CEREZYME ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
19ライソゾーム病34

19. ライソゾーム病


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
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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Recruitment_
Status
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PhaseCountries
1EUCTR2021-005402-10-DE
(EUCTR)
23/03/202205/01/2022Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 (GD3) who have reached therapeutic goals with Enzyme Replacement Therapy (ERT) - LEAP2MONO Gaucher's disease type III
MedDRA version: 24.1;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: venglustat
Product Code: SAR402671, GZ402671 or GZ / SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Product Name: venglustat
Product Code: SAR402671, GZ402671 or GZ / SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Cerezyme 400 Units Powder for concentrate for solution for infusion
INN or Proposed INN: Imiglucerase
Sanofi-aventis recherche & développementNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
2Phase 3Brazil;Germany;China;United States;Taiwan;Turkey;United Kingdom;Egypt;France;Hungary;Canada;Argentina;Japan
2EUCTR2020-003120-17-FR
(EUCTR)
18/05/202111/03/2021Venglustat in Combination with Cerezyme in Adult and Pediatric Patients with Gaucher Disease Type 3A 3-part study to evaluate the efficacy and safety of venglustat in combination with Cerezyme in adult and pediatric patients with Gaucher disease Type 3 (GD3) with open-label long-term treatment - LEAP2IT Gaucher disease type 3
MedDRA version: 20.0;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat malate
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat malate
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLNot RecruitingFemale: yes
Male: yes
12Phase 2;Phase 3United States;Taiwan;Turkey;France;Egypt;Hungary;Canada;Argentina;Brazil;Germany;China;Japan;Sweden
3EUCTR2020-003120-17-DE
(EUCTR)
12/04/202123/02/2021Venglustat in Combination with Cerezyme in Adult and Pediatric Patients with Gaucher Disease Type 3A 3-part study to evaluate the efficacy and safety of venglustat in combination with Cerezyme in adult and pediatric patients with Gaucher disease Type 3 (GD3) with open-label long-term treatment - LEAP2IT Gaucher disease type 3
MedDRA version: 20.0;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat malate
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat malate
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLNot RecruitingFemale: yes
Male: yes
12Phase 2;Phase 3United States;Taiwan;Turkey;Egypt;France;Hungary;Canada;Argentina;Brazil;Germany;China;Japan;Sweden
4EUCTR2020-003120-17-HU
(EUCTR)
06/04/202124/02/2021Venglustat in Combination with Cerezyme in Adult and Pediatric Patients with Gaucher Disease Type 3A 3-part study to evaluate the efficacy and safety of venglustat in combination with Cerezyme in adult and pediatric patients with Gaucher disease Type 3 (GD3) with open-label long-term treatment - LEAP2IT Gaucher disease type 3
MedDRA version: 20.0;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat malate
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat malate
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLNot RecruitingFemale: yes
Male: yes
12Phase 2;Phase 3United States;Taiwan;Turkey;Egypt;France;Hungary;Canada;Argentina;Brazil;Germany;China;Japan;Sweden
5NCT04656600
(ClinicalTrials.gov)
March 2, 20214/12/2020Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type ?A Single Arm, Prospective, Open Label, Multicenter Study to Evaluate Efficacy and Safety of One-year Maximum Dosage in Chinese Label of Imiglucerase Treatment in Chinese Patients Who Are Diagnosed as Gaucher Disease Type ?Gaucher's DiseaseDrug: Cerezyme® / ImigluceraseSanofiNULLRecruiting2 YearsN/AAll12Phase 4China
6NCT04787887
(ClinicalTrials.gov)
January 29, 20204/3/2021A Phase I Study to Compare Abcertin and EU-sourced Cerezyme® in Healthy VolunteersA Randomized, Double-blind, 2-treatment, 2-period, Crossover Phase I Study to Compare the PK, Safety and Tolerability of 60 IU/kg of Abcertin, and EU-sourced Cerezyme® in Healthy Volunteers Following a Single Intravenous AdministrationGaucher DiseaseDrug: Abcertin;Drug: EU-sourced CerezymeISU Abxis Co., Ltd.NULLCompleted18 Years45 YearsAll42Phase 1Australia
7EUCTR2016-000301-37-NL
(EUCTR)
06/03/201928/06/2018Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 - ELIKIDS Congenital, hereditary and neonatal diseases
MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Cerdelga
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Trade Name: Cerezyme
Product Name: Imiglucerase
INN or Proposed INN: IMIGLUCERASE
Other descriptive name: Cerezyme
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
120 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;Italy;United Kingdom;Sweden
8EUCTR2016-000301-37-GB
(EUCTR)
09/01/201917/06/2019Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 - ELIKIDS Congenital, hereditary and neonatal diseases
MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Cerdelga
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Trade Name: Cerezyme
Product Name: Imiglucerase
INN or Proposed INN: IMIGLUCERASE
Other descriptive name: Cerezyme
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
120 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;Italy;United Kingdom;Sweden
9EUCTR2016-000301-37-SE
(EUCTR)
09/10/201805/06/2018Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 - ELIKIDS Congenital, hereditary and neonatal diseases
MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Cerdelga
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Trade Name: Cerezyme
Product Name: Imiglucerase
INN or Proposed INN: IMIGLUCERASE
Other descriptive name: Cerezyme
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Product Name: Eliglustat
Product Code: GZ385660
Other descriptive name: Eliglustat Tartrate – Powder for Oral Suspension
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 3Taiwan;Spain;Turkey;Russian Federation;Italy;United Kingdom;Egypt;France;Canada;Argentina;Netherlands;Japan;Sweden
10EUCTR2016-000301-37-IT
(EUCTR)
02/07/201820/06/2018Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 - ELIKIDS Congenital, hereditary and neonatal diseases
MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Cerdelga
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Trade Name: Cerezyme
Product Name: Imiglucerase
INN or Proposed INN: IMIGLUCERASE
Other descriptive name: Cerezyme
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 3France;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;United Kingdom;Italy;Sweden
11EUCTR2016-000301-37-ES
(EUCTR)
21/06/201806/07/2018Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3 - ELIKIDS Congenital, hereditary and neonatal diseases
MedDRA version: 20.0;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Cerdelga
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Trade Name: Cerezyme
Product Name: Imiglucerase
INN or Proposed INN: IMIGLUCERASE
Other descriptive name: Cerezyme
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Product Name: Eliglustat
Product Code: GZ385660
INN or Proposed INN: Eliglustat
Other descriptive name: ELIGLUSTAT TARTRATE
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 3France;Egypt;Canada;Argentina;Spain;Turkey;Russian Federation;Netherlands;Italy;United Kingdom;Sweden
12NCT03519646
(ClinicalTrials.gov)
April 23, 201820/3/2018Eliglustat on Gaucher Disease Type IIIBEvaluation of the Safety in the Combination Usage of Cerdelga and Cerezyme in Type III Gaucher Disease Patients and the Efficacy on Soft Tissue Diseases.Gaucher Disease, Type IIIDrug: EliglustatNational Taiwan University HospitalSanofiCompleted6 YearsN/AAll4N/ATaiwan
13NCT02843035
(ClinicalTrials.gov)
January 4, 201720/7/2016Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy ExtensionA 4-part, Open-label, Multicenter, Multinational Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic, and Exploratory Efficacy of Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy ExtensionGaucher Disease Type 1;Gaucher Disease Type 3Drug: venglustat (GZ402671);Drug: imigluceraseGenzyme, a Sanofi CompanyNULLActive, not recruiting18 YearsN/AAll13Phase 2United States;Germany;Japan;United Kingdom
14EUCTR2014-002550-39-DE
(EUCTR)
20/10/201625/04/2016GZ/SAR402671 in Combination with Cerezyme in Adult Patients with Gaucher Disease Type 3A 208-week three-part, open-label, multicenter, multinational study of the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of GZ/SAR402671 in combination with Cerezyme in adult patients with Gaucher disease type 3 - LEAP Gaucher disease
MedDRA version: 20.0;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: SAR402671, GZ402671 or GZ/SAR402671
Other descriptive name: Genz-682452-AU
Product Code: SAR402671, GZ402671 or GZ/SAR402671
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
15Phase 2United States;Japan;United Kingdom;Germany
15NCT01161914
(ClinicalTrials.gov)
January 201111/7/2010The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher DiseaseA Multi-national Randomized Double Blinded Phase III Study to Evaluate the Safety and Efficacy of ISU302(Imiglucerase for Injection) or Cerezyme in Patient With Type I Gaucher DiseaseGaucher DiseaseDrug: Cerezyme®;Drug: ISU302ISU Abxis Co., Ltd.NULLWithdrawn2 Years75 YearsBoth0Phase 3NULL
16EUCTR2008-005223-28-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
17EUCTR2008-005223-28-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
18EUCTR2008-005223-28-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
19EUCTR2008-005825-12-GB
(EUCTR)
17/12/200928/04/2009A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NAA Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NA Gaucher Disease
MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease
Product Name: UPLYSO
Product Code: Taliglucerase alfa
Other descriptive name: recombinant human glucocerebrosidase (prGCD)
Protalix BiotherapeuticsNULLNot Recruiting Female: yes
Male: yes
30 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noSpain;Germany;Italy;United Kingdom
20EUCTR2008-005825-12-DE
(EUCTR)
10/12/200916/09/2009A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NAA Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NA Gaucher Disease
MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease
Product Name: UPLYSO
Product Code: Taliglucerase alfa
INN or Proposed INN: Taliglucerase alfa
Other descriptive name: Recombinant Human Glucocerebrosidase (prGCD)
Protalix BiotherapeuticsNULLNot RecruitingFemale: yes
Male: yes
30Phase 3Spain;Germany;Italy;United Kingdom
21EUCTR2008-005223-28-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]
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.NULLNot Recruiting Female: yes
Male: yes
150 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Spain;Turkey;Russian Federation;Italy;United Kingdom;Egypt;France;Czech Republic;Canada;Argentina;Brazil;Australia;Netherlands;Germany
22EUCTR2008-005223-28-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.NULLNot RecruitingFemale: yes
Male: yes
186Phase 3France;Czech Republic;Spain;Germany;Netherlands;Italy;United Kingdom
23EUCTR2008-005223-28-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.NULLNot RecruitingFemale: yes
Male: yes
96Phase 3France;Czech Republic;Spain;Germany;Netherlands;Italy;United Kingdom
24EUCTR2008-005223-28-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
25EUCTR2008-005825-12-IT
(EUCTR)
28/07/200916/09/2009: A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme) Enzyme Replacement Therapy - PB-06-002: A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme) Enzyme Replacement Therapy - PB-06-002 Gaucher disease
MedDRA version: 9.1;Level: SOC;Classification code 10029205
Product Name: Plant Cell Recombinant Glucocerebrosidase
Product Code: prGCD
PROTALIX BIOTHERAPEUTICSNULLNot RecruitingFemale: yes
Male: yes
15Phase 3Germany;United Kingdom;Spain;Italy
26EUCTR2008-005825-12-ES
(EUCTR)
04/06/200906/03/2009Ensayo clinico de fase 3, multicéntrico, en abierto y con cambio de tratamiento para evaluar la seguridad y eficacia de Glucocerebrosidasa Humana Recombinante expresada en células vegetales (prGCD) en pacientes con enfermedad de Gaucher tratados con Imiglucerasa (Cerezyme®) como terapia de reemplazo enzimatico. A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NAEnsayo clinico de fase 3, multicéntrico, en abierto y con cambio de tratamiento para evaluar la seguridad y eficacia de Glucocerebrosidasa Humana Recombinante expresada en células vegetales (prGCD) en pacientes con enfermedad de Gaucher tratados con Imiglucerasa (Cerezyme®) como terapia de reemplazo enzimatico. A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NA Enfermedad de GaucherGaucher Disease
MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease
Product Name: recombinant human glucocerebrosidase
Product Code: prGCD
Other descriptive name: recombinant human glucocerebrosidase
Protalix BiotherapeuticsNULLNot RecruitingFemale: yes
Male: yes
15Phase 3Germany;United Kingdom;Spain;Italy
27EUCTR2007-005516-61-IT
(EUCTR)
06/02/200923/03/2009Multicenter randomized study to assess the efficacy and the safety of two therapeutic regimens(high dose of imiglucerase versus co-administration of imiglucerase and miglustat) in type I Gaucher disease patients who have not responded to previous treatment with low dose imiglucerase - NDMulticenter randomized study to assess the efficacy and the safety of two therapeutic regimens(high dose of imiglucerase versus co-administration of imiglucerase and miglustat) in type I Gaucher disease patients who have not responded to previous treatment with low dose imiglucerase - ND GAUCHER disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Trade Name: CEREZYME
INN or Proposed INN: Imiglucerase
Trade Name: ZAVESCA
INN or Proposed INN: Miglustat
ISTITUTO GIANNINA GASLININULLNot RecruitingFemale: yes
Male: yes
Italy
28EUCTR2007-007164-20-ES
(EUCTR)
20/05/200821/02/2008Uso racional de los tratamientos por inhibición de sustrato y enzimático sustitutivo en pacientes con Enfermedad de Gaucher tipo 1Uso racional de los tratamientos por inhibición de sustrato y enzimático sustitutivo en pacientes con Enfermedad de Gaucher tipo 1 Tratamiento de la enfermedad de Gaucher
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Trade Name: CEREZYME
INN or Proposed INN: IMIGLUCERASA
Trade Name: MIGLUSTAT (ZAVESCA)
INN or Proposed INN: MIGLUSTAT
INSTITUTO ARAGONÉS DE CIENCIAS DE LA SALUDNULLNot RecruitingFemale: yes
Male: yes
Spain
29EUCTR2007-002840-21-GB
(EUCTR)
29/11/200711/10/2007A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - HGT-GCB-039A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - HGT-GCB-039 Type I Gaucher disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Product Name: Gene Activated Human glucocerebrosidase
Product Code: GA-GCB
INN or Proposed INN: velaglucerase alfa
Other descriptive name: Gene activated human glucocerebrosidase
Trade Name: Cerezyme
Product Name: Cerezyme
INN or Proposed INN: IMIGLUCERASE
Trade Name: Cerezyme
Product Name: Cerezyme
INN or Proposed INN: IMIGLUCERASE
Shire Human Genetic Therapies, Inc.NULLNot RecruitingFemale: yes
Male: yes
32United Kingdom;Spain;Italy
30EUCTR2007-002840-21-ES
(EUCTR)
30/10/200718/07/2007A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher DiseaseEstudio multicéntrico, aleatorizado, a doble ciego, en grupos paralelos sobre la terapia de reemplazo enzimático con Gene-Activated® glucocerebrosidasa humana (GA-GCB) en comparación con imiglucerasa en pacientes con enfermedad de Gaucher de tipo 1 - GCB039A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher DiseaseEstudio multicéntrico, aleatorizado, a doble ciego, en grupos paralelos sobre la terapia de reemplazo enzimático con Gene-Activated® glucocerebrosidasa humana (GA-GCB) en comparación con imiglucerasa en pacientes con enfermedad de Gaucher de tipo 1 - GCB039 Type I Gaucher diseaseEnfermedad de Gaucher tipo 1
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Product Name: Gene Activated Human glucocerebrosidase
Product Code: GA-GCB
Other descriptive name: Gene activated human glucocerebrosidase
Trade Name: Cerezyme
INN or Proposed INN: IMIGLUCERASE
Shire Human Genetic Therapies IncNULLNot RecruitingFemale: yes
Male: yes
32Phase 3Spain;Italy;United Kingdom
31NCT00364858
(ClinicalTrials.gov)
December 200115/8/2006Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher DiseaseA Phase IV, Multicenter, Randomized, Dose Frequency Study of the Safety and Efficacy of Cerezyme® Infusions Every Four Weeks Versus Every Two Weeks in the Maintenance Therapy of Patients With Type 1 Gaucher DiseaseGaucher Disease, Type 1;Cerebroside Lipidosis Syndrome;Glucocerebrosidase Deficiency Disease;Glucosylceramide Beta-Glucosidase Deficiency Disease;Gaucher Disease, Non-Neuronopathic FormDrug: CerezymeGenzyme, a Sanofi CompanyNULLCompleted18 YearsN/AAll95Phase 4United States;Brazil;Canada;Italy;Poland;Spain;United Kingdom
32NCT00365131
(ClinicalTrials.gov)
December 199715/8/2006A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.Gaucher Disease Type I;Cerebroside Lipidosis Syndrome;Clucocerebrosidase Deficiency Disease;Glucosylceramide Beta-Glucosidase Deficiency Disease;Gaucher Disease, Non-Neuronopathic FormDrug: Cerezyme (imiglucerase for injection)Genzyme, a Sanofi CompanyNULLCompleted10 Years65 YearsBoth40Phase 4United States
33EUCTR2020-003120-17-SE
(EUCTR)
17/02/2021Venglustat in Combination with Cerezyme in Adult and Pediatric Patients with Gaucher Disease Type 3A 3-part study to evaluate the efficacy and safety of venglustat in combination with Cerezyme in adult and pediatric patients with Gaucher disease Type 3 (GD3) with open-label long-term treatment - LEAP2IT Gaucher disease type 3
MedDRA version: 20.0;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat malate
Product Name: Venglustat
Product Code: GZ402671
INN or Proposed INN: Venglustat malate
Other descriptive name: Genz-682452-AU
Genzyme CorporationNULLNot RecruitingFemale: yes
Male: yes
12Phase 2;Phase 3United States;Taiwan;Turkey;Egypt;France;Hungary;Canada;Argentina;Brazil;Germany;China;Japan;Sweden
34EUCTR2021-005402-10-FR
(EUCTR)
27/01/2022Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 (GD3) who have reached therapeutic goals with Enzyme Replacement Therapy (ERT) - LEAP2MONO Gaucher's disease type III
MedDRA version: 24.1;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: venglustat
Product Code: SAR402671, GZ402671 or GZ / SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Product Name: venglustat
Product Code: SAR402671, GZ402671 or GZ / SAR402671
INN or Proposed INN: Venglustat malate
Other descriptive name: GZ/SAR402671
Trade Name: Cerezyme 400 Units Powder for concentrate for solution for infusion
INN or Proposed INN: Imiglucerase
Sanofi-aventis recherche & développementNULLNAFemale: yes
Male: yes
50Phase 3United States;Taiwan;Turkey;United Kingdom;France;Egypt;Hungary;Canada;Argentina;Brazil;Germany;China;Japan