19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 854 / 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191
| 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. | 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] | 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 | ||
| 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. | 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 | 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 | ||
| 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. | 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] | 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 | France;United States;Mexico;Canada;Brazil;Belgium;Spain;Israel;Netherlands;Germany;Japan;United Kingdom | ||
| 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. | 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] | 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 Kingdom;Japan;Germany;Netherlands;Israel;Spain;Belgium;Brazil;Canada;Mexico;United States;France | ||
| 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. | 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] | Product Code: SHP611 INN or Proposed INN: N/A 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;Japan;Germany;Netherlands;Brazil;Belgium;Argentina;Canada;France;United Kingdom;Italy;Israel | ||
| 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. | 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] | 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 | ||
| 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. | A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy - NA | 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] | Product Code: SHP611 INN or Proposed INN: Not Available 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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Spain;Israel;United Kingdom;France;Mexico;Canada;Belgium;Brazil;Denmark;Germany;Netherlands;Japan | ||
| 8 | NCT03771898 (ClinicalTrials.gov) | April 30, 2019 | 10/12/2018 | A Study of Intrathecal SHP611 in Participants With Late Infantile Metachromatic Leukodystrophy | A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects With Late Infantile Metachromatic Leukodystrophy | Metachromatic Leukodystrophy (MLD) | Drug: SHP611 | Shire | NULL | Active, not recruiting | 6 Months | 72 Months | All | 42 | Phase 2 | United States;Belgium;Brazil;Canada;France;Germany;Israel;Japan;Netherlands;Spain;United Kingdom;Denmark;Mexico |
| 9 | 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. | 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] | 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 | ||
| 10 | 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 | ||
| 11 | 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 | ||
| 12 | 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 | ||
| 13 | 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 | ||
| 14 | 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 |