AAV directed hLDLR gene therapy ( DrugBank: - )
1 disease
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 79 | Homozygous familial hypercholesterolemia | 1 |
79. Homozygous familial hypercholesterolemia
Clinical trials : 145 / Drugs : 114 - (DrugBank : 26) / Drug target genes : 8 - Drug target pathways : 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 | 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) | Completed | 18 Years | N/A | All | 9 | Phase 1/Phase 2 | United States;Canada;Italy;Netherlands |