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
Showing 1 to 10 of 97 diseases
| 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 | NCT05698901 (ClinicalTrials.gov) | March 31, 2023 | 16/1/2023 | Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease | Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease | Fabry Disease | Drug: Agalsidase beta | Mackay Memorial Hospital | NULL | Not yet recruiting | 18 Years | N/A | All | 150 | NULL | |
| 2 | NCT05067868 (ClinicalTrials.gov) | November 1, 2022 | 24/9/2021 | A Study of Replagal in Children and Adults With Fabry Disease in India | A Prospective, Open-label, Multicentre, Interventional, Single-arm, Phase IV Study to Evaluate the Safety and Efficacy of Agalsidase Alfa (r-DNA Origin) (Replagal™) in Indian Children and Adults With Fabry Disease A Prospective, Open-label, Multicentre, Interventional, Single-arm, Phase IV Study to Evaluate the S ... | Fabry Disease | Biological: Replagal | Shire | NULL | Recruiting | N/A | N/A | All | 5 | Phase 4 | India |
| 3 | EUCTR2018-004689-32-FI (EUCTR) | 12/10/2022 | 17/08/2021 | Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Diseas ... | A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subj ... | REPLAGAL is intended for use for patients with Fabry disease. MedDRA version: 24.1;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] REPLAGAL is intended for use for patients with Fabry disease. MedDRA version: 24.1;Level: PT;Classif ... | Trade Name: REPLAGAL ® Product Name: REPLAGAL Product Code: SHP675 INN or Proposed INN: AGALSIDASE ALFA Other descriptive name: Gene Activated a-Galactosidase A Trade Name: REPLAGAL® Product Name: REPLAGAL Product Code: SHP675 INN or Proposed INN: AGALSIDASEALFA Ot ... | Shire Human Genetic Therapies, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 45 | Phase 3 | Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Australia;Latvia;Germany;Sweden;Bosnia and Herzegovina Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Poland;Brazil;Belgium; ... | ||
| 4 | JPRN-jRCT2071220022 | 14/07/2022 | 25/06/2022 | A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry disease A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and fema ... | A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglusta ... | Fabry's disease | Drug: Venglustat (GZ402671) Pharmaceutical form: Tablet, Route of administration: Oral Drug: Agalsidase alfa Pharmaceutical form: Concentrate for solution for infusion, Route of administration: IV infusion Drug: Agalsidase beta (GZ419828) Pharmaceutical form: Powder for concentrate for solution for infusion, Route of administration: IV infusion Drug: Migalastat Pharmaceutical form: Hard capsules, Route of administration: Oral Drug: Venglustat(GZ402671) Pharmaceutical form: Tablet, Route of administration: Oral Drug: Agalsidase ... | Tanaka Tomoyuki | NULL | Recruiting | >= 18age old | <= 65age old | Both | 90 | Phase 3 | China;Italy;Republic of Korea;Japan |
| 5 | EUCTR2021-002320-20-NL (EUCTR) | 21/06/2022 | 16/03/2022 | A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry disease A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and fema ... | A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy - CARAT A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglusta ... | Fabry’s disease MedDRA version: 24.1;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] Fabry’s disease MedDRA version: 24.1;Level: PT;Classification code 10016016;Term: Fabry's disease;Sy ... | Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Trade Name: Replagal INN or Proposed INN: Agalsidase alfa Trade Name: Fabrazyme INN or Proposed INN: Agalsidase beta Trade Name: Galafold INN or Proposed INN: Migalastat Other descriptive name: Galafold Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglu ... | sanofi-aventis recherche et développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Norway;Netherlands;Germany;China;Japan;Korea, Republic of United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;D ... | ||
| 6 | EUCTR2021-002320-20-DK (EUCTR) | 05/05/2022 | 12/11/2021 | A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry disease A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and fema ... | A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy - CARAT A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglusta ... | Fabry’s disease MedDRA version: 24.1;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] Fabry’s disease MedDRA version: 24.1;Level: PT;Classification code 10016016;Term: Fabry's disease;Sy ... | Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Trade Name: Replagal INN or Proposed INN: Agalsidase alfa Trade Name: Fabrazyme INN or Proposed INN: Agalsidase beta Trade Name: Galafold INN or Proposed INN: Migalastat Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglu ... | sanofi-aventis recherche et développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Germany;Netherlands;Norway;China;Japan;Korea, Republic of United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;D ... | ||
| 7 | NCT05280548 (ClinicalTrials.gov) | May 3, 2022 | 4/3/2022 | A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Fema ... | A Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular Hypertrophy A Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglusta ... | Fabry's Disease | Drug: Venglustat (GZ402671);Drug: Agalsidase alfa;Drug: Agalsidase beta (GZ419828);Drug: Migalastat | Genzyme, a Sanofi Company | NULL | Recruiting | 18 Years | 65 Years | All | 90 | Phase 3 | China;Czechia;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Spain;Taiwan;Turkey |
| 8 | EUCTR2021-002320-20-PL (EUCTR) | 12/04/2022 | 11/01/2022 | A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry disease A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and fema ... | A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy - CARAT A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglusta ... | Fabry’s disease MedDRA version: 24.1;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] Fabry’s disease MedDRA version: 24.1;Level: PT;Classification code 10016016;Term: Fabry's disease;Sy ... | Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Trade Name: Replagal INN or Proposed INN: Agalsidase alfa Trade Name: Fabrazyme INN or Proposed INN: Agalsidase beta Trade Name: Galafold INN or Proposed INN: Migalastat Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglu ... | sanofi-aventis recherche et développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Denmark;Norway;Netherlands;Germany;China;Japan;Korea, Republic of United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Czech Republic; ... | ||
| 9 | EUCTR2021-002320-20-NO (EUCTR) | 11/04/2022 | 12/10/2021 | A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry disease A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and fema ... | A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglusta ... | Fabry’s disease MedDRA version: 24.1;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] Fabry’s disease MedDRA version: 24.1;Level: PT;Classification code 10016016;Term: Fabry's disease;Sy ... | Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Trade Name: Replagal INN or Proposed INN: Agalsidase alfa Trade Name: Fabrazyme INN or Proposed INN: Agalsidase beta Trade Name: Galafold INN or Proposed INN: Migalastat Product Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglu ... | sanofi-aventis recherche et développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Netherlands;Norway;Germany;China;Japan;Korea, Republic of United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;D ... | ||
| 10 | NCT05343715 (ClinicalTrials.gov) | October 23, 2021 | 24/1/2022 | PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion PK/PD Study of 2 AgalsidaseFormulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers ... | Comparative Pharmacokinetic and Pharmacodynamic Study Between 2 Agalsidase Beta Formulations at a Single Dose of 1 mg/kg of Agalsidase (Biosidus) and Fabrazyme (Sanofi Genzyme) as Intravenous Infusion in Male Healthy Volunteers Comparative Pharmacokinetic and Pharmacodynamic Study Between 2 AgalsidaseBeta Formulations at a Sin ... | Fabry Disease | Biological: Agalsidase beta from Biosidus 1 mg/kg;Biological: Fabrazyme (agalsidase beta) 1 mg/kg | Bio Sidus SA | NULL | Active, not recruiting | 18 Years | 40 Years | Male | 20 | Phase 1 | Argentina |