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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02322255
Registration number
NCT02322255
Ethics application status
Date submitted
8/12/2014
Date registered
23/12/2014
Date last updated
26/06/2020
Titles & IDs
Public title
A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP)
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Scientific title
A Natural History, Non-Interventional, Two-Part Study in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)
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Secondary ID [1]
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PVO-1A-001
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Fibrodysplasia Ossificans Progressiva
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Condition category
Condition code
Musculoskeletal
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Other muscular and skeletal disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Injuries and Accidents
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Other injuries and accidents
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Intervention/exposure
Study type
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
All Subjects - All subjects enrolled in the study.
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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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]).
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Assessment method [1]
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Timepoint [1]
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Month 36
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Secondary outcome [1]
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Change from baseline in physical function as assessed by range of motion.
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Assessment method [1]
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Timepoint [1]
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Month 12, Month 24, and Month 36
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Secondary outcome [2]
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Change from baseline in patient-reported use of assistive devices and adaptations.
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Assessment method [2]
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Timepoint [2]
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Month 6, Month 12, Month 18, Month 24, Month 30, and Month 36
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Secondary outcome [3]
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Change from baseline in a disease-specific patient-reported outcome measure (FOP-Physical Function Questionnaire [FOP-PFQ]).
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Assessment method [3]
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Timepoint [3]
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Month 6, Month 12, Month 18, Month 24, Month 30, and Month 36
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Secondary outcome [4]
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Change from baseline in a patient-reported measure of physical and mental health (PROMIS Global Health Scale).
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Assessment method [4]
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Timepoint [4]
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Month 6, Month 12, Month 18, Month 24, Month 30, and Month 36
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Secondary outcome [5]
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Change from baseline in biomarkers.
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Assessment method [5]
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Timepoint [5]
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Month 12, Month 24, and Month 36
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Secondary outcome [6]
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Flare-up progression as assessed by the change from baseline in heterotopic ossification at the flare-up site.
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Assessment method [6]
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Timepoint [6]
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Flare-up initiation, Flare-up Days 42 and 84
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Secondary outcome [7]
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Flare-up progression as assessed by the change from baseline in pain and swelling at the flare-up site.
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Assessment method [7]
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Timepoint [7]
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Flare-up initiation, Flare-up Days 42 and 84
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Secondary outcome [8]
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Flare-up progression as assessed by the change from baseline biomarkers.
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Assessment method [8]
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Timepoint [8]
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Flare-up initiation, Flare-up Days 42 and 84
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Secondary outcome [9]
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Flare-up progression as assessed by the change from baseline in physical function as assessed by range of motion.
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Assessment method [9]
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Timepoint [9]
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Flare-up initiation, Flare-up Days 42 and 84
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Secondary outcome [10]
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Flare-up progression as assessed by the change from baseline in a disease-specific patient-reported outcome measure (FOP-Physical Function Questionnaire [FOP-PFQ]).
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Assessment method [10]
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Timepoint [10]
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Flare-up initiation, Flare-up Days 42 and 84
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Secondary outcome [11]
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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).
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Assessment method [11]
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Timepoint [11]
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Flare-up initiation, Flare-up Days 42 and 84
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Eligibility
Key inclusion criteria
- Subjects clinically diagnosed with classical FOP with documented R206H mutation or
believed to carry the R206H mutation
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Minimum age
No limit
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Maximum age
65
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Participation in an interventional clinical research study within the 4 weeks prior to
enrollment
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Study design
Purpose
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Duration
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Selection
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Timing
Prospective
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
18/12/2014
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
9/04/2020
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Sample size
Target
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Accrual to date
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Final
114
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Recruitment in Australia
Recruitment state(s)
QLD
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Recruitment hospital [1]
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Queensland University of Technology (QUT) Institute of Health and Biomedical Innovation (IHBI) - Woolloongabba
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Recruitment postcode(s) [1]
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4102 - Woolloongabba
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Pennsylvania
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Country [3]
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Argentina
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State/province [3]
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Buenos Aires
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Country [4]
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France
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State/province [4]
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Paris
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Country [5]
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Italy
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State/province [5]
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Genoa
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Country [6]
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United Kingdom
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State/province [6]
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Middlesex
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Clementia Pharmaceuticals Inc.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief 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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02322255
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Trial related presentations / publications
Connor JM, Evans DA. Fibrodysplasia ossificans progressiva. The clinical features and natural history of 34 patients. J Bone Joint Surg Br. 1982;64(1):76-83. doi: 10.1302/0301-620X.64B1.7068725.
Zhang W, Zhang K, Song L, Pang J, Ma H, Shore EM, Kaplan FS, Wang P. The phenotype and genotype of fibrodysplasia ossificans progressiva in China: a report of 72 cases. Bone. 2013 Dec;57(2):386-91. doi: 10.1016/j.bone.2013.09.002. Epub 2013 Sep 17.
Cohen RB, Hahn GV, Tabas JA, Peeper J, Levitz CL, Sando A, Sando N, Zasloff M, Kaplan FS. The natural history of heterotopic ossification in patients who have fibrodysplasia ossificans progressiva. A study of forty-four patients. J Bone Joint Surg Am. 1993 Feb;75(2):215-9. doi: 10.2106/00004623-199302000-00008.
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Public notes
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Contacts
Principal investigator
Name
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Ipsen Medical Director
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Address
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Ipsen
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02322255
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