Osteogenesis Imperfecta (OI) Quality of Life Survey Pilot Project 2

Sponsor

University of South Florida (Other)

Overall Status

Completed

CT.gov ID

NCT02793063

Collaborator

Rare Diseases Clinical Research Network (Other), National Institutes of Health (NIH) (NIH)

300

Enrollment

Location

2.3

Duration (Months)

130.4

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to explore the patient perspective of disease burden in Osteogenesis Imperfecta (OI). Participants will complete a web-based survey of questions which are usually administered within the Patient-Reported Outcome Measurement Information System (PROMIS) and provide feedback regarding the appropriateness of the questions for someone with OI.

Condition or Disease	Intervention/Treatment	Phase
Osteogenesis Imperfecta Brittle Bone Disorders	Other: Web-based Survey

Detailed Description

Participants will be recruited through the RDCRN BBD Contact Registry. An email invitation will be sent to all RDCRN BBD Contact Registry participants that meet the inclusion criteria to complete the online PROMIS CAT instruments. The email invitation will contain a link to the informed consent form. Once participants have consented to the study, they will have access to the online instruments. Follow-up emails will be sent to participants that have not completed any of the instruments. Reminder emails will be sent to participants with partially completed instruments.

Data will be collected and stored by the RDCRN DMCC at USF.

Data from patients co-enrolled in the "Longitudinal Study of Osteogenesis Imperfecta" and the BBD RDCRN Contact Registry will be linked and compared. Additional disease specific information will be collected by the survey (e.g., information on OI type and diagnosis, height, number of fractures, concurrent medication use, utilization of any mobility aid, hearing aid, or other device that they may use.

Study Design

Study Type:

Observational

Actual Enrollment :

300 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Rare Diseases Clinical Research Network Brittle Bone Disorders Consortium Osteogenesis Imperfecta (OI) Quality of Life Survey Pilot Project 2

Study Start Date :

Jun 1, 2016

Actual Primary Completion Date :

Aug 10, 2016

Actual Study Completion Date :

Aug 10, 2016

Arms and Interventions

Arm	Intervention/Treatment
BBD Consortium Contact Registrants Osteogenesis Imperfecta patients who have self-registered at the Brittle Bone Disorders Consortium (BBD) Consortium Contact Registry, a web-based contact registry developed and supported by the Data Management and Coordinating Center (DMCC) for the Rare Diseases Clinical Research Consortium (RDCRN), located at the University of South Florida.	Other: Web-based Survey This study will occur online. Participants will be asked to complete multiple surveys one time. Participants may receive study reminder emails to complete surveys. Participants will be able to complete the surveys all at once or in multiple sessions. Participants will have approximately 3 months to complete the surveys after consenting.

Arm

Intervention/Treatment

BBD Consortium Contact Registrants

Osteogenesis Imperfecta patients who have self-registered at the Brittle Bone Disorders Consortium (BBD) Consortium Contact Registry, a web-based contact registry developed and supported by the Data Management and Coordinating Center (DMCC) for the Rare Diseases Clinical Research Consortium (RDCRN), located at the University of South Florida.

Other: Web-based Survey

This study will occur online. Participants will be asked to complete multiple surveys one time. Participants may receive study reminder emails to complete surveys. Participants will be able to complete the surveys all at once or in multiple sessions. Participants will have approximately 3 months to complete the surveys after consenting.

Outcome Measures

Primary Outcome Measures

Validated Computer Adaptive Testing (CAT) PROMIS instruments [3 months]
An online assessment will be used to obtain responses for each instrument. Scores for each are norm-based with a mean of 50 and standard deviation of 10 based upon the general population. Calibration of the standard error (SE) surrounding the reported score is possible. Default setting of 0.30 will be used for standard error with the minimum number of questions per item of 4 and maximum number of questions 8, using the computer adapted testing structure of the PROMIS instruments. Lower maximum number than the default settings will be used to avoid too many questions being given to a respondent when they do not report a diminished quality of life on any dimension. With these settings, survey respondents will be administered an average of 4-5 items per instrument and the testing among patients with OI indicate that the whole assessment will typically take 15-30 minutes in most instances.

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Enrollment in the RDCRN BBD Contact Registry English speaking Age 2 to adult

Exclusion Criteria:

Inability to provide informed consent Inability to complete instruments

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of South Florida, Health Informatics Institute	Tampa	Florida	United States	33612

Sponsors and Collaborators

University of South Florida
Rare Diseases Clinical Research Network
National Institutes of Health (NIH)

Investigators

Principal Investigator: Jeffrey Krischer, Ph.D., University of South Florida
Principal Investigator: Laura Tosi, M.D., Children's National Research Institute
Study Chair: Jeffrey Krischer, Ph.D., University of South Florida
Study Chair: Laura Tois, M.D., Children's National Research Institute