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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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11 April 2016 |
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Main ID: |
NCT02472535 |
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Date of registration:
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22/05/2015 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Study to Evaluate the Effects of MBX-8025 in Patients With HoFH
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Scientific title:
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A 12-week, Open-label, Dose-escalating, Phase 2 Study to Evaluate the Effects of MBX-8025 in Patients With Homozygous Familial Hypercholesterolemia (HoFH) |
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Date of first enrolment:
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April 2015 |
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Target sample size:
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13 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT02472535 |
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Study type:
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Interventional |
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Study design:
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Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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Phase:
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Phase 2
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Countries of recruitment
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Canada
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France
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Netherlands
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Norway
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Contacts
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Name:
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Pol F Boudes, M.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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CymaBay Therapeutics, Inc. |
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Key inclusion & exclusion criteria
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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 heterozygotes LDL-R/Apo-B).
3. 18 years of age or older.
4. Existing lipid lowering therapies (statins, cholesterol absorption inhibitors, bile
acid sequestrants, nicotinic acid and their combinations, low-density lipoprotein
(LDL) 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 (AHA).
6. Fasting LDL-C = 4.8 mmol/L (= 185.6 mg/dL) during screening.
7. For females or males 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.
Exclusion Criteria:
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 of screening.
5. Planned cardiac surgery, or planned revascularization, in the next four months.
6. Uncontrolled hypertension.
7. Aspartate transaminase (AST) or Alanine transaminase (ALT) = 3 times the Upper Limit
of Normal (ULN).
8. Unexplained creatine kinase (CK) = 5 times the upper limit of normal (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.
Age minimum:
18 Years
Age maximum:
N/A
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Homozygous Familial Hypercholesterolemia
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Intervention(s)
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Drug: MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3)
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Drug: MBX-8025 50 mg (Dose Escalation Period 1)
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Drug: MBX-8025 50 mg or 100 mg (Dose Escalation Period 2)
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Other: Run-In Period: Placebo
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Primary Outcome(s)
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LDL-C
[Time Frame: 12-Weeks]
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Secondary Outcome(s)
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Apolipoprotein A-I (Apo A-I)
[Time Frame: 12-Weeks]
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Apolipoprotein B (Apo B)
[Time Frame: 12-Weeks]
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Serum Triglyceride (TG)
[Time Frame: 12-Weeks]
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High-density lipoprotein (HDL) cholesterol [HDL-C]
[Time Frame: 12-Weeks]
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Lipoprotein
[Time Frame: 12-Weeks]
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Total Cholesterol (TC)
[Time Frame: 12-Weeks]
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Non HDL-C
[Time Frame: 12-Weeks]
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Very Low-Density Lipoprotein (VLDL)
[Time Frame: 12-Weeks]
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Apolipoprotein C-III (Apo CIII)
[Time Frame: 12-Weeks]
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Remnant-like Particle (RLP-C)
[Time Frame: 12-Weeks]
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Secondary ID(s)
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CB8025-21427
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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