A PHASE 1/2 STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFECTS ON CLINICAL OUTCOMES OF PEGTIBATINASE (TVT-058) ADMINISTERED SUBCUTANEOUSLY IN SUBJECTS WITH CYSTATHIONINE BETASYNTHASE-DEFICIENT HOMOCYSTINURIA (COMPOSE)
Classical homocystinuria is a rare genetic condition where the body cannot properly break down certain protein building blocks. This leads to high levels of a substance called homocysteine in the blood, which can cause complications with the eyes, bones, heart, and brain. This trial is testing an experimental medicine called pegtibatinase (also known as TVT-058). It is an enzyme therapy given as an injection just under the skin. The main purpose of this Phase 1/2 trial is to check whether the medicine is safe, find the right dose, and watch for any unwanted side effects. Researchers will measure how the drug moves through the body and whether it helps reduce homocysteine levels. Some groups will also be checked for improvements in bone strength, eye health, and overall quality of life. The study helps doctors learn if this could become a helpful future treatment.
At a glance
What is this study about?
Classical homocystinuria is a lifelong inherited metabolic condition. People with this condition lack enough of a working enzyme called cystathionine beta-synthase (CBS). Without this enzyme, an amino acid called homocysteine builds up to harmful levels in the blood and urine. Over time, high levels can weaken bones, cause problems with eyesight, and increase the risk of blood clots.
Pegtibatinase is an experimental medicine designed to act like the missing enzyme. It aims to break down excess homocysteine and turn it into harmless substances that the body can clear away. In this study, the medicine is given as an injection under the skin (subcutaneously).
Because this is an early-stage study (Phase 1/2), the primary focus is participant safety. The trial team will closely monitor how people feel, check their blood work, and see how their immune systems respond to the medicine. They will also look for early signs that the treatment is helping to manage the condition better than existing diets and supplements alone.
Key takeaways
- The COMPOSE trial is testing pegtibatinase, an injection for classical homocystinuria.
- The main aim is to check the medicine's safety and see how well it lowers homocysteine.
- Participants will attend clinic visits for health checks, blood tests, and scans.
- The study is mainly for adults, with some groups open to younger participants.
- Taking part is voluntary, and you can stop at any time.
Who may be eligible?
This study is for people who have been diagnosed with classical homocystinuria caused by CBS deficiency. The main part of the trial is open to adults aged 18 and older, although certain sections of the research may look at younger age groups.
To take part, participants must be able to attend regular clinic appointments and receive injections under the skin. Your medical history will be reviewed to ensure you are well enough to join safely.
You may not be eligible if you have certain other severe health problems, known allergies to similar medicines, or if you are taking part in another clinical trial at the same time. The study doctor will check all of these details with you before you join.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I have been diagnosed with classical homocystinuria (CBS deficiency).
- I am aged 18 or older (or asking on behalf of an eligible child).
- I am able to attend regular clinic visits for health checks and tests.
- I am willing to receive injections under the skin.
- I am not currently taking part in another conflicting clinical trial.
What does participation involve?
If you take part, you will receive regular injections of the study drug just under your skin. In the first part of the study, you may receive the active medicine or a dummy treatment (placebo) to ensure fair comparison, while in other parts, everyone receives the active drug.
You will need to visit the hospital or clinic regularly. During these visits, the medical team will carry out health checks including blood and urine tests, heart traces (ECGs), eye examinations, and bone density scans. You will also be asked to answer simple questions about your daily life and energy levels. The full length of the study varies depending on which group you join, but it usually lasts several months to allow researchers to carefully track your progress.
Potential risks and benefits
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Common questions
How is the medicine given?
It is given as an injection just under the skin (subcutaneously), similar to how insulin is given for diabetes.
What is the main purpose of a Phase 1/2 trial?
Phase 1/2 trials are early-stage studies designed to check if a new medicine is safe, find out what side effects it might cause, and see if it shows early signs of working.
Will I have to change my special diet?
The trial doctors will give you clear instructions about your diet and may monitor your protein intake closely to keep you safe.
Do I have to pay to take part?
No, the study medicine and all trial-related tests are provided free of charge, and travel expenses are often covered.
Can I leave the study if I change my mind?
Yes, taking part is entirely voluntary. You can withdraw at any time, and your usual healthcare will continue as normal.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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