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

Immune Deficiency, Antibody Clinical Trial

— HR-QOLPIDD  

Official title:

Construction and Validation of a Health-related Quality of Life (HR-QOL) Instrument for Patients With Primary Antibody Deficiency Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02542228
• Other study ID #: 6119806000
• Status: Completed
• Phase: N/A
• First received: August 31, 2015
• Last updated: January 12, 2017
• Start date: March 2014
• Est. completion date: September 2016

Study information

• Verified date: January 2017
• Source: University of South Florida
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical 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). 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 76
• Est. completion date: September 2016
• Est. primary completion date: January 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Common Variable Immunodeficiency
• Deficiency Diseases
• Immune Deficiency, Antibody
• Immunologic Deficiency Syndromes

Intervention

Other:
• questionnaire survey
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.

Locations

Country	Name	City	State
n/a

Sponsors (7)

Lead Sponsor	Collaborator
University of South Florida	Boston Children’s Hospital, CSL Behring, Johns Hopkins University, Midwest Immunology Clinic, Texas Children's Hospital, University of Virginia

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	one year