Efficacy and Safety of M281 in Adults With Warm Autoimmune Hemolytic Anemia
This research study is investigating a new medication called M281 for adults who have warm autoimmune hemolytic anemia (wAIHA). This condition causes the body's immune system to mistakenly attack its own red blood cells. The main goal is to find out if M281 is helpful and safe to use. Participants will receive either M281 or a placebo (a dummy treatment without medicine) for about six months. After this, all participants will receive M281 for a much longer period, nearly three years, followed by an eight-week check-up phase. The study is open to adults aged 18 and older who have been diagnosed with wAIHA for at least three months and have already received or are currently receiving treatment for it. People who are pregnant, breastfeeding, or have certain other medical conditions cannot take part.
At a glance
What is this study about?
This study is focusing on a health condition called warm autoimmune hemolytic anemia, or wAIHA. In simple terms, wAIHA is a condition where your body's defence system, known as the immune system, mistakenly attacks and destroys your own red blood cells. Red blood cells are very important for carrying oxygen around your body, so when they are destroyed too quickly, it can lead to various health problems.
The researchers want to see if a new medication, called M281, can help manage wAIHA. They will compare M281 against a 'placebo', which looks exactly like the study drug but contains no active medicine. This helps them understand whether any changes seen are truly due to the new drug or just other factors. This study is in two important stages (Phase 2 and Phase 3 of clinical trials), which means it's still being carefully tested to make sure it's both safe and works well.
Finding new and effective treatments for wAIHA is really important for improving the lives of people living with this condition. This research could potentially help us understand more about wAIHA and offer a new treatment option in the future. Remember, taking part in a study is a personal choice, and it's always good to discuss it with your doctor.
Key takeaways
- This study is testing a new medication, M281, for warm autoimmune hemolytic anemia (wAIHA).
- It targets adults aged 18 and over who have been diagnosed with wAIHA for at least 3 months and are already receiving treatment.
- The study involves comparing M281 to a placebo for six months, followed by all participants receiving M281 for a longer period.
- Participation involves regular clinic visits and close monitoring for about three and a half years.
- There are potential benefits and risks, and you can withdraw at any time.
- Always discuss your options with your doctor before deciding.
Who may be eligible?
This study is designed for adults aged 18 and over who have been diagnosed with warm autoimmune hemolytic anemia (wAIHA). To be considered, you must have had your wAIHA diagnosis for at least three months, and you should already be receiving treatment for it, or have received treatment in the past. If you have just been diagnosed and haven't started any treatment yet, this particular study isn't suitable for you.
It's also really important that you are able to understand what the study involves and are happy to give your written consent to take part. This means you'll be fully informed about everything before you agree.
However, there are certain situations where you wouldn't be able to join. For example, if you are pregnant or breastfeeding, or if your doctor thinks you have other health issues that would make it unsafe for you to participate. Also, if you have other types of autoimmune hemolytic anemia (like cold antibody or mixed type), or a different condition called paroxysmal cold hemoglobinuria, you would not be eligible for this study.
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?
- Have you been diagnosed with warm autoimmune hemolytic anemia (wAIHA) for at least 3 months?
- Are you currently receiving treatment, or have you received treatment for wAIHA in the past?
- Are you not pregnant or breastfeeding?
- Do you not have other types of autoimmune hemolytic anemia or a similar blood condition (like cold antibody AIHA)?
- Are you willing and able to understand and sign consent forms and follow study instructions?
What does participation involve?
If you decide to take part, the study will involve several stages. Initially, for about six months (24 weeks), you will either receive the new medication M281 or a placebo (a dummy drug). This part of the study is 'double-blind', meaning neither you nor your study doctor will know whether you're receiving M281 or the placebo. After these six months, all participants will then receive M281 for a much longer period, lasting about three years (144 weeks). Throughout this time, you will have regular visits to the clinic for check-ups and assessments to monitor your health and how you're responding to the treatment.
Following the treatment period, there will be a final check-up phase lasting eight weeks after your last dose of the study drug. These appointments are important for the research team to see how your body reacts after stopping the medication. The total duration of your involvement could be around three and a half years, including all treatment and follow-up checks. You'll need to attend all scheduled appointments and follow the study instructions carefully.
Potential risks and benefits
Locations (171)
- MemorialCare Medical GroupVerified postcodeFountain Valley, United States
- University of Southern CaliforniaVerified postcodeLos Angeles, United States
- Compassionate Cancer CareVerified postcodeRiverside, United States
- American Institute of ResearchVerified postcodeWhittier, United States
- University of Colorado Anschutz Medical CampusVerified postcodeAurora, United States
- GNP ResearchVerified postcodeCooper City, United States
- University of Florida College of MedicineVerified postcodeGainesville, United States
- 21st Century OncologyVerified postcodeJacksonville, United States
- Lakes ResearchVerified postcodeMiami Lakes, United States
- AdventHealth Cancer InstituteVerified postcodeOrlando, United States
- Children's Research InstituteVerified postcodeSt. Petersburg, United States
- Rush University Medical CenterVerified postcodeChicago, United States
Common questions
What is warm autoimmune hemolytic anemia (wAIHA)?
It's a condition where your immune system mistakenly attacks and destroys your own red blood cells, which are vital for carrying oxygen around your body.
What is M281?
M281 is a new medication being tested in this study to see if it can help treat wAIHA.
What is a 'placebo'?
A placebo is a dummy treatment that looks like the study drug but contains no active medicine. It helps researchers compare the real drug's effects.
How long will I be in the study?
If you join, the study will last for about three and a half years, including treatment and follow-up checks.
Can I leave the study if I change my mind?
Yes, you have the right to leave the study at any time, for any reason, and it will not affect 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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