Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (wAIHA)
This research study is investigating a new oral medication called rilzabrutinib for people in the UK aged 18 and over who have warm autoimmune hemolytic anemia (wAIHA). This condition is where your immune system mistakenly attacks and destroys your red blood cells. The study aims to understand if rilzabrutinib is effective in treating wAIHA, how safe it is, and how your body processes the medication. Participants will receive rilzabrutinib as a tablet and will be carefully monitored throughout the study. The study involves different parts, with some participants potentially continuing treatment for several years to see the long-term effects. This is a Phase 2 trial, meaning it's still in the earlier stages of testing new treatments.
At a glance
What is this study about?
This study is focused on a health condition called warm autoimmune hemolytic anemia, or wAIHA. In simple terms, this is when your body's defence system (immune system) mistakenly attacks and destroys your own red blood cells. Red blood cells are really important because they carry oxygen around your body. When too many are destroyed, it can lead to tiredness, weakness, and other symptoms. The current treatments for wAIHA don't always work for everyone or can have side effects, which is why new options are needed.
This trial is looking at a new medication called rilzabrutinib. Researchers want to find out if this medication can help reduce the destruction of red blood cells in people with wAIHA. It's a 'Phase 2' study, which means it's testing the medication in a small group of people to see if it's safe and effective before it can be studied in larger groups. This type of research is crucial for developing better treatments for conditions like wAIHA, potentially offering new hope for patients.
The study will carefully assess how well rilzabrutinib works, if it's safe for people to take, and how the body handles the medication. If the results are positive, it could lead to further research and potentially a new treatment option becoming available in the future for people living with wAIHA.
Key takeaways
- The study is for adults with warm autoimmune hemolytic anemia (wAIHA) where steroids haven't worked well.
- It's testing a new oral medication called rilzabrutinib.
- The aim is to see how safe and effective the medication is.
- Participation involves a screening period, a 24-week treatment, and potentially a longer-term option.
- You'll have regular medical checks and follow-up.
- You can withdraw from the study at any time.
Who may be eligible?
To join this study, you must be an adult aged 18 or older with a confirmed diagnosis of primary wAIHA, or wAIHA linked to lupus (SLE), as long as your lupus isn't causing other serious problems apart from skin or joint issues. It's important that previous steroid treatments for your wAIHA haven't worked well for you in the past.
There are also some general health requirements. Your general fitness level, as assessed by a doctor, should be good enough for you to take part. You also need to be up-to-date with your routine vaccinations. Your Body Mass Index (BMI) should be within a specific range, between 17.5 and 40.
However, you wouldn't be able to join if you have certain other serious health conditions, including some types of cancer, particular blood disorders like myelodysplastic syndrome, or active infections like uncontrolled Hepatitis B or HIV. You also can't be taking part in another drug trial at the same time.
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 confirmed diagnosis of wAIHA (primary or lupus-related)?
- Have previous steroid treatments for your wAIHA not been successful?
- Do you have good general fitness?
- Are you up-to-date with your routine vaccinations?
- Do you NOT have serious conditions like recent cancer, myelodysplastic syndrome, or active uncontrolled Hepatitis B/HIV?
What does participation involve?
If you join this study, you will take the study medication, rilzabrutinib, as a tablet by mouth. Before starting treatment, there will be a screening period of up to four weeks to make sure you're suitable. The main treatment phase (Part A) will then last for 24 weeks.
If you complete Part A and the treatment has shown benefit, you might be able to continue in an extended treatment period (Part B). This part could last for a considerable time, potentially up to 253 weeks, to evaluate the long-term effects of the medication. Throughout the study, you will have regular visits with the study team for health checks, blood tests, and to discuss any side effects.
After you stop taking the study medication, either at the end of Part A or Part B, there will be a safety follow-up period of 7 days to monitor your health. There will also be a special visit when the very last participant in the study reaches their 52-week mark if you are in the Core Part B period.
Potential risks and benefits
Locations (27)
- The Oncology Institute of Hope & Innovation Site Number : 8400006Verified postcodeCerritos, United States
- TOI Clinical Research LLC_ Cerritos_Investigational Site Number: 8400006Verified postcodeCerritos, United States
- TOI Clinical Research LLC_Glendale_Investigational Site Number: 8400006Verified postcodeGlendale, United States
- TOI Clinical Research LLC_Long Beach_Investigational Site Number: 8400006Verified postcodeLong Beach, United States
- University of Southern California_Investigational Site Number: 8400009Verified postcodeLos Angeles, United States
- TOI Clinical Research LLC_Santa Ana_Investigational Site Number: 8400006Verified postcodeSanta Ana, United States
- The Lundquist Institute_Investigational Site Number: 8400005Verified postcodeTorrance, United States
- TOI Clinical Research LLC_ Whittier_Investigational Site Number: 8400006Verified postcodeWhittier, United States
- Georgetown University Hospital_Investigational Site Number: 8400003Verified postcodeWashington D.C., United States
- Oncology & Hematology Associates of West Broward_Investigational Site Number: 8400002Verified postcodeTamarac, United States
- Massachusetts General Hospital_Investigational Site Number: 8400001Verified postcodeBoston, United States
- Hanush-Krankenhaus_Investigational Site Number: 0400001Verified postcodeVienna, Austria
Common questions
What is wAIHA?
wAIHA stands for warm autoimmune hemolytic anemia. It's a condition where your immune system mistakenly attacks and destroys your own red blood cells.
What is rilzabrutinib?
Rilzabrutinib is the name of the new medication being tested in this study. It's designed to be taken by mouth as a tablet.
How long will the study last?
The initial treatment period is 24 weeks. If you respond well, you might be able to continue into an extended phase which could last several years.
Will I receive other treatments during the study?
No, you cannot receive other experimental drugs or participate in another clinical trial while in this study.
What if I change my mind about participating?
You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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