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

  
23 trials found
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1NCT05634512
(ClinicalTrials.gov)
November 17, 202222/11/2022Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.Hematopoietic Cell Transplantation;Mucopolysaccharidosis Type IDrug: Laronidase therapy and a stem cell transplantMasonic Cancer Center, University of MinnesotaNULLEnrolling by invitation0 Years3 YearsAll24United States
2NCT05134571
(ClinicalTrials.gov)
October 28, 202121/10/2021China Post-marketing Surveillance (PMS) Study of Aldurazyme®A 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 CompanyNULLActive, not recruiting5 YearsN/AAll12Phase 4China
3NCT04532047
(ClinicalTrials.gov)
July 1, 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
4EUCTR2015-003031-35-GB
(EUCTR)
20/11/201527/10/2015Immune Tolerance Induction with Methotrexate in Hurler SyndromeA Single Centre Study Investigating the Safety and Efficacy of an Immune Modulation Regimen in Mitigating the Alloimmune Response to Intravenous Laronidase in Infants With Severe Mucopolysaccharidosis type I (Hurler syndrome) Prior to Haematopoietic Stem Cell Transplantation - Immune Tolerance Induction with Methotrexate in Hurler Syndrome Severe Mucopolysaccharidosis Type I (Hurler syndrome, MPS IH)
MedDRA version: 18.1;Level: LLT;Classification code 10028094;Term: Mucopolysaccharidosis IH;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Methotrexate
INN or Proposed INN: Methotrexate
Central Manchester University Hospitals NHS Foundation TrustNULLNot Recruiting Female: yes
Male: yes
4 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited Kingdom
5NCT01173016
(ClinicalTrials.gov)
May 29, 201228/7/2010Administration of IV Laronidase Post Bone Marrow Transplant in HurlerPilot Study of Administration of Intravenous Laronidase Following Allogeneic Transplantation for Hurler SyndromeHurler SyndromeDrug: LaronidaseMasonic Cancer Center, University of MinnesotaNULLCompletedN/A14 YearsAll11Phase 1United States
6NCT01572636
(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
7NCT00418821
(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/AFemale2Phase 4Italy;United States
8EUCTR2007-007003-33-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
9NCT00852358
(ClinicalTrials.gov)
June 200925/2/2009A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS IA Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis ICognitive Decline;Mucopolysaccharidosis I;Hurler-Scheie Syndrome;Scheie Syndrome;Lysosomal Storage DiseaseDrug: laronidasePatricia I. Dickson, M.D.The Ryan Foundation;BioMarin Pharmaceutical;Rare Diseases Clinical Research Network;National Center for Advancing Translational Science (NCATS);National Institute of Neurological Disorders and Stroke (NINDS);National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);University of Minnesota - Clinical and Translational Science Institute;University of California, Los AngelesCompleted6 YearsN/ABoth9N/AUnited States
10NCT00741338
(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
11NCT00638547
(ClinicalTrials.gov)
January 2, 200811/3/2008Intrathecal Enzyme Replacement for Hurler SyndromeIntrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome)Hurler SyndromeDrug: IRT LaronidaseMasonic Cancer Center, University of MinnesotaNULLCompleted6 Months3 YearsAll26Phase 1United States
12NCT00786968
(ClinicalTrials.gov)
January 200817/6/2008Extension Study of Intrathecal Enzyme Replacement Therapy for MPS IAn Extension Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis ISpinal Cord Compression;Mucopolysaccharidosis I;Hurler-Scheie Syndrome;Scheie Syndrome;Lysosomal Storage DiseaseDrug: laronidasePatricia I. Dickson, M.D.The Ryan Foundation for MPS ChildrenTerminated8 YearsN/ABoth3Phase 1United States;Finland
13EUCTR2007-001453-26-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
14EUCTR2006-005216-27-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
15NCT00258011
(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
16NCT00215527
(ClinicalTrials.gov)
November 200519/9/2005Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) IA Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis IMucopolysaccharidosis I;Lysosomal Storage Diseases;Spinal Cord CompressionDrug: laronidasePatricia I. Dickson, M.D.Ryan Foundation for MPS Children;University of California, Los Angeles;FDA Office of Orphan Products DevelopmentTerminated8 YearsN/ABoth4Phase 1United States;Finland
17NCT00144781
(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
18NCT00144768
(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
19NCT00176891
(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
20NCT00146757
(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
21NCT00146770
(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
22EUCTR2007-001163-30-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
23EUCTR2015-000585-61-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