Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruitingRare disease

Study on the Safety and Effects of Saracatinib for Patients with Fibrodysplasia Ossificans Progressiva (FOP)

Verified siteInvestigational
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare condition called Fibrodysplasia Ossificans Progressiva (FOP). FOP is a disorder where soft tissues, like muscles and tendons, gradually turn into bone, leading to restricted movement. The trial is testing a research medication known as AZD0530, also called Saracatinib, which is taken as a film-coated tablet. The purpose of the study is to evaluate the safety and effects of Saracatinib on abnormal bone formation in patients with FOP.

Participants in the study will receive either Saracatinib or a placebo, which looks like the medication but does not contain the active ingredient. The study will last for a period of 18 months, during which participants will be monitored for any new bone growth and any side effects that may occur. The study will include regular check-ups and imaging tests, such as PET and CT scans, to assess the development of new bone lesions and the overall impact of the treatment.

The trial aims to provide valuable information on how Saracatinib affects the progression of FOP and its potential to prevent new bone formation. By participating in this study, researchers hope to gain insights that could lead to better treatment options for individuals living with this challenging condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial enrollment

    Upon joining the study, participants are required to provide informed consent, confirming their understanding and willingness to comply with study procedures and restrictions.

    Participants must meet specific criteria, including age between 18-65, a clinical diagnosis of Fibrodysplasia Ossificans Progressiva (FOP), and the ability to attend study visits and complete related activities.

  2. Step 2

    Randomized controlled trial (RCT) phase

    Participants are randomly assigned to receive either the research medication AZD0530 (saracatinib) or a placebo. The medication is administered orally in the form of film-coated tablets.

    The primary goal during this phase is to assess the safety and effectiveness of AZD0530 in preventing new bone formation. This phase lasts for 26 weeks.

  3. Step 3

    Safety and efficacy assessment

    Throughout the RCT phase, the incidence and severity of any adverse events are monitored. The number of new bone lesions is evaluated using imaging techniques such as PET and CT scans.

    Participants undergo assessments to measure changes in functional mobility, including joint involvement, mouth opening, and lung capacity.

  4. Step 4

    Open-label extension phase

    After completing the RCT phase, participants enter a 6-month open-label extension where all receive AZD0530.

    The focus is on monitoring the continued safety and effectiveness of the medication, as well as any changes in bone lesions and functional mobility.

  5. Step 5

    Long-term follow-up

    Participants are followed up at weeks 52, 104, and 156 to assess long-term outcomes. This includes evaluating changes in bone lesions and functional mobility compared to baseline measurements.

    The study aims to gather data on the progression of FOP in treated versus untreated populations.

Who can join the trial?

9 criteria

  • Participants must be **male or female** and aged **18-65**.
  • Participants must have a **clinical diagnosis** of **Fibrodyplasia Ossificans Progressiva (FOP)**. This includes having a **congenital malformation** (a condition present from birth) of the **great toes** and a history of **heterotopic ossification (HO)**, which is when bone forms in places it shouldn't, like muscles or other soft tissues.
  • Participants must have a confirmed **classic-like FOP phenotype**. This means they have a specific genetic sequence known as **ACVR1R206H/+ or variant**.
  • Female participants who can have children must use a **highly effective method of contraception** (birth control) along with a **condom** or other barrier methods like a **diaphragm** or **cervical/vault caps** with **spermicidal foam/gel/film/suppository**. This is required from the time they join the study until **4 weeks after** the last dose of the study drug, unless they are practicing **true sexual abstinence**.
  • Male participants must avoid having sexual intercourse that could lead to pregnancy with women who can have children. They must use **highly effective contraceptive methods** from the time they join the study until **4 weeks after** the last dose of the study drug.
  • Male participants with a pregnant partner must use a **condom** during the study and for **4 weeks after** the last dose of the study drug.
  • Male participants are not allowed to **donate sperm** from the time they join the study until **4 weeks after** the last dose of the study drug.
  • Participants must be able to **understand and complete** the study and be willing to **sign informed consent (IC)**, which means they agree to participate after understanding the study details.
  • Participants must be able to **attend and comply** with study visits and related activities, follow all study-related rules, and be able to undergo procedures like **PET and CT imaging**. These are special scans that help doctors see inside the body.

Who cannot join the trial?

3 criteria

  • Patients who are not diagnosed with Fibrodyplasia Ossificans Progressiva (FOP) cannot participate. FOP is a rare condition where soft tissues turn into bone over time.
  • Patients who are younger than 3 years old cannot participate.
  • Patients who are part of a vulnerable population, such as those who cannot give consent or are in a dependent relationship, cannot participate.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

  • Amsterdam UMC

    Responsive

    The NetherlandsAmsterdam, The Netherlands

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

AZD0530 is a medication being studied for its potential to prevent the formation of heterotopic ossification (HO), which is an abnormal growth of bone in non-skeletal tissues. The trial aims to evaluate how effective this medication is in stopping or reducing this unwanted bone formation.

What is already known about the treatment

Saracatinib – Saracatinib is administered orally in the form of tablets. It is currently being studied in clinical trials for its potential to treat Fibrodyplasia Ossificans Progressiva (FOP), a rare and disabling condition. The main therapeutic indication for Saracatinib in this context is to prevent the formation of heterotopic ossification (HO), which is abnormal bone growth in muscles and other tissues. At the molecular level, Saracatinib works by inhibiting specific enzymes known as Src kinases, which play a role in the signaling pathways that lead to abnormal bone formation. It is classified pharmacologically as a tyrosine kinase inhibitor.

Investigated diseases

Fibrodysplasia Ossificans Progressiva (FOP) – This is a rare genetic disorder characterized by the abnormal development of bone in areas where bone should not form, such as muscles, tendons, and other connective tissues. The condition progresses through episodes of soft tissue swelling, which eventually transform into bone, leading to joint stiffness and restricted movement. Over time, these episodes can result in the formation of a second skeleton, severely limiting mobility. The progression of FOP is unpredictable, with flare-ups often triggered by trauma, viral illnesses, or even spontaneous events. As the disease advances, individuals may experience increasing difficulty with daily activities due to the ossification of soft tissues.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-515186-33-00Protocol codeSTOPFOP1Estimated enrolment18 patientsSponsorAmsterdam UMC Stichting

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.