19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

  
8 trials found
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1EUCTR2018-003291-12-IT
(EUCTR)
16/10/202021/10/2020Research 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, INCNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
42Phase 2United States;France;Canada;Spain;Brazil;Belgium;Israel;Netherlands;Germany;United Kingdom;Japan;Italy
2EUCTR2012-003775-20-IT
(EUCTR)
20/03/201715/03/2017A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injectionAn 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 IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 1;Phase 2France;Czech Republic;Brazil;Denmark;Australia;Germany;Japan;United Kingdom;Italy
3EUCTR2012-003775-20-GB
(EUCTR)
25/04/201606/06/2016A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injectionAn 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 IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 1;Phase 2France;Czech Republic;Brazil;Denmark;Australia;Germany;Japan;Italy;United Kingdom
4EUCTR2012-003775-20-CZ
(EUCTR)
06/06/201414/02/2014A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injectionAn 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 IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 1;Phase 2France;Czech Republic;Brazil;Denmark;Australia;Germany;United Kingdom;Japan;Italy
5EUCTR2012-003775-20-DE
(EUCTR)
11/09/201314/12/2012A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injectionAn 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 IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 1;Phase 2France;Czechia;Czech Republic;Brazil;Denmark;Australia;Germany;United Kingdom;Italy;Japan
6EUCTR2012-003775-20-DK
(EUCTR)
30/04/201321/12/2012A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injectionAn 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 IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 1;Phase 2France;Czechia;Czech Republic;Brazil;Australia;Denmark;Germany;United Kingdom;Italy;Japan
7NCT00633139
(ClinicalTrials.gov)
August 200729/2/2008Long-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 LeukodystrophyBiological: Recombinant human Arylsulfatase A (rhASA)ShireNULLCompleted1 Year5 YearsAll13Phase 1/Phase 2Denmark
8EUCTR2012-003775-20-FR
(EUCTR)
27/06/2013A safety and efficacy extension of study HGT-MLD-070 in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injectionAn 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 IncNULLNAFemale: yes
Male: yes
18Phase 1;Phase 2France;Czech Republic;Argentina;Brazil;Denmark;Australia;Germany;United Kingdom