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.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04656600 (ClinicalTrials.gov) | December 31, 2020 | 4/12/2020 | Phase IV Study 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 Disease | Drug: Cerezyme® / Imiglucerase | Sanofi | NULL | Not yet recruiting | 2 Years | N/A | All | 12 | Phase 4 | NULL |
| 2 | NCT01161914 (ClinicalTrials.gov) | January 2011 | 11/7/2010 | The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher Disease | A 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 Disease | Gaucher Disease | Drug: Cerezyme®;Drug: ISU302 | ISU Abxis Co., Ltd. | NULL | Withdrawn | 2 Years | 75 Years | Both | 0 | Phase 3 | NULL |
| 3 | EUCTR2008-005825-12-GB (EUCTR) | 17/12/2009 | 28/04/2009 | 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 - NA | 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 | Gaucher Disease MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | Protalix Biotherapeutics | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | Spain;Germany;Italy;United Kingdom | |||
| 4 | EUCTR2008-005825-12-DE (EUCTR) | 10/12/2009 | 16/09/2009 | 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 - NA | 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 | 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 Biotherapeutics | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | Spain;Germany;Italy;United Kingdom | ||
| 5 | EUCTR2008-005825-12-ES (EUCTR) | 04/06/2009 | 06/03/2009 | Ensayo 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 | Ensayo 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 Biotherapeutics | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 3 | Germany;United Kingdom;Spain;Italy | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT00364858 (ClinicalTrials.gov) | December 2001 | 15/8/2006 | Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease | A 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 Disease | Gaucher Disease, Type 1;Cerebroside Lipidosis Syndrome;Glucocerebrosidase Deficiency Disease;Glucosylceramide Beta-Glucosidase Deficiency Disease;Gaucher Disease, Non-Neuronopathic Form | Drug: Cerezyme | Genzyme, a Sanofi Company | NULL | Completed | 18 Years | N/A | All | 95 | Phase 4 | United States;Brazil;Canada;Italy;Poland;Spain;United Kingdom |