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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02709850 | Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS ANGPTL3-LRx in Healthy Volunteers With Elevated Triglycerides and Participants With Familial Hypercholesterolemia | PHASE1 | COMPLETED | 48 | — | — | Nov 30, 2015 | Jun 26, 2017 | Nov 19, 2020 | 1 | Canada |
The safety and tolerability of IONIS ANGPTL3-LRx will be assessed by determining the incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters by dose. Safety results in subjects dosed with IONIS ANGPTL3-LRx will be compared with those from subjects dosed with placebo.
The plasma pharmacokinetics (concentration-time results) of IONIS ANGPTL3-LRx (unconjugated and conjugated ASO) will be assessed following single and multiple-dose SC administration. The amount of IONIS ANGPTL3-LRx excreted in urine at selected 24-hour intervals will also be determined.
Changes in serum angiopoietin-like 3 (ANGPTL3) levels compared to baseline.
| Arm | Type | Description |
|---|---|---|
| Cohorts A, D: Placebo | PLACEBO_COMPARATOR | Participants received a single-dose of IONIS ANGPTL3-LRx-matching placebo subcutaneously on Day 1. |
| Cohorts A, D: IONIS ANGPTL3-LRx 20 mg | EXPERIMENTAL | Participants received a single-dose of IONIS ANGPTL3-LRx 20 milligrams (mg) subcutaneously on Day 1. |
| Cohorts A, D: IONIS ANGPTL3-LRx 120 mg | EXPERIMENTAL | Participants received a single-dose of IONIS ANGPTL3-LRx 120 mg subcutaneously on Day 1. |
| Cohorts B, C: Placebo | PLACEBO_COMPARATOR | Participants received a single-dose of IONIS ANGPTL3-LRx-matching placebo subcutaneously on Day 1. |
| Cohorts B, C: IONIS ANGPTL3-LRx 40 mg | EXPERIMENTAL | Participants received a single-dose of IONIS ANGPTL3-LRx 40 mg subcutaneously on Day 1. |
| Cohorts B, C: IONIS ANGPTL3-LRx 80 mg | EXPERIMENTAL | Participants received a single-dose of IONIS ANGPTL3-LRx 80 mg subcutaneously on Day 1. |
| Cohorts AA-DD: Placebo | PLACEBO_COMPARATOR | Participants received IONIS ANGPTL3-LRx-matching placebo subcutaneously once per week for 6 weeks. |
| Cohorts AA-DD: IONIS ANGPTL3-LRx 10 mg | EXPERIMENTAL | Participants received IONIS ANGPTL3-LRx 10 mg subcutaneously once per week for 6 weeks. |
| Cohorts AA-DD: IONIS ANGPTL3-LRx 20 mg | EXPERIMENTAL | Participants received IONIS ANGPTL3-LRx 20 mg subcutaneously once per week for 6 weeks. |
| Cohorts AA-DD: IONIS ANGPTL3-LRx 40 mg | EXPERIMENTAL | Participants received IONIS ANGPTL3-LRx 40 mg subcutaneously once per week for 6 weeks. |
| Cohorts AA-DD: IONIS ANGPTL3-LRx 60 mg | EXPERIMENTAL | Participants received IONIS ANGPTL3-LRx 60 mg subcutaneously once per week for 6 weeks. |
| Name | Type | Description |
|---|---|---|
| IONIS ANGPTL3-LRx | DRUG | - |
| Placebo | DRUG | 0.9%NaCl, water, riboflavin |
Inclusion Criteria for All Cohorts: * Must have given written informed consent and be able to comply with all study requirements * Males or females 18 to 65 years, inclusive, at the time of informed consent * Body Mass Index (BMI) ≤ 35.0 kg/m2 * Females must be non-pregnant and non-lactating, and e...