19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 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 | 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 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 | 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 | Tanaka Tomoyuki | NULL | Recruiting | >= 18age old | <= 65age old | Both | 90 | Phase 3 | China;Italy;Republic of Korea;Japan |
| 2 | 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 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 | 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 |
| 3 | NCT05206773 (ClinicalTrials.gov) | March 11, 2022 | 20/10/2021 | A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Adult Participants With Fabry Disease | A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Adults With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months | Fabry Disease | Drug: Venglustat (GZ402671);Drug: Placebo | Genzyme, a Sanofi Company | NULL | Recruiting | 18 Years | N/A | All | 114 | Phase 3 | United States;Argentina;Brazil;Canada;China;Finland;Germany;Greece;Italy;Japan;Mexico;Norway;Poland;United Kingdom |
| 4 | JPRN-jRCT2021220001 | 20/04/2020 | 13/04/2022 | A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in male and female adult participants with Fabry disease | A randomized, double-blind, placebo-controlled, 12-month Phase 3 study to evaluate the effect of venglustat on neuropathic and abdominal pain in male and female adults with Fabry disease who are treatment-naive or untreated for at least 6 months | Fabry Disease | Drug: Venglustat (GZ402671) Pharmaceutical form: Tablet, Route of administration: Oral Drug: Placebo Pharmaceutical form: Tablet, Route of administration: Oral | Tanaka Tomoyuki | NULL | Recruiting | >= 18age old | Not applicable | Both | 114 | Phase 3 | United States;Japan |
| 5 | NCT02843035 (ClinicalTrials.gov) | January 4, 2017 | 20/7/2016 | Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension | A 4-part, Open-label, Multicenter, Multinational Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic, and Exploratory Efficacy of Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension | Gaucher Disease Type 1;Gaucher Disease Type 3 | Drug: venglustat (GZ402671);Drug: imiglucerase | Genzyme, a Sanofi Company | NULL | Active, not recruiting | 18 Years | N/A | All | 13 | Phase 2 | United States;Germany;Japan;United Kingdom |