Clinical trials for new treatments
A number of new treatments for cholesterol and blood fat conditions are being researched in clinical trials. Here we explain what clinical trials are, what they involve, and why they are needed.
What is a clinical trial?
A clinical trial is a carefully designed study which looks at a new treatment to see if it works, how well it works, and what the side effects may be. New treatments may be tested against a placebo (a dummy drug), or against the best existing treatment to get a realistic idea of whether the new treatment is better.
While making lifestyle changes can help lower your cholesterol, some people need treatments to manage their cholesterol levels. It’s essential that existing and new treatments are thoroughly trialled to make sure that they’re as safe and effective as possible.
Why take part in a trial?
- You will help to treat and save lives of future generations.
- You will further medical research.
- You could be one of the first people to benefit from a new treatment.
- You may also receive payment depending on specific clinical trial.
Trials tend to happen in ‘phases’
- A phase 1 study is an early study done in small numbers of people, usually up to about 50 people. These are often focused on safety and side effects, what dose may be effective if it’s a drug trial, and how the treatment affects the body.
- A phase 2 trial is medium size and tends to include tens or sometimes hundreds of people. They aim to find the best dose of treatments, and find out more about side effects and how well the treatment works.
- A phase 3 study is larger and can include hundreds or even thousands of people. They are usually comparing the new treatment to existing treatments, or sometimes a dummy treatment (placebo).
Find out more and take part
Who is suitable to take part in a trial varies from study to study. If you find a study you might be suitable for, with your doctor to weigh up the value of taking part with any potential risks.
Here you can find out more about clinical trials and how to find trials that need participants.
NHS – The NHS has information and guidance if you are interested in taking part in a clinical trial.
Be Part of Research – find out where clinical trials are taking place for a particular medication.
The WHO – find an international clinical trials registry platform (ICTRP).
Clinicaltrials.gov – information on clinical studies around the world.
Medicines currently in clinical trials
Use the websites above to find out more about the trials for these medicines.
HEART UK are not directly involved in any of the following clinical trials and this page is for information purposes only. If you are interested in taking part in a clinical trial, please discuss this with your own specialist/healthcare professional.
CETP inhibitors
CETP is a protein that raises LDL cholesterol ("bad cholesterol") in the blood. Blocking this protein lowers LDL cholesterol and increases HDL cholesterol (sometimes called “good cholesterol”).
Previous trials have not shown that raising HDL cholesterol has a benefit in terms of cardiovascular risk, but did show that lowering LDL cholesterol reduced the risk of serious cardiovascular events such as heart attacks and strokes.
Current research drug: Obicetrapib
Lipoprotein (a) lowering therapies
Lipoprotein (a), or Lp (a) for short, is an emerging risk factor for cardiovascular disease and aortic valve disease. Your Lp (a) levels are determined by your genes and at the moment there are no medicines which effectively lower them.
New drugs are now in clinical trials which have been shown to lower Lp(a) at a genetic level, and clinical trials are aiming to find out whether reducing Lp(a) levels can help reduce the risk of cardiovascular disease.
Current research drugs:
- Pelacarsen (anti-sense oligonucleotide)
- Oliparsan (gene silencing therapy – short interfering RNA or siRNA)
- Zerlasiran (gene silencing therapy – short interfering RNA or siRNA)
PCSK9 inhibitors (oral treatment)
PCSK9 is a protein made in the liver. It destroys LDL receptors which sit on outside of cells and remove LDL cholesterol from the blood. Targeting this protein will mean there are more LDL receptors which then remove more LDL cholesterol from the blood – bringing your cholesterol levels down.
All PCSK9 inhibitors that are currently available are given by injection. Now, a once-a-day tablet that you take by mouth is in research trials.