Alirocumab Clinical Trials

What Is Alirocumab?

Alirocumab is an FDA-approved medication for the treatment of hypercholesterolemia (high blood cholesterol levels). This experimental drug, also referred to as "study drug" in some research, is primarily investigated for its effects on cholesterol. The specific mechanism of action for alirocumab is not detailed in the provided trial descriptions. It is administered as a subcutaneous injection, with one trial mentioning a 300 mg dose prior to surgery. The drug is being researched to understand its safety and effectiveness, including different dosing schedules and its use in patients who may continue or stop treatment based on clinical decisions and patient preference.

Uses and Conditions Under Study

Alirocumab is primarily studied for conditions related to high cholesterol and cardiovascular health.

• High Cholesterol and Familial Hypercholesterolemia: Alirocumab is approved for hypercholesterolemia and is extensively studied for this condition, with 41 trials for hypercholesterolemia, 6 trials for hypercholesterolaemia, and 3 trials for Heterozygous Familial Hypercholesterolemia. These conditions involve abnormally high levels of cholesterol in the blood, which can lead to serious health problems.
• Cardiovascular Diseases and Events: The drug is also investigated for its role in preventing or managing cardiovascular issues. This includes 4 trials for Acute Coronary Syndrome, 3 trials for Cardiovascular Diseases, 2 trials for Myocardial Infarction (heart attack), and 2 trials for Stroke.
• Dyslipidemias and Atherosclerosis: Alirocumab is being studied in 3 trials for Dyslipidemias, which are disorders of lipid metabolism. Additionally, it is under investigation for Atherosclerosis (3 trials) and Intracranial Atherosclerosis (3 trials), conditions where plaque builds up inside arteries.

These studies aim to evaluate how alirocumab can help improve lipid profiles and reduce the risk of cardiovascular events in patients with these conditions.

Dosing

Alirocumab has been studied in various dosage forms and administration schedules, primarily as a subcutaneous injection.

• Administration: It is typically administered via subcutaneous injection, with some trials exploring auto-injector devices.
• Strengths and Schedules: Investigational doses range from 30 mg to 300 mg. Common dosing frequencies include every 2 weeks (Q2W) and every 4 weeks (Q4W). For example, studies have explored 75 mg Q2W, 150 mg Q2W, 150 mg Q4W, and 300 mg Q4W.
• Weight-Based Dosing: Some trials have investigated weight-based dosing, such as 30 mg Q2W for participants under 50 kg and 50 mg Q2W for those 50 kg or more, or 75 mg Q4W for participants under 50 kg and 150 mg Q4W for those 50 kg or more.
• Combination Therapy: Alirocumab has been studied both as a standalone treatment and in combination with other lipid-lowering medications. This includes co-administration with statin therapy (e.g., atorvastatin, rosuvastatin), ezetimibe, and fenofibrate.

The specific dosage and schedule depend on the condition being treated and individual patient needs, as determined by a healthcare provider.

Side Effects

The most common side effect reported in clinical trials for Alirocumab was viral upper respiratory tract infection, affecting 15.3% of patients compared to 13.6% of those on placebo. Other common side effects included:

• Upper respiratory tract infection: 8.1% of patients taking Alirocumab experienced this, compared to 8.5% on placebo.
• Nasopharyngitis (common cold): 7.7% on Alirocumab versus 6.7% on placebo.
• Injection site reaction: 7.5% on Alirocumab versus 5.6% on placebo.
• Influenza (flu): 6.9% on Alirocumab versus 6.4% on placebo.
• Back pain: 6.4% on Alirocumab versus 6.2% on placebo.
• Arthralgia (joint pain): 6.4% on Alirocumab versus 7.6% on placebo.
• Myalgia (muscle pain): 5.5% on Alirocumab versus 5.1% on placebo.

These side effects were observed across multiple clinical trials involving thousands of patients.

Clinical Trial Results

Clinical trials have demonstrated the effectiveness of Alirocumab in lowering cholesterol levels, particularly low-density lipoprotein cholesterol (LDL-C), often referred to as "bad" cholesterol.

Study in Patients with Heterozygous Familial Hypercholesterolemia (HeFH) (NCT01266876)

In a study of patients with HeFH, Alirocumab significantly reduced LDL-C. After 12 weeks, patients receiving Alirocumab 150 mg every two weeks experienced an average LDL-C reduction of 102.5 mg/dL (67.9% reduction), compared to a 19.1 mg/dL reduction (10.7%) in the placebo group. Furthermore, 81.3% of patients on Alirocumab 150 mg every two weeks achieved an LDL-C level below 70 mg/dL, whereas no patients in the placebo group reached this target.

Study in Patients with Primary Hypercholesterolemia on Stable Atorvastatin Therapy (NCT01288443)

This trial evaluated Alirocumab in patients already taking atorvastatin. At Week 12, Alirocumab 150 mg every two weeks reduced LDL-C by an average of 72.4%, while the placebo group saw only a 5.1% reduction. Notably, 100.0% of patients receiving Alirocumab 150 mg every two weeks achieved an LDL-C level below 70 mg/dL, compared to 3.2% of those on placebo.

Study of Alirocumab Co-administered with High-Dose Atorvastatin (NCT01288469)

When Alirocumab was added to high-dose atorvastatin, patients experienced substantial LDL-C lowering. After 8 weeks, patients on Alirocumab plus atorvastatin 80 mg achieved an average LDL-C reduction of 73.2%. In comparison, patients on atorvastatin 80 mg alone (placebo) had a 17.3% reduction. All patients (100.0%) in the Alirocumab plus atorvastatin 80 mg group reached an LDL-C level below 70 mg/dL, versus 17.2% in the atorvastatin alone group.

ODYSSEY Long Term Study (NCT01507831)

This long-term study in high cardiovascular risk patients showed sustained benefits. After 78 weeks, Alirocumab 150 mg every two weeks reduced LDL-C by 52.4%, while the placebo group experienced a 3.6% increase. At Week 24, 79.3% of Alirocumab patients reached an LDL-C level below 70 mg/dL, compared to 8.0% on placebo. The study also observed a lower rate of major adverse cardiovascular events in the Alirocumab group (1.7%) compared to placebo (3.3%).

Open-Label Extension Study in HeFH Participants (NCT01576484)

An open-label extension study confirmed the long-term efficacy of Alirocumab. Participants who continued or started Alirocumab achieved a mean LDL-C reduction of 57.36% from their baseline in this study by the end of treatment. By the end of treatment, 85.45% of all participants achieved an LDL-C level below 70 mg/dL or a 50% reduction if their baseline LDL-C was above 70 mg/dL.

Currently Recruiting Trials

Where to Participate

Development Timeline