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
| 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 | 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 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 | 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] | Product Name: Velmanase alfa Product Code: [rhLAMAN] INN or Proposed INN: VELMANASE ALFA Other descriptive name: recombinant human alpha-mannosidase | CHIESI FARMACEUTICI S.P.A. | NULL | Not Recruiting | Female: yes Male: yes | 3 | Phase 2 | France;Denmark;Austria;Germany;Italy | ||
| 2 | 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 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 | 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] | Product Name: Velmanase alfa INN or Proposed INN: VELMANASE ALFA Other descriptive name: recombinant human alpha-mannosidase | Chiesi Farmaceutici S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 3 | Phase 2 | Austria;Denmark;Germany | ||
| 3 | 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 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 | 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] | Product Name: Velmanase alfa INN or Proposed INN: VELMANASE ALFA Other descriptive name: recombinant human alpha-mannosidase | Chiesi Farmaceutici S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 3 | Phase 2 | Denmark;Austria;Germany | ||
| 4 | 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 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 | 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] | Product Name: Velmanase alfa INN or Proposed INN: VELMANASE ALFA Other descriptive name: recombinant human alpha-mannosidase | Chiesi Farmaceutici S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 3 | Phase 2 | France;Austria;Denmark;Germany;Italy | ||
| 5 | NCT02998879 (ClinicalTrials.gov) | December 2016 | 25/11/2016 | Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis | 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 | 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 |
| 6 | 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 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 | 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] | Product Name: Velmanase alfa INN or Proposed INN: VELMANASE ALFA Other descriptive name: recombinant human alpha-mannosidase | Chiesi Farmaceutici S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 3 | Phase 2 | Austria;Denmark;Germany;Italy | ||
| 7 | 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 | Alpha-Mannosidosis | Drug: Lamazym | Zymenex A/S | NULL | Completed | N/A | N/A | All | 18 | Phase 3 | Denmark |
| 8 | 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 | 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] | Product Name: Lamazym Product Code: rhLAMAN INN or Proposed INN: recombinant human lysosomal alpha-mannosidase Other descriptive name: LAMAZYM | Zymenex A/S | NULL | Not Recruiting | Female: yes Male: yes | Denmark | ||||
| 9 | EUCTR2012-000979-17-SE (EUCTR) | 30/07/2013 | 07/06/2013 | A Placebo-Controlled phase 3 trial of repeated Lamazym treatment of subjects with Alpha-Mannosidosis | A Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Repeated Lamazym Treatment of Subjects with alpha-Mannosidosis - Phase III | Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Lamazym Product Code: rhLAMAN INN or Proposed INN: Lamazym Other descriptive name: recombinant human alpha-mannosidase | Zymenex A/S | NULL | Not Recruiting | Female: yes Male: yes | 25 | Phase 3 | Spain;Belgium;Denmark;Germany;Sweden | ||
| 10 | EUCTR2013-000321-31-DK (EUCTR) | 07/06/2013 | 07/06/2013 | A long-term safety trial of repeated Velmanase Alfa treatment of subjects with Alpha-Mannosidosis | A multi-center, un-controlled, open-labeled trial of the long-term safety of Velmanase Alfa aftercaretreatment of subjects with alpha-Mannosidosis whom previously participated in Velmanase Alfa - trials - Phase IIIb | Treatement of 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] | Product Name: Velmanase Alfa Product Code: rhLAMAN INN or Proposed INN: velmanase alfa Other descriptive name: recombinant human alpha-mannosidase | Chiesi Farmaceutici S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 8 | Phase 3 | Poland;Denmark | ||
| 11 | EUCTR2012-000979-17-BE (EUCTR) | 26/03/2013 | 07/01/2013 | A Placebo-Controlled phase 3 trial of repeated Lamazym treatment of subjects with Alpha-Mannosidosis | A Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Repeated Lamazym Treatment of Subjects with alpha-Mannosidosis - Phase III | Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Lamazym Product Code: rhLAMAN INN or Proposed INN: recombinant human alpha-mannosidase Other descriptive name: recombinant human alpha-mannosidase | Zymenex A/S | NULL | Not Recruiting | Female: yes Male: yes | 25 | Phase 3 | Spain;Belgium;Denmark;Germany;Sweden | ||
| 12 | EUCTR2012-000979-17-ES (EUCTR) | 27/02/2013 | 09/01/2013 | A Placebo-Controlled phase 3 trial of repeated Lamazym treatment of subjects with Alpha-Mannosidosis | A Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Repeated Lamazym Treatment of Subjects with alpha-Mannosidosis - Phase III | Treatment of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Lamazym Product Code: rhLAMAN INN or Proposed INN: Lamazym Other descriptive name: recombinant human alpha-mannosidase | Zymenex A/S | NULL | Not Recruiting | Female: yes Male: yes | 25 | Phase 3 | Spain;Denmark;Germany;Sweden;Belgium | ||
| 13 | EUCTR2012-000979-17-DE (EUCTR) | 17/12/2012 | 09/07/2012 | A Placebo-Controlled phase 3 trial of repeated Lamazym treatment of subjects with Alpha-Mannosidosis | A Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Repeated Lamazym Treatment of Subjects with alpha-Mannosidosis - Phase III | Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Lamazym Product Code: rhLAMAN INN or Proposed INN: rhLAMAN Other descriptive name: recombinant human alpha-mannosidase | Zymenex A/S | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 3 | Spain;Belgium;Denmark;Germany;Sweden | ||
| 14 | EUCTR2012-000979-17-DK (EUCTR) | 06/08/2012 | 06/08/2012 | A Placebo-Controlled phase 3 trial of repeated Lamazym treatment of subjects with Alpha-Mannosidosis | A Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Repeated Lamazym Treatment of Subjects with alpha-Mannosidosis - Phase III | Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Lamazym Product Code: rhLAMAN Other descriptive name: recombinant human alpha-mannosidase | Zymenex A/S | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 3 | Spain;Belgium;Denmark;Germany;Sweden | ||
| 15 | EUCTR2011-004355-40-DE (EUCTR) | 23/07/2012 | 18/01/2012 | A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis. | A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis. - Phase 2b | Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Lamazym Product Code: rhLAMAN Other descriptive name: recombinant human alpha-mannosidase | Zymenex A/S | NULL | Not Recruiting | Female: yes Male: yes | 9 | Phase 2 | Spain;Belgium;Denmark;Germany;United Kingdom | ||
| 16 | EUCTR2011-004355-40-GB (EUCTR) | 28/03/2012 | 14/02/2012 | A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis. | A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis. - Phase 2b | Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Lamazym Product Code: rhLAMAN Other descriptive name: recombinant human alpha-mannosidase | Zymenex A/S | NULL | Not Recruiting | Female: yes Male: yes | 9 | Phase 2 | Spain;Belgium;Denmark;Germany;United Kingdom | ||
| 17 | EUCTR2011-004355-40-BE (EUCTR) | 30/01/2012 | 19/01/2012 | A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis. | A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis. - Phase 2b | Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Lamazym Product Code: rhLAMAN Other descriptive name: recombinant human alpha-mannosidase | Zymenex A/S | NULL | Not Recruiting | Female: yes Male: yes | 9 | Phase 2 | Spain;Belgium;Denmark;Germany;United Kingdom | ||
| 18 | EUCTR2011-004355-40-DK (EUCTR) | 17/01/2012 | 18/01/2012 | A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis. | A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis. - Phase 2b | Treatement of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Lamazym Product Code: rhLAMAN Other descriptive name: recombinant human alpha-mannosidase | Zymenex A/S | NULL | Not Recruiting | Female: yes Male: yes | 9 | Phase 2 | Spain;Belgium;Denmark;Germany;United Kingdom | ||
| 19 | NCT01285700 (ClinicalTrials.gov) | January 2011 | 25/1/2011 | Dose Finding Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis | A Single Center, Randomized, Open-label, Multiple-dose Study of the Efficacy and Long-term Safety of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) for the Treatment of Patients With Alpha-mannosidosis. | Alpha Mannosidosis | Drug: Lamazym | Zymenex A/S | European Commission | Active, not recruiting | 5 Years | 21 Years | Both | 10 | Phase 2 | Denmark |
| 20 | EUCTR2010-022085-26-DK (EUCTR) | 05/10/2010 | 23/08/2010 | A single center, randomized, open-label, multiple-dose study of the efficacy and long-term safety of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) for the treatment of patients with alpha-mannosidosis | A single center, randomized, open-label, multiple-dose study of the efficacy and long-term safety of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) for the treatment of patients with alpha-mannosidosis | Treatment of alpha-Mannosidosis | Product Name: Lamazym Product Code: rhLAMAN INN or Proposed INN: recombinant human alpha-mannosidase | Zymenex A/S | NULL | Not Recruiting | Female: yes Male: yes | 10 | Denmark | |||
| 21 | EUCTR2010-022084-36-DK (EUCTR) | 05/10/2010 | 20/08/2010 | A single center, open-label, dose escalation study of the safety and pharmacokinetics of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) for the treatment of patients with alpha-mannosidosis. | A single center, open-label, dose escalation study of the safety and pharmacokinetics of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) for the treatment of patients with alpha-mannosidosis. | Treatment of alpha-Mannosidosis | Product Name: Lamazym Product Code: rhLAMAN INN or Proposed INN: recombinant human alpha-mannosidase | Zymenex A/S | NULL | Not Recruiting | Female: yes Male: yes | 10 | Denmark | |||
| 22 | NCT01268358 (ClinicalTrials.gov) | October 2010 | 29/12/2010 | Safety Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis | A Single Center, Open-label, Dose Escalation Study of the Safety and Pharmacokinetics of rhLAMAN (Recombinant Human Alpha-mannosidase or Lamazym) for the Treatment of Patients With Alpha-mannosidosis. | Alpha Mannosidosis | Drug: Lamazym | Zymenex A/S | European Commission | Completed | 5 Years | 20 Years | All | 10 | Phase 1 | NULL |
| 23 | EUCTR2013-000321-31-PL (EUCTR) | 21/02/2018 | A long-term safety trial of repeated Velmanase Alfa treatment of subjects with Alpha-Mannosidosis | A multi-center, un-controlled, open-labeled trial of the long-term safety of Velmanase Alfa aftercare treatment of subjects with alpha-Mannosidosis whom previously participated in Velmanase Alfa - trials - Phase IIIb | Treatement of 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] | Product Name: Velmanase Alfa Product Code: rhLAMAN INN or Proposed INN: velmanase alfa Other descriptive name: recombinant human alpha-mannosidase | Chiesi Farmaceutici S.p.A. | NULL | NA | Female: yes Male: yes | 5 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Poland;Denmark |