A Phase 2/3, Multicenter, randOmized, Double-blind, placebo-controlled, stUdy to evaLuate the safety and efficacy of Alpha-1 AntiTrypsin for the prEvention of graft versus-host disease in patients receiving hematopoietic cell transplant (MODULAATE Study)
This study, called MODULAATE, is investigating a medicine named Alpha-1 AntiTrypsin (Respreeza) to help patients who are having a stem cell transplant. The main goal is to see if this medicine can prevent a serious complication called acute graft-versus-host disease (GVHD), or at least reduce its severity. GVHD happens when the new immune cells from the donor attack the patient's body. Researchers will also be checking the safety of the medicine and how well patients recover after their transplant. This includes looking at infections, how long they need other treatments, and if the cancer comes back. We want to find better ways to support patients through this challenging treatment.
At a glance
What is this study about?
When someone has a stem cell transplant, they receive healthy new cells from a donor. While this can be life-saving, sometimes the donor cells can see the patient's body as 'foreign' and start to attack it. This serious condition is called graft-versus-host disease (GVHD). It can affect different parts of the body, like the skin, gut, or liver, and can be very uncomfortable or even life-threatening.
This study, called MODULAATE, is looking at a medicine called Alpha-1 AntiTrypsin, also known as Respreeza, to see if it can help prevent or lessen the severity of GVHD. Researchers believe this medicine might have properties that could calm down the immune system and stop it from attacking the patient’s healthy tissues. Participants will either receive this new medicine or a dummy treatment (placebo), alongside their usual care, to see if Respreeza makes a difference.
The main aim is to find out if taking Alpha-1 AntiTrypsin can reduce the chance of developing moderate to severe GVHD, or reduce its impact, in the first six months after a stem cell transplant. The study will also carefully monitor patients for any side effects and track their overall recovery, including things like infections and how long they need other medications. The hope is that this research will lead to better ways to protect patients from GVHD and improve their outcomes after a stem cell transplant.
Key takeaways
- This study investigates a new medicine (Respreeza) to prevent graft-versus-host disease (GVHD) after stem cell transplants.
- GVHD is a serious complication where donor cells attack the patient's body.
- Participants will receive either the study medicine or a placebo.
- The study will monitor safety and effectiveness for up to two years.
- Any adult undergoing a stem cell transplant might be eligible.
- The aim is to improve safety and recovery for transplant patients.
Who may be eligible?
To take part in this study, you generally need to be an adult, 18 years old or older. Both men and women can join. The most important requirement is that you are about to have, or have recently had, a stem cell transplant.
There are also other medical details that the study doctors will check to make sure the study is right and safe for you. For example, they will need to review your overall health, any other medical conditions you have, and the specific type of transplant you are receiving.
If you are interested, your transplant doctor will be able to tell you if you meet all the necessary requirements for this particular study. They can explain all the details and answer any questions you have.
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?
- Are you scheduled for or have recently had a stem cell transplant?
- Are you able to attend regular follow-up appointments?
- Are you comfortable with the possibility of receiving a placebo?
What does participation involve?
If you join this study, you would either receive the study medicine, Alpha-1 AntiTrypsin, or a dummy treatment called a placebo. The study medicine is given intravenously, meaning through a drip into your arm. You will have regular appointments and tests over a period of up to two years after your stem cell transplant. These visits will allow the medical team to closely monitor your health, check for any signs of GVHD, and look for any side effects from the treatment.
These tests may include blood samples, physical examinations, and possibly other procedures to assess how you are recovering and how the treatment is affecting you. The total duration of your active participation in the study, where we closely monitor your health, can last for up to two years, though some assessments might focus on the first few months after your transplant.
Potential risks and benefits
Locations (3)
- —UnverifiedSpain
- —UnverifiedItaly
- —UnverifiedGermany
Common questions
What is graft-versus-host disease (GVHD)?
GVHD is a serious problem after a stem cell transplant where the donor's immune cells attack the patient's body.
What is Alpha-1 AntiTrypsin (Respreeza)?
It's a medicine being tested in this study to see if it can help prevent GVHD.
Will I definitely get the new medicine?
No, you might receive the study medicine or a dummy treatment (placebo). This is decided by chance.
How long does the study last?
Your involvement in the study could last for up to two years after your stem cell transplant.
Can I stop being in the study if I want to?
Yes, you are free to stop participating at any time 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.
Discussion
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