Aav directed hldlr gene therapy (DrugBank: -)
1 disease| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 79 | 家族性高コレステロール血症（ホモ接合体） | 1 |
| 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 | NCT02651675 (ClinicalTrials.gov) | March 2016 | 4/1/2016 | A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH) | AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia (HoFH) | Biological: AAV directed hLDLR gene therapy | Regenxbio Inc. | National Heart, Lung, and Blood Institute (NHLBI) | Active, not recruiting | 18 Years | N/A | All | 12 | Phase 1;Phase 2 | United States;Canada;Italy;Netherlands |