N/A
Completed N=24
A Proof-of-concept Study to Reduce Treatment Burden in Haemophilia Participants Receiving Factor VIII and Factor IX Infusions
Hemophilia
Source: ClinicalTrials.gov NCT05437211 ↗
Enrolled (actual)
24
Serious AEs
0.0%
Results posted
Jun 2025
Primary outcomePrimary: Mean Absolute Values of Anxiety Measured by an Anxiety Visual Analogue Scale (A-VAS) Before Each Factor VIII or Factor IX Infusion in Participants and Caregivers — 0.2; 1.5; 0.0 score on a scale
Summary
The main aim is to describe how the treatment burden and challenges of participants, relatives, and doctors during regular Factor VIII and Factor IX infusions to treat hemophilia evolve using a virtual-reality (VR) based solution. The study also aims to find out how well the VR based solution is tolerated and how satisfied the participants, relatives, and doctors are with it.
The VR-based solution includes a mobile phone app and a 3D mask.
Participants will use the VR-based solution while receiving prophylactic Factor VIII and Factor IX infusions for 4 weeks at home. Participants will visit the clinic for the last infusion; at this time data will be collected from participants, relatives, and doctors
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Absolute Values of Anxiety Measured by an Anxiety Visual Analogue Scale (A-VAS) Before Each Factor VIII or Factor IX Infusion in Participants and Caregivers |
0.2; 1.5; 0.0 | — |
| PRIMARY Number of Participants With Relative Variation in Anxiety Measured by an Anxiety Visual Analogue Scale (A-VAS) Before and After Each Factor VIII or Factor IX Infusion |
0; 1; 1; 2; 0; 0 | — |
| PRIMARY Number of Participants With Decrease of 2 Points Out of 10 Points in the A-VAS Scale Before Each Factor VIII or Factor IX Infusion |
5 | — |
| PRIMARY Mean Absolute Values of the Pain Measured by a Pain Visual Analogue Scale (P-VAS) After Each Factor VIII or Factor IX Infusion in Participants |
1.4; 0.6 | — |
| PRIMARY Number of Participants With Absolute Variation of the Pain Measured by a P-VAS Scale After Each Factor VIII or Factor IX Infusion |
13; 0; 0; 1; 1; 1 | — |
| PRIMARY Number of Participants With a Decrease of 2 Points Out of 10 Points in the P-VAS Scale After Each Factor VIII or Factor IX Infusion |
4 | — |
| PRIMARY Mean Absolute Values of Anxiety Measured by an A-VAS Scale After Each Factor VIII or Factor IX Infusion in Participants and Caregivers |
0.2; 0.8; 0.0 | — |
| PRIMARY Number of Participants With a Decrease of 2 Points Out of 10 Points in the A-VAS Scale After Each Factor VIII or Factor IX Infusion |
2 | — |
| PRIMARY Absolute Variation of Caregivers and Adult Participants Quality of Life Measured With the European Quality of Life 5 Dimensions 3-Levels (EQ-5D-3L) Health State Index Score |
0.0; -0.0; 0.3; -0.0 | — |
| PRIMARY Relative Variation of Caregivers and Adult Participants Quality of Life Measured With the European Quality of Life 5 Dimensions 3-Levels (EQ-5D-3L) Health State Index Score |
7.1; -5.1; 61.0; -2.0 | — |
| PRIMARY Absolute Variation of Youth Participants Quality of Life Measured With the European Quality of Life 5 Dimensions For Youth (EQ-5D-Y) Health State Index Score |
-0.1; -0.1 | — |
| PRIMARY Relative Variation of Youth Participants Quality of Life Measured With the European Quality of Life 5 Dimensions For Youth (EQ-5D-Y) Health State Index Score |
-21.2; -12.8 | — |
| PRIMARY Number of Participants and Caregivers With a Decrease of 2 Points in the EQ-5D-3L VAS and EQ-5D-Y Score |
0; 0; 0; 0; 0; 0 | — |
| PRIMARY Absolute Variation of All Participants Anxiety Measured by State-Trait Anxiety Inventory (STAI-Y) Score |
-2.4; -1.5; -2.9; -0.5 | — |
| PRIMARY Relative Variation of All Participants Anxiety Measured by State-Trait Anxiety Inventory (STAI-Y) Score |
-6.6; -3.7; -8.1; -1.5 | — |
| PRIMARY Number of All Participants With Decrease of at Least One Level of State-Trait Anxiety Inventory Severity (STAI-S) |
3; 2; 4; 1 | — |
| PRIMARY Absolute Variation of Participants Depression Measured by Patient Health Questionnaire (Module 9) (PHQ-9) Depression Severity |
-1.3; -1.4 | — |
| PRIMARY Relative Variation of Participants Depression Measured by Patient Health Questionnaire (Module 9) (PHQ-9) Depression Severity |
-13.7; 51.9 | — |
| PRIMARY Number of Participants With Decrease of at Least One Level of Depression Severity Measured by PHQ-9 Scale |
2; 3 | — |
| PRIMARY Number of Participants With Absolute Adherence to Infusions Measured by 4-Item Morisky Medication Adherence Scale (MMAS-4) Scores |
7; 6 | — |
| PRIMARY Number of Participants With Changes in Adherence to Infusions Measured by MMAS-4 by Classes |
0; 0; 6; 3; 7; 6 | — |
| PRIMARY Number of Participants Who Scored 4 (High Adherence) Using MMAS-4 Scale at Week 4 |
7; 6 | — |
| PRIMARY Number of Participants and Caregivers With Adherence to VR-Based Solutions at Week 4 |
1; 0; 6; 5; 6; 4 | — |
| PRIMARY Number of Participants With a Patients' Global Impression of Change (PGIC) Score Greater Than (>) 3 Among Participants and Caregivers |
7; 4; 2; 2 | — |
| SECONDARY Number of Participants, With Satisfaction, Willingness to Continue the VR-Based Solution Measured by 4-point Likert Scale Score More Than and Equal to (>=3) Among Participants and Caregivers |
10; 9; 2; 4 | — |
| SECONDARY Number of Participants With Preference and No-preference for the VR-Based Solution Using a Binary Question Among Participants and Caregivers |
5; 7; 8; 2; 2; 4 | — |
| SECONDARY Number of Incidents With the VR-Based Solution |
3; 1 | — |
| SECONDARY Number of Participants With Related Adverse Events (AEs) and Serious Adverse Events (SAEs) to the Infusions of Factor VIII or Factor IX or VR-Based Solution |
0; 0; 0; 0 | — |
Eligibility Criteria
Inclusion criteria
- Male participant over the age of 6 years, with diagnosed Congenital Hemophilia A or B, whatever the severity is
- Participant under long-term prophylaxis with intravenous Factor VIII or Factor IX infusions
- Participant (or the legal guardians if participant age <18 years) able and willing to give written informed consent and to comply with the requirements of the study protocol
- Participant affiliated to the national social security or beneficiary to such insurance
Exclusion criteria
- Participant with known or suspected hypersensitivity to virtual-reality based tools
- Participant with central venous line for the administration of Factor VIII or Factor IX
- Participant (and the legal guardians if participant age <18 years) with history of unreliability or non-cooperation (including for completion of self-reported questionnaires)
- Participant (and the legal guardians if participant age <18 years) with insufficient comprehension of French language
- Participant taking part in another clinical trial
- Participant deprived of his liberty by judicial or administrative order
NB: participants with haemophilia A having participated in the test phase of the study could be included in the clinical study itself after a period of 4 months minimum with no use of the virtual-reality based solution.
Data sourced from ClinicalTrials.gov (NCT05437211). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.