19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
| 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 | EUCTR2011-005682-20-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 | |||
| 4 | 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 | ||
| 5 | 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 | ||
| 6 | 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 | |||
| 7 | 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 | ||
| 8 | 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 | 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;Norway;Germany;Netherlands;Japan;Korea, Republic of | ||
| 9 | 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 | ||
| 10 | EUCTR2011-003197-84-GB (EUCTR) | 22/11/2011 | 13/09/2011 | 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: 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 | 15 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Italy;United Kingdom | ||
| 11 | 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 | ||
| 12 | 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 | ||
| 13 | 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 | ||
| 14 | 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 | ||
| 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 | ||
| 16 | EUCTR2010-021048-16-GB (EUCTR) | 07/10/2010 | 17/06/2010 | A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | A Multicenter, Multinational, Open-Label, 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: 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: 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 | 20 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | 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 | |||
| 18 | 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 |