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
| 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 | NCT05238324 (ClinicalTrials.gov) | March 2023 | 7/12/2021 | Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II | A Phase 1 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of HMI-203 in ERT-Treated Adults With Mucopolysaccharidosis Type II (MPS II) (juMPStart Trial) | Mucopolysaccharidosis II | Biological: Genetic HMI-203 | Homology Medicines, Inc | NULL | Recruiting | 18 Years | 45 Years | Male | 9 | Phase 1 | United States;Canada |
| 2 | NCT05222906 (ClinicalTrials.gov) | April 18, 2022 | 20/1/2022 | Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 | A Phase 3, Multicenter, Multinational, Randomized, Double-blind, Double-dummy, Active-comparator Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 (GD3) Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ERT) | Gaucher's Disease Type III | Drug: Venglustat;Drug: imiglucerase | Genzyme, a Sanofi Company | NULL | Recruiting | 12 Years | N/A | All | 40 | Phase 3 | United States;Argentina;Canada;China;France;Germany;Hungary;Japan |
| 3 | EUCTR2021-005402-10-DE (EUCTR) | 23/03/2022 | 05/01/2022 | Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 | A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 (GD3) who have reached therapeutic goals with Enzyme Replacement Therapy (ERT) - LEAP2MONO | Gaucher's disease type III MedDRA version: 24.1;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: venglustat Product Code: SAR402671, GZ402671 or GZ / SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Product Name: venglustat Product Code: SAR402671, GZ402671 or GZ / SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Trade Name: Cerezyme 400 Units Powder for concentrate for solution for infusion INN or Proposed INN: Imiglucerase | Sanofi-aventis recherche & développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 2 | Phase 3 | Brazil;Germany;China;United States;Taiwan;Turkey;United Kingdom;Egypt;France;Hungary;Canada;Argentina;Japan | ||
| 4 | NCT05152914 (ClinicalTrials.gov) | November 1, 2021 | 29/11/2021 | Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2 | Intravitreal Enzyme Replacement Therapy to Prevent Retinal Disease Progression in Children With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) | Neuronal Ceroid Lipofuscinosis Type 2 | Drug: Cerliponase Alfa | David L Rogers, MD | NULL | Enrolling by invitation | 24 Months | 72 Months | All | 5 | Phase 1/Phase 2 | United States |
| 5 | NCT04532047 (ClinicalTrials.gov) | July 1, 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 |
| 6 | NCT04353466 (ClinicalTrials.gov) | January 1, 2017 | 13/4/2020 | Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs | An Open-label, Investigator Initiated Clinical Trial to Asses Impact of Elelyso on Bone Involvement in Patients With Gaucher Disease Currently Treated With Other ERTs | Gaucher Disease, Type 1 | Procedure: quantitative chemical shift imaging (QCSI);Drug: Elelyso | Shaare Zedek Medical Center | Pfizer | Completed | 18 Years | N/A | All | 30 | N/A | NULL |
| 7 | NCT02520934 (ClinicalTrials.gov) | July 2015 | 6/8/2015 | Miglustat on Gaucher Disease Type IIIB | Evaluation of Combination Therapy With Miglustat and Enzyme Replacement Therapy on Gaucher Disease Type IIIB | Gaucher Disease | Drug: Miglustat;Drug: ERT | National Taiwan University Hospital | Actelion | Active, not recruiting | 6 Years | N/A | All | 19 | N/A | Taiwan |
| 8 | NCT02969200 (ClinicalTrials.gov) | April 2015 | 2/11/2016 | Fabry: Renal Function During Long-term ERT by 51Cr-EDTA Clearance | Fabry Disease: Renal Function During Long-term Enzyme Replacement Therapy Evaluated by Gold Standard GFR 51Cr-EDTA Clearance | Fabry Disease | Drug: Enzyme replacement therapy | Ulla Feldt-Rasmussen | NULL | Completed | N/A | N/A | All | 52 | Denmark | |
| 9 | NCT02930655 (ClinicalTrials.gov) | February 1, 2015 | 10/10/2016 | A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease | A Single-center, Open-label, Randomized, Versus a Control Group, Phase 1b Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Oral Lucerastat in Adult Subjects With Fabry Disease Receiving Enzyme Replacement Therapy | Fabry Disease | Drug: Lucerastat;Drug: Enzyme replacement therapy (ERT) | Idorsia Pharmaceuticals Ltd. | NULL | Completed | 18 Years | N/A | All | 14 | Phase 1 | Germany |
| 10 | NCT02228460 (ClinicalTrials.gov) | November 2014 | 27/8/2014 | Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease | A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Enzyme Replacement Therapy (ERT) Treatment-naïve Adult Male Patients Diagnosed With Fabry Disease | Fabry Disease | Drug: GZ/SAR402671 | Genzyme, a Sanofi Company | NULL | Completed | 18 Years | 49 Years | Male | 11 | Phase 2 | United States;France;Poland;Russian Federation;United Kingdom;Czech Republic;Czechia |
| 11 | EUCTR2013-005324-41-GB (EUCTR) | 07/10/2014 | 30/06/2014 | Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ402671 in Treatment-naïve Adult Male Patients with Fabry Disease | A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Enzyme Replacement Therapy (ERT) Treatment-naïve Adult Male Patients Diagnosed with Fabry Disease | Fabry disease MedDRA version: 17.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: GZ402671 / SAR402671 Other descriptive name: Genz-682452-AA Product Code: GZ402671 / SAR402671 Other descriptive name: Genz-682452-AA | Genzyme Corporation | NULL | Not Recruiting | Female: no Male: yes | 8 | Phase 2 | France;United States;Czech Republic;Poland;Russian Federation;United Kingdom | ||
| 12 | EUCTR2013-005324-41-CZ (EUCTR) | 19/09/2014 | 03/07/2014 | Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ402671 in Treatment-naïve Adult Male Patients with Fabry Disease | A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Enzyme Replacement Therapy (ERT) Treatment-naïve Adult Male Patients Diagnosed with Fabry Disease | Fabry disease MedDRA version: 17.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: GZ402671 / SAR402671 Other descriptive name: Genz-682452-AA Product Code: GZ402671 / SAR402671 Other descriptive name: Genz-682452-AA | Genzyme Corporation | NULL | Not Recruiting | Female: no Male: yes | 8 | Phase 2 | France;United States;Czech Republic;Poland;Russian Federation;United Kingdom | ||
| 13 | EUCTR2013-002554-78-GB (EUCTR) | 29/08/2013 | 09/09/2013 | An Extension of the PB-102-F01 study: A clinical study in patients with Fabry disease to assess the safety, tolerability, and the body processing of the medication PRX102, which will be given as an infusion. | An Extension of Phase 1/2, Open-Label, Dose Ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 38 Weeks (9 Months) to Adult Fabry Patients - PB-102-F02: Extension of the PB-102-F01 study (PRX-102 for ERT in Fabry Disease) | Fabry disease MedDRA version: 16.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: PRX-102 Product Code: PRX-102 INN or Proposed INN: PRX-102 | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 1;Phase 2 | United States;Paraguay;Argentina;Spain;Australia;Israel;Chile;United Kingdom | ||
| 14 | EUCTR2012-004786-40-GB (EUCTR) | 10/05/2013 | 07/01/2013 | A clinical study in patients with Fabry disease to assess the safety, tolerability, and the body processing of the medication PRX-102, which will be given as an infusion | A Phase 1/2, Open Label, Dose Ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 12 Weeks to Adult Fabry Patients - PB-102-F01: PRX-102 for ERT in patients with Fabry disease | Fabry disease MedDRA version: 17.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: PRX-102 Product Code: PRX-102 INN or Proposed INN: PRX-102 | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 1/2 | United States;Paraguay;Argentina;Spain;Australia;Israel;Chile;United Kingdom | ||
| 15 | EUCTR2010-022709-16-GB (EUCTR) | 23/08/2012 | 21/05/2012 | A study to find out if an investigational new drug called AT1001 can help people with Fabry disease and if it is safe for use in combination with enzyme replacement therapy (ERT). | AN OPEN-LABEL PHASE 2A STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT1001 (MIGALASTAT HYDROCHLORIDE) AND AGALSIDASE IN SUBJECTS WITH FABRY DISEASE | Fabry disease MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 18 | Phase 2 | Belgium;United Kingdom | ||
| 16 | EUCTR2010-022636-37-DE (EUCTR) | 09/05/2012 | 10/01/2012 | The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease | A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT | Fabry disease MedDRA version: 15.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Hungary;Germany;Turkey;Switzerland;France;Italy;Austria;Australia;Brazil;Israel;United Kingdom;Slovakia;Russian Federation;Taiwan;Mexico;Argentina;Belgium;Denmark;Japan;United States;Greece;Poland | ||
| 17 | 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 | |
| 18 | EUCTR2010-022636-37-IT (EUCTR) | 16/12/2011 | 13/03/2012 | A Randomized, Open-Label, Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT - AT1001-012 | A Randomized, Open-Label, Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT - AT1001-012 | Fabry disease MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrocloride Product Code: AT1001 INN or Proposed INN: migalastat hydrocloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Trade Name: REPLAGAL INN or Proposed INN: Agalsidase alfa Other descriptive name: NA Trade Name: FABRAZYME INN or Proposed INN: Agalsidase beta Other descriptive name: NA | AMICUS THERAPEUTICS, INC | NULL | Not Recruiting | Female: yes Male: yes | 50 | United States;Taiwan;Greece;Turkey;Austria;Russian Federation;Italy;Switzerland;United Kingdom;Mexico;Argentina;Brazil;Belgium;Denmark;Australia;Germany;Japan | |||
| 19 | EUCTR2010-022636-37-GR (EUCTR) | 02/11/2011 | 23/09/2011 | The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease | A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT | Fabry disease MedDRA version: 14.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | United States;Taiwan;Slovakia;Greece;Turkey;Austria;Israel;Russian Federation;Italy;Switzerland;United Kingdom;France;Hungary;Mexico;Argentina;Brazil;Poland;Belgium;Denmark;Australia;Germany;Japan | ||
| 20 | EUCTR2010-022709-16-BE (EUCTR) | 12/09/2011 | 10/08/2011 | A study to find out if an investigational new drug called AT1001 can help people with Fabry disease and if it is safe for use in combination with enzyme replacement therapy (ERT). | AN OPEN-LABEL PHASE 2A STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT1001 (MIGALASTAT HYDROCHLORIDE) AND AGALSIDASE IN SUBJECTS WITH FABRY DISEASE | Fabry disease MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 24 | Phase 2 | France;Canada;Belgium;Australia;Netherlands;United Kingdom;Switzerland | ||
| 21 | NCT01218659 (ClinicalTrials.gov) | September 8, 2011 | 6/10/2010 | Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease | A Randomized, Open-Label Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT | Fabry Disease | Drug: migalastat hydrochloride;Biological: agalsidase | Amicus Therapeutics | NULL | Completed | 16 Years | 74 Years | All | 68 | Phase 3 | United States;Australia;Austria;Belgium;Brazil;Denmark;France;Italy;Japan;United Kingdom;Argentina;Germany;Greece;Poland;Switzerland;Taiwan;Turkey |
| 22 | EUCTR2010-022636-37-AT (EUCTR) | 03/08/2011 | 27/07/2011 | The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease | A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT | Fabry disease MedDRA version: 16.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 68 | Phase 3 | France;United States;Greece;Brazil;Belgium;Denmark;Australia;Austria;Germany;Japan;Italy;United Kingdom | ||
| 23 | EUCTR2010-022636-37-DK (EUCTR) | 03/05/2011 | 06/04/2011 | The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease | A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT | Fabry disease MedDRA version: 16.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 68 | Phase 3 | United States;France;Greece;Belgium;Brazil;Austria;Australia;Denmark;Germany;United Kingdom;Japan;Italy | ||
| 24 | EUCTR2010-022636-37-GB (EUCTR) | 15/04/2011 | 22/12/2010 | The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease | A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT | Fabry disease MedDRA version: 16.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 68 | Phase 3 | United States;France;Greece;Belgium;Brazil;Denmark;Austria;Australia;Germany;Japan;Italy;United Kingdom | ||
| 25 | EUCTR2010-022636-37-BE (EUCTR) | 18/01/2011 | 16/12/2010 | The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease | A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT | Fabry disease MedDRA version: 16.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 68 | Phase 3 | United States;France;Greece;Brazil;Belgium;Denmark;Austria;Australia;Germany;United Kingdom;Japan;Italy | ||
| 26 | NCT00954460 (ClinicalTrials.gov) | October 2009 | 5/8/2009 | Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease | Multicenter Open-Label Treatment Protocol to Observe the Safety of Gene-Activated™ Human Glucocerebrosidase (GA-GCB, Velaglucerase Alfa) ERT in Newly Diagnosed or Previously Treated (With Imiglucerase) Patients With Type 1 Gaucher Disease | Gaucher Disease, Type 1 | Drug: velaglucerase alfa | Shire | NULL | Approved for marketing | 3 Years | N/A | All | United States | ||
| 27 | NCT00635427 (ClinicalTrials.gov) | May 2008 | 6/3/2008 | An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease | An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease | Gaucher Disease, Type 1 | Biological: VPRIV® | Shire | NULL | Completed | 2 Years | N/A | All | 95 | Phase 3 | United States;Argentina;India;Israel;Korea, Republic of;Paraguay;Poland;Russian Federation;Spain;Tunisia;United Kingdom |
| 28 | NCT00553631 (ClinicalTrials.gov) | January 2008 | 1/11/2007 | Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease | A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared With Imiglucerase in Patients With Type I Gaucher Disease | Gaucher Disease, Type 1 | Biological: velaglucerase alfa;Biological: imiglucerase | Shire | NULL | Completed | 2 Years | N/A | All | 34 | Phase 3 | United States;Argentina;India;Israel;Paraguay;Russian Federation;Spain;Tunisia;United Kingdom |
| 29 | EUCTR2005-001651-37-ES (EUCTR) | 12/04/2006 | 25/01/2006 | A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease | A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease | Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol:1BACKGROUND AND RATIONALE1.1Disease | Trade Name: Zavesca Product Name: Miglustat Product Code: OGT918 INN or Proposed INN: miglustat | Actelion Pharmaceuticals Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 4 | Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy | ||
| 30 | EUCTR2005-001651-37-IT (EUCTR) | 06/02/2006 | 14/03/2006 | A phase IV, open label, non comparative, multicenter study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch form enzyme replacement therapy ERT in adult patients with stable type 1 Gaucher disease - ND | A phase IV, open label, non comparative, multicenter study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch form enzyme replacement therapy ERT in adult patients with stable type 1 Gaucher disease - ND | Gaucher disease is the most common of the glycosphingolipids storage disease and has autosomal recessive inheritance. MedDRA version: 6.1;Level: PT;Classification code 10018048 | Trade Name: ZAVESCA 84CPS 100MG INN or Proposed INN: Miglustat | Actelion Registration Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 4 | Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy | ||
| 31 | EUCTR2005-001651-37-GB (EUCTR) | 27/10/2005 | 03/10/2005 | A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease | A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease | Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol: 1BACKGROUND AND RATIONALE 1.1Disease | Trade Name: Zavesca Product Name: Miglustat Product Code: OGT918 INN or Proposed INN: miglustat | Actelion Pharmaceuticals Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): yes | Hungary;Czech Republic;Spain;Germany;Italy;United Kingdom | ||
| 32 | NCT00391625 (ClinicalTrials.gov) | February 2005 | 20/10/2006 | Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT) | An Open-Label Extension of Study TKT025 Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A Enzyme Replacement Therapy | Gaucher Disease | Drug: GA-GCB | Shire | NULL | Completed | 18 Years | N/A | All | 10 | Phase 1/Phase 2 | Israel;Romania;Serbia;Former Serbia and Montenegro |
| 33 | 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 |
| 34 | EUCTR2021-005402-10-FR (EUCTR) | 27/01/2022 | Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 | A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 (GD3) who have reached therapeutic goals with Enzyme Replacement Therapy (ERT) - LEAP2MONO | Gaucher's disease type III MedDRA version: 24.1;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: venglustat Product Code: SAR402671, GZ402671 or GZ / SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Product Name: venglustat Product Code: SAR402671, GZ402671 or GZ / SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Trade Name: Cerezyme 400 Units Powder for concentrate for solution for infusion INN or Proposed INN: Imiglucerase | Sanofi-aventis recherche & développement | NULL | NA | Female: yes Male: yes | 50 | Phase 3 | United States;Taiwan;Turkey;United Kingdom;France;Egypt;Hungary;Canada;Argentina;Brazil;Germany;China;Japan | |||
| 35 | EUCTR2008-005825-12-Outside-EU/EEA (EUCTR) | 10/04/2012 | A multi-centre, open-label, switchover trial to assess safety and efficacy of taliglucerase alfa in adult and paediatric patients with Gaucher disease who are currently being treated with imiglucerase enzyme replacement therapy (ERT). | A multi-centre, open-label, switchover trial to assess safety and efficacy of taliglucerase alfa in adult and paediatric patients with Gaucher disease who are currently being treated with imiglucerase enzyme replacement therapy (ERT). | Gaucher disease MedDRA version: 14.1;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Taliglucerase alfa - Recombinant human glucocerebrosidase Product Code: prGCD Other descriptive name: TALIGLUCERASE ALFA | Protalix Biotherapeutics | NULL | NA | Female: yes Male: yes | 30 | Canada;Israel;United Kingdom;United States |