19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
Showing 1 to 10 of 27 diseases
| 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 | EUCTR2018-003291-12-GR (EUCTR) | 27/07/2021 | 06/07/2021 | 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 Function Classification in Metachromatic Leukodystrophy (GMFC-MLD) compared with matched historical control data in children with MLD. Research study to determine the effects of an investigational drug, SHP611 on patients with with Lat ... | A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subject ... | 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] Late Metachromatic Leukodystrophy (MLD) MedDRA version: 20.0;Level: PT;Classification code 10067609; ... | Product Code: SHP611 Other descriptive name: RECOMBINANT HUMAN ARYLSULFATASE A | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 2 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Brazil;Belgium;Germany;Netherlands;Japan United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Brazil;Belgium;German ... | ||
| 2 | EUCTR2018-003291-12-IT (EUCTR) | 16/10/2020 | 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. Research study to determine the effects of an investigational drug, SHP611 on patients with with Lat ... | A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy - ---- A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subject ... | 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] Late Metachromatic Leukodystrophy (MLD) MedDRA version: 20.0;Level: PT;Classification code 10067609; ... | Trade Name: -- Product Name: --- Product Code: [SHP611] INN or Proposed INN: Recombinant Human Arylsulfatase A (rhASA) Other descriptive name: RECOMBINANT HUMAN ARYLSULFATASE A Trade Name: -- Product Name: --- Product Code: [SHP611] INN or Proposed INN: Recombinant Human Arylsulf ... | SHIRE HUMAN GENETIC THERAPIES, INC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 2 | United States;France;Canada;Spain;Brazil;Belgium;Israel;Netherlands;Germany;United Kingdom;Japan;Italy United States;France;Canada;Spain;Brazil;Belgium;Israel;Netherlands;Germany;United Kingdom;Japan;Ita ... | ||
| 3 | EUCTR2018-003291-12-DE (EUCTR) | 08/01/2020 | 06/06/2019 | 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 Function Classification in Metachromatic Leukodystrophy (GMFC-MLD) compared with matched historical control data in children with MLD. Research study to determine the effects of an investigational drug, SHP611 on patients with with Lat ... | A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subject ... | 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] Late Metachromatic Leukodystrophy (MLD) MedDRA version: 20.0;Level: PT;Classification code 10067609; ... | Product Code: SHP611 Other descriptive name: RECOMBINANT HUMAN ARYLSULFATASE A | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ( ... | France;United States;Mexico;Canada;Brazil;Belgium;Spain;Israel;Netherlands;Germany;Japan;United Kingdom France;United States;Mexico;Canada;Brazil;Belgium;Spain;Israel;Netherlands;Germany;Japan;United King ... | ||
| 4 | EUCTR2018-003291-12-GB (EUCTR) | 12/11/2019 | 18/06/2019 | 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 Function Classification in Metachromatic Leukodystrophy (GMFC-MLD) compared with matched historical control data in children with MLD. Research study to determine the effects of an investigational drug, SHP611 on patients with with Lat ... | A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subject ... | 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] Late Metachromatic Leukodystrophy (MLD) MedDRA version: 20.0;Level: PT;Classification code 10067609; ... | Product Code: SHP611 Other descriptive name: RECOMBINANT HUMAN ARYLSULFATASE A | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 2 | France;United States;Mexico;Canada;Brazil;Belgium;Spain;Israel;Netherlands;Germany;Japan;United Kingdom France;United States;Mexico;Canada;Brazil;Belgium;Spain;Israel;Netherlands;Germany;Japan;United King ... | ||
| 5 | EUCTR2018-003291-12-NL (EUCTR) | 24/10/2019 | 10/04/2019 | 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 Function Classification in Metachromatic Leukodystrophy (GMFC-MLD) compared with matched historical control data in children with MLD. Research study to determine the effects of an investigational drug, SHP611 on patients with with Lat ... | A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subject ... | 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] Late Metachromatic Leukodystrophy (MLD) MedDRA version: 20.0;Level: PT;Classification code 10067609; ... | Product Code: SHP611 INN or Proposed INN: N/A Other descriptive name: RECOMBINANT HUMAN ARYLSULFATASE A Product Code: SHP611 INN or Proposed INN: N/A Other descriptive name: RECOMBINANT HUMAN ARYLSULFATASE ... | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 2 | United States;Japan;United Kingdom;Spain;Greece;Canada;Netherlands;Belgium;Brazil;Denmark;Mexico;Italy;Israel;France;Germany United States;Japan;United Kingdom;Spain;Greece;Canada;Netherlands;Belgium;Brazil;Denmark;Mexico;Ita ... | ||
| 6 | EUCTR2018-003291-12-BE (EUCTR) | 20/05/2019 | 18/04/2019 | 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 Function Classification in Metachromatic Leukodystrophy (GMFC-MLD) compared with matched historical control data in children with MLD. Research study to determine the effects of an investigational drug, SHP611 on patients with with Lat ... | A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subject ... | 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] Late Metachromatic Leukodystrophy (MLD) MedDRA version: 20.0;Level: PT;Classification code 10067609; ... | Product Code: SHP611 Other descriptive name: RECOMBINANT HUMAN ARYLSULFATASE A | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 42 | Phase 2 | United States;France;Mexico;Canada;Spain;Brazil;Belgium;Israel;Netherlands;Germany;United Kingdom;Japan United States;France;Mexico;Canada;Spain;Brazil;Belgium;Israel;Netherlands;Germany;United Kingdom;Ja ... | ||
| 7 | EUCTR2018-003291-12-ES (EUCTR) | 16/05/2019 | 12/04/2019 | 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 Function Classification in Metachromatic Leukodystrophy (GMFC-MLD) compared with matched historical control data in children with MLD. Research study to determine the effects of an investigational drug, SHP611 on patients with with Lat ... | A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy - NA A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subject ... | 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] Late Metachromatic Leukodystrophy (MLD) MedDRA version: 20.0;Level: PT;Classification code 10067609; ... | Product Code: SHP611 INN or Proposed INN: Not Available Other descriptive name: RECOMBINANT HUMAN ARYLSULFATASE A Product Code: SHP611 INN or Proposed INN: Not Available Other descriptive name: RECOMBINANT HUMAN ARYL ... | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 35 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ( ... | United States;Spain;Israel;United Kingdom;France;Mexico;Canada;Belgium;Brazil;Denmark;Germany;Netherlands;Japan United States;Spain;Israel;United Kingdom;France;Mexico;Canada;Belgium;Brazil;Denmark;Germany;Nether ... | ||
| 8 | EUCTR2018-003291-12-FR (EUCTR) | 11/04/2019 | 20/02/2019 | 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 Function Classification in Metachromatic Leukodystrophy (GMFC-MLD) compared with matched historical control data in children with MLD. Research study to determine the effects of an investigational drug, SHP611 on patients with with Lat ... | A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subject ... | 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] Late Metachromatic Leukodystrophy (MLD) MedDRA version: 20.0;Level: PT;Classification code 10067609; ... | Product Code: SHP611 Other descriptive name: RECOMBINANT HUMAN ARYLSULFATASE A | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 35 | Phase 2 | United States;Spain;Israel;United Kingdom;France;Mexico;Canada;Belgium;Brazil;Denmark;Germany;Netherlands;Japan United States;Spain;Israel;United Kingdom;France;Mexico;Canada;Belgium;Brazil;Denmark;Germany;Nether ... | ||
| 9 | 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 A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy r ... | 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 An Open-label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal ... | 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] Treatment of Metachromatic Leukodystrophy MedDRA version: 19.1;Level: PT;Classification code 1006760 ... | Product Code: SHP611 INN or Proposed INN: Not available Other descriptive name: Recombinant Human Arylsulfatase A (rhASA) Product Code: SHP611 INN or Proposed INN: Not available Other descriptive name: Recombinant Human Aryl ... | 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 | ||
| 10 | 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 A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy r ... | 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 An Open-label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal ... | 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] Treatment of Metachromatic Leukodystrophy MedDRA version: 20.0;Level: PT;Classification code 1006760 ... | Product Code: SHP611 INN or Proposed INN: Not available Other descriptive name: Recombinant Human Arylsulfatase A (rhASA) Product Code: SHP611 INN or Proposed INN: Not available Other descriptive name: Recombinant Human Aryl ... | 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 |