Venetoclax as treatment of acute graft-versus-host disease in patients after allogeneic hematopoietic stem cell transplantation in a Phase-II dose escalation pilot study.
This research is a Phase II study looking into Venclyxto (also known as Venetoclax) tablets for people who have developed acute graft-versus-host disease (aGVHD) that affects their skin after an allogeneic hematopoietic stem cell transplant. This condition happens when the transplanted cells attack the patient's own body. The main goal is to find the maximum tolerated dose (MTD) of Venclyxto, which means finding a dose that is both effective and has manageable side effects over 28 and 56 days. Researchers will also be checking how safe the medicine is, how well it helps improve the skin condition, and its overall effects on the body and quality of life.
At a glance
What is this study about?
This clinical trial is designed to investigate a new medication called Venclyxto (also known as Venetoclax) for patients who have developed acute graft-versus-host disease (aGVHD) affecting their skin. This condition can sometimes occur after an allogeneic hematopoietic stem cell transplant, a procedure where a patient receives healthy stem cells from a donor. In aGVHD, the new donor cells recognise the patient’s body as ‘foreign’ and begin to attack it, leading to symptoms like skin rashes.
The main purpose of this study is to find the best dose of Venclyxto for treating skin aGVHD. This is called the 'maximum tolerated dose' (MTD), which is the highest dose that can be given without causing too many severe side effects. The study will closely watch how patients react to different doses of Venclyxto over the first 28 and 56 days to determine this. Understanding the right dose is crucial for future, larger studies.
Beyond finding the right dose, researchers will also be carefully monitoring several other aspects. They will look at how safe Venclyxto is, how much it helps to improve the skin aGVHD, and whether it prevents the condition from getting worse. The study will also explore how Venclyxto affects the body at a cellular level, gather information on how it impacts patients' overall survival, and assess their quality of life during treatment. This comprehensive approach helps researchers understand the full picture of Venclyxto's potential as a treatment.
Key takeaways
- This study is testing a new medicine, Venclyxto, for skin-related acute graft-versus-host disease (aGVHD).
- The main goal is to find the safest and most effective dose of Venclyxto.
- It's for adults (18+) who have aGVHD of the skin after a stem cell transplant.
- Participation involves taking Venclyxto, regular hospital visits, and health checks.
- You will be closely monitored for side effects and how your condition responds.
- You can stop participating at any time without affecting your standard care.
Who may be eligible?
This study is open to adults aged 18 years and older. Both men and women are welcome to participate. The key requirement is that you must have been diagnosed with acute graft-versus-host disease (aGVHD) that is affecting your skin, following a stem cell transplant.
There will be other medical criteria that a doctor will need to check to make sure the study is a safe and appropriate option for you. These criteria are put in place to protect your safety and ensure the results of the study are clear and accurate.
- Are you 18 years old or older?
- Have you had an allogeneic stem cell transplant?
- Have you been diagnosed with acute graft-versus-host disease (aGVHD) that affects your skin?
- Are you able to attend regular hospital appointments for check-ups?
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 will be given Venclyxto tablets at a specific dose. This is a "dose escalation" study, meaning that the dose might gradually increase for different groups of participants to find the best level. You will have regular visits to the hospital for check-ups, blood tests, and possibly skin biopsies (where a small sample of skin is taken). These appointments are important for monitoring how you are responding to the medication, checking for any side effects, and assessing your overall health. You will also be asked to complete questionnaires about your quality of life and how you are feeling. The study will closely follow you for at least 56 days, with ongoing monitoring beyond that to understand the long-term effects. The total duration of your participation will depend on how you respond to the treatment and the study's follow-up plan.
Potential risks and benefits
Locations (1)
- —Austria
Common questions
What is acute graft-versus-host disease (aGVHD)?
It's a condition where new donor cells from a stem cell transplant see the patient's body as foreign and attack it, often causing reactions in the skin.
What is Venclyxto (Venetoclax)?
It's a medicine being tested in this study to see if it can help treat skin aGVHD after a stem cell transplant.
What does 'Phase II study' mean?
It means this is one of the earlier stages of drug testing. Researchers are trying to find the best dose and see how well the medicine works compared to its side effects.
Will I know which dose of Venclyxto I'm getting?
Yes, in this type of study, the dose you receive will be part of a planned escalation, which your study doctor will discuss with you.
What is a 'maximum tolerated dose'?
It's the highest dose of a drug that can be given without causing too many severe side effects. Finding this dose is a key goal of this early study.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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