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Trial record 69 of 154 for:    "familial hypercholesterolemia"

Study to Evaluate the Effects of MBX-8025 in Patients With HoFH

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ClinicalTrials.gov Identifier: NCT02472535
Recruitment Status : Completed
First Posted : June 16, 2015
Last Update Posted : March 9, 2016
Sponsor:
Information provided by (Responsible Party):
CymaBay Therapeutics, Inc.

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

Condition or disease Intervention/treatment Phase
Homozygous Familial Hypercholesterolemia Other: Run-In Period: Placebo Drug: MBX-8025 50 mg (Dose Escalation Period 1) Drug: MBX-8025 50 mg or 100 mg (Dose Escalation Period 2) Drug: MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3) Phase 2

Detailed Description:

Open-label, single arm, non-controlled, dose ascending (50 mg/day, 100 mg/day and 200 mg/day) with three consecutive dose escalation periods.

After signing an informed consent subject will enter a screening period and a run-in stabilization period. At the end of run-in period patients will enter treatment phase. MBX-8025 in ascending doses (50 mg, 100 mg, and 200 mg) will be given within three consecutive 4 weeks periods, for a total of 12 weeks. At the end of treatment, subjects will enter a follow-up period.


Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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 Start Date : April 2015
Actual Primary Completion Date : February 2016
Actual Study Completion Date : February 2016

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: placebo, MBX-8025 50 mg, 100 mg or 200 mg capsules Other: Run-In Period: Placebo
2 capsules, once a day for two weeks

Drug: MBX-8025 50 mg (Dose Escalation Period 1)

1 capsule once a day for 4 weeks

(MBX-8025 50 mg capsule)


Drug: MBX-8025 50 mg or 100 mg (Dose Escalation Period 2)

1 capsule once a day for 4 weeks

(MBX-8025 50 mg or 100 mg capsule)


Drug: MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3)

1 or 2 capsules once a day for 4 weeks

(MBX-8025 50 mg, 100 mg or two (2) 100 mg capsules)





Primary Outcome Measures :
  1. LDL-C [ Time Frame: 12-Weeks ]
    Absolute and percentage (%) reduction in serum LDL-C at any point from baseline through Week 16.


Secondary Outcome Measures :
  1. Total Cholesterol (TC) [ Time Frame: 12-Weeks ]
    Absolute and percentage change at any point from baseline through Week 16

  2. High-density lipoprotein (HDL) cholesterol [HDL-C] [ Time Frame: 12-Weeks ]
    Absolute and percentage change at any point from baseline through Week 16

  3. Very Low-Density Lipoprotein (VLDL) [ Time Frame: 12-Weeks ]
    Absolute and percentage change at any point from baseline through Week 16

  4. Non HDL-C [ Time Frame: 12-Weeks ]
    Absolute and percentage change at any point from baseline through Week 16

  5. Remnant-like Particle (RLP-C) [ Time Frame: 12-Weeks ]
    Absolute and percentage change at any point from baseline through Week 16

  6. Apolipoprotein B (Apo B) [ Time Frame: 12-Weeks ]
    Absolute and percentage change at any point from baseline through Week 16

  7. Apolipoprotein A-I (Apo A-I) [ Time Frame: 12-Weeks ]
    Absolute and percentage change at any point from baseline through Week 16

  8. Lipoprotein [ Time Frame: 12-Weeks ]
    Absolute and percentage change at any point from baseline through Week 16

  9. Serum Triglyceride (TG) [ Time Frame: 12-Weeks ]
    Absolute and percentage change at any point from baseline through Week 16

  10. Apolipoprotein C-III (Apo CIII) [ Time Frame: 12-Weeks ]
    Absolute and percentage change at any point from baseline through Week 16


Other Outcome Measures:
  1. C reactive protein hs-(CRP) [ Time Frame: 12-Weeks ]
    Absolute and percentage change at any point from baseline through Week 16

  2. MBX-8025 Plasma Concentration Levels [ Time Frame: 12-Weeks ]
    Blood samples for the plasma concentration determination of MBX-8025 and its metabolites (M1, M2 and M3) collected pre-dose at the following visits: 4, 5, 6, 7, 8 and 9.

  3. Safety Measures: Number of Participants with Adverse Events as a Measure of Safety [ Time Frame: 12-Weeks ]
    Complete characterization of Adverse Events (AE), Biochemistry and Hematology

  4. Proprotein convertase subtilisin/kexin type 9 (PCSK-9) [ Time Frame: 12-Weeks ]
    Absolute and percentage change at any point from baseline through Week 16 for the following: Proprotein convertase subtilisin/kexin type 9 (PCSK-9)



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02472535


Locations
Canada, Quebec
Ecogene-21
Chicoutimi, Quebec, Canada, G7H 7K9
Montreal Heart Institute
Montreal, Quebec, Canada, H1T 1C8
France
Endocrinologie metabolisme et prevention cardiovasulaire, Institut E3M et IHU cardiometabolique (ICAN), Hôpital Pitié Salpêtrière
Paris, France, 75 013
Netherlands
Radbound UMC
Nijmegen, Netherlands, 6525 GA
Norway
Lipidklinikken, Oslo Universitetssykehus
Oslo, Norway, N-0373
Sponsors and Collaborators
CymaBay Therapeutics, Inc.
Investigators
Study Chair: Pol F Boudes, M.D. CymaBay Therapeutics, Inc.

Responsible Party: CymaBay Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT02472535     History of Changes
Other Study ID Numbers: CB8025-21427
First Posted: June 16, 2015    Key Record Dates
Last Update Posted: March 9, 2016
Last Verified: March 2016

Keywords provided by CymaBay Therapeutics, Inc.:
HoFH

Additional relevant MeSH terms:
Hypercholesterolemia
Hyperlipoproteinemia Type II
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias