19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
| 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 | 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 | ||
| 2 | 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 | ||
| 3 | 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 | ||
| 4 | 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 | ||
| 5 | EUCTR2010-020199-45-GB (EUCTR) | 08/06/2011 | 06/05/2011 | 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 drug substance | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 162 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Portugal;United States;Qatar;Saudi Arabia;Taiwan;Spain;Colombia;Italy;United Kingdom;Switzerland;France;Canada;Argentina;Poland;Brazil;Denmark;Australia;Netherlands;Germany;Norway;Japan;Korea, Republic of |