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.
| 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 | NCT04532047 (ClinicalTrials.gov) | December 1, 2020 | 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 | Not yet recruiting | 18 Years | 50 Years | Female | 10 | Phase 1 | United States |
| 2 | 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 | |
| 3 | NCT00418821 (ClinicalTrials.gov) | October 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 |
| 4 | 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 | |||
| 5 | 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 |
| 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 |
| 6 | 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 | ||||
| 7 | 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 | |||
| 8 | 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 |
| 9 | 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 |
| 10 | 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 |
| 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 |
| 11 | 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 |
| 12 | 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 |
| 13 | 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 |
| 14 | NCT00912925 (ClinicalTrials.gov) | December 2000 | 2/6/2009 | Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I | A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Multinational, Clinical Study of Recombinant Human Alpha L-Iduronidase In Patients With Mucopolysaccharidosis I | Mucopolysaccharidosis I;Hurlers Syndrome;Hurler-Scheie Syndrome | Biological: rhIDU (recombinant human-Alpha-L-Iduronidase);Biological: Placebo | Genzyme, a Sanofi Company | BioMarin/Genzyme LLC | Completed | 5 Years | N/A | All | 45 | Phase 3 | United States;Canada;Germany |
| 15 | 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 | ||||
| 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 |
| 16 | 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 |