Active not recruitingPHASE2INTERVENTIONAL

Saracatinib Trial TO Prevent FOP

This research study, called the Saracatinib Trial, is looking into a new medication (AZD0530) for a rare condition called Fibrodysplasia Ossificans Progressiva, or FOP. FOP causes extra bone to grow in soft tissues like muscles, tendons, and ligaments, making movement difficult. The study wants to find out if this new drug can help stop this extra bone growth. It involves 20 adults in Europe who have FOP. For the first six months, some participants will receive the drug and others will receive a dummy pill (placebo). Then, all participants will receive the active drug for another year. Researchers will use special scans to measure any changes in bone growth.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Amsterdam UMC, location VUmc

Enrolment target

Start

05 Aug 2020

Estimated completion

30 Nov 2026

Fibrodysplasia Ossificans Progressiva

What is this study about?

This study is called the 'Saracatinib Trial TO Prevent FOP'. FOP stands for Fibrodysplasia Ossificans Progressiva, which is a very rare genetic condition. If you have FOP, your body mistakenly grows extra bone in places where it shouldn't, like in your muscles, tendons, and ligaments. This extra bone can cause pain, stiffness, and make it hard to move.

The main goal of this study is to see if a new drug, called Saracatinib (also known as AZD0530), can help stop new bone from forming in people with FOP. Currently, there isn't a cure for FOP, so finding treatments that can slow down or prevent this extra bone growth is very important for improving the lives of people living with the condition.

This is a 'Phase 2' study, which means it's an early stage of testing. The researchers are looking at how safe the drug is and if it shows promise in preventing new bone growth. This trial is being run in several hospitals across Europe, and it's a 'controlled' study, meaning some people will get the drug and some will get a placebo (a dummy pill) to fairly compare the effects.

Key takeaways

This study is testing a new drug (Saracatinib/AZD0530) for Fibrodysplasia Ossificans Progressiva (FOP).
It aims to see if the drug can prevent new bone from growing in soft tissues.
The study involves 20 adults with FOP, aged 18-65.
Participants will take a pill daily for 18 months (initially drug or placebo, then drug for everyone).
Regular hospital visits and special scans (CT/PET) are required.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To be considered for this study, you must be an adult with FOP, aged between 18 and 65 years old. You need to have been officially diagnosed with FOP, including having the specific foot malformations often seen in FOP and a history of extra bone growth, confirmed by a genetic test.

It's very important that you can understand what the study involves and are happy to give your permission to take part. You must also be able to attend all the study appointments, follow the study instructions, and be able to have the special scans (like CT and PET scans) that are part of the research.

There are also some reasons why you wouldn't be able to join. For example, if you are pregnant or breastfeeding, or if you have certain other serious health problems affecting your heart, lungs, kidneys, or mental health. You also must be willing to follow strict rules about contraception during the study, both for men and women, to prevent pregnancy.

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 aged between 18 and 65 years old?
Do you have a confirmed diagnosis of FOP, including typical toe changes and a history of extra bone growth?
Are you able to attend regular hospital visits and have special scans (CT/PET)?
Are you able and willing to use highly effective contraception if applicable, and follow all study rules?
Are you currently not pregnant or breastfeeding?
Do you not have any other serious health conditions that would prevent your participation?

Answer every question to see your result.

What does participation involve?

If you join this study, it will last for 18 months. For the first six months, you will take a pill once a day. You'll either get the study drug (AZD0530) or a matching dummy pill (placebo). Neither you nor your study doctor will know which you are receiving during this time. After these first six months, everyone in the study will then receive the actual drug (AZD0530) in a pill form, once a day, for another 12 months.

Throughout the study, you'll need to attend regular hospital visits. These visits will involve various checks, including physical examinations, blood tests, and special scans. The scans, called low-dose CT and PET scans, are important to measure any changes in bone growth. These tests help the researchers understand how the drug is working. You'll need to commit to attending all these appointments and following the study's instructions carefully.

Potential risks and benefits

Participating in a clinical trial like this could potentially offer some benefits, such as early access to a new experimental treatment that might help slow down your FOP. You would also be contributing valuable information that could help others with FOP in the future. However, there are also potential risks, as the drug is still being tested and its full effects are not yet known. Side effects could occur, and some procedures like scans involve radiation, though at low doses. You will be closely monitored by medical staff throughout the study. It's important to remember that you are free to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (3)

Klinikum Garmish-Partenkirchen
Verified postcode
Garmisch-Partenkirchen, Germany
Amsterdam University Medical Center
Verified postcode
Amsterdam, Netherlands
Royal National Orthopaedic Hospital
Verified postcode
London, United Kingdom

Common questions

What is FOP?

FOP, or Fibrodysplasia Ossificans Progressiva, is a rare condition where extra bone grows in soft tissues like muscles, making movement difficult.

What is the new drug being tested?

The new drug is called Saracatinib, also known as AZD0530. It's hoped to help prevent new bone growth in FOP.

Will I definitely get the actual drug?

For the first 6 months, you might get the drug or a dummy pill. After that, everyone will receive the active drug for a further 12 months.

How long will the study last?

The study will last for a total of 18 months, with regular hospital visits during that time.

What does 'Phase 2' mean?

Phase 2 means it's an early stage study to check how safe the drug is and if it shows promise in treating the condition.