Heterozygous Familial HypercholesterolaemiaPhase 3AKKANCT01410383
Efficacy and Safety Study of Eprotirome in HeFH Patients Who Are on Optimal Standard of Care
A Phase 3 study testing Eprotirome in people with Heterozygous Familial Hypercholesterolaemia, run by Karo Bio AB.
Trial summary
Eprotirome is a liver selective thyroid hormone that can reduce several independent risk factors for cardiovascular disease, while an euthyroid state is preserved in the extrahepatic tissue. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Heterozygous Familial Hypercholesterolaemia
- What is being tested?
- Eprotirome
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 236 participants
- Where
- No sites listed yet
- Study dates
- Oct 2011to Oct 2014
- Phase
- Phase 3
- 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
- Patients with confirmed HeFH and 18 years of age at screening
- Presence of clinical atherosclerotic disease that confers high risk for CAD events together with an LDL-C more than 2 mmol/L (more than 80 mg/dL)
- Presence of risk factors for CVD (other than the HeFH diagnosis) together with an LDL-C more than 2.5 mmol/L (more than 100 mg/dL)
- On an optimal standard of care, defined as being on a stable dose of statin (rosuvastatin, atorvastatin, or simvastatin) with or without ezetimibe for 8 weeks prior to randomisation
Who cannot join
- Significant health problems in recent past including heart failure,cardiac electrophysiologic instability, rheumatoid arthritis, thyroid dysfunction, liver disease, cancer, secondary dyslipidaemia
About this study
Official title: A Placebo-controlled, Double-blind, Randomised, Parallel-group, Long Term Phase III Trial Assessing the Safety and Efficacy of Eprotirome in Patients With Heterozygous Familial Hypercholesterolaemia Who Are on Optimal Standard of Care
Eprotirome is a liver selective thyroid hormone that can reduce several independent risk factors for cardiovascular disease, while an euthyroid state is preserved in the extrahepatic tissue.
The purpose of this Phase III study is to assess the long-term efficacy and safety of Eprotirome in Patients with heterozygous Familial Hypercholesterolaemia who are on optimal standard of care.
- Being tested
- Placebo, Eprotirome
- Keywords
- hypercholesterolemia,, dyslipidemia,, thyroid,, safety,, Heterozygous Familial Hypercholesterolaemia, coronary artery disease
- Medical subject headings
- Hypercholesterolemia, Dyslipidemias, Thyroid Diseases, Coronary Artery Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
Tablets taken daily
EprotiromeDrug
Also called KB2115
Tablets taken daily
EprotiromeDrug
Also called KB2115
Tablets taken daily
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
| Eprotirome I | Experimental | Not described Receives: Drug: Eprotirome |
| Eprotirome II | Experimental | Not described Receives: Drug: Eprotirome |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent reduction in low-density lipoprotein cholesterol (LDL-C) from baseline
Measured: 12 weeks treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Aug 2011 (estimated)
- Study startOct 2011
- Primary completionOct 2013 (estimated)
- Study completionOct 2014 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Heterozygous Familial HypercholesterolaemiaStudies of the same condition, open and under way.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 236 participants (actual)
Sponsor and investigators
- Lead sponsor
- Karo Bio AB
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- John Kastelein, Professor, Principal investigator, Academic Medical Center (AMC) of the University of Amsterdam
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01410383
- Sponsor's protocol ID
- KBT 009
- Acronym
- AKKA
- Status last verified
- Aug 2012
- First submitted
- 13 Jul 2011
- Last updated
- 3 Sep 2012
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Sjouke B, Langslet G, Ceska R, Nicholls SJ, Nissen SE, Ohlander M, Ladenson PW, Olsson AG, Hovingh GK, Kastelein JJ. Eprotirome in patients with familial hypercholesterolaemia (the AKKA trial): a randomised, double-blind, placebo-controlled phase 3 study. Lancet Diabetes Endocrinol. 2014 Jun;2(6):455-63. doi: 10.1016/S2213-8587(14)70006-3. Epub 2014 Feb 18.
PubMed 24731671
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Placebo-controlled, Double-blind, Randomised, Parallel-group, Long Term Phase III Trial Assessing the Safety and Efficacy of Eprotirome in Patients With Heterozygous Familial Hypercholesterolaemia Who Are on Optimal Standard of Care
- ClinicalTrials.gov ID
- NCT01410383
- Acronym / sponsor ID
- AKKA / KBT 009
- Keywords
- hypercholesterolemia,, dyslipidemia,, thyroid,, safety,, Heterozygous Familial Hypercholesterolaemia, coronary artery disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 236 participants (actual)
- Lead sponsor
- Karo Bio ABIndustry (pharma, biotech, devices)
- Study start
- Oct 2011
- Primary completion
- Oct 2013 (estimated)
- Study completion
- Oct 2014 (estimated)
- First posted
- 5 Aug 2011 (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.
Eprotirome is a liver selective thyroid hormone that can reduce several independent risk factors for cardiovascular disease, while an euthyroid state is preserved in the extrahepatic tissue.
The purpose of this Phase III study is to assess the long-term efficacy and safety of Eprotirome in Patients with heterozygous Familial Hypercholesterolaemia who are on optimal standard of care.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Placebo |
| Eprotirome I | Experimental | Drug: Eprotirome |
| Eprotirome II | Experimental | Drug: Eprotirome |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Patients with confirmed HeFH and 18 years of age at screening
- Presence of clinical atherosclerotic disease that confers high risk for CAD events together with an LDL-C more than 2 mmol/L (more than 80 mg/dL)
- Presence of risk factors for CVD (other than the HeFH diagnosis) together with an LDL-C more than 2.5 mmol/L (more than 100 mg/dL)
- On an optimal standard of care, defined as being on a stable dose of statin (rosuvastatin, atorvastatin, or simvastatin) with or without ezetimibe for 8 weeks prior to randomisation
Key exclusion criteria (1 listed)
- Significant health problems in recent past including heart failure,cardiac electrophysiologic instability, rheumatoid arthritis, thyroid dysfunction, liver disease, cancer, secondary dyslipidaemia
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 |
|---|---|---|
| PlaceboTablets taken daily | Drug | Placebo |
| EprotiromeAlso: KB2115Tablets taken daily | Drug | Eprotirome I |
| EprotiromeAlso: KB2115Tablets taken daily | Drug | Eprotirome II |
Primary outcome
Percent reduction in low-density lipoprotein cholesterol (LDL-C) from baseline
Time frame: 12 weeks treatment
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Sep 2012.
- Derived from this study: PubMed 24731671Sjouke B, Langslet G, Ceska R, Nicholls SJ, Nissen SE, Ohlander M, Ladenson PW, Olsson AG, Hovingh GK, Kastelein JJ. Eprotirome in patients with familial hypercholesterolaemia (the AKKA trial): a randomised, double-blind, placebo-controlled phase 3 study. Lancet Diabetes Endocrinol. 2014 Jun;2(6):455-63. doi: 10.1016/S2213-8587(14)70006-3. Epub 2014 Feb 18.
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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.
John Kastelein, Professor principal investigator
Academic Medical Center (AMC) of the University of Amsterdam
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Eprotirome in people with Heterozygous Familial Hypercholesterolaemia, run by Karo Bio AB.
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 4 inclusion and 1 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: stopped early, as of the sponsor's last update on 3 Sep 2012. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 was stopped early. Results are not available in the source record.
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?
Percent reduction in low-density lipoprotein cholesterol (LDL-C) from baseline (time frame: 12 weeks treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01410383, which also lists 1 linked publication.
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 3 Sep 2012.