A Phase 1/2, First-in-Human, Multicenter, Ascending Single-Dose and Multi-Dose Study to Assess the Safety of DIAG723, a Novel Bispecific ALK-1 and BMPRII Agonist Antibody in Adult Patients with Hereditary Hemorrhagic Telangiectasia (DIAMOND trial)
Hereditary haemorrhagic telangiectasia (HHT) is an inherited condition that causes fragile, tangled blood vessels. This often leads to frequent and heavy nosebleeds, low blood counts (anaemia), and a regular need for iron treatments or blood transfusions. The DIAMOND trial is testing a new medicine called DIAG723 in humans for the first time. The main goal is to check its safety, look for side effects, and find the right dose. Participants may receive DIAG723 or a harmless salt-water mixture (placebo). Doctors will carefully track participants' general health, heart activity, and blood tests. They will also see if the medicine helps reduce the severity of nosebleeds, raises red blood cell levels, and improves day-to-day quality of life. This trial is an important early step in finding better treatments for adults with HHT.
Em resumo
Sobre o que é este estudo?
Hereditary haemorrhagic telangiectasia (HHT) is a genetic disorder that affects how blood vessels form. Normally, blood flows from arteries into tiny capillaries before moving into veins. In people with HHT, some arteries connect directly to veins without capillaries in between. These fragile connections can bleed very easily, commonly causing severe nosebleeds, iron deficiency anaemia, and abnormal blood vessels in organs such as the lungs or liver.
The DIAMOND trial is evaluating a new laboratory-developed medicine called DIAG723. It is designed to encourage blood vessels to grow and repair themselves more normally. Because this is a 'first-in-human' study, researchers are testing it in people for the very first time. They need to find out how safe the medicine is, what side effects it might cause, and what dose works best.
During the trial, doctors will also check whether DIAG723 helps ease HHT symptoms. They will monitor whether it cuts down the number and duration of nosebleeds, reduces the need for iron supplements or blood transfusions, and improves participants' overall energy and quality of life.
Taking part in early research like this helps scientists understand whether new treatments have potential. While this trial focuses mainly on safety, the findings will guide future research into whether DIAG723 could become an effective long-term treatment for people with HHT.
Pontos principais
- The DIAMOND trial is testing an experimental medicine (DIAG723) for adults with HHT.
- This is a first-in-human trial focused primarily on checking safety, finding the best dose, and monitoring side effects.
- Researchers will track whether the medicine reduces nosebleeds and lowers the need for blood transfusions or iron.
- Participants will receive regular health checks, blood tests, and heart monitoring throughout the study.
- Taking part is completely voluntary, and you can withdraw at any point.
Quem pode ser elegível?
This trial is open to adults aged 18 and older who have been diagnosed with hereditary haemorrhagic telangiectasia (HHT). Both men and women can take part.
Because this is an early-stage safety trial, the study doctors will carry out detailed health checks before you can join. They will look closely at your medical history, current medicines, and general health to make sure it is safe for you to take part.
You may not be able to join if you have certain other serious medical conditions, are pregnant or breastfeeding, or are taking treatments that could interfere with the study medicine. The trial team will review your full medical details to confirm whether this study is suitable for you.
Este estudo pode ser adequado para você?
Responda a estas perguntas rápidas para ver se você pode ser elegível. Este é apenas um guia — a decisão final é da equipe de pesquisa.
- You are aged 18 or older.
- You have a confirmed diagnosis of hereditary haemorrhagic telangiectasia (HHT).
- You are willing to attend regular hospital appointments for check-ups and tests.
- You are able to keep a diary of your nosebleeds and symptoms.
- You are not currently taking part in another clinical drug trial (or are willing to check with the study team).
O que envolve a participação?
If you decide to join, you will first attend a screening visit to check your general health, blood counts, and heart rhythm. If you are eligible, you will receive either the study medicine (DIAG723) or a salt-water solution (placebo) given through a drip into a vein. Because this trial tests increasing doses, you may receive a single dose or several doses over a set schedule.
You will need to attend regular clinic visits so the medical team can monitor your health. These visits will involve blood and urine tests, blood pressure and temperature checks, physical examinations, and heart traces (ECGs). You will also be asked to keep a record of your nosebleeds and fill in questionnaires about your daily wellbeing.
The trial team will follow your progress closely throughout the treatment and for a set follow-up period afterwards. The exact number of visits and total time in the study will be explained in detail by the study team before you agree to join.
Riscos e benefícios potenciais
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Perguntas frequentes
What is a 'first-in-human' study?
It means the medicine has been tested thoroughly in laboratories, but this is the first time it is being given to people. These studies start with very small doses and monitor participants closely to check safety.
Will I definitely receive the study medicine?
Not necessarily. Some participants may receive a harmless salt-water drip (a placebo) instead of the active medicine so doctors can fairly compare the results.
Can I leave the trial if I change my mind?
Yes. Taking part is completely your choice. You are free to leave the study at any time, for any reason, and your regular medical care will continue as normal.
Will this trial cure my HHT?
No, this trial is not a cure. It is an early safety study designed to test how well the body tolerates DIAG723 and whether it can help manage symptoms like nosebleeds.
Does taking part cost anything?
No, the study medicine and all trial-related tests are provided free of charge. Clinical trials often offer help with travel expenses for clinic visits.
Como saber mais
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
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