19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05134571 (ClinicalTrials.gov) | October 28, 2021 | 21/10/2021 | 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 China | 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 China | Mucopolysaccharidosis I | Drug: Laronidase | Genzyme, a Sanofi Company | NULL | Recruiting | 5 Years | N/A | All | 12 | Phase 4 | China |
| 2 | NCT04532047 (ClinicalTrials.gov) | April 7, 2021 | 19/8/2020 | In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases | In 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 Disease | Drug: Aldurazyme (laronidase) | University of California, San Francisco | Duke University | Recruiting | 18 Years | 50 Years | Female | 10 | Phase 1 | United States |
| 3 | EUCTR2015-003031-35-GB (EUCTR) | 20/11/2015 | 27/10/2015 | Immune Tolerance Induction with Methotrexate in Hurler Syndrome | A 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 Trust | NULL | Not 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): no | United Kingdom | ||
| 4 | NCT01173016 (ClinicalTrials.gov) | May 29, 2012 | 28/7/2010 | Administration of IV Laronidase Post Bone Marrow Transplant in Hurler | Pilot Study of Administration of Intravenous Laronidase Following Allogeneic Transplantation for Hurler Syndrome | Hurler Syndrome | Drug: Laronidase | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | 14 Years | All | 11 | Phase 1 | United States |
| 5 | NCT01572636 (ClinicalTrials.gov) | March 28, 2012 | 4/4/2012 | Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome | MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH). | Mucopolysaccharidosis Type IH;MPS I;Hurler Syndrome | Drug: Laronidase | Masonic Cancer Center, University of Minnesota | NULL | Terminated | N/A | N/A | All | 20 | United States | |
| 6 | NCT00418821 (ClinicalTrials.gov) | October 22, 2010 | 3/1/2007 | A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants | A 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 | Mucopolysaccharidosis I;Hurler's Syndrome;Hurler-Scheie Syndrome;Scheie | Biological: Aldurazyme (laronidase) | Genzyme, a Sanofi Company | BioMarin/Genzyme LLC | Recruiting | N/A | N/A | Female | 10 | Phase 4 | Italy;United States |
| 7 | EUCTR2007-007003-33-IT (EUCTR) | 16/09/2010 | 27/12/2010 | A 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 | A 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 BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: no | 10 | Italy | |||
| 8 | NCT00852358 (ClinicalTrials.gov) | June 2009 | 25/2/2009 | A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I | A Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I | Cognitive Decline;Mucopolysaccharidosis I;Hurler-Scheie Syndrome;Scheie Syndrome;Lysosomal Storage Disease | Drug: laronidase | Patricia 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 Angeles | Completed | 6 Years | N/A | Both | 9 | N/A | United States |
| 9 | NCT00741338 (ClinicalTrials.gov) | September 2008 | 13/8/2008 | Immune 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 I | Biological: Laronidase;Drug: Cyclosporine A (CsA);Drug: Azathioprine (Aza) | Genzyme, a Sanofi Company | BioMarin/Genzyme LLC | Completed | N/A | 5 Years | All | 7 | Phase 1/Phase 2 | Brazil;Russian Federation;Ukraine |
| 10 | NCT00638547 (ClinicalTrials.gov) | January 2, 2008 | 11/3/2008 | Intrathecal Enzyme Replacement for Hurler Syndrome | Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome) | Hurler Syndrome | Drug: IRT Laronidase | Masonic Cancer Center, University of Minnesota | NULL | Completed | 6 Months | 3 Years | All | 26 | Phase 1 | United States |
| 11 | NCT00786968 (ClinicalTrials.gov) | January 2008 | 17/6/2008 | Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I | An Extension Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I | Spinal Cord Compression;Mucopolysaccharidosis I;Hurler-Scheie Syndrome;Scheie Syndrome;Lysosomal Storage Disease | Drug: laronidase | Patricia I. Dickson, M.D. | The Ryan Foundation for MPS Children | Terminated | 8 Years | N/A | Both | 3 | Phase 1 | United States;Finland |
| 12 | EUCTR2007-001453-26-NL (EUCTR) | 02/07/2007 | 13/11/2009 | 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. | 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 | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Netherlands | ||||
| 13 | EUCTR2006-005216-27-FI (EUCTR) | 26/03/2007 | 14/03/2007 | Selkäytimeen 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 | Selkäytimeen 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 Adolescents | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Finland | |||
| 14 | NCT00258011 (ClinicalTrials.gov) | December 2005 | 22/11/2005 | Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease | A Safety Confirmatory Study of JC0498 (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients | Mucopolysaccharidosis I;Hurler Syndrome;Hurler-Scheie Syndrome;Scheie Syndrome | Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) | Genzyme, a Sanofi Company | BioMarin/Genzyme LLC | Completed | N/A | N/A | All | 3 | Phase 3 | Japan |
| 15 | NCT00215527 (ClinicalTrials.gov) | November 2005 | 19/9/2005 | Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I | A Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I | Mucopolysaccharidosis I;Lysosomal Storage Diseases;Spinal Cord Compression | Drug: laronidase | Patricia I. Dickson, M.D. | Ryan Foundation for MPS Children;University of California, Los Angeles;FDA Office of Orphan Products Development | Terminated | 8 Years | N/A | Both | 4 | Phase 1 | United States;Finland |
| 16 | NCT00144781 (ClinicalTrials.gov) | December 2004 | 2/9/2005 | A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease | A Multicenter, Multinational, Randomized, Dose-Optimization Study of the Safety and Pharmacodynamic Response of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I | Mucopolysaccharidosis I;Hurler's Syndrome;Hurler-Scheie Syndrome;Scheie Syndrome | Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) | Genzyme, a Sanofi Company | BioMarin/Genzyme LLC | Completed | N/A | N/A | All | 34 | Phase 4 | Brazil;Canada |
| 17 | NCT00144768 (ClinicalTrials.gov) | July 2004 | 2/9/2005 | A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients | A 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 Syndrome | Drug: laronidase | Genzyme, a Sanofi Company | BioMarin/Genzyme LLC | Completed | N/A | N/A | Both | 25 | Phase 4 | United States |
| 18 | NCT00176891 (ClinicalTrials.gov) | March 2004 | 12/9/2005 | Stem Cell Transplant w/Laronidase for Hurler | Phase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH) | Mucopolysaccharidosis I;Hurler Syndrome | Procedure: Stem Cell Transplant;Drug: Laronidase ERT | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | 7 Years | All | 25 | Phase 2 | United States |
| 19 | NCT00146757 (ClinicalTrials.gov) | October 2002 | 2/9/2005 | A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old | A 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 Old | Mucopolysaccharidosis I;Hurler Syndrome;Hurler-Scheie Syndrome;Scheie Syndrome | Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) | Genzyme, a Sanofi Company | BioMarin/Genzyme LLC | Completed | N/A | 5 Years | All | 20 | Phase 2 | France;Germany;Netherlands;United Kingdom |
| 20 | NCT00146770 (ClinicalTrials.gov) | May 2001 | 2/9/2005 | Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients | A Multicenter, Multinational, Open-Label Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I | Mucopolysaccharidosis I;Hurler's Syndrome;Hurler-Scheie Syndrome;Scheie Syndrome | Biological: Aldurazyme;Biological: placebo | Genzyme, a Sanofi Company | BioMarin/Genzyme LLC | Completed | N/A | N/A | All | 45 | Phase 3 | United States;Brazil;Canada;Germany;Italy;Netherlands;United Kingdom |
| 21 | EUCTR2007-001163-30-Outside-EU/EEA (EUCTR) | 11/05/2015 | A 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 Company | NULL | NA | Female: yes Male: yes | 12 | Brazil;Russian Federation | ||||
| 22 | EUCTR2015-000585-61-Outside-EU/EEA (EUCTR) | 18/05/2015 | A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients | A 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 Company | NULL | NA | Female: yes Male: yes | 6 | United States |