Hemophilia aNCT06369740
Case and Use Scenario Study to Gain Knowledge on the User Needs for a Medical Device for Hemophilia a Monitoring
An observational study of Hemophilia a, run by Enzyre B.V. at 1 site in United States.
Trial summary
This observational study consists of two parts. In part one, case scenario focus groups with hemophilia A patients and healthcare providers (HCPs) will be held. This parts aims to identify potential use scenarios of a point of care (POC) in vitro medical for patients with hemophilia A. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hemophilia a
- What is being tested?
- Case scenarios - questionnaire, Case scenarios - Focus groups and Use scenarios - Usability test
- How many people
- 74 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Aug 2024to 25 Aug 2024
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients or caregivers of patients with hemophilia A of any severity (mild, moderate, severe) aged 12 years and older are considered. Patients have tbe able to speak English and have give informed consent. In patients ages 12-17, parents will also have to give informed consent. Patients are not allowed to participate in both the use scenarios and the case scenarios parts of the study. Healthcare providers specialized in hemophilia care are also included. This could be hematologists, nurse practitioners, physician assistents, nurses and pharmacists.
Who may be able to join
- Patients:
- With Hemophilia A diagnosis
- All Hemophilia A severity categories are eligible (mild, moderate, or severe)
- Aged 12 years and older
- For children aged 12 through 17 their parent must provide permission by signing the informed consent form
- Consider English their primary language (capable of communicating in English verbally and in writing)
Show all 15Show fewer
- Capable of giving informed consent or assent
- Family member(s) of patients:
- Related to a patient with Hemophilia A (mild, moderate, or severe)
- Aged 18 years and older
- Consider English their primary language (capable of communicating in English verbally and in writing)
- Capable of giving informed consent or assent
- Healthcare professionals:
- Healthcare workers specialized in hemophilia care. (e.g. hematologists, nurse practitioners, physician assistants, nurses, and pharmacists)
- Capable of giving informed consent
Who cannot join
- Patients:
- Without a diagnosis of Hemophilia A
- With acquired Hemophilia A
- Younger than 12 years of age
- Unable to communicate in English
- Incapable of giving consent or assent for themselves
Show all 32Show fewer
- Specifically for the case scenario focus group
- Unwilling to consent to voice recording
- Participating in the use scenario part of the study
- Specifically for the use scenario part of the study
- Unwilling to consent to video recording
- Participating in the case scenario part of the study
- Family members:
- No relation to a patient with Hemophilia A (mild, moderate, or severe)
- Related to a patient with acquired Hemophilia A
- Younger than 18 years of age
- Unable to communicate in English
- Incapable of giving consent or assent for themselves
- Specifically for the case scenario focus group
- Unwilling to consent to voice recording
- Participating in the use scenario part of the study
- Specifically for the use scenario part of the study
- Unwilling to consent to video recording
- Participating in the case scenario part of the study
- Healthcare professionals:
- Without any prior hematological experience
- Not licensed as hematologists, nurse practitioners, physician assistants, nurses or pharmacists
- Unable to communicate in English
- Incapable of giving their consent to participate
- Specifically for the case scenario part of the study • Unwilling to consent to voice recording
- Specifically for the use scenario part of the study
- Unwilling to consent to video recording
Trial locations and contacts
1 site in 1 country.
United States1 site
Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana, 46260
About this study
This observational study consists of two parts.
In part one, case scenario focus groups with hemophilia A patients and healthcare providers (HCPs) will be held. This parts aims to identify potential use scenarios of a point of care (POC) in vitro medical for patients with hemophilia A. The main questions it aims to answer are:
Read more
- How is coagulation lab testing for patients with hemophilia A currently organized?
- What is the interest and what are desired alternatives of a POC in-vitro diagnostic medical device for patients with hemophilia A?
Part two of the study consists of a use scenario study in which patients with hemophilia A and HCPs will evaluate two types of non-functional mock-ups of a POC in vitro medical device. The main goal of this part is to evaluate the usability of the current prototypes of the POC device.
- Conditions
- Hemophilia a
- Being tested
- Case scenarios - questionnaire, Case scenarios - Focus groups, Use scenarios - Usability test, Use scenarios - interview
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Case scenarios - questionnaireOther
Participants fill in a questionnaire on the current care of hemophilia A and envisioned use scenarios of a point-of-care diagnostic device for hemophilia A.
Case scenarios - Focus groupsOther
Participants partake in focus groups on the current care for hemophilia A, the envisioned use scenarios of point-of-care diagnostic device for hemophilia A and desired features of such a diagnostic device.
Use scenarios - Usability testOther
Participants are asked to perform basic tasks with the mock-ups with minimal instructions, they will also be given a graphic of the user interface (GUI).
Use scenarios - interviewOther
The participants will be interviewed and asked for their opinions and experiences with the mock-ups.
| Study group | Role | What they receive |
|---|---|---|
| Hemophilia A patients - case scenario focus groups | 60 patients with hemophilia A aged 12 years and older that partake in the case scenario focus groups Receives: Other: Case scenarios - questionnaire; Other: Case scenarios - Focus groups | |
| Healthcare providers - case scenario focus groups | 30 healthcare providers with experience in the field of hemophilia A that partake in the case scenario focus groups Receives: Other: Case scenarios - questionnaire; Other: Case scenarios - Focus groups | |
| Hemophilia A patients - use scenarios | 6 patients with hemophilia A aged 12 years and older that partake in the use scenarios usability evaluation Receives: Other: Use scenarios - Usability test; Other: Use scenarios - interview | |
| Healthcare providers - use scenarios | 6 healthcare providers with experience in the field of hemophilia A that partake in the use scenarios usability evaluation Receives: Other: Use scenarios - Usability test; Other: Use scenarios - interview |
What is being measured
The primary outcome is the main question the study is designed to answer.
Current management of Hemophilia A coagulation lab testing
Themes regarding the current management of Hemophilia A coagulation lab testing
Measured: One day
Potential case scenarios for a point of care in-vitro diagnostic device for hemophilia A
Measured: One day
Usability issues of the current POC in-vitro diagnostic device prototypes
Overview of identified usability issues
Measured: one day
Secondary outcomes (3)
User preferences for the point of care in-vitro diagnostic device
Measured: One day
Problems with current Hemophilia A coagulation monitoring
Measured: One day
Potential benefits of a POC device for home use, near-patient use or in clinic use
Measured: One day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2024
- Study start22 Aug 2024
- Primary completion25 Aug 2024
- Study completion25 Aug 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia aStudies of the same condition, open and under way.
- 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
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 74 participants (actual)
Sponsor and investigators
- Lead sponsor
- Enzyre B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Indiana Hemophilia &Thrombosis Center, Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06369740
- Sponsor's protocol ID
- ENZ20-2024-INDIANAPOLIS
- Status last verified
- Oct 2024
- First submitted
- 12 Apr 2024
- Last updated
- 29 Oct 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Case and Use Scenario Study to Gain Knowledge on the User Needs for a Medical Device for Hemophilia a Monitoring
- ClinicalTrials.gov ID
- NCT06369740
- Acronym / sponsor ID
- ENZ20-2024-INDIANAPOLIS
- Conditions
- Hemophilia a
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 74 participants (actual)
- Lead sponsor
- Enzyre B.V.Industry (pharma, biotech, devices)
- Collaborators
- Indiana Hemophilia &Thrombosis Center, Inc.
- Study start
- 22 Aug 2024 (actual)
- Primary completion
- 25 Aug 2024 (actual)
- Study completion
- 25 Aug 2024 (actual)
- First posted
- 17 Apr 2024 (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.
This observational study consists of two parts.
In part one, case scenario focus groups with hemophilia A patients and healthcare providers (HCPs) will be held. This parts aims to identify potential use scenarios of a point of care (POC) in vitro medical for patients with hemophilia A. The main questions it aims to answer are:
- How is coagulation lab testing for patients with hemophilia A currently organized?
- What is the interest and what are desired alternatives of a POC in-vitro diagnostic medical device for patients with hemophilia A?
Part two of the study consists of a use scenario study in which patients with hemophilia A and HCPs will evaluate two types of non-functional mock-ups of a POC in vitro medical device. The main goal of this part is to evaluate the usability of the current prototypes of the POC device.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Hemophilia A patients - case scenario focus groups | – | Other: Case scenarios - questionnaire, Other: Case scenarios - Focus groups60 patients with hemophilia A aged 12 years and older that partake in the case scenario focus groups |
| Healthcare providers - case scenario focus groups | – | Other: Case scenarios - questionnaire, Other: Case scenarios - Focus groups30 healthcare providers with experience in the field of hemophilia A that partake in the case scenario focus groups |
| Hemophilia A patients - use scenarios | – | Other: Use scenarios - Usability test, Other: Use scenarios - interview6 patients with hemophilia A aged 12 years and older that partake in the use scenarios usability evaluation |
| Healthcare providers - use scenarios | – | Other: Use scenarios - Usability test, Other: Use scenarios - interview6 healthcare providers with experience in the field of hemophilia A that partake in the use scenarios usability evaluation |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients or caregivers of patients with hemophilia A of any severity (mild, moderate, severe) aged 12 years and older are considered. Patients have tbe able to speak English and have give informed consent. In patients ages 12-17, parents will also have to give informed consent. Patients are not allowed to participate in both the use scenarios and the case scenarios parts of the study. Healthcare providers specialized in hemophilia care are also included. This could be hematologists, nurse practitioners, physician assistents, nurses and pharmacists.
Key inclusion criteria (15 listed)
- With Hemophilia A diagnosis
- All Hemophilia A severity categories are eligible (mild, moderate, or severe)
- Aged 12 years and older
- For children aged 12 through 17 their parent must provide permission by signing the informed consent form
- Consider English their primary language (capable of communicating in English verbally and in writing)
Key exclusion criteria (32 listed)
- Without a diagnosis of Hemophilia A
- With acquired Hemophilia A
- Younger than 12 years of age
- Unable to communicate in English
- Incapable of giving consent or assent for themselves
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 |
|---|---|---|
| Case scenarios - questionnaireParticipants fill in a questionnaire on the current care of hemophilia A and envisioned use scenarios of a point-of-care diagnostic device for hemophilia A. | Other | Healthcare providers - case scenario focus groups, Hemophilia A patients - case scenario focus groups |
| Case scenarios - Focus groupsParticipants partake in focus groups on the current care for hemophilia A, the envisioned use scenarios of point-of-care diagnostic device for hemophilia A and desired features of such a diagnostic device. | Other | Healthcare providers - case scenario focus groups, Hemophilia A patients - case scenario focus groups |
| Use scenarios - Usability testParticipants are asked to perform basic tasks with the mock-ups with minimal instructions, they will also be given a graphic of the user interface (GUI). | Other | Healthcare providers - use scenarios, Hemophilia A patients - use scenarios |
| Use scenarios - interviewThe participants will be interviewed and asked for their opinions and experiences with the mock-ups. | Other | Healthcare providers - use scenarios, Hemophilia A patients - use scenarios |
Primary outcome
Current management of Hemophilia A coagulation lab testing
Time frame: One day
Themes regarding the current management of Hemophilia A coagulation lab testing
Potential case scenarios for a point of care in-vitro diagnostic device for hemophilia A
Time frame: One day
Usability issues of the current POC in-vitro diagnostic device prototypes
Time frame: one day
Overview of identified usability issues
3 secondary outcomes
User preferences for the point of care in-vitro diagnostic device
Time frame: One day
Problems with current Hemophilia A coagulation monitoring
Time frame: One day
Potential benefits of a POC device for home use, near-patient use or in clinic use
Time frame: One day
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 29 Oct 2024.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 Hemophilia a, run by Enzyre B.V. at 1 site in United States.
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 15 inclusion and 32 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 29 Oct 2024. 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 United States. 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?
Current management of Hemophilia A coagulation lab testing (time frame: One day). 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?
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 29 Oct 2024.