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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02472535 | Study to Evaluate the Effects of MBX-8025 in Patients With HoFH | PHASE2 | COMPLETED | 13 | — | — | Apr 1, 2015 | Feb 1, 2016 | Mar 9, 2016 | 5 | Canada, France +2 |
Absolute and percentage (%) reduction in serum LDL-C at any point from baseline through Week 16.
| Arm | Type | Description |
|---|---|---|
| placebo, MBX-8025 50 mg, 100 mg or 200 mg capsules | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| Run-In Period: Placebo | OTHER | 2 capsules, once a day for two weeks |
| MBX-8025 50 mg (Dose Escalation Period 1) | DRUG | 1 capsule once a day for 4 weeks (MBX-8025 50 mg capsule) |
| MBX-8025 50 mg or 100 mg (Dose Escalation Period 2) | DRUG | 1 capsule once a day for 4 weeks (MBX-8025 50 mg or 100 mg capsule) |
| MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3) | DRUG | 1 or 2 capsules once a day for 4 weeks (MBX-8025 50 mg, 100 mg or two (2) 100 mg capsules) |
Inclusion Criteria: 1. Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements. 2. Male or female with HoFH confirmed by genotype (two mutant alleles at the LDL-Receptor (LDL-R) gene locus or double h...