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NCT Number: NCT02322255

A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP)

Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in abnormal bone formation in muscles, tendons, and ligaments. Flare-ups begin early in life and may occur spontaneously or after soft tissue trauma, vaccinations, or influenza infections. Recurrent flare-ups progressively restrict movement by locking joints leading to cumulative loss of function and disability. This 3-year, non-interventional, two-part, natural history study is designed to gain insight into total body HO, FOP disease progression, the impact of FOP on subjects' physical functioning, and clinical features and biomarkers that may be useful in the diagnosis and monitoring of disease progression. This natural history study will also provide important information to inform the design of subsequent interventional trials.

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Key information

Conditions

Fibrodysplasia Ossificans Progressiva Muscular Diseases Musculoskeletal Diseases Myositis Myositis Ossificans Ossification, Heterotopic Pathologic Processes Pathological Conditions, Signs and Symptoms

Age range

Up to 65 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Hospital Italiano de Buenos Aires, Department of Pediatrics, Buenos Aires, Argentina

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About this study

This is a multi-center, natural history, non-interventional, longitudinal study in subjects with classic FOP. A thorough baseline examination will be performed to determine the current status of disease in each subject. In Part A, two imaging modalities assessed total body HO at baseline, and the optimal method (low-dose whole body CT scan [excluding head]) will be employed in Part B for the balance of the study. Progression will be assessed at annual in-clinic visits (ie, at Months 12, 24, and 36) at which time the procedures conducted at the baseline visit will be repeated. In addition, site personnel will telephone subjects midway between the annual visits (ie, at Months 6, 18, and 30).

During the 36-month follow-up period, at least one new flare-up (with a maximum of one per year) will be carefully studied. An in-clinic visit will be performed within 14 days following the subject's identification of his/her flare-up. Additional visits at Day 42 and Day 84 (after the initial flare-up clinic visit) will be performed. An additional future visit may be scheduled after Day 84 at the discretion of the Principal Investigator (PI) for prolonged flare-ups. However, subjects with an eligible flare-up may elect to participate in an ongoing Clementia interventional study rather than continue in this natural history study.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Subjects clinically diagnosed with classical FOP with documented R206H mutation or believed to carry the R206H mutation

Exclusion criteria

  • Participation in an interventional clinical research study within the 4 weeks prior to enrollment

Treatment and study plan

Primary outcomes

  1. Change from baseline in the total body burden of heterotopic ossification as assessed by the optimal imaging modality (low-dose whole body CT [excluding head]).

    Time frame: Month 36

Secondary outcomes

  1. Change from baseline in physical function as assessed by range of motion.

    Time frame: Month 12, Month 24, and Month 36

  2. Change from baseline in patient-reported use of assistive devices and adaptations.

    Time frame: Month 6, Month 12, Month 18, Month 24, Month 30, and Month 36

  3. Change from baseline in a disease-specific patient-reported outcome measure (FOP-Physical Function Questionnaire [FOP-PFQ]).

    Time frame: Month 6, Month 12, Month 18, Month 24, Month 30, and Month 36

  4. Change from baseline in a patient-reported measure of physical and mental health (PROMIS Global Health Scale).

    Time frame: Month 6, Month 12, Month 18, Month 24, Month 30, and Month 36

  5. Change from baseline in biomarkers.

    Time frame: Month 12, Month 24, and Month 36

  6. Flare-up progression as assessed by the change from baseline in heterotopic ossification at the flare-up site.

    Time frame: Flare-up initiation, Flare-up Days 42 and 84

  7. Flare-up progression as assessed by the change from baseline in pain and swelling at the flare-up site.

    Time frame: Flare-up initiation, Flare-up Days 42 and 84

  8. Flare-up progression as assessed by the change from baseline biomarkers.

    Time frame: Flare-up initiation, Flare-up Days 42 and 84

  9. Flare-up progression as assessed by the change from baseline in physical function as assessed by range of motion.

    Time frame: Flare-up initiation, Flare-up Days 42 and 84

  10. Flare-up progression as assessed by the change from baseline in a disease-specific patient-reported outcome measure (FOP-Physical Function Questionnaire [FOP-PFQ]).

    Time frame: Flare-up initiation, Flare-up Days 42 and 84

  11. Flare-up progression as assessed by the change from baseline in a patient-reported outcome measure of physical and mental health (PROMIS Global Health Scale).

    Time frame: Flare-up initiation, Flare-up Days 42 and 84

Sponsors and collaborators

Lead sponsor

Clementia Pharmaceuticals Inc.

Industry

Registry information

Official study title

A Natural History, Non-Interventional, Two-Part Study in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)

Important dates

Study start
2014
Primary completion
2020
Study completion
2020
First posted
Dec 23, 2014
Registry last updated
Jun 26, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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