19. Lysosomal storage disease
784 clinical trials,   673 drugs   (DrugBank: 101 drugs),   68 drug target genes,   184 drug target pathways

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"
20 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT01747980
(ClinicalTrials.gov)
March 20135/12/2012Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease PatientsAn Exploratory, Open-label Study to Evaluate the Safety of PRX-112 and Pharmacokinetics of Oral prGCD (Plant Recombinant Human Glucocerebrosidase) in Gaucher PatientsGaucher DiseaseDrug: PRX-112ProtalixNULLCompleted18 YearsN/ABoth16Phase 1Israel
2EUCTR2011-003685-32-ES
(EUCTR)
27/01/201213/10/2011NAA Multicenter Extension Study of Taliglucerase alfa in Adult Subjects with Gaucher Disease - NA Gaucher disease
MedDRA version: 14.0;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: recombinant human glucerebrosidase
Product Code: Taliglucerase Alfa
INN or Proposed INN: Taliglucerase alfa
Other descriptive name: prGCD
Protalix BiotherapeuticsNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25Serbia;Canada;Mexico;Chile;Spain;Italy;South Africa;Israel
3EUCTR2011-003676-37-ES
(EUCTR)
15/11/201114/09/2011NAA Multicenter Extension Study of Taliglucerase alfa in Pediatric Subjects with Gaucher Disease - NA Gaucher disease
MedDRA version: 14.0;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: recombinant human glucerebrosidase
Product Code: Taliglucerase Alfa
INN or Proposed INN: Taliglucerase alfa
Other descriptive name: prGCD
Protalix BiotherapeuticsNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
15Spain;South Africa;Israel
4EUCTR2008-005825-12-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
Protalix BiotherapeuticsNULLNot Recruiting Female: yes
Male: yes
30Phase 3Spain;Germany;Italy;United Kingdom
5EUCTR2008-005825-12-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2008-005825-12-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
7EUCTR2008-005826-35-GB
(EUCTR)
24/06/200914/04/2009A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher DiseaseA Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Gaucher Disease
MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease
Protalix BiotherapeuticsNULLNot Recruiting Female: yes
Male: yes
30Phase 3Spain;Italy;United Kingdom
8EUCTR2008-005826-35-IT
(EUCTR)
19/06/200930/06/2009A multicenter, double-blind, extension trial of two parallel dose groups of Plant cell expressed recombinant human glucocerebrosidase (prGCD) in patients with Gaucher disiease. - PB-06-003A multicenter, double-blind, extension trial of two parallel dose groups of Plant cell expressed recombinant human glucocerebrosidase (prGCD) in patients with Gaucher disiease. - PB-06-003 Gaucher DiseaseProduct Name: Plant Cell Recombinant Glucocerebrosidase
Product Code: prGCD
Product Name: Plant Cell Recombinant Glucocerebrosidase
Product Code: prGCD
PROTALIX BIOTHERAPEUTICSNULLNot RecruitingFemale: yes
Male: yes
30United Kingdom;Spain;Italy
9EUCTR2008-005826-35-ES
(EUCTR)
04/06/200907/04/2009Ensayo clínico de extensión, multicéntrico, doble ciego con dos grupos paralelos de dosis de glucocerebrosidasa humana recombinante expresada en células vegetales (prGCD) en pacientes con enfermedad de Gaucher A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher DiseaseEnsayo clínico de extensión, multicéntrico, doble ciego con dos grupos paralelos de dosis de glucocerebrosidasa humana recombinante expresada en células vegetales (prGCD) en pacientes con enfermedad de Gaucher A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease 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
Product Name: recombinant human glucocerebrosidase
Product Code: prGCD
Other descriptive name: recombinant human glucocerebrosidase
Protalix BiotherapeuticsNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30United Kingdom;Spain;Italy
10EUCTR2008-005825-12-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2007-000498-42-IT
(EUCTR)
13/11/200819/03/2009A phase III Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two parallel Dose Groups of Plant CElle Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease - NDA phase III Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two parallel Dose Groups of Plant CElle Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease - ND Gaucher Disease
MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease
Product Name: Plant Cell Recombinant Glucocerebrosidase
Product Code: prGCD
INN or Proposed INN: GLUCOCEREBROSIDASE
Product Name: Plant Cell Recombinant Glucocerebrosidase
Product Code: prGCD
INN or Proposed INN: GLUCOCEREBROSIDASE
PROTALIX BIOTHERAPEUTICSNULLNot RecruitingFemale: yes
Male: yes
30Phase 3United Kingdom;Spain;Italy
12EUCTR2007-000498-42-ES
(EUCTR)
08/10/200810/07/2008Ensayo clinico de fase III, multicéntrico, aleatorizado, doble ciego; para evaluar la seguridad y eficacia de dos grupos paralelos de dosis de glucocerebrosidasa humana recombinante expresada en células vegetales (prGCD) en pacientes con enfermedad de Gaucher.A phase III Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease. - NAEnsayo clinico de fase III, multicéntrico, aleatorizado, doble ciego; para evaluar la seguridad y eficacia de dos grupos paralelos de dosis de glucocerebrosidasa humana recombinante expresada en células vegetales (prGCD) en pacientes con enfermedad de Gaucher.A phase III Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease. - NA Enfermedad de Gaucher.Gaucher 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
Product Name: recombinant human glucocerebrosidase
Product Code: prGCD
Other descriptive name: recombinant human glucocerebrosidase
Protalix BiotherapeuticsNULLNot RecruitingFemale: yes
Male: yes
30Phase 3Spain;Italy;United Kingdom
13NCT00705939
(ClinicalTrials.gov)
June 200825/6/2008Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension TrialA Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher DiseaseGaucher DiseaseDrug: Taliglucerase alfaPfizerNULLCompleted18 YearsN/AAll45Phase 3United States;Australia;Canada;Chile;Israel;South Africa;Spain;United Kingdom
14EUCTR2007-000498-42-GB
(EUCTR)
08/05/200807/01/2008A Phase III Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease - NAA Phase III Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease - NA Gaucher disease
MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease
Protalix BiotherapeuticsNULLNot Recruiting Female: yes
Male: yes
30Phase 3Spain;Italy;United Kingdom
15NCT00376168
(ClinicalTrials.gov)
August 200712/9/2006A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher DiseaseA Phase III, Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher DiseaseGaucher DiseaseDrug: Plant cell expressed recombinant glucocerebrosidase (prGCD)PfizerNULLCompleted18 YearsN/AAll32Phase 3United States;Canada;Chile;Israel;Italy;South Africa;Spain;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT00962260
(ClinicalTrials.gov)
July 200618/8/2009Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher DiseaseAn Open-label Expanded Access Trial of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease Who Require Enzyme Replacement TherapyGaucher DiseaseDrug: Plant cell expressed recombinant glucocerebrosidase (prGCD)PfizerNULLNo longer available18 YearsN/AAllUnited States;Israel
17NCT00258778
(ClinicalTrials.gov)
November 200523/11/2005Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)A Phase I, Non-Randomized, Open Label, Single Dose-Escalation Safety Study of Recombinant Human Glucocerebrosidase (prGCD) in Healthy VolunteersGaucher DiseaseDrug: Human Glucocerebrosidase (prGCD)ProtalixNULLCompleted18 Years45 YearsBoth6Phase 1Israel
18EUCTR2007-000498-42-Outside-EU/EEA
(EUCTR)
10/04/2012A double-blind, randomised efficacy and safety study of taliglucerase alfa enzyme replacement therapy in children and adolescents with Gaucher disease (non-neuronopathic and chronic neuronopathic)A double-blind, randomised efficacy and safety study of taliglucerase alfa enzyme replacement therapy in children and adolescents with Gaucher disease (non-neuronopathic and chronic neuronopathic) Gaucher disease
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: Taliglucerase alfa - Recombinant human glucocerebrosidase
Product Code: prGCD
Other descriptive name: TALIGLUCERASE ALFA
Protalix BiotherapeuticsNULLNAFemale: yes
Male: yes
10Israel;United States
19EUCTR2008-005825-12-Outside-EU/EEA
(EUCTR)
10/04/2012A multi-centre, open-label, switchover trial to assess safety and efficacy of taliglucerase alfa in adult and paediatric patients with Gaucher disease who are currently being treated with imiglucerase enzyme replacement therapy (ERT).A multi-centre, open-label, switchover trial to assess safety and efficacy of taliglucerase alfa in adult and paediatric patients with Gaucher disease who are currently being treated with imiglucerase enzyme replacement therapy (ERT). Gaucher disease
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: Taliglucerase alfa - Recombinant human glucocerebrosidase
Product Code: prGCD
Other descriptive name: TALIGLUCERASE ALFA
Protalix BiotherapeuticsNULLNAFemale: yes
Male: yes
30Canada;Israel;United Kingdom;United States
20EUCTR2008-005826-35-Outside-EU/EEA
(EUCTR)
10/04/2012A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher DiseaseA Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease Gaucher disease
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: Taliglucerase alfa - Recombinant human glucocerebrosidase
Product Code: prGCD
Other descriptive name: TALIGLUCERASE ALFA
Protalix BiotherapeuticsNULLNAFemale: yes
Male: yes
30Australia;Canada;Chile;Israel;South Africa;Spain;United Kingdom;United States