Developing a Patient-Reported Outcome (PRO) Screening Measure for Infections and Measuring Quality of Life in Hematological Patients With Secondary Immunodeficiency (SID) Across the Treatment Trajectory - The PRO SID Project (PRO-SID)

February 11, 2025 updated by: Medical University Innsbruck

The goal of this observational study is to improve the treatment of hematological patients with secondary immunodeficiency (SID) by developing a patient-reported outcome (PRO) instrument to detect clinically diagnosed infections. The study focuses on adults diagnosed with chronic lymphocytic leukemia (CLL) or multiple myeloma (MM) who are at increased risk of infections due to SID.

The main questions it aims to answer are:

  1. Can a newly developed PRO screening tool for infection-related symptoms reliably detect infections in patients with SID?
  2. How does the health-related quality of life change over the treatment course

Participants will:

  • Complete daily electronic PRO questionnaires (ePRO) to monitor infection-related symptoms
  • Complete ePRO health-related quality of life questionnaires every 1.5 months
  • Participate in study visits every three months to ensure documentation of clinical data

Study Overview

Status

Recruiting

Conditions

Detailed Description

The aim of this observational study is to develop an ePRO symptom monitoring tool to identify infections among patients with MM or CLL who recently started an anticancer therapy.

This multicenter trial, conducted in participating sites in Germany in Austria, targets a total of 120 adult patients to complete daily ePRO questions in a mobile app regarding current infection-related symptoms.

Objectives:

Primary:

To develop and evaluate the diagnostic accuracy of a newly developed PRO screening measure for detecting clinically diagnosed infections in hematological patients with SID.

Secondary:

To measure QoL over time using validated instruments such as the EORTC QLQ-C30 and disease-specific modules (QLQ-MY20 for MM and QLQ-CLL17 for CLL).

Explore whether PRO-based infection detection prompts subsequent initiation of immunoglobulin replacement therapy (IgRT) and examine QoL before and after its initiation.

Exploratory Objectives:

  • To evaluate the completion and adherence rates for ePRO assessments.
  • To investigate the predictive value of the PRO screening tool for infection incidence.

To develop a reliable item list to monitor and detect infections, it is necessary to establish a high-quality measurement of infections as criterion. We developed the item list based on items from the European Organisation for Research and Treatment of Cancer (EORTC) Item Library and in line with international best-practice recommendations for item list development. The development comprised a comprehensive review of the literature and existing measures measuring infection-related symptoms, as well as physician input on which domains and symptoms were the most relevant to assess to monitor for infections in this population.

Clinicians will also document any diagnosed infections and relevant clinical data throughout study visits, which take place every three months. These include updates on treatment regimens, disease staging, and infection history.

The app was developed based on the Computer-based Health Evaluation System (CHES) software platform, which allowed for real-time symptom tracking and data integration. The software has been used extensively and also been tested with patients with hematological diseases in the past. For the PRO-SID app, we developed study-specific content, which comprised information in lay language on the diseases (MM and CLL), information on infections and SID, and information on the study itself. The native app (iOS and Android) prompts patients to complete the item list once daily via push-reminders (up to two reminders).

Usability of the app and understanding of the item list within the daily ePRO assessment on infection symptoms has been assessed within a designed feasibility study according to best practice guidelines (data to be published separately). After completion of the feasibility study, we implemented changes to the app based on patients' feedback and answers in the evaluation questionnaires in order to improve the app for the final study.

Patient Safety and Ethics:

The study is conducted in compliance with the Declaration of Helsinki and Good Clinical Practice standards. Informed consent is obtained, and all participant data is de-identified and securely stored to ensure confidentiality.

Study Type

Observational

Enrollment (Estimated)

120

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Austria
      • Graz, Austria, 8010
        • Not yet recruiting
        • Medizinische Universitat Graz
        • Contact:
      • Kufstein, Austria, 6330
        • Not yet recruiting
        • BKH Kufstein
        • Contact:
    • Tyrolia
      • Innsbruck, Tyrolia, Austria, 6020
        • Recruiting
        • Medizinische Universitat Innsbruck
        • Contact:
  • Germany
      • Berlin, Germany, 14195
        • Not yet recruiting
        • Onkologischer Schwerpunkt am Oskar-Helene-Heim
        • Contact:
      • Garmisch-Partenkirchen, Germany, 82467
        • Not yet recruiting
        • Klinikum Garmisch-Partenkirchen
        • Contact:

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

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Probability Sample

Study Population

The study population are subjects with MM or CLL and a SID who have recently started a anticancer therapy in Germany and Austria.

Description

Inclusion Criteria:

  • Adult patient (>=18 years of age)
  • Access to an internet device (e.g., personal computer or tablet to use a web-based platform, or smartphone where the study app can be installed - all common iOS and Android systems)
  • German-speaking
  • Diagnosis of multiple myeloma or chronic lymphocytic leukemia
  • Secondary immunodeficiency (defined as: recurrent infections, infections requiring inpatient treatment, hypogammaglobulinemia, neutropenia and/or lymphopenia on differential blood cell counts, deficit in lymphocyte subsets as assessed by flow cytometry)

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

Cohorts and Interventions

Group / Cohort
Patients with MM or CLL at risk of infectious complications due to SID

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Diagnostic accuracy of the ePRO monitoring instrument for infection-related symptoms
Time Frame: From enrollment until study discontinuation or up until study completion (day 365), which ever occurs first
Evaluation of diagnostic accuracy of the daily ePRO screening measure to detect infections as diagnosed by a clinician (CTCAE).
From enrollment until study discontinuation or up until study completion (day 365), which ever occurs first

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Changes in health-related quality of life measured with the EORTC QLQ-C30
Time Frame: From enrollment until study discontinuation or up until study completion (day 365), which ever occurs first
Changes in health-related quality of life over the study period (from inclusion to six and 12 months) measured with the EORTC Core Quality of Life questionnaire (EORTC QLQ-C30).
From enrollment until study discontinuation or up until study completion (day 365), which ever occurs first
Changes in health-related quality of life measured with the EORTC QLQ-MY20
Time Frame: From enrollment until study discontinuation or up until study completion (day 365), which ever occurs first
Changes in health-related quality of life over the study period (from inclusion to six and 12 months) measured with the EORTC disease-specific module QLQ-MY20 - a 20 item instrument designed to assess the quality of life of patients with multiple myeloma.
From enrollment until study discontinuation or up until study completion (day 365), which ever occurs first
2. Changes in health-related quality of life measured with the EORTC QLQ-CLL17
Time Frame: From enrollment until study discontinuation or up until study completion (day 365), which ever occurs first
Description: Changes in health-related quality of life over the study period (from inclusion to six and 12 months) measured with the EORTC disease-specific module QLQ-CLL17 - a 17 item instrument designed to assess the quality of life of patients with chronic lymphocytic leukemia.
From enrollment until study discontinuation or up until study completion (day 365), which ever occurs first

Collaborators and Investigators

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

Sponsor

Medical University Innsbruck

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 (Actual)

February 3, 2025

Primary Completion (Estimated)

July 1, 2026

Study Completion (Estimated)

July 1, 2026

Study Registration Dates

First Submitted

January 23, 2025

First Submitted That Met QC Criteria

February 11, 2025

First Posted (Actual)

March 25, 2025

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

February 11, 2025

Last Verified

January 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 1179/2024
  • ISR-2022-200331 (Other Grant/Funding Number: Takeda Pharma Ges.m.b.H and Takeda Pharma Vertrieb GmbH & Co. KG as Investigator Initiated Research)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Sharing Time Frame

Beginning with publication and ending 10 years after the publication of results

IPD Sharing Access Criteria

Open access available

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

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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