Homozygous Familial Hypercholesterolemia
Conditions
Interventions
Product Name: MBX-8025
Product Code: MBX-8025
Pharmaceutical Form: Capsule
INN or Proposed INN: MBX-8025
CAS Number: 928821-40-3
Current Sponsor code: MBX-8025
Concentration unit: mg milligram(s)
Conc
Sponsors
CymaBay Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 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
Exclusion criteria
Exclusion criteria: 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Primary: To evaluate the effect of MBX-8025 on Low Density Lipoprotein Cholesterol (LDL-C). ;Secondary Objective: To evaluate the effects of MBX-8025 on other lipid parameters To evaluate the safety and tolerability of MBX-8025 in patients with HoFH. To evaluate steady-state trough plasma levels of MBX-8025 and its metabolites, M1, M2 and M3 Exploratory: To evaluate the effects of MBX-8025 on proprotein convertase subtilisin/kexin type 9 (PCSK9) and high sensitivity C reactive protein (hs-CRP). ;Primary end point(s): The effect of MBX-8025 on Serum LDL-C.;Timepoint(s) of evaluation of this end point: All visits after first drug administration: visit 4-10 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The effect of MBX-8025 on other lipid parameters, safety and tolerability and PK of MBX-8025 and it's metabolites.;Timepoint(s) of evaluation of this end point: All visits after first drug administration: visit 4-10 | — |
Countries
France, Netherlands, Norway
Contacts
Public ContactPol Boudes
CymaBay Therapeutics, Inc.
Outcome results
None listed