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

CymaBay Therapeutics, Inc.0 个研究点目标入组 13 人开始时间: 2015年5月19日最近更新: 2020年11月30日

适应症

试验速览

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•Inclusions:
•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 mutants 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 AHA.
•6. Fasting LDL-C = 4.8 mmol/L (= 185.6 mg/dL) 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 study and for at least two weeks after the last dose.
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 7
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 1

•Exclusions:
•1. Treatment with lomitapide or mipomersen within two months of screening.
•2. Heart Failure (HF) with New York Heart Association (NYHA) class III and class IV or a left ventricular ejection fraction (LVEF) of less than 30%.
•3. Uncontrolled cardiac arrhythmia during the past three months of screening.
•4. Myocardial infarction, unstable angina, percutaneous coronary intervention, coronary artery bypass graft or stroke during the past three months prior to Screening Visit.
•5. Planned cardiac surgery, or planned revascularization, in the next four months.
•6. Uncontrolled hypertension.
•7. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) = 3 times the upper limit of normal (ULN).
•8. Unexplained creatine kinase (CK) = 5 times the ULN.
•9. For females, pregnancy or breast-feeding.
•10. Any other condition(s) that would compromise the safety of the patient or compromise the quality of the clinical study as judged by the Investigator and/or Medical Monitor

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