To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva
This research study is focused on Fibrodysplasia Ossificans Progressiva (FOP), a very rare condition where soft tissues turn into bone over time. The study is testing a new drug, INCB000928, to see if it can help slow down or stop this process. We will also check if the medication is safe to use and how well people tolerate it. This is a "Phase 2" study, meaning we are exploring its effectiveness and safety in more detail after initial tests. Participants will be given either the new drug or a placebo (a dummy pill with no medicine) in a way that neither they nor the doctors know who gets what. This helps us accurately compare the real effects of the drug. The information gained will be vital for developing future treatments for FOP.
At a glance
What is this study about?
This study is all about understanding and potentially treating Fibrodysplasia Ossificans Progressiva (FOP). FOP is a very rare genetic condition that causes muscle and other connective tissues to gradually turn into bone, leading to a permanent loss of movement. It's a challenging condition, and currently, there aren't many effective treatments available.
Researchers are testing a new medication called INCB000928. The main goal is to see if this drug can help people with FOP. We want to find out if it can reduce the formation of new bone, ease symptoms, and generally improve the lives of those affected. We will also carefully check if the medication is safe to use and if people can take it without too many unwanted side effects. This kind of research is essential for finding better ways to manage and potentially even cure FOP in the future.
This specific study is called a "Phase 2" trial. This means we're moving past the very first safety checks and are now looking more closely at how well the drug works and its overall safety in a larger group of people. Participants will be randomly assigned to receive either the new drug or a placebo – a tablet that looks the same but contains no active medicine. This 'blinded' approach helps us get the most accurate results by preventing anyone's expectations from affecting the outcome. The study is a crucial step in bringing new hope to the FOP community.
Key takeaways
- Tests a new drug (INCB000928) for Fibrodysplasia Ossificans Progressiva (FOP).
- Aims to check if the drug is effective and safe.
- Compares the new drug to a placebo (dummy pill).
- Open to both adults and children aged 2 and up.
- Involves regular clinic visits, blood tests, and special body scans.
- Your participation helps advance FOP research, but no guaranteed personal benefit.
Who may be eligible?
To join this study, you need to have a confirmed diagnosis of Fibrodysplasia Ossificans Progressiva (FOP). We're looking for both males and females. The study includes different age groups, starting from children as young as 2 years old, up to adults aged 99.
There are also some important safety considerations. For instance, you shouldn't be pregnant or breastfeeding. If you're able to have children, or capable of fathering them, you'll need to agree to use effective birth control throughout the study. Your FOP symptoms should also be at a level that allows you to safely take part in the study and undergo necessary scans.
There are certain health conditions or situations that would prevent you from joining, such as having certain infections like HIV, Hepatitis B, or C. The study team will review all your medical information carefully to make sure it's safe and appropriate for you to participate.
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.
- Do I have a diagnosis of FOP?
- Am I aged between 2 and 99 years old?
- Am I willing to use birth control if I can have children?
- Can I have body scans without needing to be put to sleep?
- Do I have any serious infections like HIV or Hepatitis?
What does participation involve?
Taking part in this study would involve regular visits to the clinic for check-ups and assessments. You would be given either the new study medication, INCB000928, or a placebo (a tablet with no active medicine) to take regularly, as instructed by the study team. Neither you nor your study doctor will know which one you are receiving – this is to ensure the results are as fair and accurate as possible.
Throughout the study, you'll have specific tests, including low-dose body scans (CT scans) that help us see any changes in your bones, but these won't involve your head. You'll also have blood tests and other examinations to monitor your health and how the medication is affecting you. We won't ask you to be put to sleep (intubated) for any of these scans. The total duration of your involvement in the study will be explained in detail by the study team, including how long you'll be taking the medication and how many follow-up appointments you'll have.
Potential risks and benefits
Locations (24)
- Mayo Clinic RochesterVerified postcodeRochester, United States· Recruiting
- Children'S Hospital of PhiladelphiaVerified postcodePhiladelphia, United States· Recruiting
- Penn Medicine - Perelman Center For Advanced MedicineVerified postcodePhiladelphia, United States· Active not recruiting
- Hospital Italiano de Buenos AiresVerified postcodeBuenos Aires, Argentina· Active not recruiting
- Royal North Shore HospitalVerified postcodeSt Leonards, Australia· Completed
- Murdoch Children'S Research InstituteVerified postcodeParkville, Australia· Active not recruiting
- Albert Einstein Israelite HospitalVerified postcodeSão Paulo, Brazil· Active not recruiting
- University Health Network Toronto General HospitalVerified postcodeToronto, Canada· Active not recruiting
- Centro de Estudios ReumatologicosVerified postcodeSantiago, Chile· Active not recruiting
- Beijing Childrens Hospital Capital Medical UniversityVerified postcodeBeijing, China· Recruiting
- Tongji Hospital of Tongji UniversityVerified postcodeShanghai, China· Recruiting
- Shanghai Childrens Medical CenterVerified postcodeShanghai, China· Recruiting
Common questions
What is FOP?
FOP (Fibrodysplasia Ossificans Progressiva) is a rare condition where soft tissues like muscles and tendons gradually turn into bone, leading to movement problems.
What is INCB000928?
INCB000928 is a new medication being tested to see if it can help slow down or stop the extra bone formation in people with FOP.
Will I get the real medicine or a dummy pill?
You will be randomly given either the new medicine or a placebo (a dummy pill), and neither you nor your doctor will know which one you are receiving.
What is a 'Phase 2' study?
A Phase 2 study checks how well a new medicine works and continues to monitor its safety after initial testing in smaller groups.
Can children join this study?
Yes, children from 2 years old up to adults are included in different age groups within the study.
How to find out more
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Always speak to your GP or specialist before deciding to take part in a study.
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