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 | EUCTR2012-003775-20-IT (EUCTR) | 20/03/2017 | 15/03/2017 | A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injection | An Open-label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients with Metachromatic Leukodystrophy | Treatment of Metachromatic Leukodystrophy MedDRA version: 19.1;Level: PT;Classification code 10067609;Term: Metachromatic leukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Code: SHP611 INN or Proposed INN: Not available Other descriptive name: Recombinant Human Arylsulfatase A (rhASA) | Shire Human Genetics Therapies Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 1;Phase 2 | France;Czech Republic;Brazil;Denmark;Australia;Germany;Japan;United Kingdom;Italy | ||
| 2 | EUCTR2012-003775-20-GB (EUCTR) | 25/04/2016 | 06/06/2016 | A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injection | An Open-label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients with Metachromatic Leukodystrophy | Treatment of Metachromatic Leukodystrophy MedDRA version: 20.0;Level: PT;Classification code 10067609;Term: Metachromatic leukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Code: SHP611 INN or Proposed INN: Not available Other descriptive name: Recombinant Human Arylsulfatase A (rhASA) | Shire Human Genetics Therapies Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 1;Phase 2 | France;Czech Republic;Brazil;Denmark;Australia;Germany;Japan;Italy;United Kingdom | ||
| 3 | EUCTR2012-003775-20-CZ (EUCTR) | 06/06/2014 | 14/02/2014 | A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injection | An Open-label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients with Metachromatic Leukodystrophy | Treatment of Metachromatic Leukodystrophy MedDRA version: 20.0;Level: PT;Classification code 10067609;Term: Metachromatic leukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: SHP611 INN or Proposed INN: Not available Other descriptive name: Recombinant Human Arylsulfatase A (rhASA) | Shire Human Genetics Therapies Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 1;Phase 2 | France;Czech Republic;Brazil;Denmark;Australia;Germany;United Kingdom;Japan;Italy | ||
| 4 | EUCTR2012-003775-20-DE (EUCTR) | 11/09/2013 | 14/12/2012 | A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injection | An Open-label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients with Metachromatic Leukodystrophy | Treatment of Metachromatic Leukodystrophy MedDRA version: 20.0;Level: PT;Classification code 10067609;Term: Metachromatic leukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Code: SHP611 INN or Proposed INN: Not available Other descriptive name: Recombinant Human Arylsulfatase A (rhASA) | Shire Human Genetics Therapies Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 1;Phase 2 | France;Czech Republic;Brazil;Denmark;Australia;Germany;Japan;Italy;United Kingdom | ||
| 5 | EUCTR2012-003775-20-DK (EUCTR) | 30/04/2013 | 21/12/2012 | A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injection | An Open-label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients with Metachromatic Leukodystrophy | Treatment of Metachromatic Leukodystrophy MedDRA version: 20.0;Level: PT;Classification code 10067609;Term: Metachromatic leukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Code: SHP611 INN or Proposed INN: Not available Other descriptive name: Recombinant Human Arylsulfatase A (rhASA) | Shire Human Genetics Therapies Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 1;Phase 2 | France;Czechia;Czech Republic;Brazil;Australia;Denmark;Germany;United Kingdom;Japan;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 | EUCTR2011-002044-28-DE (EUCTR) | 14/06/2012 | 28/06/2011 | A safety and efficacy study in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injection | A Phase I/II, Multicenter, Open-label, Dose Escalation Study of HGT-1110 Administered Intrathecally in Children with Metachromatic Leukodystrophy | Treatment of Metachromatic Leukodystrophy MedDRA version: 19.0;Level: PT;Classification code 10067609;Term: Metachromatic leukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Code: HGT-1110 INN or Proposed INN: N/A Other descriptive name: Recombinant Human Arylsulfatase A (rhASA, | Shire Human Genetics Therapies Inc | NULL | Not Recruiting | Female: yes Male: yes | 24 | Phase 1;Phase 2 | France;Brazil;Denmark;Australia;Germany;Japan | ||
| 7 | EUCTR2011-002044-28-DK (EUCTR) | 25/11/2011 | 16/08/2011 | A safety and efficacy study in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injection | A Phase I/II, Multicenter, Open-label, Dose Escalation Study of HGT-1110 Administered Intrathecally in Children with Metachromatic Leukodystrophy | Treatment of Metachromatic Leukodystrophy MedDRA version: 19.0;Level: PT;Classification code 10067609;Term: Metachromatic leukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Code: HGT-1110 INN or Proposed INN: N/A Other descriptive name: Recombinant Human Arylsulfatase A (rhASA, | Shire Human Genetics Therapies Inc | NULL | Not Recruiting | Female: yes Male: yes | 24 | Phase 1;Phase 2 | France;Brazil;Australia;Denmark;Germany;Japan | ||
| 8 | EUCTR2008-000084-41-BE (EUCTR) | 03/03/2010 | 15/12/2009 | A multi-center open label extension study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) treatment in patients with Late Infantile Metachromatic Leukodystrophy (MLD) | A multi-center open label extension study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) treatment in patients with Late Infantile Metachromatic Leukodystrophy (MLD) | Metachromatic Leukodystrophy (MLD) in late infantile patients MedDRA version: 9.1;Level: LLT;Classification code 10024381;Term: Leukodystrophy | Product Name: HGT-1111 Product Code: rhASA Other descriptive name: recombinant human arylsulfatase A | Shire Human Genetic Therapies Inc | NULL | Not Recruiting | Female: yes Male: yes | 18 | Denmark;Belgium;France;Italy | |||
| 9 | EUCTR2008-000084-41-FR (EUCTR) | 01/12/2009 | 05/08/2009 | A multi-center open label extension study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) treatment in patients with Late Infantile Metachromatic Leukodystrophy (MLD) | A multi-center open label extension study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) treatment in patients with Late Infantile Metachromatic Leukodystrophy (MLD) | Metachromatic Leukodystrophy (MLD) in late infantile patients MedDRA version: 9.1;Level: LLT;Classification code 10024381;Term: Leukodystrophy | Product Name: HGT-1111 Product Code: rhASA Other descriptive name: recombinant human arylsulfatase A | Shire Human Genetic Therapies Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 18 | Belgium;Denmark;France;Italy | |||
| 10 | EUCTR2008-000084-41-IT (EUCTR) | 25/08/2009 | 11/08/2009 | A Multi-center, Open-Label Extension Study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) Treatment in Patients with Late Infantile Metachromatic Leukodystrophy (MLD) - ND | A Multi-center, Open-Label Extension Study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) Treatment in Patients with Late Infantile Metachromatic Leukodystrophy (MLD) - ND | metachromatic leukodystrophy (MLD) in late infantile patients MedDRA version: 9.1;Level: PT;Classification code 10024381 | Product Name: HGT-1111 | Shire Human Genetic Therapies Inc | NULL | Not Recruiting | Female: yes Male: yes | 18 | Belgium;Denmark;France;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 |
| 11 | 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 |
| 12 | 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 | ||||
| 13 | EUCTR2008-000084-41-DK (EUCTR) | 15/05/2008 | 15/01/2008 | A multi-center open label extension study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) treatment in patients with Late Infantile Metachromatic Leukodystrophy (MLD) | A multi-center open label extension study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) treatment in patients with Late Infantile Metachromatic Leukodystrophy (MLD) | Metachromatic Leukodystrophy (MLD) in late infantile patients MedDRA version: 9.1;Level: LLT;Classification code 10024381;Term: Leukodystrophy | Product Name: HGT-1111 Product Code: rhASA INN or Proposed INN: Not available Other descriptive name: recombinant human arylsulfatase A | Shire Human Genetic Therapies Inc | NULL | Not Recruiting | Female: yes Male: yes | France;Belgium;Denmark;Italy | ||||
| 14 | 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 | |||
| 15 | NCT00681811 (ClinicalTrials.gov) | February 2008 | 19/5/2008 | Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD | A Multi-center, Open-Label Extension Study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) Treatment in Patients With Late Infantile Metachromatic Leukodystrophy (MLD) | Late Infantile Metachromatic Leukodystrophy | Drug: HGT-1111 | Shire | NULL | Terminated | 3 Years | 6 Years | All | 11 | 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 |
| 16 | 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 |
| 17 | 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 |
| 18 | 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 | |||
| 19 | EUCTR2018-003291-12-IT (EUCTR) | 21/10/2020 | Research study to determine the effects of an investigational drug, SHP611 on patients with with Late Infantile Metachromatic Leukodystrophy (MLD) specially the gross motor function, using the Gross Motor FunctionClassification in Metachromatic Leukodystrophy (GMFC-MLD) compared with matched historical control data in children with MLD. | A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy - ---- | Late Metachromatic Leukodystrophy (MLD) MedDRA version: 20.0;Level: PT;Classification code 10067609;Term: Metachromatic leukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: -- Product Name: --- Product Code: [SHP611] INN or Proposed INN: Recombinant Human Arylsulfatase A (rhASA) Other descriptive name: RECOMBINANT HUMAN ARYLSULFATASE A | SHIRE HUMAN GENETIC THERAPIES, INC | NULL | NA | Female: yes Male: yes | 42 | Phase 2 | United States;France;Canada;Spain;Brazil;Belgium;Israel;Netherlands;Germany;United Kingdom;Japan;Italy | |||
| 20 | EUCTR2012-003775-20-FR (EUCTR) | 27/06/2013 | A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injection | An Open-label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients with Metachromatic Leukodystrophy | Treatment of Metachromatic Leukodystrophy MedDRA version: 16.0;Level: PT;Classification code 10067609;Term: Metachromatic leukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Code: HGT-1110 INN or Proposed INN: Not available Other descriptive name: Recombinant Human Arylsulfatase A (rhASA) | Shire Human Genetics Therapies Inc | NULL | NA | Female: yes Male: yes | 18 | Phase 1;Phase 2 | France;Czech Republic;Argentina;Brazil;Denmark;Australia;Germany;United Kingdom |