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Saracatinib Trial TO Prevent FOP | Clinical Trials | Clareo Health

RECRUITINGPHASE2

Saracatinib Trial TO Prevent FOP

NCT04307953•Amsterdam UMC, location VUmc

Summary

This is a phase 2 study, designed as a European multicentre 6-month double blind random-ized controlled trial (RCT) of AZD0530 versus matched placebo, followed by a 12 month trial comparing open-label extended AZD0530 treatment with historical control data. Study population: Male and female adult patients aged 18 years and older with a diagnosis of FOP who meet the inclusion (active disease) and exclusion criteria will be eligible for participation in this study. The total number of enrolled patients will be 20. Intervention: Patients will be randomized to receive either AZD0530 100mg once daily or matched placebo, taken orally for the first 6 months, immediately followed by an open-label extension in which all patients will receive AZD0530 100mg once daily oral dose for a further 12 months. Endpoints: Endpoints include objective change in heterotopic bone volume measured by low-dose whole-body computer tomography (CT) , \[18F\] NaF Positron Emission Tomography (PET) activity and patient reported outcome measures.

Eligibility

Age

18 - 65 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Male or female aged 18-65 with a clinical diagnosis of FOP at screening, including congenital malformation of the great toes and a history of spontaneous or injury-induced heterotopic ossification (HO), and have a confirmed classic FOP phenotype by the documentation of an ACVR1R206H/+ or variant genomic sequence.
•1. Female participants who are women of child-bearing potential will be required to use a highly effective method of contraception as defined in section 5.4, in combination with a condom or diaphragm or cervical/vault caps with spermicidal foam/gel/film/suppository), from the time of enrolment until 4 weeks after final dose of study drug, unless practicing true sexual abstinence as defined in section 5.4.
•2. Male participants will be required to avoid procreative sexual intercourse with women of child-bearing potential from time of enrollment until 4 weeks after final dose of study drug through use of highly effective contraceptive methods. Male participants with a pregnant female partner will be required to use a condom for the duration of the study and for 4 weeks final dose of study drug. Male study participants will not be permitted to donate sperm for from the time of enrolment and until 4 weeks after final dose of study drug.
•2. Participants will have to be able to understand and complete study and willing to sign informed consent (IC). They have to be able to attend and comply with the study visits and related activities, adhere to all study-related restrictions, and able to undergo procedures such as PET and CT imaging.

Exclusion Criteria

•1. Not willing to strictly adhere to the reproductive restrictions as defined in section 5.4
•2. Women who are pregnant or breast-feeding (from the time 3 months prior to 4 weeks after completion of participation in the study)
•3. The presence of significant concomitant illness or history of significant illness such as cardiac, respiratory, renal, rheumatologic, neurologic, psychiatric, endocrine, metabolic, lymphatic disease, or infectious disease, that might confound the results of the study or pose additional risk to the patient;
•4. Evidence of active bleeding (including hematuria or hematochezia,) acute or chronic gastrointestinal illness, inflammatory bowel disease, or mucositis
•5. Malignant disease / cancer requiring treatment in the past 3 years (except some primary non melanoma skin cancer);
•6. Severely impaired renal function defined as estimated glomerular filtration rate \<30 mL/min/1.73 m2 calculated by the Modification of Diet in Renal Disease equation;
•7. Showing uncontrolled diabetes mellitus with an HbA1C \> 9%;
•8. Significant viral illness or active infections at screening or randomisation; Subjects should not have subacute or acute fevers of \>101 degrees F at time of screening or randomisation
•9. Evidence of prolonged QT interval at screening or randomization (defined as QTc of \>450 ms) .or known congenital long-QT syndrome.
•10. Neutropenia defined as an absolute neutrophil count of \<1,500/µl,
+8 more criteria

Locations (3)

Klinikum Garmish-Partenkirchen

Garmisch-Partenkirchen, Germany

Amsterdam University Medical Center

Amsterdam, Netherlands

Royal National Orthopaedic Hospital

London, United Kingdom

Key Dates

Start Date

August 5, 2020

Primary Completion Date

May 6, 2025

Completion Date

May 6, 2025

Last Updated

May 3, 2024

Enrollment

ESTIMATED participants

Conditions

Fibrodysplasia Ossificans Progressiva

Interventions

AZD0530 Difumarate

DRUG

Matching placebo

DRUG

Contacts

Vincent A. Verheij, MD

CONTACT

+31204444444 v.a.verheij@amsterdamumc.nl

Elisabeth MW Eekhoff, MD, PhD

CONTACT

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COMPLETEDPHASE3

A Rollover Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged ≥14 Years With Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed the Relevant Parent Studies.

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: May 3, 2024Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Saracatinib Trial TO Prevent FOP

Inclusion Criteria

Exclusion Criteria

A Study to Assess Safety, Tolerability and Efficacy of Garetosmab Versus Placebo Administered Intravenously (IV) in Adult Participants With Fibrodysplasia Ossificans Progressiva (FOP)

A Rollover Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged ≥14 Years With Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed the Relevant Parent Studies.

An International Cross-sectional Survey to Evaluate the Burden of Fibrodysplasia Ossificans Progressiva (FOP) on Patients and Their Families.

Urine Sample Collection From FOP Patients