Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) 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 |
79. 家族性高コレステロール血症（ホモ接合体）
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 | NCT04080050 (ClinicalTrials.gov) | September 30, 2019 | 3/9/2019 | 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 | Homozygous Familial Hypercholesterolemia (HoFH) | Drug: Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy | REGENXBIO Inc. | NULL | Active, not recruiting | 18 Years | N/A | All | 8 | United States;Canada;Netherlands;Italy |