Construction of a Health-related Quality of Life (HR-QOL) Questionnaire for Patients With Primary Antibody Deficiency Disease (HR-QOLPIDD)

January 12, 2017 updated by: University of South Florida

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
76

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with primary antibody immune deficiency with x-linked agammaglobulinemia and common variable immune deficiency

Description

Inclusion Criteria:

  1. Subjects with documented x-linked agammaglobulinemia, or common variable immunodeficiency, autosomal recessive agammaglobulinemia
  2. Subjects must be able to read and speak English
  3. Willing to sign consent and follow study schedule.
  4. Subjects 18 years to 65 years of age.
  5. Subject must be receiving Ig treatment for diagnosis.

Exclusion Criteria:

  1. Subject or guardian unwilling to sign consent or adhere to study schedule.
  2. Pregnancy,
  3. Severe concurrent medical conditions which would prevent treatment or assessment, including significant hematological, renal, lung or liver dysfunction or malignancies
  4. Presence of any other medical condition, which in the opinion of the investigator might interfere with performance or interpretation of this study
  5. Can not read or understand English

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
one year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
University of South Florida

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Johns Hopkins University
Baylor College of Medicine
Boston Children's Hospital
CSL Behring
University of Virginia
Midwest Immunology Clinic

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 1, 2014

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 1, 2016

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 1, 2016

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 31, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 2, 2015

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 4, 2015

Study Record Updates

Last Update Posted (Estimate)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 13, 2017

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 12, 2017

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 6119806000

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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