;India;Mexico;Canada;Jordan;Bulgaria;Netherlands;Tunisia
12EUCTR2008-005223-28-NL
(EUCTR)
04/11/200901/07/2009A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic Goals with Enzyme Replacement Therapy - ENCOREA Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic Goals with Enzyme Replacement Therapy - ENCORE Gaucher Disease type I
MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Trade Name: Cerezyme
Product Name: Cerezyme
Product Code: Imiglucerase
INN or Proposed INN: IMIGLUCERASE
Other descriptive name: Recombinant human derived macrophage-targeted ß-Glucocerebrosidase
Genzyme Europe B.V.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
186Phase 3Czech Republic;Germany;United Kingdom;Netherlands;France;Spain;Italy
13EUCTR2008-005222-37-NL
(EUCTR)
04/11/200910/06/2009A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 - ENGAGEA Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 - ENGAGE Gaucher Disease type I
MedDRA version: 13.1;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat
Other descriptive name: not available
Genzyme Europe B.V.NULLNot RecruitingFemale: yes
Male: yes
28Phase 3Bulgaria;Netherlands;United Kingdom
14NCT00891202
(ClinicalTrials.gov)
November 200930/4/2009A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 (ENGAGE)Gaucher Disease, Type 1Drug: Eliglustat tartrate;Drug: PlaceboGenzyme, a Sanofi CompanyNULLCompleted16 YearsN/AAll40Phase 3United States;Bulgaria;Canada;Colombia;India;Israel;Lebanon;Mexico;Russian Federation;Serbia;Tunisia;United Kingdom;Chile;Georgia;Jordan;Netherlands;Saudi Arabia
15EUCTR2008-005223-28-FR
(EUCTR)
22/10/200905/08/2009A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have been Stabilized with Cerezyme - ENCOREA Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have been Stabilized with Cerezyme - ENCORE Gaucher Disease type I
MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease
Product Name: Genz-112638
Product Code: Genz-112638
Trade Name: Cerezyme
Product Name: Cerezyme
Product Code: imiglucerase
INN or Proposed INN: IMIGLUCERASE
Other descriptive name: Recombinant human derived macrophage-targeted ß-Glucocerebrosidase
Genzyme Europe B.V.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
96Phase 3Czech Republic;Germany;United Kingdom;Netherlands;France;Spain;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2008-005223-28-CZ
(EUCTR)
23/09/200921/08/2009A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic goals with Enzyme Replacement Therapy - ENCOREA Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic goals with Enzyme Replacement Therapy - ENCORE Gaucher Disease type I
MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease
Product Name: Genz-112638
Product Code: Genz-112638
INN or Proposed INN: Eliglustat tartrate
Other descriptive name: not available
Trade Name: Cerezyme
Product Name: Cerezyme
Product Code: Imiglucerase
INN or Proposed INN: IMIGLUCERASE
Other descriptive name: recombinant human derived macrophage-targeted ß-glucocerebrosidase
Genzyme Europe B.V.NULLNot RecruitingFemale: yes
Male: yes
186Phase 3Germany;United Kingdom;Czech Republic;Netherlands;France;Spain;Italy
17NCT00943111
(ClinicalTrials.gov)
September 200920/7/2009A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)Gaucher Disease, Type 1Drug: Eliglustat tartrate;Drug: ImigluceraseGenzyme, a Sanofi CompanyNULLCompleted18 YearsN/AAll160Phase 3United States;Argentina;Australia;Brazil;Canada;Egypt;France;Germany;Italy;Russian Federation;Spain;United Kingdom;Czech Republic;Netherlands;Turkey
18EUCTR2005-004732-42-IT
(EUCTR)
12/10/200610/07/2007A Phase 2, Open-Label, Multi-Center Study Evaluating the Efficacy, Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 Patients - NDA Phase 2, Open-Label, Multi-Center Study Evaluating the Efficacy, Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 Patients - ND patients with diagnosis of Gaucher Type I disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Product Name: Genz-112638Genzyme Europe BVNULLNot RecruitingFemale: yes
Male: yes
20Phase 2Italy
19NCT00358150
(ClinicalTrials.gov)
June 200627/7/2006A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher PatientsA Phase 2, Open-Label, Multi-Center Study Evaluating the Efficacy, Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 PatientsGaucher Disease, Type 1;Cerebroside Lipidosis Syndrome;Glucocerebrosidase Deficiency Disease;Glucosylceramide Beta-Glucosidase Deficiency Disease;Gaucher Disease, Non-Neuronopathic FormDrug: Eliglustat tartrateGenzyme, a Sanofi CompanyNULLCompleted18 Years65 YearsAll26Phase 2United States;Argentina;Israel;Italy;Mexico;Russian Federation