Immune Deficiency, AntibodyHR-QOLPIDDNCT02542228
Construction of a Health-related Quality of Life (HR-QOL) Questionnaire for Patients With Primary Antibody Deficiency Disease
An observational study of Immune Deficiency, Antibody, run by University of South Florida.
Trial summary
This research is being done to construct a survey questionnaire, a quality of life tool, to determine the health, well-being, ability to perform daily activities, and physical, social and emotional functioning in participants with primary immunodeficiency disorders (PIDD). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Immune Deficiency, Antibody
- What is being tested?
- questionnaire survey
- How many people
- 76 participants
- Where
- No sites listed yet
- Study dates
- Mar 2014to Sep 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with primary antibody immune deficiency with x-linked agammaglobulinemia and common variable immune deficiency
Who may be able to join
- Subjects with documented x-linked agammaglobulinemia, or common variable immunodeficiency, autosomal recessive agammaglobulinemia
- Subjects must be able to read and speak English
- Willing to sign consent and follow study schedule.
- Subjects 18 years to 65 years of age.
- Subject must be receiving Ig treatment for diagnosis.
Who cannot join
- Subject or guardian unwilling to sign consent or adhere to study schedule.
- Pregnancy,
- Severe concurrent medical conditions which would prevent treatment or assessment, including significant hematological, renal, lung or liver dysfunction or malignancies
- Presence of any other medical condition, which in the opinion of the investigator might interfere with performance or interpretation of this study
- Can not read or understand English
About this study
Official title: Construction and Validation of a Health-related Quality of Life (HR-QOL) Instrument for Patients With Primary Antibody Deficiency Disease
This research is being done to construct a survey questionnaire, a quality of life tool, to determine the health, well-being, ability to perform daily activities, and physical, social and emotional functioning in participants with primary immunodeficiency disorders (PIDD). This quality of life (QOL) tool will help physicians understand the effects of PIDD on a person's health. These quality of life surveys are important tools for physicians to measure outcomes for satisfaction and effects of treatment.
Read the detailed description
A preliminary patient and clinician-generated scale will be validated and modified during a multicenter scale validity study involving 85 patients from 5 sites. Feasibility surveys have been conducted and available sites that can be included in this study include Boston Children's/ Harvard, Midwest Immunology, Johns Hopkins, Children's Hospital at Baylor University, and the University of South Florida at All Children's Hospital Johns Hopkins Medicine. Patients will be enrolled at a clinic non-infusion visit. The purpose of the study will be explained and informed consent obtained. A case report form (CRF) of their clinical condition and demographics will be completed based on the information obtained by the local study coordinator during the interview at the time of the visit. The HR-QOL PIDD survey will be administered to the patient by the site PI or the study coordinator. A second survey will be handed to the patient to take home with them and completed at 48-120 hrs. after the clinic visit. The study coordinator will contact the patient during this period to ensure that the patient completes this second survey and returns it to the local study site. If a patient is on SCIg they will be asked to take the survey 3 days after the SC infusion (if weekly), and complete the paired survey 48-72 hrs later (before another SCIg infusion is scheduled). The study coordinator will obtain an interim history for the period between the paired surveys to ensure that, in general, the condition of the patient has not changed that could invalidate the validation process, and this information will be documented on the addendum case report form (CRF). Patient can return the surveys to the site study coordinator either by mail, fax or electronically by scanning the documents. Paired surveys will be returned to the central study site (USF St Petersburg) for data input.
- Conditions
- Immune Deficiency, Antibody
- Being tested
- questionnaire survey
- Keywords
- Immune Deficiency, antibody, Quality of Life survey
- Medical subject headings
- Immunologic Deficiency Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
questionnaire surveyOther
The HR-QOL PIDD survey will be administered to the patient by the site PI or the study coordinator. A second survey will be handed to the patient to take home with them and completed at 48-120 hrs. after the clinic visit. The study coordinator will contact the patient during this period to ensure that the patient completes this second survey and returns it to the local study site. A case report form (CRF) of their clinical condition and demographics will be collected at the time of informed consent.
What is being measured
The primary outcome is the main question the study is designed to answer.
Develop and validate a disease specific Quality of Life Questionnaire for patients with primary antibody immune deficiency disease (PIDD)
A 28 item questionnaire will be administered to patients with antibody immune deficiency using a 5 point Likert type scale. The questions were generated by clinical immunologist, infusion nurses with feedback from patients. The questions cover all of the SF-36 QOL instrument except body pain. Paired questionnaires were collected 48 to 120 hrs apart from each patient. Rasch analysis will be used to analysis the performance of the questionnaire for disordered items, lack of discrimination between item questions, overlap of questions, and the spread of the 5 point scale that will lead to item reduction. The primary outcome is to generate a validated HR-QOL questionnaire for patients with primary antibody deficiency that will be useful for clinical care and potentially for clinical trials.
Measured: one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Sep 2015 (estimated)
- Study startMar 2014
- Primary completionJan 2016
- Study completionSep 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Immune Deficiency, AntibodyStudies 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
- 76 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of South Florida
- Sponsor type
- University, hospital or other
- Collaborators
- University of Virginia, Boston Children's Hospital, Baylor College of Medicine, Johns Hopkins University, Midwest Immunology Clinic, CSL Behring
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02542228
- Sponsor's protocol ID
- 6119806000
- Acronym
- HR-QOLPIDD
- Status last verified
- Jan 2017
- First submitted
- 31 Aug 2015
- Last updated
- 13 Jan 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
Ballow M, Conaway MR, Sriaroon P, Rachid RA, Seeborg FO, Duff CM, Bonilla FA, Younger MEM, Shapiro R, Burns TM. Construction and validation of a novel disease-specific quality-of-life instrument for patients with primary antibody deficiency disease (PADQOL-16). J Allergy Clin Immunol. 2017 Jun;139(6):2007-2010.e8. doi: 10.1016/j.jaci.2016.11.029. Epub 2017 Jan 5. No abstract available.
PubMed 28065678
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Construction and Validation of a Health-related Quality of Life (HR-QOL) Instrument for Patients With Primary Antibody Deficiency Disease
- ClinicalTrials.gov ID
- NCT02542228
- Acronym / sponsor ID
- HR-QOLPIDD / 6119806000
- Conditions
- Immune Deficiency, Antibody
- Keywords
- Immune Deficiency, antibody, Quality of Life survey
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 76 participants (actual)
- Lead sponsor
- University of South FloridaUniversity, hospital or other
- Collaborators
- University of Virginia, Boston Children's Hospital, Baylor College of Medicine, Johns Hopkins University, Midwest Immunology Clinic, CSL Behring
- Study start
- Mar 2014
- Primary completion
- Jan 2016 (actual)
- Study completion
- Sep 2016 (actual)
- First posted
- 4 Sep 2015 (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.
This research is being done to construct a survey questionnaire, a quality of life tool, to determine the health, well-being, ability to perform daily activities, and physical, social and emotional functioning in participants with primary immunodeficiency disorders (PIDD). This quality of life (QOL) tool will help physicians understand the effects of PIDD on a person's health. These quality of life surveys are important tools for physicians to measure outcomes for satisfaction and effects of treatment.
Detailed description
A preliminary patient and clinician-generated scale will be validated and modified during a multicenter scale validity study involving 85 patients from 5 sites. Feasibility surveys have been conducted and available sites that can be included in this study include Boston Children's/ Harvard, Midwest Immunology, Johns Hopkins, Children's Hospital at Baylor University, and the University of South Florida at All Children's Hospital Johns Hopkins Medicine. Patients will be enrolled at a clinic non-infusion visit. The purpose of the study will be explained and informed consent obtained. A case report form (CRF) of their clinical condition and demographics will be completed based on the information obtained by the local study coordinator during the interview at the time of the visit. The HR-QOL PIDD survey will be administered to the patient by the site PI or the study coordinator. A second survey will be handed to the patient to take home with them and completed at 48-120 hrs. after the clinic visit. The study coordinator will contact the patient during this period to ensure that the patient completes this second survey and returns it to the local study site. If a patient is on SCIg they will be asked to take the survey 3 days after the SC infusion (if weekly), and complete the paired survey 48-72 hrs later (before another SCIg infusion is scheduled). The study coordinator will obtain an interim history for the period between the paired surveys to ensure that, in general, the condition of the patient has not changed that could invalidate the validation process, and this information will be documented on the addendum case report form (CRF). Patient can return the surveys to the site study coordinator either by mail, fax or electronically by scanning the documents. Paired surveys will be returned to the central study site (USF St Petersburg) for data input.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with primary antibody immune deficiency with x-linked agammaglobulinemia and common variable immune deficiency
Key inclusion criteria (5 listed)
- Subjects with documented x-linked agammaglobulinemia, or common variable immunodeficiency, autosomal recessive agammaglobulinemia
- Subjects must be able to read and speak English
- Willing to sign consent and follow study schedule.
- Subjects 18 years to 65 years of age.
- Subject must be receiving Ig treatment for diagnosis.
Key exclusion criteria (5 listed)
- Subject or guardian unwilling to sign consent or adhere to study schedule.
- Pregnancy,
- Severe concurrent medical conditions which would prevent treatment or assessment, including significant hematological, renal, lung or liver dysfunction or malignancies
- Presence of any other medical condition, which in the opinion of the investigator might interfere with performance or interpretation of this study
- Can not read or understand English
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 |
|---|---|---|
| questionnaire surveyThe HR-QOL PIDD survey will be administered to the patient by the site PI or the study coordinator. A second survey will be handed to the patient to take home with them and completed at 48-120 hrs. after the clinic visit. The study coordinator will contact the patient during this period to ensure that the patient completes this second survey and returns it to the local study site. A case report form (CRF) of their clinical condition and demographics will be collected at the time of informed consent. | Other | – |
Primary outcome
Develop and validate a disease specific Quality of Life Questionnaire for patients with primary antibody immune deficiency disease (PIDD)
Time frame: one year
A 28 item questionnaire will be administered to patients with antibody immune deficiency using a 5 point Likert type scale. The questions were generated by clinical immunologist, infusion nurses with feedback from patients. The questions cover all of the SF-36 QOL instrument except body pain. Paired questionnaires were collected 48 to 120 hrs apart from each patient. Rasch analysis will be used to analysis the performance of the questionnaire for disordered items, lack of discrimination between item questions, overlap of questions, and the spread of the 5 point scale that will lead to item reduction. The primary outcome is to generate a validated HR-QOL questionnaire for patients with primary antibody deficiency that will be useful for clinical care and potentially for clinical trials.
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 13 Jan 2017.
- Derived from this study: PubMed 28065678Ballow M, Conaway MR, Sriaroon P, Rachid RA, Seeborg FO, Duff CM, Bonilla FA, Younger MEM, Shapiro R, Burns TM. Construction and validation of a novel disease-specific quality-of-life instrument for patients with primary antibody deficiency disease (PADQOL-16). J Allergy Clin Immunol. 2017 Jun;139(6):2007-2010.e8. doi: 10.1016/j.jaci.2016.11.029. Epub 2017 Jan 5. No abstract available.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of Immune Deficiency, Antibody, run by University of South Florida.
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 5 inclusion and 5 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 13 Jan 2017. Status can change between updates, so confirm with the study contact before making plans.
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?
Develop and validate a disease specific Quality of Life Questionnaire for patients with primary antibody immune deficiency disease (PIDD) (time frame: one year). 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 NCT02542228, which also lists 1 linked publication.
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 13 Jan 2017.