Homozygous familial hypercholesterolemia
Conditions
Interventions
Sponsors
CymaBay Therapeutics Inc
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: 1. Written informed consent. 2. Male or female with HoFH confirmed by genotype (two mutant alleles at the LDL-Receptor gene locus) 3. 18 years of age or older 4. Existing lipid lowering therapies (statins, cholesterol absorption inhibitors, bile acid sequestrants, nicotinic acid and their combinations, LDL-C apheresis) on a stable regimen for at least four weeks before Screening Visit. 5. Stable lipid lowering diet compatible with a Step I diet of the American Heart Association. 6. Fasting LDL-C >= 4.8 mmol/L during screening. 7. For females of reproductive potential, use of at least one barrier contraceptive and a second effective birth control method during the study and for at least two weeks after the last dose. For male subjects, use of appropriate contraception (e.g., condoms) so their female partners of reproductive potential do not become pregnant during the use of study medication and for at least two weeks after the last dose.
Exclusion criteria
Exclusion criteria: 1. Treatment with lomitapide or mipomersen within two months of screening. 2. Heart Failure NYHA class III-IV or LVEF =3 times the ULN. 8. Unexplained CK >=5 times the ULN. 9. For females, pregnancy or breast-feeding.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| LDL-C. | — |
Secondary
| Measure | Time frame |
|---|---|
| Total cholesterol, HDL-C, VLDL-C, non-HDL-cholesterol, apolipoprotein B, apolipoproteïi A-I, lipoprotein (a), triglycerides, apolipoprotein C-III, RLP-cholesterol. Adverse events. Steady-state trough plasma levels of MBX-8025 and its metabolites. | — |
Countries
France, Netherlands, Norway
Outcome results
None listed