Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Aplastic Anemia Clinical Trial

— ePRO-AA-PNH  

Official title:

Electronic Patient-reported Outcome Monitoring in Patients With Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria - a Pilot Study

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04128943
• Other study ID #: 2019-01563; me18Drexler
• Status: Completed
• Start date: November 1, 2019
• Est. completion date: July 6, 2020

Study information

• Verified date: September 2020
• Source: University Hospital, Basel, Switzerland
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 9
• Est. completion date: July 6, 2020
• Est. primary completion date: July 6, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Aplastic Anemia
• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria

Intervention

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
• 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
• 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.
• 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

Locations

Country	Name	City	State
Switzerland	Division of Hematology, University Hospital Basel	Basel

Sponsors (3)

Lead Sponsor	Collaborator
University Hospital, Basel, Switzerland	Novartis, ProPatient foundation of the University Hospital Basel

Country where clinical trial is conducted

Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	recruitment with the ePRO application (number)	Analyses of usability of the ePRO application by assessing recruitment with the ePRO application	Day 1= Day of inclusion
Primary	usability of the ePRO application	Descriptive analyses of usability of the ePRO application by assessing data collection with the ePRO application	Weekly assessments from Day 1= Day of inclusion until Day 180 (+/- 7 days) = last day of testing phase
Primary	acceptability of the ePRO application	Descriptive analyses of acceptability of the ePRO application by assessing app utilization	Weekly assessments from Day 1= Day of inclusion until Day 180 (+/- 7 days) = last day of testing phase
Secondary	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)	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.	at day 1= Day of inclusion and at day 180 (+/- 7 days) = last day of testing phase