19. ライソゾーム病
[臨床試験数:784,薬物数:673(DrugBank:101),標的遺伝子数:68,標的パスウェイ数:184

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"
16 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04532047
(ClinicalTrials.gov)
December 1, 202019/8/2020In Utero Enzyme Replacement Therapy for Lysosomal Storage DiseasesIn Utero Enzyme Replacement Therapy (ERT) for Prenatally Diagnosed Lysosomal Storage Disorders (LSDs).MPS I;MPS II;MPS IVA;MPS VI;Mps VII;Gaucher Disease, Type 2;Gaucher Disease, Type 3;Pompe Disease Infantile-Onset;Wolman DiseaseDrug: Aldurazyme (laronidase)University of California, San FranciscoDuke UniversityNot yet recruiting18 Years50 YearsFemale10Phase 1United States
2NCT01572636
(ClinicalTrials.gov)
March 28, 20124/4/2012Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler SyndromeMT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH).Mucopolysaccharidosis Type IH;MPS I;Hurler SyndromeDrug: LaronidaseMasonic Cancer Center, University of MinnesotaNULLTerminatedN/AN/AAll20United States
3NCT00418821
(ClinicalTrials.gov)
October 20103/1/2007A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed InfantsA Multicenter, Multinational, Open-Label Study of the Effects of Aldurazyme (Laronidase) Treatment on Lactation in Women With Mucopolysaccharidosis I (MPS I) and Their Breastfed InfantsMucopolysaccharidosis I;Hurler's Syndrome;Hurler-Scheie Syndrome;ScheieBiological: Aldurazyme (laronidase)Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCRecruitingN/AN/AFemale10Phase 4Italy;United States
4EUCTR2007-007003-33-IT
(EUCTR)
16/09/201027/12/2010A Multicenter, Multinational, Open-Label Study of the Effects of Aldurazyme(laronidase) Treatment on Lactation in Women with Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants - NDA Multicenter, Multinational, Open-Label Study of the Effects of Aldurazyme(laronidase) Treatment on Lactation in Women with Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants - ND mucocopolysaccharidoses I
MedDRA version: 9.1;Level: LLT;Classification code 10056886
Trade Name: ALDURAZYME*INFUS 1FL 5ML 500U
INN or Proposed INN: Laronidase
Genzyme Europe BVNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: no
10Italy
5NCT00741338
(ClinicalTrials.gov)
September 200813/8/2008Immune Tolerance Study With Aldurazyme® (Laronidase)A Trial of Antigen-specific Immune Tolerance Induction in Mucopolysaccharidosis I (MPS I) Patients Initiating Enzyme Replacement Therapy With Aldurazyme® (Laronidase)Mucopolysaccharidosis IBiological: Laronidase;Drug: Cyclosporine A (CsA);Drug: Azathioprine (Aza)Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedN/A5 YearsAll7Phase 1;Phase 2Brazil;Russian Federation;Ukraine
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2007-001453-26-NL
(EUCTR)
02/07/200713/11/2009Natural course, effects of enzyme therapy and health economic aspects in patients with mucopolysaccharidosis type I, II and VI. Long-term folloe-up of untreated patients and patients receiving commercially available Aldurazyme, Elaprase and Naglazyme.Natural course, effects of enzyme therapy and health economic aspects in patients with mucopolysaccharidosis type I, II and VI. Long-term folloe-up of untreated patients and patients receiving commercially available Aldurazyme, Elaprase and Naglazyme. Mucopolysaccharidosis type I, II and VI.
MedDRA version: 12.0;Level: LLT;Classification code 10028095;Term: Mucopolysaccharidosis IV
MedDRA version: 12.0;Classification code 10056886;Term: Mucopolysaccharidosis I
MedDRA version: 12.0;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II
Trade Name: Elaprase
Product Name: idursulfase
INN or Proposed INN: IDURSULFASE
Other descriptive name: Elaprase
Trade Name: Naglazyme
Product Name: Naglazyme
INN or Proposed INN: GALSULFASE
Trade Name: Aldurazyme
Product Name: Aldurazyme
INN or Proposed INN: LARONIDASE
Other descriptive name: Aldurazyme
NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
50Netherlands
7EUCTR2006-005216-27-FI
(EUCTR)
26/03/200714/03/2007Selkäytimeen annettu entsyymikorvaus mukopolysakkaridoosi I:n selkäytimen puristuman hoidossa. Kansainvälinen monikeskustutkimus. A study of intrathecal enzyme replacement therapy for spinal cord compression in mucopolysaccharidosis I, MIRC-001. - Intrathecal enzyme replacement in MPS ISelkäytimeen annettu entsyymikorvaus mukopolysakkaridoosi I:n selkäytimen puristuman hoidossa. Kansainvälinen monikeskustutkimus. A study of intrathecal enzyme replacement therapy for spinal cord compression in mucopolysaccharidosis I, MIRC-001. - Intrathecal enzyme replacement in MPS I Mucopolysaccharidosis I H/S, lysosomal storage disease with clinical manifestations such as progressive joint stiffness, growth retardation, corneal clouding, hepatosplenomegaly, cardiac and respiratory dysfunction, and in severe fore, mental retardation
MedDRA version: 9.1;Level: LLT;Classification code 10056887;Term: Mucopolysaccharidosis IH/S
Trade Name: Aldurazyme
Other descriptive name: LARONIDASE
HUS, Hospital for Children and AdolescentsNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
10Finland
8NCT00258011
(ClinicalTrials.gov)
December 200522/11/2005Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) DiseaseA Safety Confirmatory Study of JC0498 (Laronidase) in Mucopolysaccharidosis I (MPS I) PatientsMucopolysaccharidosis I;Hurler Syndrome;Hurler-Scheie Syndrome;Scheie SyndromeBiological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedN/AN/AAll3Phase 3Japan
9NCT00144781
(ClinicalTrials.gov)
December 20042/9/2005A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) DiseaseA Multicenter, Multinational, Randomized, Dose-Optimization Study of the Safety and Pharmacodynamic Response of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis IMucopolysaccharidosis I;Hurler's Syndrome;Hurler-Scheie Syndrome;Scheie SyndromeBiological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedN/AN/AAll34Phase 4Brazil;Canada
10NCT00144768
(ClinicalTrials.gov)
July 20042/9/2005A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated PatientsA Multicenter, Multinational, Open-Label Study of Anti-Laronidase Antibody Formation and Urinary GAG Levels in Patients With Mucopolysaccharidosis I (MPS I) Being Treated With Aldurazyme® (Laronidase).Mucopolysaccharidosis I;Hurler's Syndrome;Hurler-Scheie Syndrome;Scheie's SyndromeDrug: laronidaseGenzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedN/AN/ABoth25Phase 4United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT00176891
(ClinicalTrials.gov)
March 200412/9/2005Stem Cell Transplant w/Laronidase for HurlerPhase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH)Mucopolysaccharidosis I;Hurler SyndromeProcedure: Stem Cell Transplant;Drug: Laronidase ERTMasonic Cancer Center, University of MinnesotaNULLCompletedN/A7 YearsAll25Phase 2United States
12NCT00146757
(ClinicalTrials.gov)
October 20022/9/2005A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years OldA Phase II Open-Label Clinical Trial of Recombinant Human Alpha-L-iduronidase (Aldurazyme®) to Evaluate the Safety and Pharmacokinetics in Mucopolysaccharidosis I (MPS I) Patients Less Than 5 Years OldMucopolysaccharidosis I;Hurler Syndrome;Hurler-Scheie Syndrome;Scheie SyndromeBiological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedN/A5 YearsAll20Phase 2France;Germany;Netherlands;United Kingdom
13NCT00146770
(ClinicalTrials.gov)
May 20012/9/2005Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) PatientsA Multicenter, Multinational, Open-Label Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis IMucopolysaccharidosis I;Hurler's Syndrome;Hurler-Scheie Syndrome;Scheie SyndromeBiological: Aldurazyme;Biological: placeboGenzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedN/AN/AAll45Phase 3United States;Brazil;Canada;Germany;Italy;Netherlands;United Kingdom
14NCT00912925
(ClinicalTrials.gov)
December 20002/6/2009Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) IA Randomized, Double-Blind, Placebo-Controlled, Multicenter, Multinational, Clinical Study of Recombinant Human Alpha L-Iduronidase In Patients With Mucopolysaccharidosis IMucopolysaccharidosis I;Hurlers Syndrome;Hurler-Scheie SyndromeBiological: rhIDU (recombinant human-Alpha-L-Iduronidase);Biological: PlaceboGenzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompleted5 YearsN/AAll45Phase 3United States;Canada;Germany
15EUCTR2015-000585-61-Outside-EU/EEA
(EUCTR)
18/05/2015A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated PatientsA Multicenter, Multinational, Open-Label Study of Anti-Laronidase Antibody Formation and Urinary GAG Levels in Patients with Mucopolysaccaridosis I (MPS I) Being Treated with Aldurazyme® (laronidase) Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie SyndromeScheie's Syndrome
MedDRA version: 18.0;Level: PT;Classification code 10056886;Term: Mucopolysaccharidosis I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Laronidase
INN or Proposed INN: LARONIDASE
Genzyme, a Sanofi CompanyNULLNAFemale: yes
Male: yes
6United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2007-001163-30-Outside-EU/EEA
(EUCTR)
11/05/2015A trial of antigen-specific immune tolerance induction in mucopolysaccharidosis I (MPS I) patients initiating enzyme replacement therapy with Aldurazyme® (laronidase)A trial of antigen-specific immune tolerance induction in mucopolysaccharidosis I (MPS I) patients initiating enzyme replacement therapy with Aldurazyme® (laronidase) Mucopolysaccharidosis I
MedDRA version: 18.0;Level: PT;Classification code 10056886;Term: Mucopolysaccharidosis I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Laronidase
INN or Proposed INN: LARONIDASE
Genzyme, a Sanofi CompanyNULLNAFemale: yes
Male: yes
12Brazil;Russian Federation