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

Completed

• Conditions: Osteogenesis Imperfecta; Brittle Bone Disorders
• Interventions: Other: Web-based Survey

• Registration Number: NCT02793063
• Lead Sponsor: University of South Florida

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.

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 Timeline

• Study First Submitted: 2016-05-26
• Study Start: 2016-06
• Study First Submitted QC: 2016-06-02
• Study First Posted: 2016-06-08
• Primary Completion: 2016-08-10
• Study Completion: 2016-08-10
• Status Verified: 2017-03
• Last Update Submitted: 2018-03-05
• Last Update Posted: 2018-03-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 300

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
BBD Consortium Contact Registrants	Web-based Survey	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.

Primary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

University of South Florida, Health Informatics Institute; 🇺🇸 Tampa, Florida, United States