A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH) (study CB8025-21427) - CB8025-21427 (CymaBay)

CymaBay Therapeutics Inc0 个研究点目标入组 3 人开始时间: 待定最近更新: 2024年2月28日

适应症

试验速览

暂无简介。

•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.

•1. Treatment with lomitapide or mipomersen within two months of screening.
•2. Heart Failure NYHA class III-IV or LVEF <30%.
•3. Uncontrolled cardiac arrhythmia during the past three months of screening.
•4. Myocardial infarction, unstable angina, PCI, CABG or stroke during the past three months prior to screening.
•5. Planned cardiac surgery, or revascularization, in the next four months.
•6. Uncontrolled hypertension.
•7.AST or ALT >=3 times the ULN.
•8. Unexplained CK >=5 times the ULN.
•9. For females, pregnancy or breast-feeding.

发起方