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-ganglio ... show all
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 | NCT01303146 (ClinicalTrials.gov) | October 2008 | 23/2/2011 | Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation | and Safety of METAZYM (Recombinant Human Arylsulfatase A or rhASA) for the Treatment of Patients With Late Infantile MLD Who Had Previously Hematopoietic Stem Cell Transplantation | Metachromatic Leukodystrophy | Drug: rhARSA | Assistance Publique - Hôpitaux de Paris | European Leukodystrophy Association;Zymenex A/S;Shire | Completed | 6 Months | N/A | Both | 1 | Phase 2 | France |
| 2 | EUCTR2007-006345-40-DK (EUCTR) | 26/08/2008 | 14/01/2008 | A single center, open-label, non-randomized, uncontrolled, multiple-dose study of the efficacy and long-term safety of Metazym (recombinant human arylsulfatase A or rhASA) for the treatment of patients with late infantile metachromatic leukodystrophy | A single center, open-label, non-randomized, uncontrolled, multiple-dose study of the efficacy and long-term safety of Metazym (recombinant human arylsulfatase A or rhASA) for the treatment of patients with late infantile metachromatic leukodystrophy | Metachromatic Leukodystrophy (MLD) in late infantile patients MedDRA version: 9.1;Level: LLT;Classification code 10024381;Term: Leukodystrophy | Product Name: Metazym Product Code: rhASA Other descriptive name: recombinant human arylsulfatase A | Shire Pharmaceutical Ireland Limited | NULL | Not Recruiting | Female: yes Male: yes | Denmark | ||||
| 3 | EUCTR2007-007165-20-DK (EUCTR) | 26/02/2008 | 14/01/2008 | A single center, open-label, non-randomized, uncontrolled, multiple-dose study of the efficacy and safety of Metazym (recombinant human arylsulfatase A or rhASA) for the treatment of MLD patients with high residual level of voluntary function | A single center, open-label, non-randomized, uncontrolled, multiple-dose study of the efficacy and safety of Metazym (recombinant human arylsulfatase A or rhASA) for the treatment of MLD patients with high residual level of voluntary function | Metachromatic Leukodystrophy (MLD) in late infantile patients MedDRA version: 9.1;Level: LLT;Classification code 10024381;Term: Leukodystrophy | Product Name: Metazym Product Code: rhASA Other descriptive name: recombinant human arylsulfatase A | Shire Pharmaceuticals Ireland Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Phase 2 | Denmark | |||
| 4 | NCT00633139 (ClinicalTrials.gov) | August 2007 | 29/2/2008 | Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD) | A Single Center, Open-Label, Non-Randomized, Uncontrolled, Multiple-Dose, Dose Escalation Study of the Safety, Pharmacokinetics, Efficacy and Long Term Safety of HGT-1111 (Recombinant Human Arylsulfatase A [rhASA, Metazym]) for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD) | Late Infantile Metachromatic Leukodystrophy | Biological: Recombinant human Arylsulfatase A (rhASA) | Shire | NULL | Completed | 1 Year | 5 Years | All | 13 | Phase 1;Phase 2 | Denmark |
| 5 | NCT00418561 (ClinicalTrials.gov) | January 22, 2007 | 4/1/2007 | Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD) | A Single Center, Open-label, Non-randomized, Uncontrolled, Multiple-dose, Dose Escalation Study of the Safety, Pharmacokinetics and Efficacy of Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD) | Metachromatic Leukodystrophy (MLD) | Drug: rhASA | Shire | NULL | Completed | 1 Year | 5 Years | All | 13 | Phase 1 | 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 |
| 6 | EUCTR2006-005341-11-DK (EUCTR) | 28/12/2006 | 08/11/2006 | A single center, open-label, non-randomized, uncontrolled, multiple-dose, dose escalation study of the safety, pharmacokinetics and efficacy of Metazym (recombinant human arylsulfatase A or rhASA) for the treatment of patients with late infantile metachromatic leukodystrophy (MLD) | A single center, open-label, non-randomized, uncontrolled, multiple-dose, dose escalation study of the safety, pharmacokinetics and efficacy of Metazym (recombinant human arylsulfatase A or rhASA) for the treatment of patients with late infantile metachromatic leukodystrophy (MLD) | Late infantile metachromatic leukodystrophy (MLD) MedDRA version: 9.1;Level: LLT;Classification code 10024381;Term: Leukodystrophy | Product Name: Metazym Product Code: rhASA INN or Proposed INN: Arylsulfatase A Other descriptive name: Metazym | Shire Pharmaceuticals Ireland Limited | NULL | Not Recruiting | Female: yes Male: yes | 12 | Denmark |