79. 家族性高コレステロール血症（ホモ接合体） Homozygous familial hypercholesterolemia Clinical trials / Disease details
臨床試験数 : 145 / 薬物数 : 114 - (DrugBank : 26) / 標的遺伝子数 : 8 - 標的パスウェイ数 : 17
| 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 | EUCTR2019-004496-39-NL (EUCTR) | 12/08/2020 | 13/11/2020 | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 | Adults with homozygous familial hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: AAV8.TBG.hLDLR Product Code: AAV8.TBG.hLDLR | REGENXBIO Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 2 | United States;Canada;Netherlands;Italy | ||
| 2 | EUCTR2016-001446-25-IT (EUCTR) | 02/10/2017 | 06/05/2021 | This is a study to investigate if gene therapy is safe in people with homozygous familial hypercholesterolemia (also called HoFH). | AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) | Adults with homozygous familial hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: AAV8.TBG.hLDLR Product Code: AAV8.TBG.hLDLR Product Name: prednisone Product Code: prednisone | REGENXBIO Inc. | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 1;Phase 2 | United States;Canada;Netherlands;Italy | ||
| 3 | EUCTR2016-001446-25-NL (EUCTR) | 20/09/2017 | 12/09/2016 | This is a study to investigate if gene therapy is safe in people with homozygous familial hypercholesterolemia (also called HoFH). | AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) | Adults with homozygous familial hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: AAV8.TBG.hLDLR Product Code: AAV8.TBG.hLDLR | University of Pennsylvania | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 1;Phase 2 | United States;Canada;Netherlands;Italy |