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Trial record 6 of 34 for:    fop

A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP)

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.
 
ClinicalTrials.gov Identifier: NCT02322255
Recruitment Status : Active, not recruiting
First Posted : December 23, 2014
Last Update Posted : August 1, 2019
Sponsor:
Information provided by (Responsible Party):
Clementia Pharmaceuticals Inc.

Tracking Information
First Submitted Date December 8, 2014
First Posted Date December 23, 2014
Last Update Posted Date August 1, 2019
Study Start Date December 2014
Estimated Primary Completion Date October 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: December 17, 2015)
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 ]
Original Primary Outcome Measures
 (submitted: December 19, 2014)
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 ]
Change History Complete list of historical versions of study NCT02322255 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: December 19, 2014)
  • 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 ]
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP)
Official Title A Natural History, Non-Interventional, Two-Part Study in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)
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.
Detailed Description

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.

Study Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
Blood and urine samples for biomarker and proteomic analysis.
Sampling Method Non-Probability Sample
Study Population Individuals with classic FOP (R206H mutation).
Condition Fibrodysplasia Ossificans Progressiva
Intervention Not Provided
Study Groups/Cohorts All Subjects
All subjects enrolled in the study.
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Estimated Enrollment
 (submitted: December 17, 2015)
100
Original Estimated Enrollment
 (submitted: December 19, 2014)
50
Estimated Study Completion Date October 2019
Estimated Primary Completion Date October 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria

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

Sex/Gender
Sexes Eligible for Study: All
Ages up to 65 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Argentina,   Australia,   France,   Italy,   United Kingdom,   United States
Removed Location Countries Canada
 
Administrative Information
NCT Number NCT02322255
Other Study ID Numbers PVO-1A-001
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Clementia Pharmaceuticals Inc.
Study Sponsor Clementia Pharmaceuticals Inc.
Collaborators Not Provided
Investigators
Study Director: Donna Grogan, MD Clementia Pharmaceuticals Inc.
PRS Account Clementia Pharmaceuticals Inc.
Verification Date July 2019