A Phase 1/2, open-label, 2-arm study evaluating BLU-263 as monotherapy and in combination with azacitidine, in patients with KIT altered hematologic malignancies
This research study is investigating a new medication called BLU-263 for people with a rare type of blood cancer known as Advanced Systemic Mastocytosis. The study has two main parts. In one part, BLU-263 will be given by itself to see what dose is safe and effective. In another part, BLU-263 will be combined with an existing drug called azacitidine to see if they work better together. Researchers will carefully check for any side effects and how well the treatment helps control the cancer. This information will help doctors understand if BLU-263 could be a useful new treatment option.
At a glance
What is this study about?
This study is designed to learn more about a new medication called BLU-263 for a rare blood condition called Advanced Systemic Mastocytosis (AdvSM). In this condition, certain cells in the body, called mast cells, grow too much and can cause various health problems. The aim of this research is to see if BLU-263 is a safe and effective way to manage this condition.
The study is split into two main sections. In the first section, doctors will give BLU-263 on its own (that's called 'monotherapy'). They'll start with small doses and slowly increase them to find the best and safest amount to give. They will be looking very closely for any side effects and how the body reacts to the drug. In the second section, BLU-263 will be given alongside an existing medicine called azacitidine. This is to see if combining the two drugs works better and is still safe.
Throughout the study, the research team will keep a close eye on participants. They'll check for any unwanted effects and examine how well the treatment is working against the AdvSM. This includes checking blood tests, vital signs, and performing other medical assessments. The information gathered will be crucial in determining if BLU-263, either alone or with azacitidine, could become a helpful treatment for people living with Advanced Systemic Mastocytosis.
Key takeaways
- This study is testing a new drug for Advanced Systemic Mastocytosis.
- It will look at the drug alone and in combination with another medicine.
- The main goals are to find a safe dose and see how well it works.
- Participants will have regular medical check-ups and tests.
- You can stop participating at any time.
Who may be eligible?
To be considered for this study, like in all research, there are certain requirements that you need to meet. This helps make sure the study is right for you and that the results are reliable.
First, you must be at least 18 years old. There is no upper age limit for taking part. The study is open to both men and women.
Specifically for your health condition, you must have a diagnosis of Advanced Systemic Mastocytosis. There will be other medical criteria that the study doctors will discuss with you to ensure your health status is suitable for this particular treatment approach.
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.
- Are you 18 years old or older?
- Do you have a diagnosis of Advanced Systemic Mastocytosis?
- Are you willing to attend regular hospital or clinic visits?
- Are you comfortable with receiving a new, investigational drug?
What does participation involve?
If you decide to take part in this study, you will either receive BLU-263 by itself or BLU-263 combined with azacitidine. The treatment will involve regular visits to the hospital or clinic for drug administration, typically over cycles of treatment. During these visits, you will have several tests, including blood samples, checks of your heart's electrical activity (ECGs), and measurements of your vital signs like blood pressure and pulse. These tests are important for monitoring your safety and how well the treatment is working.
The duration of your participation will depend on how you respond to the treatment and the phase of the study you are in. You will have regular follow-up appointments, and the overall time you are involved in the study could range from several months to potentially longer, allowing researchers to see the long-term effects of the treatment. The research team will explain the full schedule of visits and assessments to you in detail.
Potential risks and benefits
Locations (6)
- —UnverifiedBelgium
- —UnverifiedSpain
- —UnverifiedFrance
- —UnverifiedNetherlands
- —UnverifiedNorway
- —UnverifiedGermany
Common questions
What is Advanced Systemic Mastocytosis?
It's a rare blood condition where special cells, called mast cells, grow too much in various parts of the body.
What is BLU-263?
BLU-263 is a new drug being tested in this study to see if it can help treat Advanced Systemic Mastocytosis.
Will I get both drugs or just one?
The study has two parts: one where BLU-263 is given alone, and another where it's combined with an existing drug called azacitidine. The study team will tell you which one you'd be in.
How long will I be in the study?
Your involvement in the study will depend on how you respond to the treatment, but it could range from several months or possibly longer.
Can I leave the study if I change my mind?
Yes, you can leave the study at any time, for any reason, without affecting your regular medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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