Aplastic AnemiaePRO-AA-PNHNCT04128943
Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
An observational study of Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, run by University Hospital, Basel, Switzerland at 1 site in Switzerland.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- symptom questionnaire, recording of vital signs and European Organisation for Research and Treatment of Cancer (EORTC) quality of life questionnaire (QLQ)-C30 questionnaire
- How many people
- 9 participants
- Where
- 1 siteSwitzerland
- Study dates
- 1 Nov 2019to 6 Jul 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: All patients treated at the University Hospital Basel for AA and/or PNH
Who may be able to join
- AA (acquired and hereditary) and/or PNH patients
- Minimal level of computer literacy with prior email experience and access to an Internet connection
Who cannot join
- Mental alteration or psychiatric disease that can compromise written informed consent or adherence to the protocol and monitoring of the trial
Trial locations and contacts
1 site in 1 country.
Switzerland1 site
Division of Hematology, University Hospital Basel
Basel, 4031
About this study
Official title: Electronic Patient-reported Outcome Monitoring in Patients With Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria - a Pilot Study
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.
- Being tested
- symptom questionnaire, recording of vital signs, European Organisation for Research and Treatment of Cancer (EORTC) quality of life questionnaire (QLQ)-C30 questionnaire, interview
- Keywords
- electronic patient-reported outcomes, electronic health (eHealth) technology, app utilization, ePRO application
- Medical subject headings
- Anemia, Aplastic, Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
symptom questionnaireOther
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 signsOther
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 questionnaireOther
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.
interviewOther
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
What is being measured
The primary outcome is the main question the study is designed to answer.
recruitment with the ePRO application (number)
Analyses of usability of the ePRO application by assessing recruitment with the ePRO application
Measured: Day 1= Day of inclusion
usability of the ePRO application
Descriptive analyses of usability of the ePRO application by assessing data collection with the ePRO application
Measured: Weekly assessments from Day 1= Day of inclusion until Day 180 (+/- 7 days) = last day of testing phase
acceptability of the ePRO application
Descriptive analyses of acceptability of the ePRO application by assessing app utilization
Measured: Weekly assessments from Day 1= Day of inclusion until Day 180 (+/- 7 days) = last day of testing phase
Secondary outcomes (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)
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.
Measured: at day 1= Day of inclusion and at day 180 (+/- 7 days) = last day of testing phase
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Oct 2019
- Study start1 Nov 2019
- Primary completion6 Jul 2020
- Study completion6 Jul 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by University Hospital, Basel, SwitzerlandThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Basel, Switzerland
- Sponsor type
- University, hospital or other
- Collaborators
- ProPatient foundation of the University Hospital Basel, Novartis
- Responsible party
- Sponsor
- Investigators
- Beatrice Drexler, Dr. med, Principal investigator, Division of Hematology, University Hospital Basel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04128943
- Sponsor's protocol ID
- 2019-01563; me18Drexler
- Other IDs
- 2019
- Acronym
- ePRO-AA-PNH
- Status last verified
- Sep 2020
- First submitted
- 15 Oct 2019
- Last updated
- 5 Oct 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Banziger S, Weisshaar K, Arokoski R, Gerull S, Halter J, Rovo A, Bargetzi M, Goede JS, Senft Y, Valenta S, Passweg JR, Drexler B. Feasibility of electronic patient-reported outcome monitoring and self-management program in aplastic anemia and paroxysmal nocturnal hemoglobinuria-a pilot study (ePRO-AA-PNH). Ann Hematol. 2023 Jan;102(1):199-208. doi: 10.1007/s00277-022-05012-5. Epub 2022 Nov 3.
PubMed 36326854
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Electronic Patient-reported Outcome Monitoring in Patients With Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria - a Pilot Study
- ClinicalTrials.gov ID
- NCT04128943
- Acronym / sponsor ID
- ePRO-AA-PNH / 2019-01563; me18Drexler
- Keywords
- electronic patient-reported outcomes, electronic health (eHealth) technology, app utilization, ePRO application
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- University Hospital, Basel, SwitzerlandUniversity, hospital or other
- Collaborators
- ProPatient foundation of the University Hospital Basel, Novartis
- Study start
- 1 Nov 2019 (actual)
- Primary completion
- 6 Jul 2020 (actual)
- Study completion
- 6 Jul 2020 (actual)
- First posted
- 16 Oct 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- All patients treated at the University Hospital Basel for AA and/or PNH
Key inclusion criteria (2 listed)
- AA (acquired and hereditary) and/or PNH patients
- Minimal level of computer literacy with prior email experience and access to an Internet connection
Key exclusion criteria (1 listed)
- Mental alteration or psychiatric disease that can compromise written informed consent or adherence to the protocol and monitoring of the trial
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| symptom questionnaireA 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 signsRecord 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 questionnaireThe 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 | – |
| interviewInterview 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 | Other | – |
Primary outcome
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
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
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
1 secondary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2020.
- Derived from this study: PubMed 36326854Banziger S, Weisshaar K, Arokoski R, Gerull S, Halter J, Rovo A, Bargetzi M, Goede JS, Senft Y, Valenta S, Passweg JR, Drexler B. Feasibility of electronic patient-reported outcome monitoring and self-management program in aplastic anemia and paroxysmal nocturnal hemoglobinuria-a pilot study (ePRO-AA-PNH). Ann Hematol. 2023 Jan;102(1):199-208. doi: 10.1007/s00277-022-05012-5. Epub 2022 Nov 3.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Beatrice Drexler, Dr. med principal investigator
Division of Hematology, University Hospital Basel
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, run by University Hospital, Basel, Switzerland at 1 site in Switzerland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Switzerland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
recruitment with the ePRO application (number) (time frame: Day 1= Day of inclusion). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04128943, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Oct 2020.