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 | 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 | 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 |
| 5 | 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 | ||
| 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 | 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 |
| 7 | 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 | ||||
| 8 | 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 | ||
| 9 | 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 | 9 | Phase 3 | Poland;Denmark | ||
| 10 | 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 | ||
| 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 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | Phase 3 | Spain;Denmark;Germany | ||
| 12 | 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 | ||
| 13 | 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 | ||
| 14 | 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 2b | Spain;Belgium;Denmark;Germany;United Kingdom | ||
| 15 | 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 2b | Spain;Belgium;Denmark;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 |
| 16 | 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 2b | Spain;Belgium;Denmark;Germany;United Kingdom | ||
| 17 | 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 | ||
| 18 | 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 |
| 19 | 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 | |||
| 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 | |||
| 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 | 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 |