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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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)
Scientific title
A Natural History, Non-Interventional, Two-Part Study in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)
Secondary ID [1] 0 0
PVO-1A-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fibrodysplasia Ossificans Progressiva 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Injuries and Accidents 0 0 0 0
Other injuries and accidents

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
All Subjects - All subjects enrolled in the study.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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]).
Timepoint [1] 0 0
Month 36
Secondary outcome [1] 0 0
Change from baseline in physical function as assessed by range of motion.
Timepoint [1] 0 0
Month 12, Month 24, and Month 36
Secondary outcome [2] 0 0
Change from baseline in patient-reported use of assistive devices and adaptations.
Timepoint [2] 0 0
Month 6, Month 12, Month 18, Month 24, Month 30, and Month 36
Secondary outcome [3] 0 0
Change from baseline in a disease-specific patient-reported outcome measure (FOP-Physical Function Questionnaire [FOP-PFQ]).
Timepoint [3] 0 0
Month 6, Month 12, Month 18, Month 24, Month 30, and Month 36
Secondary outcome [4] 0 0
Change from baseline in a patient-reported measure of physical and mental health (PROMIS Global Health Scale).
Timepoint [4] 0 0
Month 6, Month 12, Month 18, Month 24, Month 30, and Month 36
Secondary outcome [5] 0 0
Change from baseline in biomarkers.
Timepoint [5] 0 0
Month 12, Month 24, and Month 36
Secondary outcome [6] 0 0
Flare-up progression as assessed by the change from baseline in heterotopic ossification at the flare-up site.
Timepoint [6] 0 0
Flare-up initiation, Flare-up Days 42 and 84
Secondary outcome [7] 0 0
Flare-up progression as assessed by the change from baseline in pain and swelling at the flare-up site.
Timepoint [7] 0 0
Flare-up initiation, Flare-up Days 42 and 84
Secondary outcome [8] 0 0
Flare-up progression as assessed by the change from baseline biomarkers.
Timepoint [8] 0 0
Flare-up initiation, Flare-up Days 42 and 84
Secondary outcome [9] 0 0
Flare-up progression as assessed by the change from baseline in physical function as assessed by range of motion.
Timepoint [9] 0 0
Flare-up initiation, Flare-up Days 42 and 84
Secondary outcome [10] 0 0
Flare-up progression as assessed by the change from baseline in a disease-specific patient-reported outcome measure (FOP-Physical Function Questionnaire [FOP-PFQ]).
Timepoint [10] 0 0
Flare-up initiation, Flare-up Days 42 and 84
Secondary outcome [11] 0 0
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).
Timepoint [11] 0 0
Flare-up initiation, Flare-up Days 42 and 84

Eligibility
Key inclusion criteria
- Subjects clinically diagnosed with classical FOP with documented R206H mutation or believed to carry the R206H mutation
Minimum age
No limit
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Participation in an interventional clinical research study within the 4 weeks prior to enrollment

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
18/12/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
9/04/2020
Sample size
Target
Accrual to date
Final
114
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
Queensland University of Technology (QUT) Institute of Health and Biomedical Innovation (IHBI) - Woolloongabba
Recruitment postcode(s) [1] 0 0
4102 - Woolloongabba
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Pennsylvania
Country [3] 0 0
Argentina
State/province [3] 0 0
Buenos Aires
Country [4] 0 0
France
State/province [4] 0 0
Paris
Country [5] 0 0
Italy
State/province [5] 0 0
Genoa
Country [6] 0 0
United Kingdom
State/province [6] 0 0
Middlesex

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Clementia Pharmaceuticals Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Ipsen Medical Director
Address 0 0
Ipsen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT02322255