Clinical trial NCT02793063: Osteogenesis Imperfecta (OI) Quality of Life Survey Pilot Project 2
Official title: Rare Diseases Clinical Research Network Brittle Bone Disorders Consortium Osteogenesis Imperfecta (OI) Quality of Life Survey Pilot Project 2
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Osteogenesis Imperfecta
Brittle Bone Disorders
Keywords: Brittle Bone
Osteogenesis Imperfecta
BBD
OI
Congenital Abnormalities
Congenital Bone Disorder
Lobstein Syndrome
Lead sponsor: University of South Florida
Sponsor class: OTHER
Collaborators: name: Rare Diseases Clinical Research Network
name: National Institutes of Health (NIH)
First posted: 2016-06-08
Enrollment: 300
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with Osteogenesis Imperfecta who are enrolled in the RDCRN BBD Contact Registry

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.

Eligibility criteria: 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

Primary outcomes:
measure: Validated Computer Adaptive Testing (CAT) PROMIS instruments
timeFrame: 3 months
Secondary outcomes:
Other outcomes:
Locations:
city: Tampa
country: United States
facility: University of South Florida, Health Informatics Institute
state: Florida
Study officials:
affiliation: University of South Florida
name: Jeffrey Krischer, Ph.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's National Research Institute
name: Laura Tosi, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: University of South Florida
name: Jeffrey Krischer, Ph.D.
role: STUDY_CHAIR
affiliation: Children's National Research Institute
name: Laura Tois, M.D.
role: STUDY_CHAIR
References:
citation: 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.
citation: 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.