A Study Evaluating the Safety and Efficacy of Inhaled AP01 in Participants With Progressive Pulmonary Fibrosis
This study is investigating a new inhaled medicine called AP01 for people living with a lung condition called progressive pulmonary fibrosis (PPF). PPF causes scarring in the lungs that gets worse over time. The study will compare AP01, given in two different doses, to a dummy medicine (placebo) to see if it's safe and helps improve or slow down the lung condition. Participants will also continue their usual treatments. It's a 'double-blind' study, meaning neither you nor your doctor will know if you're getting AP01 or the placebo. The study will last for about 52 weeks (one year) and aims to include up to 300 adults.
At a glance
What is this study about?
This study is looking into a new inhaled medicine called AP01. It's designed for people who have progressive pulmonary fibrosis (PPF). Pulmonary fibrosis is a condition where your lungs become scarred, making it harder to breathe. 'Progressive' means the scarring is getting worse over time. Doctors call this an 'interstitial lung disease' or ILD, which covers a group of lung conditions that cause scarring.
The main goal of this study is to find out if AP01 is safe and if it can help people with PPF. Researchers also want to see if it works better than a dummy medicine (called a placebo) when added to the usual care you might already be receiving. The study is called 'double-blind,' which means that neither you nor your study doctor will know whether you are receiving the active AP01 medicine or the placebo. This helps ensure the results are as fair and unbiased as possible. The study will also test two different doses of AP01 to see which one might be more effective.
Over 52 weeks (about one year), the researchers will carefully monitor participants to see how their lung function changes, how they feel, and if they experience any side effects. By comparing the groups who receive AP01 with the group receiving the placebo, they hope to understand if AP01 could be a helpful new treatment option for people with this challenging lung condition.
Key takeaways
- This study is testing a new inhaled medicine (AP01) for progressive pulmonary fibrosis (PPF).
- It aims to see if AP01 is safe and helps slow down lung scarring.
- Participants will receive either AP01 (high or low dose) or a placebo, plus their usual care.
- The study is 'double-blind,' meaning neither you nor your doctor will know which you receive.
- It lasts for about one year and involves regular clinic visits for health checks and tests.
Who may be eligible?
To join this study, you need to be at least 18 years old. The main requirement is that you have been diagnosed with progressive pulmonary fibrosis (PPF). This means your lung scarring is getting worse over time, even with your current treatments. Your doctor will use specific tests, including lung function tests and X-rays or scans of your chest, to confirm that your PPF meets the study's definition of getting worse.
For example, your lung function tests (like FVC, which measures how much air you can breathe out) must show a certain level of decline over the last two years, or your breathing symptoms must have worsened, or scans must show more scarring. There are also specific thresholds for your lung function — for instance, your FVC must be at least 45% of what's expected for someone your age and size, and other lung measurements must also be within certain ranges. Your study doctor will explain all these details and check if you meet these specific health requirements during the screening stage of the study.
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 been diagnosed with progressive pulmonary fibrosis (PPF)?
- Have your lung function tests shown your condition has worsened over the past two years, or have your breathing symptoms or scans gotten worse?
- Are your current lung function tests at or above certain levels set by the study?
What does participation involve?
If you decide to take part, you'll be one of up to 300 people in the study. You will be randomly assigned to one of three groups: one group will receive a high dose of the inhaled medicine AP01, another group a low dose of AP01, and the third group will receive a placebo (a dummy medicine that looks just like AP01 but contains no active drug). You will continue to receive your usual medical care for your lung condition alongside the study medicine.
The study will last for about 52 weeks (one year). During this time, you'll have several visits to the study clinic. At these visits, you'll likely have physical exams, blood tests, lung function tests (like breathing into a machine), and possibly chest scans or X-rays to monitor your lung condition and check for any side effects. The study medicine, whether AP01 or placebo, will be given to you as an inhalation, which you will take at home as instructed. Throughout the study, the research team will be there to support you and answer any questions you have.
Potential risks and benefits
Locations (152)
- University of Alabama at BirminghamVerified postcodeBirmingham, United States· Recruiting
- Mayo Clinic- ScottsdaleVerified postcodeScottsdale, United States· Recruiting
- University of Southern CaliforniaVerified postcodeLos Angeles, United States· Recruiting
- Cedars-SinaiVerified postcodeLos Angeles, United States· Recruiting
- UCLAVerified postcodeLos Angeles, United States· Recruiting
- Newport Native MD, Inc.Verified postcodeNewport Beach, United States· Recruiting
- Paradigm Clinical Research - ReddingVerified postcodeRedding, United States· Recruiting
- University of California - San FranciscoVerified postcodeSan Francisco, United States· Recruiting
- University of Colorado, Anschutz Medical CampusVerified postcodeAurora, United States· Recruiting
- National Jewish HealthVerified postcodeDenver, United States· Recruiting
- UCONN HealthVerified postcodeFarmington, United States· Recruiting
- Yale UniversityVerified postcodeNew Haven, United States· Recruiting
Common questions
What is progressive pulmonary fibrosis?
It's a lung condition where scarring in the lungs gets worse over time, making it harder to breathe. It's also known as progressive fibrotic interstitial lung disease.
What is AP01?
AP01 is a new medicine being tested, given by inhaling it. It contains a drug called pirfenidone, which is sometimes used for lung scarring, but here it's given in a different way.
What does 'placebo-controlled' mean?
It means some participants will receive the active medicine, while others will receive a dummy medicine (placebo) that looks the same but has no drug in it. This helps doctors see if the real medicine is actually working.
How long will I be in the study?
The study will last about 52 weeks, which is roughly one year.
Can I continue my usual lung treatments?
Yes, you will continue your standard medical care for your lung condition alongside the study medicine.
How to find out more
Craig S. Conoscenti, MD
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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