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

NCT02793063 | Completed

• 2 other identifiers: BBD7702U01TR001263
• Study Type: observational
• Target: 300
• Locations: 1 country
• Sites: 1

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.

Conditions

Osteogenesis Imperfecta; Brittle Bone Disorders

Keywords

Brittle Bone; Osteogenesis Imperfecta; BBD; OI; Congenital Abnormalities; Congenital Bone Disorder; Lobstein Syndrome

Outcome Measures

Primary Outcomes (1)

• Validated Computer Adaptive Testing (CAT) PROMIS instruments

  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.

  3 months

Study Arms (1)

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

Interventions

Web-based Survey OTHER

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.

BBD Consortium Contact Registrants

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with Osteogenesis Imperfecta who are enrolled in the RDCRN BBD Contact Registry

You may qualify if:

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

You may not qualify if:

• Inability to provide informed consent Inability to complete instruments

Sponsors & Collaborators

• University of South Florida: lead
• Rare Diseases Clinical Research Network: collaborator
• National Institutes of Health (NIH): collaborator

Study Sites (1)

University of South Florida, Health Informatics Institute

Tampa, Florida, 33612, United States

Location

Related Publications (2)

• Richesson RL, Young K, Lloyd J, Adams T, Guillette H, Malloy J, Krischer JP. An automated communication system in a Contact Registry for persons with rare diseases: tools for retaining potential clinical research participants. AMIA Annu Symp Proc. 2007 Oct 11:1094.

  PMID: 18694191 BACKGROUND

• Richesson RL, Lee HS, Cuthbertson D, Lloyd J, Young K, Krischer JP. An automated communication system in a contact registry for persons with rare diseases: scalable tools for identifying and recruiting clinical research participants. Contemp Clin Trials. 2009 Jan;30(1):55-62. doi: 10.1016/j.cct.2008.09.002. Epub 2008 Sep 7.

  PMID: 18804556 RESULT

MeSH Terms

Conditions

Osteogenesis Imperfecta; Congenital Abnormalities

Condition Hierarchy (Ancestors)

Osteochondrodysplasias; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Study Officials

• Jeffrey Krischer, Ph.D.

  University of South Florida

  PRINCIPAL INVESTIGATOR

• Laura Tosi, M.D.

  Children's National Research Institute

  PRINCIPAL INVESTIGATOR

• Jeffrey Krischer, Ph.D.

  University of South Florida

  STUDY CHAIR

• Laura Tois, M.D.

  Children's National Research Institute

  STUDY CHAIR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 26, 2016
• First Posted: June 8, 2016
• Study Start: June 1, 2016
• Primary Completion: August 10, 2016
• Study Completion: August 10, 2016
• Last Updated: March 6, 2018

Record last verified: 2017-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)