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

Status: Completed
Location: See all (7) locations...
Study Type: Observational
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.

Eligibility
Participation Requirements
Sex: All
Maximum Age: 65
Healthy Volunteers: No
View:

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

Locations
United States
California
University of California San Francisco, Division of Endocrinology and Metabolism
San Francisco
Pennsylvania
University of Pennsylvania, Center for FOP & Related Bone Disorders
Philadelphia
Other Locations
Argentina
Hospital Italiano de Buenos Aires, Department of Pediatrics
Buenos Aires
Australia
Queensland University of Technology (QUT) Institute of Health and Biomedical Innovation (IHBI)
Woolloongabba
France
Hôpital Necker-Enfants Malades, Department of Genetics
Paris
Italy
Gaslini Institute, Unit of Rare Diseases, Department of Pediatrics
Genoa
United Kingdom
The Royal National Orthopaedic Hospital, Brockley Hill
Stanmore
Time Frame
Start Date: December 18, 2014
Completion Date: April 9, 2020
Participants
Target number of participants: 114
Treatments
All Subjects
All subjects enrolled in the study.
Authors
Frederick S Kaplan, Matthew Brown, Umesh Masharani, Mona Al Mukaddam, Edward Hsiao, Maria De Los Angeles Britos, Sateesh Shankaranarayana, Caroline Michot, Sylvain Breton, Valerie Cormier-Daire, Benjamin Jacobs, Maja DiRocco, Richard Keen, Zulf Mughal, Carmen L DeCunto, Genevieve Baujat
Sponsors
Leads: Clementia Pharmaceuticals Inc.

This content was sourced from clinicaltrials.gov

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