A Phase 2 study to assess the efficacy and safety of 2 dosage regimens of oral fidrisertib (IPN60130) for the treatment of fibrodysplasia ossificans progressiva in male and female paediatric and adult participants.
This research study is looking into a new medication called fidrisertib (also known as IPN60130) for people, both young and old, who have a rare condition called Fibrodysplasia Ossificans Progressiva (FOP). FOP causes extra bone to grow in soft tissues, which can be very painful and limit movement. The main goal is to find out if this new medicine can help slow down or stop this extra bone from forming, and if it can make flare-ups happen less often. Researchers will also be checking carefully to make sure the medicine is safe. Some people in the study will be given the actual medicine, while others will receive a dummy pill, called a placebo, to compare the effects.
At a glance
What is this study about?
This study is called a 'Phase 2' trial. This means that the new medicine, fidrisertib, has already been tested in a small number of people and found to be generally safe. Now, we're looking to see if it actually works for people with Fibrodysplasia Ossificans Progressiva (FOP) and to understand the best dose to use. FOP is a very rare condition where muscles, tendons, and ligaments gradually turn into bone, which can cause pain and severe limitations in movement.
The main aim of this study is to see if fidrisertib can help reduce the amount of new bone that forms in people with FOP over a year. We'll be using special low-dose X-rays to measure any changes. We also want to see if the medicine can reduce how often people experience painful flare-ups and how long they last. Safety is always a top priority, so we'll be closely monitoring for any side effects from the medicine.
By comparing participants who receive fidrisertib with those who receive a dummy pill (placebo), we can get a clearer picture of the medicine's effects. This research is incredibly important because it could lead to new ways to manage FOP and improve the lives of those living with this challenging condition.
Key takeaways
- This study tests a new oral medicine (fidrisertib) for Fibrodysplasia Ossificans Progressiva (FOP).
- It aims to see if the medicine can reduce new bone growth and lessen flare-ups.
- Participants will receive either fidrisertib or a placebo (dummy pill).
- The study involves regular clinic visits, physical exams, blood tests, and X-rays over 12 months.
- It's a Phase 2 study, meaning the medicine's effectiveness and safety are being further investigated.
- Participants must be 18 years or older with an FOP diagnosis.
Who may be eligible?
This study is open to both men and women, ranging from 18 years old up to any age, who have been diagnosed with Fibrodysplasia Ossificans Progressiva (FOP). The study team will need to review your medical history to make sure that taking part would be safe and appropriate for you.
Because this is a research study and safety is key, there may be certain medical conditions or medications that would prevent someone from joining. For example, conditions that might interact with the study drug, or other health issues that could make it difficult to accurately measure the drug's effects, might mean someone isn't eligible.
If you're interested, the best first step is to talk to your doctor. They can help you understand if your specific health situation aligns with the study's requirements and can also discuss any questions you might have about FOP treatments in general.
- I have been diagnosed with Fibrodysplasia Ossificans Progressiva (FOP).
- I am 18 years old or older.
- I am able to visit the clinic for regular check-ups and tests.
- I am willing to take a study capsule every day for 12 months.
- I am willing to undergo regular X-rays and have blood/urine samples taken.
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part in this study, you would be asked to take a capsule by mouth every day. You'll either receive the active study medicine, fidrisertib, or a placebo (a dummy pill that looks just like the study medicine but contains no active drug). You won't know which one you're taking, and neither will the study team at first. This helps us get accurate results.
Throughout the study, you'll have regular visits to the clinic for various checks. These will include physical exams, checking your vital signs (like blood pressure and heart rate), measuring your weight and height, and taking blood and urine samples for lab tests. You'll also have special low-dose X-ray scans (called WBCT) to look at any changes in bone growth. For women who could become pregnant, regular pregnancy tests will be part of the routine. We'll also ask about any other medicines you're taking and if you've had any new health problems or flare-ups. The study will last for a total of 12 months.
Potential risks and benefits
Locations (8)
- —Sweden
- —Germany
- —France
- —Netherlands
- —Spain
- —Belgium
- —Italy
- —Portugal
Common questions
What is Fibrodysplasia Ossificans Progressiva (FOP)?
FOP is a very rare genetic disorder where soft tissues, like muscles and tendons, gradually turn into bone, causing pain and restricted movement.
What is a 'placebo'?
A placebo is a dummy pill that looks exactly like the study medicine but doesn't contain any active drug. It helps researchers compare the effects of the real medicine.
Will I know if I'm getting the real medicine or the placebo?
No, neither you nor the study team will know at first. This is called 'blinding' and helps ensure the study results are as fair and accurate as possible.
How long will I need to be in the study?
If you decide to join, you will be part of the study for about 12 months, which includes regular check-ups and tests.
What are flare-ups?
In FOP, flare-ups are episodes where a sudden new area of inflammation can appear, often leading to rapid new bone formation and pain.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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