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 | NCT03204370 (ClinicalTrials.gov) | February 1, 2018 | 7/6/2017 | Natural History of Atypical Morquio A Disease | Natural History of Atypical Morquio A Disease: a 5-years Prospective Study in a Series of 9 Adult Patients Followed in a Single Expert Center | Mucopolysaccharidosis IV A | Drug: Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM] | GOIZET | BioMarin Pharmaceutical | Recruiting | 18 Years | N/A | All | 9 | France | |
| 2 | NCT02294877 (ClinicalTrials.gov) | September 2014 | 28/10/2014 | A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) | A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) | Mucopolysaccharidosis IV Type A;Morquio A Syndrome;MPS IVA | Drug: Vimizim® (elosulfase alfa) | BioMarin Pharmaceutical | ICON plc | Recruiting | N/A | N/A | All | 583 | United States;Australia;Austria;Belgium;Canada;Czechia;Denmark;France;Germany;Ireland;Italy;Malaysia;Netherlands;Poland;Portugal;Puerto Rico;Taiwan;United Kingdom | |
| 3 | EUCTR2010-020199-45-ES (EUCTR) | 23/07/2013 | 09/04/2013 | A Phase 3 Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)Extensión de un estudio multicéntrico, multinacional para evaluar la eficacia y seguridad a largo plazo de BMN 110 en pacientes con mucopolisacaridosis IVA (síndrome de Morquio A) | Mucopolysaccharidosis Type IVA MedDRA version: 16.0;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS) Product Code: BMN 110 INN or Proposed INN: Not Available Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 162 | Phase 3 | Portugal;United States;Qatar;Saudi Arabia;Taiwan;Spain;Colombia;Italy;United Kingdom;France;Canada;Argentina;Brazil;Denmark;Australia;Netherlands;Germany;Norway;Japan;Korea, Republic of | ||
| 4 | EUCTR2010-020199-45-DK (EUCTR) | 04/07/2012 | 24/05/2012 | A Phase 3 Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) | Mucopolysaccharidosis Type IVA MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS) Product Code: BMN 110 INN or Proposed INN: Not Available Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 162 | Phase 3 | Portugal;United States;Qatar;Saudi Arabia;Taiwan;Spain;Colombia;Italy;United Kingdom;France;Canada;Argentina;Brazil;Denmark;Australia;Netherlands;Germany;Norway;Japan;Korea, Republic of | ||
| 5 | EUCTR2011-005703-33-DE (EUCTR) | 19/06/2012 | 31/01/2012 | A Clinical Trial Study to Evaluate how Effective and Safe is the Drug BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation | A Phase 2, Open-label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation | Mucopolysaccharidosis Type IVA MedDRA version: 16.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS) Product Code: BMN 110 INN or Proposed INN: Not Available Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | United States;Germany;United Kingdom;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 | EUCTR2011-005682-20-DE (EUCTR) | 19/06/2012 | 31/01/2012 | A Pilot Study to Evaluate the Safety and Physiological Effects of Two Doses of BMN 110 in MPS IVA Patients | A Randomized, Double-Blind, Pilot Study of the Safety and Physiological Effects of Two Doses of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) | Mucopolysaccharidosis Type IVA MedDRA version: 16.0;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: BMN 110 Product Code: N/A INN or Proposed INN: Not Available Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 25 | United States;Canada;Germany;United Kingdom | |||
| 7 | EUCTR2011-005703-33-GB (EUCTR) | 29/05/2012 | 01/03/2012 | A Clinical Trial Study to Evaluate how Effective and Safe is the Drug BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation | A Phase 2, Open-label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation | Mucopolysaccharidosis Type IVA MedDRA version: 16.0;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS) Product Code: BMN 110 INN or Proposed INN: Not Available Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | United States;Germany;Italy;United Kingdom | ||
| 8 | EUCTR2011-005682-20-GB (EUCTR) | 19/03/2012 | 21/03/2012 | A Pilot Study to Evaluate the Safety and Physiological Effects of Two Doses of BMN 110 in MPS IVA Patients | A Randomized, Double-Blind, Pilot Study of the Safety and Physiological Effects of Two Doses of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) | Mucopolysaccharidosis Type IVA MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: BMN 110 Product Code: N/A INN or Proposed INN: Not Available Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 25 | United States;Canada;Germany;United Kingdom | |||
| 9 | EUCTR2011-003197-84-IT (EUCTR) | 13/03/2012 | 12/03/2012 | A Study to Evaluate the Safety and Efficacy of BMN 110 in MPS IVA Patients Less Than 5 Years of Age | A Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age with Mucopolysaccharidosis IVA (Morquio A Syndrome) | Mucopolysaccharidosis Type IVA MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: BMN 110 Product Code: NA Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance | BIOMARIN PHARMACEUTICAL INC. | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | United States;United Kingdom;Italy | ||
| 10 | EUCTR2010-020198-18-NO (EUCTR) | 03/01/2012 | 19/09/2011 | A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) | Mucopolysaccharidosis Type IVA MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: BMN 110 Product Code: N/A INN or Proposed INN: N/A Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance | BioMarin Pharmaceutical Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 162 | Phase 3 | Portugal;Germany;Colombia;Switzerland;Netherlands;France;Norway;Italy;Australia;Brazil;United Kingdom;Canada;Saudi Arabia;Denmark;Taiwan;Argentina;Korea, Republic of;Qatar;Japan;United States;Poland | ||
| 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 |
| 11 | EUCTR2010-020198-18-DK (EUCTR) | 01/12/2011 | 17/10/2011 | A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) | Mucopolysaccharidosis Type IVA MedDRA version: 14.0;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: BMN 110 Product Code: N/A INN or Proposed INN: N/A Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 162 | Phase 3 | Portugal;United States;Qatar;Saudi Arabia;Taiwan;Colombia;Italy;Switzerland;United Kingdom;France;Canada;Argentina;Poland;Brazil;Denmark;Australia;Netherlands;Germany;Norway;Japan;Korea, Republic of | ||
| 12 | EUCTR2010-020198-18-DE (EUCTR) | 12/08/2011 | 14/02/2011 | A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) | Mucopolysaccharidosis Type IVA MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: BMN 110 Product Code: N/A INN or Proposed INN: N/A Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance Product Name: BMN 110 Product Code: N/A INN or Proposed INN: N/A Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 162 | Phase 3 | United States;Portugal;Qatar;Taiwan;Saudi Arabia;Colombia;United Kingdom;Switzerland;Italy;France;Canada;Argentina;Poland;Brazil;Australia;Denmark;Germany;Netherlands;Norway;Japan;Korea, Republic of | ||
| 13 | EUCTR2010-020198-18-IT (EUCTR) | 19/07/2011 | 14/06/2011 | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) - ND | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) - ND | Mucopolysaccharidosis Type IVA MedDRA version: 13.1;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders | Product Name: BMN 110 Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance | BIOMARIN PHARMACEUTICAL INC. | NULL | Not Recruiting | Female: yes Male: yes | 162 | Phase 3 | Portugal;Germany;United Kingdom;Denmark;Norway;Italy | ||
| 14 | EUCTR2010-020198-18-PT (EUCTR) | 01/07/2011 | 19/04/2011 | A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) | Mucopolysaccharidosis Type IVA MedDRA version: 14.0;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: BMN 110 Product Code: N/A INN or Proposed INN: N/A Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 162 | Phase 3 | United Kingdom;France;Canada;Argentina;Poland;Brazil;Denmark;Australia;Netherlands;Germany;Norway;Japan;Korea, Republic of;Portugal;United States;Qatar;Saudi Arabia;Taiwan;Colombia;Italy;Switzerland | ||
| 15 | EUCTR2010-020198-18-GB (EUCTR) | 30/12/2010 | 15/11/2010 | A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) | Mucopolysaccharidosis Type IVA MedDRA version: 14.0;Level: PT;Classification code 10028095;Term: Mucopolysaccharidosis IV;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: BMN 110 Product Code: N/A INN or Proposed INN: N/A Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 162 | Phase 3 | United States;Portugal;Qatar;Taiwan;Saudi Arabia;Spain;Turkey;Colombia;Switzerland;United Kingdom;Italy;France;Canada;Argentina;Poland;Brazil;Australia;Denmark;Norway;Germany;Netherlands;Japan;Korea, Republic of | ||
| 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 |
| 16 | EUCTR2010-020198-18-FR (EUCTR) | 01/02/2011 | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) | Mucopolysaccharidosis Type IVA MedDRA version: 12.1;Level: LLT;Classification code 10028095;Term: Mucopolysaccharidosis IV MedDRA version: 12.1;Level: PT;Term: Mucopolysaccharidosis IV | Product Name: BMN 110 Product Code: N/A INN or Proposed INN: N/A Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance | BioMarin Pharmaceutical Inc | NULL | NA | Female: yes Male: yes | 162 | Phase 3 | Portugal;France;Denmark;Norway;Germany;Netherlands;Italy;United Kingdom | |||
| 17 | EUCTR2010-020198-18-NL (EUCTR) | 31/01/2011 | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/week and 2.0 mg/kg/every other week BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) | Mucopolysaccharidosis Type IVA MedDRA version: 12.1;Level: LLT;Classification code 10028095;Term: Mucopolysaccharidosis IV MedDRA version: 12.1;Level: PT;Term: Mucopolysaccharidosis IV | Product Name: BMN 110 Product Code: N/A INN or Proposed INN: N/A Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 162 | Phase 3 | Portugal;France;Denmark;Norway;Germany;Netherlands;Italy;United Kingdom |