19. Lysosomal storage disease Clinical trials / Disease details


Clinical trials : 854 Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191

  
17 trials found
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1NCT05134571
(ClinicalTrials.gov)
October 28, 202121/10/2021A Phase 4, Single-arm, Open-label Safety and Efficacy Study of Aldurazyme® (Laronidase) as Enzyme Replacement Therapy in Participants With Mucopolysaccharidosis I (MPS I) in ChinaA Phase 4, Single-arm, Open-label Safety and Efficacy Study of Aldurazyme® (Laronidase) as Enzyme Replacement Therapy in Participants With Mucopolysaccharidosis I (MPS I) in ChinaMucopolysaccharidosis IDrug: LaronidaseGenzyme, a Sanofi CompanyNULLRecruiting5 YearsN/AAll12Phase 4China
2NCT04532047
(ClinicalTrials.gov)
April 7, 202119/8/2020In 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 UniversityRecruiting18 Years50 YearsFemale10Phase 1United States
3NCT01572636
(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
4NCT00418821
(ClinicalTrials.gov)
October 22, 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
5EUCTR2007-007003-33-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
6NCT00741338
(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
7EUCTR2007-001453-26-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
8EUCTR2006-005216-27-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
9NCT00258011
(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
10NCT00144781
(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
11NCT00144768
(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
12NCT00176891
(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
13NCT00146757
(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
14NCT00146770
(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
15NCT00912925
(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
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
17EUCTR2015-000585-61-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