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Trial record 6 of 29 for:    fop

A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP)

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02322255
First Posted: December 23, 2014
Last Update Posted: March 23, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Clementia Pharmaceuticals Inc.
December 8, 2014
December 23, 2014
March 23, 2017
December 2014
October 2019   (Final data collection date for primary outcome measure)
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 ]
Change from baseline in the total body burden of heterotopic ossification as assessed by the optimal imaging modality (either low-dose whole body CT [excluding head] or DEXA scans). [ Time Frame: Month 36 ]
Complete list of historical versions of study NCT02322255 on ClinicalTrials.gov Archive Site
  • Change from baseline in physical function as assessed by range of motion. [ Time Frame: Month 12, Month 24, and Month 36 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • Change from baseline in biomarkers. [ Time Frame: Month 12, Month 24, and Month 36 ]
  • 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 ]
  • 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 ]
  • Flare-up progression as assessed by the change from baseline biomarkers. [ Time Frame: Flare-up initiation, Flare-up Days 42 and 84 ]
  • 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 ]
  • 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 ]
  • 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 ]
Same as current
Not Provided
Not Provided
 
A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP)
A Natural History, Non-Interventional, Two-Part Study in Subjects With 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.

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.

Observational
Observational Model: Case-Only
Time Perspective: Prospective
Not Provided
Retention:   Samples With DNA
Description:
Blood and urine samples for biomarker and proteomic analysis.
Non-Probability Sample
Individuals with classic FOP (R206H mutation).
Fibrodysplasia Ossificans Progressiva
Not Provided
All Subjects
All subjects enrolled in the study.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
100
October 2019
October 2019   (Final data collection date for primary outcome measure)

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

Sexes Eligible for Study: All
up to 65 Years   (Child, Adult)
No
Contact information is only displayed when the study is recruiting subjects
Argentina,   Australia,   France,   Italy,   United Kingdom,   United States
Canada
 
NCT02322255
PVO-1A-001
No
Not Provided
Not Provided
Clementia Pharmaceuticals Inc.
Clementia Pharmaceuticals Inc.
Not Provided
Study Director: Donna Grogan, MD Clementia Pharmaceuticals Inc.
Clementia Pharmaceuticals Inc.
December 2016