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 | 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 | NCT01966029 (ClinicalTrials.gov) | July 2013 | 10/9/2013 | BMN 110 Phase 3B in Australian Patients | A Multicenter Open-Label, Phase 3B Study to Evaluate the Efficacy and Safety of BMN 110 in Australian Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | Mucopolysaccharidosis IVA (Morquio A Syndrome) | Drug: BMN 110 | BioMarin Pharmaceutical | NULL | Completed | N/A | N/A | All | 13 | Phase 3 | Australia |
| 3 | NCT01697319 (ClinicalTrials.gov) | August 2012 | 17/9/2012 | Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients 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 IVA;Morquio A Syndrome;MPS IVA | Drug: BMN 110 | BioMarin Pharmaceutical | NULL | Terminated | 5 Years | N/A | All | 16 | Phase 2 | United States;Germany;United Kingdom |
| 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 | EUCTR2011-005682-20-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 | |||
| 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 | EUCTR2011-005703-33-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 | ||
| 7 | EUCTR2010-020199-45-DE (EUCTR) | 14/06/2012 | 12/09/2011 | A phase 3 extension study to evaluate the long term efficacy and safety of BMN 110 in patients with Mucopolysaccharidosis IV A (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) - NA | 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 Nacetylgalactosamine- 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;Turkey;Colombia;Italy;Switzerland;United Kingdom;France;Canada;Argentina;Brazil;Poland;Denmark;Australia;Netherlands;Germany;Norway;Japan;Korea, Republic of | ||
| 8 | 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 | ||
| 9 | NCT01609062 (ClinicalTrials.gov) | April 2012 | 24/5/2012 | Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome | 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 IVA;Morquio A Syndrome;MPS IVA | Drug: BMN 110 | BioMarin Pharmaceutical | NULL | Terminated | 7 Years | N/A | All | 25 | Phase 2 | United States;Canada;Germany;United Kingdom |
| 10 | 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 | |||
| 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 | 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 | ||
| 12 | EUCTR2010-020199-45-PT (EUCTR) | 02/03/2012 | 29/12/2011 | A phase 3 extension study to evaluate the long term efficacy and safety of BMN 110 in patients with Mucopolysaccharidosis IV A (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) - NA | 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 Nacetylgalactosamine- 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 | Portugal;United States;Qatar;Saudi Arabia;Taiwan;Spain;Turkey;Colombia;Italy;Switzerland;United Kingdom;France;Canada;Argentina;Brazil;Poland;Denmark;Australia;Netherlands;Germany;Norway;Japan;Korea, Republic of | |||
| 13 | 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 | ||
| 14 | EUCTR2010-020199-45-IT (EUCTR) | 20/12/2011 | 27/12/2011 | A phase 3 extension study to evaluate the long term efficacy and safetyof BMN 110 in patients with Mucopolysaccharidosis IV A (Morquio Asyndrome) | A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with MucopolysaccharidosisIVA (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: BM110 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;Korea, Democratic People's Republic of;Turkey;Colombia;United Kingdom;Italy;Switzerland;Canada;Argentina;Brazil;Denmark;Australia;Germany;Japan | ||
| 15 | 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 | ||
| 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 | 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] | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | United States;Italy;United Kingdom | |||
| 17 | NCT01515956 (ClinicalTrials.gov) | October 2011 | 22/12/2011 | Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) | 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 IVA;Morquio A Syndrome;MPS IVA | Drug: BMN 110 | BioMarin Pharmaceutical | NULL | Completed | N/A | 5 Years | All | 15 | Phase 2 | United States;Italy;Taiwan;United Kingdom |
| 18 | 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 | ||
| 19 | 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 | ||
| 20 | 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 | ||
| 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 |
| 21 | NCT01415427 (ClinicalTrials.gov) | July 2011 | 8/8/2011 | Long-Term Efficacy and Safety Extension Study 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 IV A;Morquio A Syndrome;MPS IVA | Drug: BMN 110 - Weekly;Drug: BMN 110 - Every Other Week | BioMarin Pharmaceutical | NULL | Completed | 5 Years | N/A | All | 173 | Phase 3 | United States;Argentina;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom |
| 22 | 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] | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 162 | 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 | ||||
| 23 | NCT01275066 (ClinicalTrials.gov) | February 2011 | 10/1/2011 | A Double-Blind Study to Evaluate the Efficacy and Safety of 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) | MPS IV A | Drug: BMN 110 Weekly;Drug: Placebo;Drug: BMN 110 Every Other Week | BioMarin Pharmaceutical | NULL | Completed | 5 Years | N/A | All | 177 | Phase 3 | United States;Argentina;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Portugal;Qatar;Saudi Arabia;Taiwan;United Kingdom;Australia;Norway;Poland;Switzerland;Turkey |
| 24 | 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 | ||
| 25 | NCT01242111 (ClinicalTrials.gov) | November 2010 | 28/10/2010 | 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) | MPS IV A;Mucopolysaccharidosis IVA;Morquio A Syndrome | Drug: BMN 110 | BioMarin Pharmaceutical | NULL | Terminated | N/A | N/A | All | 20 | Phase 1;Phase 2 | United Kingdom |
| 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 |
| 26 | 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] | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 20 | United Kingdom | ||||
| 27 | NCT00884949 (ClinicalTrials.gov) | April 2009 | 10/4/2009 | A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA | A Phase 1/2, Multicenter, Open-label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome) | MPS IV A | Drug: BMN 110 | BioMarin Pharmaceutical | NULL | Completed | 5 Years | 18 Years | All | 20 | Phase 1;Phase 2 | United Kingdom |
| 28 | EUCTR2008-007365-23-GB (EUCTR) | 12/03/2009 | 14/04/2009 | A Phase 1/2, Multicenter, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Subjects with Mucopolysaccharidosis IVA (Morquio Syndrome) | A Phase 1/2, Multicenter, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Subjects with Mucopolysaccharidosis IVA (Morquio Syndrome) | Mucopolysaccharidosis Type IV A MedDRA version: 9.1;Level: LLT;Classification code 10028095;Term: Mucopolysaccharidosis IV | Product Name: BMN 110 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 | Phase 1;Phase 2 | United Kingdom | |||
| 29 | NCT01858103 (ClinicalTrials.gov) | January 2009 | 15/5/2013 | BMN 110 US Expanded Access Program | A Multicenter, Open-label BMN 110 US Expanded Access Program (BMN 110 US EAP) to Provide BMN 110 to Patients Diagnosed With MPS IVA | Mucopolysaccharidosis IVA;Morquio A Syndrome;MPS IVA | Drug: BMN 110 | BioMarin Pharmaceutical | NULL | Approved for marketing | N/A | N/A | Both | N/A | United States;Puerto Rico | |
| 30 | 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 | |||
| 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 |
| 31 | 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 |