An Open-label, Single-arm, Multicenter Pilot Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy and Safety of Pegcetacoplan in Patients with Transplant-associated Thrombotic Microangiopathy (TA-TMA) After Hematopoietic Stem Cell Transplantation (HSCT)
This study is for adults over 18 who have developed a serious condition called Transplant-associated Thrombotic Microangiopathy (TA-TMA) after having a stem cell transplant. TA-TMA can cause problems with tiny blood vessels and organs. This study is testing a new medicine called Pegcetacoplan. Researchers want to understand how this medicine moves through the body, how much is needed, and if it helps improve the signs and symptoms of TA-TMA. They will also look at how safe it is and if it leads to better health outcomes for patients. This is an early-stage study, meaning it's one of the first times this medicine is being tested in a group of patients for TA-TMA.
At a glance
What is this study about?
This study is exploring a new medicine called Pegcetacoplan for a condition called Transplant-associated Thrombotic Microangiopathy, or TA-TMA for short. TA-TMA is a serious problem that can sometimes happen after a stem cell transplant. In TA-TMA, tiny blood vessels can get damaged, which can lead to issues with different organs in the body.
The main goal of this study is to find out more about how Pegcetacoplan works in the body of people with TA-TMA. This includes understanding how the body absorbs, uses, and removes the medicine. We'll also be looking closely to see if the medicine is safe and if it helps improve the condition. This is an 'open-label' study, which means both you and your doctors will know you are receiving the study medicine.
We want to see if Pegcetacoplan can help improve the signs of TA-TMA, such as better blood test results and improvements in how patients feel. This includes checking if there are positive changes in things like kidney function, breathing, stomach/bowel problems, and brain-related issues. We'll also be looking at overall health and how long people stay well after treatment. This is a Phase II study, which means it’s an important step to see if the medicine shows promise before it can be tested in larger groups of patients.
Key takeaways
- This study investigates a new medicine, Pegcetacoplan, for TA-TMA after stem cell transplant.
- It aims to understand how the medicine works, if it's safe, and if it helps patients get better.
- Participation involves receiving the medicine and regular health check-ups for up to 24 weeks, plus follow-up.
- The study is for adults aged 18 and over who have been diagnosed with TA-TMA.
- Potential benefits include access to a new treatment; risks include possible side effects.
- You can withdraw from the study at any time without penalty.
Who may be eligible?
To be able to take part in this study, you must be at least 18 years old. This study is open to both men and women.
The most important requirement is that you have been diagnosed with Transplant-associated Thrombotic Microangiopathy (TA-TMA) specifically after having received a stem cell transplant.
There might be other health conditions or medicines that could prevent you from joining, as the researchers need to make sure the study is safe and appropriate for everyone involved. Your doctor will be able to discuss these more detailed criteria with you.
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 had a stem cell transplant?
- Have you been diagnosed with Transplant-associated Thrombotic Microangiopathy (TA-TMA)?
- Are you able to attend regular hospital appointments?
What does participation involve?
If you decide to take part in this study, you will be given the study medicine, Pegcetacoplan. The study will involve regular visits to the hospital or clinic for blood tests and examinations. These tests will help the doctors understand how the medicine is working in your body and if your TA-TMA is improving. They will also keep a close eye on your health to make sure the medicine is safe for you.
Your participation will involve taking the study medication and undergoing various assessments, including blood samples and health reviews, for up to 24 weeks. After this initial treatment period, there will be continued monitoring of your health. The total duration of your involvement could extend beyond 24 weeks for follow-up appointments.
Potential risks and benefits
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Common questions
What is TA-TMA?
TA-TMA is a serious condition that can sometimes happen after a stem cell transplant, causing damage to small blood vessels and organs.
What is Pegcetacoplan?
Pegcetacoplan is the name of the new medicine being tested in this study for people with TA-TMA.
Will I know if I'm getting the study medicine?
Yes, this is an 'open-label' study, which means you and your doctors will know you are receiving Pegcetacoplan.
How long will I be in the study?
The main treatment period is up to 24 weeks, but there will be follow-up appointments after that.
Can I leave the study if I change my mind?
Yes, you can choose to leave the study at any time without affecting your 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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