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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
20 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2012-003775-20-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
2EUCTR2012-003775-20-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
3EUCTR2012-003775-20-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
4EUCTR2012-003775-20-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;Czech Republic;Brazil;Denmark;Australia;Germany;Japan;Italy;United Kingdom
5EUCTR2012-003775-20-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;Japan;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2011-002044-28-DE
(EUCTR)
14/06/201228/06/2011A safety and efficacy study in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injectionA 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 IncNULLNot RecruitingFemale: yes
Male: yes
24Phase 1;Phase 2France;Brazil;Denmark;Australia;Germany;Japan
7EUCTR2011-002044-28-DK
(EUCTR)
25/11/201116/08/2011A safety and efficacy study in Children with Metachromatic Leukodystrophy recieving enzyme (HGT-1110) replacement by intrathecal injectionA 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 IncNULLNot RecruitingFemale: yes
Male: yes
24Phase 1;Phase 2France;Brazil;Australia;Denmark;Germany;Japan
8EUCTR2008-000084-41-BE
(EUCTR)
03/03/201015/12/2009A 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 IncNULLNot RecruitingFemale: yes
Male: yes
18Denmark;Belgium;France;Italy
9EUCTR2008-000084-41-FR
(EUCTR)
01/12/200905/08/2009A 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 IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
18Belgium;Denmark;France;Italy
10EUCTR2008-000084-41-IT
(EUCTR)
25/08/200911/08/2009A Multi-center, Open-Label Extension Study of HGT-1111 (Recombinant Human Arylsulfatase A or rhASA) Treatment in Patients with Late Infantile Metachromatic Leukodystrophy (MLD) - NDA 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-1111Shire Human Genetic Therapies IncNULLNot RecruitingFemale: yes
Male: yes
18Belgium;Denmark;France;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT01303146
(ClinicalTrials.gov)
October 200823/2/2011Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantationand Safety of METAZYM (Recombinant Human Arylsulfatase A or rhASA) for the Treatment of Patients With Late Infantile MLD Who Had Previously Hematopoietic Stem Cell TransplantationMetachromatic LeukodystrophyDrug: rhARSAAssistance Publique - Hôpitaux de ParisEuropean Leukodystrophy Association;Zymenex A/S;ShireCompleted6 MonthsN/ABoth1Phase 2France
12EUCTR2007-006345-40-DK
(EUCTR)
26/08/200814/01/2008A 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 leukodystrophyA 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
Denmark
13EUCTR2008-000084-41-DK
(EUCTR)
15/05/200815/01/2008A 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 IncNULLNot RecruitingFemale: yes
Male: yes
France;Belgium;Denmark;Italy
14EUCTR2007-007165-20-DK
(EUCTR)
26/02/200814/01/2008A 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 functionA 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 LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 2Denmark
15NCT00681811
(ClinicalTrials.gov)
February 200819/5/2008Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLDA 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 LeukodystrophyDrug: HGT-1111ShireNULLTerminated3 Years6 YearsAll11Phase 2Denmark
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT00633139
(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
17NCT00418561
(ClinicalTrials.gov)
January 22, 20074/1/2007Metazym 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: rhASAShireNULLCompleted1 Year5 YearsAll13Phase 1Denmark
18EUCTR2006-005341-11-DK
(EUCTR)
28/12/200608/11/2006A 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 LimitedNULLNot RecruitingFemale: yes
Male: yes
12Denmark
19EUCTR2018-003291-12-IT
(EUCTR)
21/10/2020Research 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, INCNULLNAFemale: yes
Male: yes
42Phase 2United States;France;Canada;Spain;Brazil;Belgium;Israel;Netherlands;Germany;United Kingdom;Japan;Italy
20EUCTR2012-003775-20-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