Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency
This research study aims to find out more about the long-term safety of a medicine called Alpha-1 MP. It's for people in the UK who have a genetic lung condition called Alpha-1 Antitrypsin Deficiency (AATD), which can cause emphysema. Participants will receive Alpha-1 MP as a weekly drip (intravenous infusion) for two years. This study is an extension of an earlier trial and is specifically designed to collect more information on safety over a longer period. Doctors will monitor participants closely to check for any side effects and to see how the treatment affects their lungs and overall health. It's important for understanding how best to help people with AATD in the future.
At a glance
What is this study about?
This research is looking at a treatment called Alpha-1 MP for a lung condition known as Alpha-1 Antitrypsin Deficiency (AATD). People with AATD don't have enough of an important protein that protects their lungs, which can lead to damage like emphysema. This study aims to gather more information on how safe Alpha-1 MP is when given over a longer period.
This particular study is an extension of a previous trial. This means that people who took part in the first study, called GTi1201, and meet certain requirements, are invited to continue with this new phase. The main goal is to collect two more years of safety information for Alpha-1 MP, which is given through a drip.
Understanding the long-term safety of treatments like Alpha-1 MP is crucial. It helps doctors and researchers learn more about how to best manage AATD and improve the health and quality of life for people living with this condition. The information gathered here will contribute to a better understanding of this treatment for future patients.
Key takeaways
- This study is for people with Alpha-1 Antitrypsin Deficiency (AATD).
- It aims to understand the long-term safety of Alpha-1 MP treatment.
- Participants will receive weekly Alpha-1 MP infusions for two years.
- The study primarily includes individuals who participated in a previous trial.
- Regular health checks and lung monitoring will be part of the study.
- You can discuss participation and withdrawal with your doctor at any time.
Who may be eligible?
To be considered for this study, you would generally need to be between 20 and 72 years old.
The main requirement is that you have already completed a previous study called GTi1201. There are also specific criteria if your lung function, measured by something called FEV1, showed a certain drop during that earlier study.
There are several reasons why you might not be able to join. These include having certain serious health conditions like severe heart failure or liver scarring, or if you have certain types of cancer. You also can't take part if you're pregnant, breastfeeding, or could become pregnant and aren't using effective contraception. If you have any metal objects in your body that could affect a CT scan, you might also be excluded. Lastly, if you have certain viral infections like Hepatitis or HIV, as shown by recent tests, you wouldn't be able to join.
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 between 20 and 72 years old?
- Did you complete the previous GTi1201 study?
- Are you able to undergo a CT scan?
- Do you have severe heart failure, liver scarring, or certain cancers?
- Are you pregnant, breastfeeding, or unwilling to use effective contraception if you could become pregnant?
- Do you have recent positive tests for certain serious viral infections (e.g., Hepatitis, HIV)?
What does participation involve?
If you join this study, your involvement will last for about two years. The study begins with a screening visit, which for most people will be the same as their final visit from the previous study (GTi1201). Immediately after this, you will start receiving the treatment.
You will have weekly infusions (drips) of Alpha-1 MP into your vein. This will happen for a total of 104 weeks (two years). Throughout this time, the medical team will keep a close eye on your health. They will check for any side effects, review any other medications you are taking, and perform regular physical examinations (though not breast or private area exams). They will also take blood and urine samples and might do pregnancy tests if you are a woman of child-bearing age.
To see how the treatment is affecting your lungs, you will have regular lung function tests, CT scans of your lungs, and carbon monoxide diffusing capacity tests. You'll also be asked to complete questionnaires about your quality of life. The team will also track any flare-ups of your lung condition. After the two years of treatment, you will have a final end-of-study visit.
Potential risks and benefits
Locations (28)
- Grifols Investigative SiteVerified postcodePhoenix, United States
- Grifols Investigative SiteVerified postcodeMiami, United States
- Grifols Investigative SiteVerified postcodeWilmington, United States
- Grifols Investigative SiteVerified postcodePortland, United States
- Grifols Investigative SiteVerified postcodeCharleston, United States
- Grifols Investigative SiteVerified postcodeTyler, United States
- Grifols Investigative SiteVerified postcodeDarlinghurst, Australia
- Grifols Investigative SiteVerified postcodeAdelaide, Australia
- Grifols Investigative SiteVerified postcodeChermside, Australia
- Grifols Investigative SiteVerified postcodeFitzroy, Australia
- Grifols Investigative SiteVerified postcodeNedlands, Australia
- Grifols Investigative SiteVerified postcodeHalifax, Canada
Common questions
What is Alpha-1 Antitrypsin Deficiency (AATD)?
AATD is a genetic condition where your body doesn't make enough of a protein that protects your lungs, which can lead to lung damage like emphysema.
What is Alpha-1 MP?
Alpha-1 MP is a treatment designed to replace the missing protein in people with AATD to help protect their lungs.
How will I receive the treatment?
You will receive Alpha-1 MP through a weekly drip (intravenous infusion) into your vein.
How long will I be in the study?
Your participation will last for approximately two years, including weekly treatments and regular check-ups.
What kind of tests will I have?
You'll have physical exams, blood and urine tests, lung function tests, and CT scans of your lungs to monitor your health and the treatment's effects.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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