Efficacy and Safety Study of Eprotirome in HeFH Patients Who Are on Optimal Standard of Care

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

Status

Terminated

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01410383

Acronym

AKKA

Enrollment

236

Registered

2011-08-05

Start date

2011-10-31

Completion date

2014-10-31

Last updated

2012-09-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Heterozygous Familial Hypercholesterolaemia

Keywords

hypercholesterolemia,, dyslipidemia,, thyroid,, safety,, Heterozygous Familial Hypercholesterolaemia, coronary artery disease

Brief 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. 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.

Interventions

DRUGPlacebo

Tablets taken daily

DRUGEprotirome

Tablets taken daily

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

TRIPLE (Subject, Caregiver, Investigator)

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

* 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

Exclusion criteria

* Significant health problems in recent past including heart failure,cardiac electrophysiologic instability, rheumatoid arthritis, thyroid dysfunction, liver disease, cancer, secondary dyslipidaemia

Design outcomes

Primary

Measure	Time frame
Percent reduction in low-density lipoprotein cholesterol (LDL-C) from baseline	12 weeks treatment

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Mar 21, 2026