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 | 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 |
| 2 | 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 |
| 3 | 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 multi-center, un-controlled, open-labeled trial of the long-term safety of Lamazym aftercare treatment of subjects with alpha-Mannosidosis | 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: Lamazym Product Code: rhLAMAN INN or Proposed INN: Velmanase alfa Other descriptive name: LAMAZYM | Chiesi Farmaceutici S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 13 | Phase 3 | France;Denmark | ||
| 4 | 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 | ||||
| 5 | NCT01908712 (ClinicalTrials.gov) | August 2013 | 16/7/2013 | Lamazym Aftercare Study FR Designed to Provide Treatment for French Patients | A Multi-center, Un-controlled, Open-labeled Trial of the Long-term Safety of Lamazym Aftercare Treatment of Subjects With Alpha-Mannosidosis Whom Previously Participated in Lamazym Trials | Alpha-Mannosidosis | Drug: Lamazym | Chiesi Farmaceutici S.p.A. | NULL | Enrolling by invitation | N/A | N/A | All | 10 | Phase 3 | France |
| 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 | 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 | ||
| 7 | EUCTR2013-000336-97-FR (EUCTR) | 26/06/2013 | 28/09/2015 | A trial of the long-term safety of Lamazym aftercare treatment of subjects with alpha-Mannosidosis who previously participated in Lamazym-trials | A multi-center, un-controlled, open-labeled trial of the long-term safety of Lamazym aftercare treatment of subjects with alpha-Mannosidosis who previously participated in Lamazym-trials | Treatment of Alpha-Mannosidosis;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Zymenex A/S | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 7 | Phase 3 | France;Denmark | |||
| 8 | NCT01908725 (ClinicalTrials.gov) | June 2013 | 12/6/2013 | Lamazym Aftercare Study | A Single-center, Un-controlled, Open-labeled Trial of the Long-term Safety of Lamazym Aftercare Treatment of Subjects With Alpha-Mannosidosis Whom Previously Participated in Lamazym Trials | Alpha-Mannosidosis | Drug: Lamazym | Chiesi Farmaceutici S.p.A. | NULL | Active, not recruiting | N/A | N/A | All | 5 | Phase 3 | Denmark |
| 9 | 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 | ||
| 10 | 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 | ||
| 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-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 | ||
| 12 | 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 | ||
| 13 | NCT01681953 (ClinicalTrials.gov) | August 2012 | 22/8/2012 | 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. | Alpha-Mannosidosis | Drug: Lamazym;Drug: Placebo | Zymenex A/S | European Commission | Completed | 5 Years | 35 Years | All | 25 | Phase 3 | Denmark;France;Germany;Poland;United Kingdom |
| 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-ES (EUCTR) | 19/04/2012 | 17/04/2012 | 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: alfa-manosidadas humana recombinante | Zymenex A/S | NULL | Not Recruiting | Female: yes Male: yes | 9 | Phase 2b | Belgium;Spain;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-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 | ||
| 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 2b | 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 | NCT01681940 (ClinicalTrials.gov) | January 2012 | 22/8/2012 | 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 | Alpha-Mannosidosis | Drug: Lamazym | Zymenex A/S | European Commission | Completed | 5 Years | 21 Years | All | 10 | Phase 2 | Belgium;Denmark;Spain;United Kingdom |
| 20 | 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 |
| 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 | 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 | |||
| 22 | 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 | |||
| 23 | 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 |