Exploratory study to evaluate the safety and tolerability of tamoxifen citrate in the treatment of cystic fibrosis in patients without mutations currently eligible for therapy with CFTR modulator drugs Protocol Code: CRCFC-TAMOXI063
This study is exploring a medicine called TAMOXENE (20 mg tablets) for adults with cystic fibrosis (CF). It's for people whose CF isn't caused by specific gene changes that are usually treated with newer CF medicines. The main goal is to find out if TAMOXENE is safe and what side effects it might cause over 24 weeks. This includes looking at serious side effects and reasons why someone might stop taking the medicine. We'll also be carefully checking for specific problems like blood clots, liver issues, or worsening lung problems. Beyond safety, the study will also look at whether the medicine helps improve breathing, reduces flare-ups or hospital stays, impacts quality of life, or changes other CF symptoms like weight and sweat test results.
At a glance
What is this study about?
This study is testing a medicine called TAMOXENE to see if it can help adults with cystic fibrosis (CF). CF is a condition that mainly affects the lungs, making it hard to breathe, and can affect other parts of the body too. This particular study is for people who have CF but can't be treated with some of the newer, more common CF medicines because their specific genetic mutation (a change in their genes) isn't one that those medicines target.
The main purpose of this study is to make sure TAMOXENE is safe to use for CF patients and to understand any side effects it might cause. Researchers will carefully watch all participants for any health problems that come up during the study, especially serious ones. They will also look specifically for issues that can sometimes be linked to CF or this type of medication, such as blood clots, changes in liver function, or worsening lung symptoms.
Beyond safety, the study also aims to see if TAMOXENE could offer other benefits. Researchers will be checking if it helps people breathe better, reduces the number of times their CF gets worse (called 'exacerbations'), or decreases the need for hospital stays. They'll also look at how it affects everyday life, how much antibiotics people need, changes in weight, and other measures of CF health like sweat test results. This is an early-stage study, meaning it's one of the first times this medicine is being looked at for CF in this group of patients.
Key takeaways
- This study is for adults with cystic fibrosis who can't use other modern CF medicines.
- It's exploring a medicine called TAMOXENE to check its safety and side effects over 24 weeks.
- Researchers will also see if TAMOXENE helps with breathing, flare-ups, and quality of life.
- Participation involves taking pills and having regular health check-ups and tests.
- You can stop participating in the study at any time.
Who may be eligible?
This study is looking for adults aged 18 and over, of any gender. It's specifically for people who have cystic fibrosis but do not have the type of genetic changes that usually qualify them for treatment with some of the newer, advanced CF medicines.
There might be other health requirements or medicines you're currently taking that could affect whether you can join. These details would be discussed with you by the study team if you express interest.
Essentially, if you're an adult with CF and your doctors have told you that you're not suitable for certain modern CF treatments due to your specific genetic makeup, this study might be for you.
- Are you 18 years old or older?
- Do you have cystic fibrosis?
- Have your doctors told you that you don't have the specific genetic mutations that qualify you for modern CF modulator drugs?
- Are you able to take a daily tablet?
- Are you willing to attend regular appointments for 24 weeks?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you join this study, you would be taking TAMOXENE 20 mg tablets. The study will last for 24 weeks, which is about six months. During this time, you would have regular visits with the study team. These visits would involve checking your overall health, discussing any new symptoms or problems, and having tests done.
Tests might include checking your breathing, taking blood samples to look at your liver and other body functions, and other assessments related to CF. Your doctors will also keep track of how many times your CF symptoms worsen or if you need to go to the hospital. You'll be asked about your quality of life and medicine use throughout the study to see how the treatment might be affecting you.
Potential risks and benefits
Locations (1)
- —Italy
Common questions
What is TAMOXENE?
TAMOXENE is a medicine being tested to see if it can help adults with cystic fibrosis who don't qualify for other newer CF treatments.
How long will the study last?
If you join, the study treatment period will be about 24 weeks, which is approximately six months.
What is a 'side effect'?
A side effect is any unwanted or unexpected health problem or change that happens during a study, which might or might not be caused by the medicine being tested.
Will I know if the medicine is working for me?
The study team will monitor many aspects of your health, like your breathing and how often you get sick, and discuss your progress with you throughout the study.
Can I stop participating if I don't like it?
Yes, you can choose to leave the study at any time, for any reason, and your regular medical care will not be affected.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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