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

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What is this study about?

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.

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 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 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 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 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 Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name City Country Status
Krahbpzq Geubdmrznlglopysvkkuvz Grnm Garmisch-Partenkirchen Germany
Aylfubnpu Uri Amsterdam The Netherlands

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Germany Germany
Not recruiting
01.11.2019
The Netherlands The Netherlands
Not recruiting
01.11.2019

Trial locations

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.

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 ID:
2024-515186-33-00
Protocol code:
STOPFOP1
NCT ID:
NCT04307953
Trial Phase:
Therapeutic exploratory (Phase II)

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