A Phase 3, Parallel-group Treatment, Blinded, Randomized, Placebo-controlled Study to Assess the Efficacy and Safety of Pegtibatinase Administered Subcutaneously in Addition to Standard of Care in Participants with Classical Homocystinuria due to Cystathionine Beta Synthase Deficiency (HARMONY)
This research study, called HARMONY, is investigating a new medicine named Pegtibatinase for individuals who have Classical Homocystinuria, a rare genetic disorder where the body has trouble breaking down certain proteins, leading to high levels of a substance called tHcy in the blood. This condition can cause serious health problems. The study will compare Pegtibatinase, given as an injection under the skin, with a dummy treatment (placebo) to see if it effectively lowers tHcy levels when added to their usual care. Researchers will also be checking how safe the new treatment is. The main goal is to find out if Pegtibatinase can significantly reduce these harmful tHcy levels over a 12-week period.
At a glance
What is this study about?
This study, called HARMONY, is for individuals living with a rare genetic condition known as Classical Homocystinuria. This condition happens when your body has a problem with a specific enzyme, leading to a build-up of a substance called homocystine (or tHcy) in your blood. High levels of tHcy can be harmful and lead to various health issues. The aim of this study is to see if a new medication, Pegtibatinase, can help to lower these unhealthy tHcy levels when taken alongside your regular, ongoing treatment.
Imagine this study like an experiment. Some people will receive the new medicine, Pegtibatinase, while others will get a non-active solution, like plain saline (saltwater), which looks exactly the same but doesn't contain any medicine. This is called a placebo. Neither you, nor your study doctor, will know if you're receiving the active medicine or the placebo. This helps us get clear and unbiased results to understand if Pegtibatinase truly makes a difference. The medicine is given as an injection under your skin.
Researchers will closely monitor your tHcy levels in your blood over several weeks. They want to compare how much these levels change in people taking Pegtibatinase versus those taking the placebo. They will also be very careful to record any side effects or changes in your health to make sure the new treatment is safe. Your participation could help us learn if Pegtibatinase could be a valuable new treatment option for Classical Homocystinuria in the future.
Key takeaways
- Tests a new medicine (Pegtibatinase) for Classical Homocystinuria.
- Aims to lower harmful tHcy levels in the blood.
- Compares the new medicine to a dummy treatment (placebo).
- Involves regular blood tests and health checks.
- Participation is voluntary, and you can withdraw anytime.
Who may be eligible?
To be part of this study, you need to be an adult, meaning 18 years old or older. The study is open to both men and women.
Beyond these basic requirements, there will be other specific medical checks to make sure the study is a good fit for you and that taking part would be safe. For example, the researchers will need to confirm you have Classical Homocystinuria due to a specific enzyme deficiency (Cystathionine Beta Synthase Deficiency).
Your doctor will look at your full medical history and current health to determine if you meet all the detailed criteria for joining the HARMONY study. It's important that your health situation matches what the study is looking for to ensure accurate results and your safety.
- Are you 18 years old or older?
- Do you have a diagnosis of Classical Homocystinuria?
- Are you currently on standard treatment for your condition?
- Are you able to attend regular clinic visits and have blood tests?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part in this study, you'll be attending several clinic visits over a period of time. During these visits, the study team will take blood samples to measure your tHcy levels and other important health markers. You will also have regular check-ups to monitor your general health and see if the medication is having any effects.
You will receive either the investigational medication, Pegtibatinase, or a placebo (which looks the same but contains no active drug). Both will be given as an injection under your skin, and the study team will show you how this is done. You will continue to take your usual prescribed treatments for Classical Homocystinuria throughout the study.
The initial part of the study where the main comparisons are made will last for about 12 weeks. After this, there will be further follow-up visits, potentially extending for several more weeks (up to 24 weeks or more), to keep an eye on your progress and any long-term effects. The total duration of your participation will be explained to you in detail by the study team.
Potential risks and benefits
Locations (8)
- —Belgium
- —Ireland
- —Spain
- —Italy
- —Germany
- —France
- —Portugal
- —Poland
Common questions
What is Classical Homocystinuria?
It's a rare genetic condition where your body can't properly process certain proteins, leading to harmful levels of a substance called tHcy in your blood.
What is Pegtibatinase?
It's a new medicine being tested in this study, designed to help lower the high levels of tHcy in people with Classical Homocystinuria.
What does 'placebo-controlled' mean?
It means some participants will get the actual medicine, while others will get a dummy treatment that looks identical but doesn't contain any active drug. This helps researchers compare the effects accurately.
How is the study medication given?
The medication (or placebo) is given as a shot just under the skin (subcutaneous injection).
How long does the main part of the study last?
The main part where the treatment is compared lasts about 12 weeks, with further follow-up for up to 24 weeks or more.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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