19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 854 / 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191
Showing 1 to 10 of 20 diseases
| 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 Natural History of Atypical Morquio A Disease: a 5-years Prospective Study in a Series of 9 Adult Pa ... | Mucopolysaccharidosis IV A | Drug: Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM] | GOIZET | BioMarin Pharmaceutical | Active, not recruiting | 18 Years | N/A | All | 10 | 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 United States;Australia;Austria;Belgium;Canada;Czechia;Denmark;France;Germany;Ireland;Italy;Malaysia ... | |
| 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 Phase 3 Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with ... | 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) A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 1 ... | 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] Mucopolysaccharidosis Type IVA MedDRA version: 16.0;Level: PT;Classification code 10028095;Term: Muc ... | 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 Product Name: recombinant human N-acetylgalactosamine-6-sulfatase(rhGALNS) Product Code: BMN 110 INN o ... | 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 Portugal;United States;Qatar;Saudi Arabia;Taiwan;Spain;Colombia;Italy;United Kingdom;France;Canada;A ... | ||
| 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 Phase 3 Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with ... | A Multicenter, Multinational, 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 1 ... | 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] Mucopolysaccharidosis Type IVA MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Muc ... | 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 Product Name: recombinant human N-acetylgalactosamine-6-sulfatase(rhGALNS) Product Code: BMN 110 INN o ... | 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 Portugal;United States;Qatar;Saudi Arabia;Taiwan;Spain;Colombia;Italy;United Kingdom;France;Canada;A ... | ||
| 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 Clinical Trial Study to Evaluate how Effective and Safe is the Drug BMN 110 in Patients with Mucop ... | 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 A Phase 2, Open-label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patient ... | 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] Mucopolysaccharidosis Type IVA MedDRA version: 16.1;Level: PT;Classification code 10028095;Term: Muc ... | 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 Product Name: recombinant human N-acetylgalactosamine-6-sulfatase(rhGALNS) Product Code: BMN 110 INN o ... | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | United States;Germany;United Kingdom;Italy | ||
| 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 Pilot Study to Evaluate the Safety and Physiological Effects of Two Doses of BMN 110 in MPS IVA Pa ... | 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) A Randomized, Double-Blind, Pilot Study of the Safety and Physiological Effects of Two Doses of BMN ... | 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] Mucopolysaccharidosis Type IVA MedDRA version: 16.0;Level: PT;Classification code 10028095;Term: Muc ... | 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 Product Name: BMN 110 Product Code: N/A INN or Proposed INN: Not Available Other descriptive name: reco ... | 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 Clinical Trial Study to Evaluate how Effective and Safe is the Drug BMN 110 in Patients with Mucop ... | 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 A Phase 2, Open-label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patient ... | 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] Mucopolysaccharidosis Type IVA MedDRA version: 16.0;Level: PT;Classification code 10028095;Term: Muc ... | 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 Product Name: recombinant human N-acetylgalactosamine-6-sulfatase(rhGALNS) Product Code: BMN 110 INN o ... | 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 Pilot Study to Evaluate the Safety and Physiological Effects of Two Doses of BMN 110 in MPS IVA Pa ... | 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) A Randomized, Double-Blind, Pilot Study of the Safety and Physiological Effects of Two Doses of BMN ... | 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] Mucopolysaccharidosis Type IVA MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Muc ... | 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 Product Name: BMN 110 Product Code: N/A INN or Proposed INN: Not Available Other descriptive name: reco ... | 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) A Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 i ... | 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] Mucopolysaccharidosis Type IVA MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Muc ... | Product Name: BMN 110 Product Code: NA Other descriptive name: recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance Product Name: BMN 110 Product Code: NA Other descriptive name: recombinant human N-acetylgalactosamine ... | 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 Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysacchar ... | 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 th ... | 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] Mucopolysaccharidosis Type IVA MedDRA version: 14.1;Level: PT;Classification code 10028095;Term: Muc ... | 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 hu ... | 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 United States;Portugal;Qatar;Taiwan;Saudi Arabia;Colombia;United Kingdom;Switzerland;Italy;France;Ca ... |