19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
Showing 1 to 10 of 31 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 |
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1 | NCT04031066 (ClinicalTrials.gov) | January 11, 2021 | 17/7/2019 | Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis Interventional Study to Assess Efficacy and Safety of Velmanase Alfain Patients With Alpha Mannosido ... | A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel Group, Phase 3 Study to Evaluate the Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel Group, Phase 3 Study to Evalua ... | Alpha-Mannosidosis | Drug: Velmanase Alfa;Drug: Placebo | Chiesi Farmaceutici S.p.A. | NULL | Withdrawn | N/A | N/A | All | 0 | Phase 3 | NULL |
2 | EUCTR2016-001988-36-IT (EUCTR) | 12/01/2018 | 05/11/2020 | A study, conducted in several sites, with a duration of 2 years to evaluate the Safety and Efficacy of a new drug named velmanase alfa used in children with Alpha-Mannosidosis below 6 years of age A study, conducted in several sites, with a duration of 2 years to evaluate the Safety and Efficacy ... | A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated velmanase alfa (recombinant human alpha-mannosidase) Treatment in Pediatric Patients below 6 years of age with Alpha-Mannosidosis - A study, conducted in several sites, with a duration of 2 years to evaluate the Safety and Efficacy A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated ... | Alfa-mannosidosis MedDRA version: 20.0;Level: LLT;Classification code 10032658;Term: Other specified disorders of carbohydrate transport and metabolism;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Alfa-mannosidosis MedDRA version: 20.0;Level: LLT;Classification code 10032658;Term: Other specified ... | Product Name: Velmanase alfa Product Code: [rhLAMAN] INN or Proposed INN: VELMANASE ALFA Other descriptive name: recombinant human alpha-mannosidase Product Name: Velmanase alfa Product Code: [rhLAMAN] INN or Proposed INN: VELMANASE ALFA Other descript ... | CHIESI FARMACEUTICI S.P.A. | NULL | Not Recruiting | Female: yes Male: yes | 3 | Phase 2 | France;Denmark;Austria;Germany;Italy | ||
3 | EUCTR2016-001988-36-DE (EUCTR) | 09/08/2017 | 14/02/2017 | A study, conducted in several sites, with a duration of 2 years to evaluate the Safety and Efficacy of a new drug named velmanase alfa used in children with Alpha-Mannosidosis below 6 years of age A study, conducted in several sites, with a duration of 2 years to evaluate the Safety and Efficacy ... | A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated velmanase alfa (recombinant human alpha-mannosidase) Treatment in Pediatric Patients below 6 years of age with Alpha-Mannosidosis A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated ... | Alfa-mannosidosis MedDRA version: 20.0;Level: LLT;Classification code 10032658;Term: Other specified disorders of carbohydrate transport and metabolism;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Alfa-mannosidosis MedDRA version: 20.0;Level: LLT;Classification code 10032658;Term: Other specified ... | Product Name: Velmanase alfa INN or Proposed INN: VELMANASE ALFA Other descriptive name: recombinant human alpha-mannosidase Product Name: Velmanase alfa INN or Proposed INN: VELMANASE ALFA Other descriptive name: recombinant h ... | Chiesi Farmaceutici S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 3 | Phase 2 | Austria;Denmark;Germany | ||
4 | EUCTR2016-001988-36-AT (EUCTR) | 10/04/2017 | 22/02/2017 | A study, conducted in several sites, with a duration of 2 years to evaluate the Safety and Efficacy of a new drug named velmanase alfa used in children with Alpha-Mannosidosis below 6 years of age A study, conducted in several sites, with a duration of 2 years to evaluate the Safety and Efficacy ... | A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated velmanase alfa (recombinant human alpha-mannosidase) Treatment in Pediatric Patients below 6 years of age with Alpha-Mannosidosis A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated ... | Alfa-mannosidosis MedDRA version: 20.0;Level: LLT;Classification code 10032658;Term: Other specified disorders of carbohydrate transport and metabolism;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Alfa-mannosidosis MedDRA version: 20.0;Level: LLT;Classification code 10032658;Term: Other specified ... | Product Name: Velmanase alfa INN or Proposed INN: VELMANASE ALFA Other descriptive name: recombinant human alpha-mannosidase Product Name: Velmanase alfa INN or Proposed INN: VELMANASE ALFA Other descriptive name: recombinant h ... | Chiesi Farmaceutici S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 3 | Phase 2 | Denmark;Austria;Germany | ||
5 | EUCTR2016-001988-36-FR (EUCTR) | 27/02/2017 | 18/09/2020 | A study, conducted in several sites, with a duration of 2 years to evaluate the Safety and Efficacy of a new drug named velmanase alfa used in children with Alpha-Mannosidosis below 6 years of age A study, conducted in several sites, with a duration of 2 years to evaluate the Safety and Efficacy ... | A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated velmanase alfa (recombinant human alpha-mannosidase) Treatment in Pediatric Patients below 6 years of age with Alpha-Mannosidosis A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated ... | Alfa-mannosidosis MedDRA version: 20.0;Level: LLT;Classification code 10032658;Term: Other specified disorders of carbohydrate transport and metabolism;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Alfa-mannosidosis MedDRA version: 20.0;Level: LLT;Classification code 10032658;Term: Other specified ... | Product Name: Velmanase alfa INN or Proposed INN: VELMANASE ALFA Other descriptive name: recombinant human alpha-mannosidase Product Name: Velmanase alfa INN or Proposed INN: VELMANASE ALFA Other descriptive name: recombinant h ... | Chiesi Farmaceutici S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 3 | Phase 2 | France;Austria;Denmark;Germany;Italy | ||
6 | NCT02998879 (ClinicalTrials.gov) | December 2016 | 25/11/2016 | Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis Trial on Safety and Efficacy of Velmanase AlfaTreatment in Pediatric Patients With Alpha-Mannosidosi ... | A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated Velmanase Alfa (Recombinant Human Alpha-mannosidase) Treatment in Pediatric Patients Below 6 Years of Age With Alpha-Mannosidosis A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated ... | Alpha-Mannosidosis | Drug: Velmanase Alfa (e.g. Lamazym) | Chiesi Farmaceutici S.p.A. | Cromsource | Completed | N/A | 6 Years | All | 5 | Phase 2 | Austria;Denmark;France;Germany;Italy |
7 | EUCTR2016-001988-36-DK (EUCTR) | 05/10/2016 | 11/07/2016 | A study, conducted in several sites, with a duration of 2 years to evaluate the Safety and Efficacy of a new drug named velmanase alfa used in children with Alpha-Mannosidosis below 6 years of age A study, conducted in several sites, with a duration of 2 years to evaluate the Safety and Efficacy ... | A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated velmanase alfa (recombinant human alpha-mannosidase) Treatment in Pediatric Patients below 6 years of age with Alpha-Mannosidosis A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated ... | Alfa-mannosidosis MedDRA version: 20.0;Level: LLT;Classification code 10032658;Term: Other specified disorders of carbohydrate transport and metabolism;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Alfa-mannosidosis MedDRA version: 20.0;Level: LLT;Classification code 10032658;Term: Other specified ... | Product Name: Velmanase alfa INN or Proposed INN: VELMANASE ALFA Other descriptive name: recombinant human alpha-mannosidase Product Name: Velmanase alfa INN or Proposed INN: VELMANASE ALFA Other descriptive name: recombinant h ... | Chiesi Farmaceutici S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 3 | Phase 2 | Austria;Denmark;Germany;Italy | ||
8 | NCT02478840 (ClinicalTrials.gov) | February 2015 | 9/2/2015 | Evaluation of Long-term Efficacy of Treatment With Lamazym | A Single Center, Open Label Clinical Trial Investigating the Long-term Efficacy of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) Treatment in Subjects With Alpha-Mannosidosis Who Previously Participated in Lamazym Trials A Single Center, Open Label Clinical Trial Investigating the Long-term Efficacy of rhLAMAN(Recombina ... | Alpha-Mannosidosis | Drug: Lamazym | Zymenex A/S | NULL | Completed | N/A | N/A | All | 18 | Phase 3 | Denmark |
9 | EUCTR2013-000336-97-DK (EUCTR) | 30/01/2015 | 03/12/2014 | A trial of the long-term safety of Lamazym aftercare treatment of subjects with alpha-Mannosidosis who previously participated in Lamazym-trials A trial of the long-term safety of Lamazymaftercare treatment of subjects with alpha-Mannosidosis wh ... | A multi-center, un-controlled, open-labeled trial of the long-term safety of Lamazym aftercare treatment of subjects with alpha-Mannosidosis A multi-center, un-controlled, open-labeled trial of the long-term safety of Lamazymaftercare treatm ... | Alpha-Mannosidosis MedDRA version: 20.0;Level: LLT;Classification code 10032658;Term: Other specified disorders of carbohydrate transport and metabolism;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Alpha-Mannosidosis MedDRA version: 20.0;Level: LLT;Classification code 10032658;Term: Other specifie ... | Product Name: Lamazym Product Code: rhLAMAN INN or Proposed INN: Velmanase alfa Other descriptive name: LAMAZYM Product Name: Lamazym Product Code: rhLAMAN INN or Proposed INN: Velmanase alfa Other descriptive name: ... | Chiesi Farmaceutici S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 13 | Phase 3 | France;Denmark | ||
10 | EUCTR2014-003950-15-DK (EUCTR) | 22/01/2015 | 25/11/2014 | Medical research trial for testing drug treatment of alpha-Mannosidosis | A single center, open label clinical trial investigating the long-term efficacy of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) treatment in subjects with alpha-Mannosidosis who previously participated in Lamazym trials A single center, open label clinical trial investigating the long-term efficacy of rhLAMAN(recombina ... | alpha-Mannosidosis MedDRA version: 17.1;Level: LLT;Classification code 10032658;Term: Other specified disorders of carbohydrate transport and metabolism;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] alpha-Mannosidosis MedDRA version: 17.1;Level: LLT;Classification code 10032658;Term: Other specifie ... | Product Name: Lamazym Product Code: rhLAMAN INN or Proposed INN: recombinant human lysosomal alpha-mannosidase Other descriptive name: LAMAZYM Product Name: Lamazym Product Code: rhLAMAN INN or Proposed INN: recombinant human lysosomal alpha-man ... | Zymenex A/S | NULL | Not Recruiting | Female: yes Male: yes | Denmark |