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Summary

for people ages up to 65 years
at San Francisco, California and other locations
study started
estimated completion:

Description

Summary

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.

Official Title

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

Details

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.

Keywords

Fibrodysplasia Ossificans Progressiva Natural History Study Non-interventional Study Observational Study Heterotopic ossification FOP disease progression FOP flare-up progression Clementia Myositosis Ossificans Progressiva Munchmeyer's Disease FOP Palovarotene

Eligibility

You can join if…

Open to people ages up to 65 years

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

You CAN'T join if...

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

Locations

  • University of Pennsylvania, Center for FOP & Related Bone Disorders
    Philadelphia, Pennsylvania, 19104, United States
  • The Royal National Orthopaedic Hospital, Brockley Hill
    Stanmore, Middlesex, HA7 4LP, United Kingdom
  • Hôpital Necker-Enfants Malades, Department of Genetics
    Paris, France
  • Hospital Italiano de Buenos Aires, Department of Pediatrics
    Buenos Aires, Argentina
  • Gaslini Institute, Unit of Rare Diseases, Department of Pediatrics
    Genoa, Italy
  • Queensland University of Technology (QUT) Institute of Health and Biomedical Innovation (IHBI)
    Woolloongabba, Queensland, 4102, Australia

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Clementia Pharmaceuticals Inc.
Links
website for the International FOP Association
Click here for more information about this study: A Natural History, Non-Intervention Study in Subjects with Fibrodysplasia Ossificans Progressiva (FOP)
ID
NCT02322255
Study Type
Observational
Last Updated
December 1, 2016