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Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (ePRO-AA-PNH)

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ClinicalTrials.gov Identifier: NCT04128943
Recruitment Status : Completed
First Posted : October 16, 2019
Last Update Posted : October 5, 2020
Sponsor:
Collaborators:
ProPatient foundation of the University Hospital Basel
Novartis
Information provided by (Responsible Party):
University Hospital, Basel, Switzerland

Brief Summary:
Aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are interrelated and very rare diseases. Therefore, little data about clinical characteristics, especially the variety of symptoms in the course of the respective disease are available. As a consequence, patients may be left on their own between infrequent follow-ups at a specialist center. A web-based symptom-monitoring application can support selfmanagement and patient empowerment and promotes a patient- centered interdisciplinary team approach in the context of a "disease management program". This pilot study is to investigate usability and feasibility of the electronic Patient-Reported Outcome (ePRO) application in AA/PNH by assessing recruitment, app utilization, data collection, functionality, acceptability after using and working with the ePRO application.

Condition or disease Intervention/treatment
Aplastic Anemia Paroxysmal Nocturnal Hemoglobinuria Other: symptom questionnaire Other: recording of vital signs Other: European Organisation for Research and Treatment of Cancer (EORTC) quality of life questionnaire (QLQ)-C30 questionnaire Other: interview

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Study Type : Observational
Actual Enrollment : 9 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Electronic Patient-reported Outcome Monitoring in Patients With Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria - a Pilot Study
Actual Study Start Date : November 1, 2019
Actual Primary Completion Date : July 6, 2020
Actual Study Completion Date : July 6, 2020



Intervention Details:
  • Other: symptom questionnaire

    A weekly filled in symptom questionnaire for reported symptoms of AA and/or PNH (yes/no, grade, date). Grading according to current grading systems (Common Terminology Criteria for Adverse Events,CTCAE).

    Data collection and processing via the ePRO application by Kaiku Health Ltd

  • Other: recording of vital signs
    Record function for vital parameters (blood pressure, temperature and pulse); Data collection and processing via the ePRO application by Kaiku Health Ltd
  • Other: European Organisation for Research and Treatment of Cancer (EORTC) quality of life questionnaire (QLQ)-C30 questionnaire
    The EORTC QLQ-C30 incorporates nine multi-item scales: five functional scales (Physical, Role, Cognitive, Emotional and Social Functioning); three symptom scales (Fatigue, Pain and Nausea/Vomiting); and a Global Health Status/QoL scale. Six single item scales are also included (Dyspnoea, Insomnia, Appetite Loss, Constipation, Diarrhoea and Financial Difficulties). All of the scales range in score from 0 to 100. A high score for a functional scale represents a high/healthy level of functioning whereas a high score for a symptom scale or item represents a high level of symptomatology or problems.
  • Other: interview
    Interview with the patients and responsible medical team by phone or during hospital visit about functional problems regarding the application (duration: 15-30 min) to avoid incorrect use of the application, at week 3 and month 3 and month 6


Primary Outcome Measures :
  1. recruitment with the ePRO application (number) [ Time Frame: Day 1= Day of inclusion ]
    Analyses of usability of the ePRO application by assessing recruitment with the ePRO application

  2. usability of the ePRO application [ Time Frame: Weekly assessments from Day 1= Day of inclusion until Day 180 (+/- 7 days) = last day of testing phase ]
    Descriptive analyses of usability of the ePRO application by assessing data collection with the ePRO application

  3. acceptability of the ePRO application [ Time Frame: Weekly assessments from Day 1= Day of inclusion until Day 180 (+/- 7 days) = last day of testing phase ]
    Descriptive analyses of acceptability of the ePRO application by assessing app utilization


Secondary Outcome Measures :
  1. Change in quality of life assessed by the European Organisation for Research and Treatment of Cancer (EORTC) quality of life questionnaire (QLQ)-C30 questionnaire (EORTC QLQ-C30 questionnaire) [ Time Frame: at day 1= Day of inclusion and at day 180 (+/- 7 days) = last day of testing phase ]
    Change in quality of life assessed by the EORTC QLQ-C30 questionnaire. The EORTC QLQ-C30 incorporates nine multi-item scales: five functional scales (Physical, Role, Cognitive, Emotional and Social Functioning); three symptom scales (Fatigue, Pain and Nausea/Vomiting); and a Global Health Status/QoL scale. Six single item scales are also included (Dyspnoea, Insomnia, Appetite Loss, Constipation, Diarrhoea and Financial Difficulties). All of the scales range in score from 0 to 100. A high score for a functional scale represents a high/healthy level of functioning whereas a high score for a symptom scale or item represents a high level of symptomatology or problems.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
All patients treated at the University Hospital Basel for AA and/or PNH
Criteria

Inclusion Criteria:

  • AA (acquired and hereditary) and/or PNH patients
  • Minimal level of computer literacy with prior email experience and access to an Internet connection

Exclusion Criteria:

  • Mental alteration or psychiatric disease that can compromise written informed consent or adherence to the protocol and monitoring of the trial

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04128943


Locations
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Switzerland
Division of Hematology, University Hospital Basel
Basel, Switzerland, 4031
Sponsors and Collaborators
University Hospital, Basel, Switzerland
ProPatient foundation of the University Hospital Basel
Novartis
Investigators
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Principal Investigator: Beatrice Drexler, Dr. med Division of Hematology, University Hospital Basel
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Responsible Party: University Hospital, Basel, Switzerland
ClinicalTrials.gov Identifier: NCT04128943    
Other Study ID Numbers: 2019-01563; me18Drexler
First Posted: October 16, 2019    Key Record Dates
Last Update Posted: October 5, 2020
Last Verified: September 2020

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by University Hospital, Basel, Switzerland:
electronic patient-reported outcomes
electronic health (eHealth) technology
app utilization
ePRO application
Additional relevant MeSH terms:
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Hemoglobinuria
Anemia
Anemia, Aplastic
Hemoglobinuria, Paroxysmal
Hematologic Diseases
Bone Marrow Diseases
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Anemia, Hemolytic
Myelodysplastic Syndromes