Osteogenesis ImperfectaNCT02793063
Osteogenesis Imperfecta (OI) Quality of Life Survey Pilot Project 2
An observational study of Osteogenesis Imperfecta and Brittle Bone Disorders, run by University of South Florida at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Osteogenesis Imperfecta, Brittle Bone Disorders
- What is being tested?
- Web-based Survey
- How many people
- 300 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2016to 10 Aug 2016
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with Osteogenesis Imperfecta who are enrolled in the RDCRN BBD Contact Registry
Who may be able to join
- Enrollment in the RDCRN BBD Contact Registry English speaking Age 2 to adult
Who cannot join
- Inability to provide informed consent Inability to complete instruments
Trial locations and contacts
1 site in 1 country.
United States1 site
University of South Florida, Health Informatics Institute
Tampa, Florida, 33612
About this study
Official title: Rare Diseases Clinical Research Network Brittle Bone Disorders Consortium Osteogenesis Imperfecta (OI) Quality of Life Survey Pilot Project 2
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.
Read the 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.
- Conditions
- Osteogenesis Imperfecta, Brittle Bone Disorders
- Being tested
- Web-based Survey
- Keywords
- Brittle Bone, Osteogenesis Imperfecta, BBD, OI, Congenital Abnormalities, Congenital Bone Disorder, Lobstein Syndrome
- Medical subject headings
- Osteogenesis Imperfecta, Congenital Abnormalities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Web-based SurveyOther
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Other: Web-based Survey |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2016 (estimated)
- Study startJun 2016
- Primary completion10 Aug 2016
- Study completion10 Aug 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Osteogenesis ImperfectaStudies of the same condition, open and under way.
- More trials run by University of South FloridaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 300 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of South Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Rare Diseases Clinical Research Network, National Institutes of Health (NIH)
- Responsible party
- Sponsor
- Investigators
- Jeffrey Krischer, Ph.D., Principal investigator, University of South FloridaLaura Tosi, M.D., Principal investigator, Children's National Research InstituteJeffrey Krischer, Ph.D., Study chair, University of South FloridaLaura Tois, M.D., Study chair, Children's National Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02793063
- Sponsor's protocol ID
- BBD7702
- Other IDs
- U01TR001263
- Status last verified
- Mar 2017
- First submitted
- 26 May 2016
- Last updated
- 6 Mar 2018
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
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.
PubMed 18694191Richesson 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.
PubMed 18804556
Study at a glance
From the registry record, last updated by the sponsor on 6 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Rare Diseases Clinical Research Network Brittle Bone Disorders Consortium Osteogenesis Imperfecta (OI) Quality of Life Survey Pilot Project 2
- ClinicalTrials.gov ID
- NCT02793063
- Acronym / sponsor ID
- BBD7702
- Conditions
- Osteogenesis Imperfecta, Brittle Bone Disorders
- Keywords
- Brittle Bone, Osteogenesis Imperfecta, BBD, OI, Congenital Abnormalities, Congenital Bone Disorder, Lobstein Syndrome
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 300 participants (actual)
- Lead sponsor
- University of South FloridaUniversity, hospital or other
- Collaborators
- Rare Diseases Clinical Research Network, National Institutes of Health (NIH)
- Study start
- Jun 2016
- Primary completion
- 10 Aug 2016 (actual)
- Study completion
- 10 Aug 2016 (actual)
- First posted
- 8 Jun 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BBD Consortium Contact Registrants | – | Other: Web-based SurveyOsteogenesis 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. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with Osteogenesis Imperfecta who are enrolled in the RDCRN BBD Contact Registry
Key inclusion criteria (1 listed)
- Enrollment in the RDCRN BBD Contact Registry English speaking Age 2 to adult
Key exclusion criteria (1 listed)
- Inability to provide informed consent Inability to complete instruments
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Web-based SurveyThis 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. | Other | BBD Consortium Contact Registrants |
Primary outcome
Validated Computer Adaptive Testing (CAT) PROMIS instruments
Time frame: 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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Mar 2018.
- Background: PubMed 18694191Richesson 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.
- Results publication: PubMed 18804556Richesson 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jeffrey Krischer, Ph.D. principal investigator
University of South Florida
Laura Tosi, M.D. principal investigator
Children's National Research Institute
Jeffrey Krischer, Ph.D. study chair
University of South Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Osteogenesis Imperfecta and Brittle Bone Disorders, run by University of South Florida at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Validated Computer Adaptive Testing (CAT) PROMIS instruments (time frame: 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02793063, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Mar 2018; AzTrials last refreshed its copy on 7 Nov 2026.