Homozygous Familial HypercholesterolemiaPhase 2NCT02472535
Study to Evaluate the Effects of MBX-8025 in Patients With HoFH
A Phase 2 study testing MBX-8025 50 mg (Dose Escalation Period 1), MBX-8025 50 mg or 100 mg (Dose Escalation Period 2) and MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3) in people with Homozygous Familial Hypercholesterolemia, run by Gilead Sciences at 5 sites in 4 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Homozygous Familial Hypercholesterolemia
- What is being tested?
- MBX-8025 50 mg (Dose Escalation Period 1), MBX-8025 50 mg or 100 mg (Dose Escalation Period 2) and MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 13 participants
- Where
- 5 sitesin 4 countries
- Study dates
- Apr 2015to Feb 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
- 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).
- 18 years of age or older.
- 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.
- Stable lipid lowering diet compatible with a Step I diet of the American Heart Association (AHA).
- Fasting LDL-C ≥ 4.8 mmol/L (≥ 185.6 mg/dL) during screening.
- 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.
Who cannot join
- Treatment with lomitapide or mipomersen within two months of screening.
- 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%.
- Uncontrolled cardiac arrhythmia during the past three months of screening.
- Myocardial infarction, unstable angina, percutaneous coronary intervention, coronary artery bypass graft or stroke during the past three months of screening.
- Planned cardiac surgery, or planned revascularization, in the next four months.
- Uncontrolled hypertension.
Show all 10Show fewer
- Aspartate transaminase (AST) or Alanine transaminase (ALT) ≥ 3 times the Upper Limit of Normal (ULN).
- Unexplained creatine kinase (CK) ≥ 5 times the upper limit of normal (ULN).
- For females, pregnancy or breast-feeding.
- 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.
Trial locations and contacts
5 sites in 4 countries.
5 sites in 4 countries
Canada2 sites
Ecogene-21
Chicoutimi, Quebec, G7H 7K9
Montreal Heart Institute
Montreal, Quebec, H1T 1C8
France1 site
Endocrinologie metabolisme et prevention cardiovasulaire, Institut E3M et IHU cardiometabolique (ICAN), Hôpital Pitié Salpêtrière
Paris, 75 013
About this study
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)
A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH).
Read the 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.
- Conditions
- Homozygous Familial Hypercholesterolemia
- Being tested
- Run-In Period: Placebo, MBX-8025 50 mg (Dose Escalation Period 1), MBX-8025 50 mg or 100 mg (Dose Escalation Period 2), MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3)
- Keywords
- HoFH
- Medical subject headings
- Homozygous Familial Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Run-In Period: PlaceboOther
2 capsules, once a day for two weeks
MBX-8025 50 mg (Dose Escalation Period 1)Drug
1 capsule once a day for 4 weeks
(MBX-8025 50 mg capsule)
MBX-8025 50 mg or 100 mg (Dose Escalation Period 2)Drug
1 capsule once a day for 4 weeks
(MBX-8025 50 mg or 100 mg capsule)
MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3)Drug
1 or 2 capsules once a day for 4 weeks
(MBX-8025 50 mg, 100 mg or two (2) 100 mg capsules)
| Study group | Role | What they receive |
|---|---|---|
| placebo, MBX-8025 50 mg, 100 mg or 200 mg capsules | Experimental | Not described Receives: 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) |
What is being measured
The primary outcome is the main question the study is designed to answer.
LDL-C
Absolute and percentage (%) reduction in serum LDL-C at any point from baseline through Week 16.
Measured: 12-Weeks
Secondary outcomes (10)
Total Cholesterol (TC)
Absolute and percentage change at any point from baseline through Week 16
Measured: 12-Weeks
High-density lipoprotein (HDL) cholesterol [HDL-C]
Absolute and percentage change at any point from baseline through Week 16
Measured: 12-Weeks
Very Low-Density Lipoprotein (VLDL)
Absolute and percentage change at any point from baseline through Week 16
Measured: 12-Weeks
Non HDL-C
Absolute and percentage change at any point from baseline through Week 16
Measured: 12-Weeks
Remnant-like Particle (RLP-C)
Absolute and percentage change at any point from baseline through Week 16
Measured: 12-Weeks
Apolipoprotein B (Apo B)
Absolute and percentage change at any point from baseline through Week 16
Measured: 12-Weeks
Apolipoprotein A-I (Apo A-I)
Absolute and percentage change at any point from baseline through Week 16
Measured: 12-Weeks
Lipoprotein
Absolute and percentage change at any point from baseline through Week 16
Measured: 12-Weeks
Serum Triglyceride (TG)
Absolute and percentage change at any point from baseline through Week 16
Measured: 12-Weeks
Apolipoprotein C-III (Apo CIII)
Absolute and percentage change at any point from baseline through Week 16
Measured: 12-Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jun 2015 (estimated)
- Study startApr 2015
- Primary completionFeb 2016
- Study completionFeb 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Homozygous Familial HypercholesterolemiaStudies of the same condition, open and under way.
- More trials run by Gilead SciencesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gilead Sciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pol F Boudes, M.D., Study chair, Gilead Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02472535
- Sponsor's protocol ID
- CB8025-21427
- Status last verified
- Mar 2016
- First submitted
- 22 May 2015
- Last updated
- 9 Mar 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT02472535
- Acronym / sponsor ID
- CB8025-21427
- Conditions
- Homozygous Familial Hypercholesterolemia
- Keywords
- HoFH
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Gilead SciencesIndustry (pharma, biotech, devices)
- Study start
- Apr 2015
- Primary completion
- Feb 2016 (actual)
- Study completion
- Feb 2016 (actual)
- First posted
- 16 Jun 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH).
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| placebo, MBX-8025 50 mg, 100 mg or 200 mg capsules | Experimental | 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) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
- 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).
- 18 years of age or older.
- 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.
- Stable lipid lowering diet compatible with a Step I diet of the American Heart Association (AHA).
Key exclusion criteria (10 listed)
- Treatment with lomitapide or mipomersen within two months of screening.
- 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%.
- Uncontrolled cardiac arrhythmia during the past three months of screening.
- Myocardial infarction, unstable angina, percutaneous coronary intervention, coronary artery bypass graft or stroke during the past three months of screening.
- Planned cardiac surgery, or planned revascularization, in the next four months.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Run-In Period: Placebo2 capsules, once a day for two weeks | Other | placebo, MBX-8025 50 mg, 100 mg or 200 mg capsules |
| MBX-8025 50 mg (Dose Escalation Period 1)1 capsule once a day for 4 weeks (MBX-8025 50 mg capsule) | Drug | placebo, MBX-8025 50 mg, 100 mg or 200 mg capsules |
| 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 | placebo, MBX-8025 50 mg, 100 mg or 200 mg capsules |
| 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) | Drug | placebo, MBX-8025 50 mg, 100 mg or 200 mg capsules |
Primary outcome
LDL-C
Time frame: 12-Weeks
Absolute and percentage (%) reduction in serum LDL-C at any point from baseline through Week 16.
10 secondary outcomes
Total Cholesterol (TC)
Time frame: 12-Weeks
High-density lipoprotein (HDL) cholesterol [HDL-C]
Time frame: 12-Weeks
Very Low-Density Lipoprotein (VLDL)
Time frame: 12-Weeks
Non HDL-C
Time frame: 12-Weeks
Remnant-like Particle (RLP-C)
Time frame: 12-Weeks
Apolipoprotein B (Apo B)
Time frame: 12-Weeks
Apolipoprotein A-I (Apo A-I)
Time frame: 12-Weeks
Lipoprotein
Time frame: 12-Weeks
Serum Triglyceride (TG)
Time frame: 12-Weeks
Apolipoprotein C-III (Apo CIII)
Time frame: 12-Weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2016.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pol F Boudes, M.D. study chair
Gilead Sciences
5 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MBX-8025 50 mg (Dose Escalation Period 1), MBX-8025 50 mg or 100 mg (Dose Escalation Period 2) and MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3) in people with Homozygous Familial Hypercholesterolemia, run by Gilead Sciences at 5 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Mar 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Canada, France, Netherlands, Norway. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
LDL-C (time frame: 12-Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Mar 2016.