Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00280995 | Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy | PHASE2 | COMPLETED | 12 | — | — | Jan 1, 2006 | Aug 1, 2007 | Aug 3, 2016 | 6 | United States, Netherlands |
| Arm | Type | Description |
|---|---|---|
| Cohort A | EXPERIMENTAL | Loading doses followed by weekly maintenance doses |
| Cohort B | EXPERIMENTAL | Loading doses followed by weekly maintenance doses |
| Cohort C | EXPERIMENTAL | Loading doses followed by weekly maintenance doses |
| Cohort D | EXPERIMENTAL | Loading doses followed by extended weekly maintenance doses |
| Name | Type | Description |
|---|---|---|
| ISIS 301012 | DRUG | 100 mg subcutaneous injections on days 1, 4, 8, 11, 15, 22, 29, and 36 |
Inclusion Criteria: * Weight ≥ 40 kg for Cohorts A, B, \& C; Weight ≥ 50 kg for Cohort D * Diagnosis of Homozygous Familial Hypercholesterolemia. * Female must be non-pregnant and non-lactating. * On stable lipid lowering therapy for at least 4 weeks. * Lipid values that meet the pre-specified crit...