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"
6 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT01303146
(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
2EUCTR2007-006345-40-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
3EUCTR2007-007165-20-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
4NCT00633139
(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
5NCT00418561
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2006-005341-11-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