Study of the Efficacy and Safety of Inhaled Treprostinil in Subjects With Progressive Pulmonary Fibrosis (TETON-PPF)
This study, called TETON-PPF, is looking into a new inhaled medicine called treprostinil for people who have progressive pulmonary fibrosis (PPF) or other types of interstitial lung disease that are getting worse. PPF is a serious lung condition where lung tissue becomes scarred, making it harder to breathe. The main goal is to find out if this medicine is both safe and works well to help people with PPF over a 52-week period. Participants will be randomly divided: half will receive the active inhaled medicine, and the other half will receive a placebo (a dummy treatment). This helps researchers fairly compare how well the new medicine works. The study will monitor lung function, symptoms, and overall health.
At a glance
What is this study about?
This study, known as TETON-PPF, is focusing on a lung condition called progressive pulmonary fibrosis (PPF). Fibrosis means scarring, and in PPF, the lung tissue becomes scarred and stiff over time, making it increasingly difficult to breathe. This study is for people whose lung disease is generally getting worse despite existing treatments. The researchers want to see if an inhaled medicine called treprostinil can help. This medicine is given through a special device that turns it into a fine mist you breathe in.
The study aims to understand two main things: first, if the inhaled treprostinil is safe for people with PPF, and second, if it's effective in slowing down the progression of the disease or improving symptoms. To do this fairly, some participants will receive the active medicine, while others will receive a placebo – a treatment that looks and feels the same but contains no active medicine. This is a common way to test new treatments, as it helps doctors accurately judge the real effects of the medicine.
This kind of research is really important because it could lead to new ways to help people living with progressive pulmonary fibrosis. By taking part, volunteers contribute to medical knowledge that could ultimately improve treatment options for many others in the future.
Key takeaways
- Tests an inhaled medicine for progressive lung scarring.
- Compares active medicine to a dummy treatment (placebo).
- Study lasts for 52 weeks with regular clinic visits.
- Aims to check safety and effectiveness for breathing and lung function.
- Participation is voluntary; you can withdraw at any time.
Who may be eligible?
To join this study, you need to be at least 18 years old. You must have had a recent scan (within the last year) that shows signs of lung scarring affecting more than 10% of your lungs. Most importantly, your doctors must confirm you have progressive pulmonary fibrosis, which means your lung condition has been getting worse in the past two years, even with standard treatments.
Your doctors will look for specific signs that your condition is progressing, such as a noticeable drop in your breathing test results (FVC), worsening breathing difficulties combined with a smaller drop in FVC, or if scans show more scarring along with breathing problems. Your lung function also needs to be at a certain level (at least 45% predicted FVC) at the start of the study.
Crucially, you should either be taking existing fibrosis medicines like nintedanib or pirfenidone for at least 90 days and plan to continue, or not be taking them at all and not planning to start them during the study. If you're on other medications that affect your immune system, you'll generally need to have been on them for at least 120 days and your doctors must believe your condition is not responding well to them. There are also specific requirements for women who could become pregnant.
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 or older?
- Do you have a diagnosis of progressive pulmonary fibrosis (PPF)?
- Has your lung condition been getting worse in the last two years despite treatment?
- Is your lung function (FVC) at least 45% based on a recent test?
- Are you established on your current fibrosis medication or not planning to start new ones?
What does participation involve?
If you join the study, you'll be randomly assigned to either receive the inhaled treprostinil or a placebo (a dummy medicine). You'll start by breathing in the study medicine four times a day, taking 3 breaths each time. Over time, your dose will be gradually increased, if you tolerate it well, up to a target of 12 breaths, four times a day. You will use a special nebulizer device to take the medicine.
Over the course of 52 weeks, you'll need to attend 6 visits at the clinic. These will be at weeks 4, 8, 16, 28, 40, and 52. During these visits, the study team will carry out various tests to check your lung function (like spirometry and lung diffusion capacity), ask about your symptoms and quality of life using questionnaires, and take blood samples. They will also monitor your vital signs, check for any side effects, and perform ECGs (heart traces).
After completing the 52-week study, you might have the option to continue receiving the treprostinil medicine in an 'open-label' extension study, where everyone receives the active drug.
Potential risks and benefits
Locations (159)
- UAB Lung Health CenterVerified postcodeBirmingham, United States· Recruiting
- Banner University Medical Center Phoenix Lung InstituteVerified postcodePhoenix, United States· Recruiting
- Norton Thoracic InstituteVerified postcodePhoenix, United States· Recruiting
- Peter Morton Medical BuildingVerified postcodeLos Angeles, United States· Recruiting
- NewportNativeMD, Inc.Verified postcodeNewport Beach, United States· Recruiting
- University of California Irvine Medical CenterVerified postcodeOrange, United States· Recruiting
- Paradigm Clinical ResearchVerified postcodeRedding, United States· Recruiting
- UC Davis Health Medical CenterVerified postcodeSacramento, United States· Recruiting
- Paradigm Clinical ResearchVerified postcodeSan Diego, United States· Recruiting
- Stanford University Medical CenterVerified postcodeStanford, United States· Recruiting
- Georgetown University HospitalVerified postcodeWashington D.C., United States· Recruiting
- Ascension Medical Group St. Vincent's Lung InstituteVerified postcodeJacksonville, United States· Recruiting
Common questions
What is progressive pulmonary fibrosis?
It's a serious lung condition where lung tissue becomes scarred, making it progressively harder to breathe over time.
What is 'inhaled treprostinil'?
It's a study medicine that you breathe in through a special device, aiming to help manage progressive pulmonary fibrosis.
What is a 'placebo'?
A placebo is a dummy treatment that looks and feels like the real medicine but has no active ingredients. It's used to compare against the active drug.
How long will the study last?
The main part of the study will last for 52 weeks, which is about one year. You may then have an option to continue in an extension study.
Will I know if I'm getting the real medicine or the placebo?
No, this is a 'double-blind' study, meaning neither you nor your study doctors will know whether you're receiving the active medicine or the placebo.
How to find out more
United Therapeutics Global Medical Information
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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