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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02265952 | Study of REGN1500 in Participants With Homozygous Familial Hypercholesterolemia (HoFH) | PHASE2 | COMPLETED | 9 | — | — | Feb 4, 2015 | Jul 23, 2018 | Dec 9, 2019 | 5 | United States, Canada +1 |
Percent change was reported for low-density lipoprotein cholesterol (LDL-C) from baseline (week 0) up to week 4. LDL-C was measured using conventional units mg/dL. The efficacy analysis set included all participants in the safety analysis set (SAF) who had baseline and at least 1 post-baseline measure of the lipid panel in the main study period.
| Arm | Type | Description |
|---|---|---|
| Open-label | EXPERIMENTAL | Open-label REGN1500 |
| Name | Type | Description |
|---|---|---|
| REGN1500 250 mg SC/15 mg/kg IV/450 mg SC | DRUG | Participants received single dose of REGN1500 subcutaneous (SC) injection of 250 milligrams (mg) at Week 0 (Day 1), followed by single dose of REGN1500 intravenous (IV) injection of 15 milligrams per kilogram (mg/kg) at Week 2 (Day 15) and then followed by 4 doses of REGN1500 SC injection of 450 mg once weekly starting from Week 12 (Day 85). Only the first 2 enrolled participants received 4 weekly REGN1500 450 mg SC doses at weeks 12, 13, 14, and 15 per the protocol. This dose regimen was removed under protocol amendment 4. Participants were followed for a period of 24 weeks (through Week 26 \[Day 183\]) after the last dose of study drug in the main study period. |
Inclusion Criteria: 1. Men and women ≥18 years of age at the time of the screening visit 2. Diagnosis of homozygous familial hypercholesterolemia (HoFH) 3. Willing to consistently maintain usual diet for the duration of the study Exclusion Criteria: 1. Background medical lipid modifying therapy t...