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The Fibrodysplasia Ossificans Progressiva (FOP) Connection Registry

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ClinicalTrials.gov Identifier: NCT02745158
Recruitment Status : Recruiting
First Posted : April 20, 2016
Last Update Posted : August 17, 2017
Sponsor:
Information provided by (Responsible Party):
The International FOP Association

Tracking Information
First Submitted Date March 6, 2016
First Posted Date April 20, 2016
Last Update Posted Date August 17, 2017
Actual Study Start Date July 2015
Estimated Primary Completion Date July 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: April 15, 2016)
  • Patient Reported Changes in New Bone Growth Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]
    The patient is able to report changes in new bone growth among 27 joints and body locations.
  • Patient Reported Changes in Episodic Flare-Ups Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]
    The patient is able to report changes in episodic flare-ups among 27 joints and body locations.
  • Patient Reported Changes in Mobility Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]
    The patient is able to report changes in mobility among 27 joints and body locations.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: April 15, 2016)
  • Patient Reported Changes in Health Resource Utilization Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]
    The number of visits to a Physician and a Dentist, as well as the number of hospitalizations and reasons for the hospitalizations, are used to assess health resource utilization.
  • Patient Reported Changes in Signs Related to the Ear Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]
    The patient is able to report new or continuing health issues related to the ears.
  • Patient Reported Changes in Signs Related to the Skin Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]
    The patient is able to report new or continuing health issues related to the skin.
  • Patient Reported Changes in Signs Related to the Central Nervous System Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]
    The patient is able to report new or continuing health issues related to the central nervous system.
  • Patient Reported Changes in Signs Related to the Pulmonary System Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]
    The patient is able to report new or continuing health issues related to the pulmonary system.
  • Patient Reported Changes in Signs Related to the Gastrointestinal System Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]
    The patient is able to report new or continuing health issues related to the gastrointestinal system.
  • Patient Reported Changes in Signs Related to the Cardiovascular System Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]
    The patient is able to report new or continuing health issues related to the cardiovascular system.
  • Patient Reported Changes in Signs Related to the Renal System Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]
    The patient is able to report new or continuing health issues related to the renal system.
  • Patient Reported Changes in Signs Related to the Endocrine System Using a Patient-Directed Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]
    The patient is able to report new or continuing health issues related to the endocrine system.
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures
 (submitted: April 15, 2016)
  • Patient Reported Changes in Activities of Daily Living Using the FOP-Physical Functioning Questionnaire (PFQ) [ Time Frame: Baseline, then every six months for up to 10 years ]
  • Patient Reported Changes in Overall Health Using the PROMIS Global Health Scale [ Time Frame: Baseline, then every six months for up to 10 years ]
  • Patient Reported Changes in the Need for Assistive Devices, Aids, Attendants, and Adaptations Using a Survey Developed by the IFOPA [ Time Frame: Baseline, then every six months for up to 10 years ]
    The patient is able to respond Yes or No to questions related to his/her need for Assistive Devices, Aids, Attendants, and Adaptations as a result of his/her FOP disease.
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title The Fibrodysplasia Ossificans Progressiva (FOP) Connection Registry
Official Title FOP Connection: A Global Registry for the Fibrodysplasia Ossificans Progressiva Community
Brief Summary The Fibrodysplasia Ossificans Progressiva (FOP) Connection Registry is a global, non-interventional, voluntary database that captures demographic and disease data directly from FOP patients and their caregivers via a secure, web-based patient portal. A physician portal (in development) will allow physicians to enter clinical data about their patients. The objectives are to organize the international FOP community for participation in clinical trials; to enable FOP patients worldwide to report data in a shared forum; to improve the collective understanding of FOP natural history; and to advance the understanding of FOP treatment outcomes.
Detailed Description

The FOP Connection Registry is a global, non-interventional, voluntary database that captures demographic and disease data directly from FOP patients and their caregivers via a secure, web-based patient portal. No experimental intervention is involved. The objectives are to organize the international FOP community for participation in clinical trials; to enable FOP patients worldwide to report data in a shared forum; to improve the collective understanding of FOP natural history; and to advance the understanding of FOP treatment outcomes.

The Registry collects data through two sources. First, the Patient Portal (launched in July 2015) allows FOP patients and caregivers to enter information about their experiences living with FOP. Second, the Physician Portal (in development) will allow physicians to enter clinical data about patients under their care. The Registry will be capable of including data on specific marketed therapies under the direction and control of a sponsoring pharmaceutical company. Key identifiers will link the physician-reported data with the Patient Portal data.

Participants must have a confirmed diagnosis of FOP and the participant (or a parent or legal guardian) must be willing and able to provide written informed consent. There are no exclusion criteria. Data collected in the Patient Portal include: patient demographics and diagnosis pathway; medical and dental care; clinical research participation and biospecimen donation; heterotopic ossification (bone growth and episodic flare-ups); other signs and symptoms by body system; patient-reported outcomes (physical functioning, pain, fatigue, and general health); and assistive devices, aids, attendants, and adaptations. Because the Registry is designed to accommodate participants along a broad spectrum of FOP disease severity, most of the data fields are optional, allowing participants a high degree of flexibility in how much information they contribute, which also minimizes participant burden.

After completing the informed consent, participants enter their baseline (historical) data. Participants will be encouraged to update their information at least twice per year. Participants may withdraw their consent at any time without prejudice or providing an explanation. The Registry has no pre-specified end date and will continue for as long as it is sustainable and useful to the FOP community.

Study Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration 10 Years
Biospecimen Not Provided
Sampling Method Probability Sample
Study Population Individuals with Fibrodysplasia Ossificans Progressive (FOP) from the international FOP community will be invited to join the Registry.
Condition Fibrodysplasia Ossificans Progressiva (FOP)
Intervention Not Provided
Study Groups/Cohorts FOP Patients
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: April 15, 2016)
800
Original Estimated Enrollment Same as current
Estimated Study Completion Date December 2025
Estimated Primary Completion Date July 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Participants must have a confirmed diagnosis of FOP.
  • Participants (or a parent or legal guardian) must be willing and able to provide written informed consent.

Exclusion Criteria:

  • There are no exclusion criteria.
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Neal S Mantick, BS, MS 1-617-910-8508 registry@fopconnection.org
Contact: Betsy Bogard, BS, MS 1-407-365-4194 registry@fopconnection.org
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02745158
Other Study ID Numbers IFOPA-REG-001
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement
Plan to Share IPD: Yes
Plan Description: Formal data sharing agreements are planned between the International FOP Association and biopharmaceutical companies doing research in Fibrodysplasia Ossificans Progressiva (FOP). Publications are also planned with biopharmaceutical companies and physician researchers.
Responsible Party The International FOP Association
Study Sponsor The International FOP Association
Collaborators Not Provided
Investigators
Study Director: Betsy Bogard, BS, MS The International FOP Association
PRS Account The International FOP Association
Verification Date March 2016