A Proof-of-concept Study to Reduce Treatment Burden in Haemophilia Participants Receiving Factor VIII and Factor IX Infusions
- Conditions
- Hemophilia
- Interventions
- Device: Virtual-reality (VR) Based Solution
- Registration Number
- NCT05437211
- Lead Sponsor
- Takeda
- Brief 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
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 24
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description VR-based Experimental Group Virtual-reality (VR) Based Solution Participants will receive Factor VIII or Factor IX infusion using a VR-based solution. The VR-based solution will be provided in a medical device CE marked and specifically developed by Deepsen, a French company specializing in e-health solutions to reduce pain. It includes both a mobile phone application (for an explanation on infusions) and a 3D mask to be used during infusions.
- Primary Outcome Measures
Name Time Method Change from Baseline in Anxiety of Participants and Relatives Using an Anxiety Visual Analogue Scale (A-VAS) Score Before Each Factor VIII or Factor IX Infusion Baseline up to Week 4 The anxiety of participants and relatives before each Factor VIII or Factor IX infusion will be measured with a 10-points VAS. The A-VAS is a 10 centimeter (cm) line with anchor statements on the left (no anxiety) and on the right (extreme anxiety). An increase in score from 0 to 10 indicates a worsening of anxiety.
Change from Baseline in Anxiety of Participants and Relatives Using an A-VAS Score After Each Factor VIII or Factor IX Infusion Baseline up to Week 4 The anxiety of participants and relatives after each Factor VIII or Factor IX infusion will be measured with a 10-points VAS. The A-VAS is a 10 cm line with anchor statements on the left (no anxiety) and on the right (extreme anxiety). An increase in score from 0 to 10 indicates a worsening of anxiety.
Percent Change from Baseline in Anxiety of Participants and Relatives Using an A-VAS Score After Each Factor VIII or Factor IX Infusion Baseline up to Week 4 The anxiety of participants and relatives after each Factor VIII or Factor IX infusion will be measured with a 10-points VAS. The A-VAS is a 10 cm line with anchor statements on the left (no anxiety) and on the right (extreme anxiety). An increase in score from 0 to 10 indicates a worsening of anxiety.
Number of Participants and Relatives With a Decrease of 2 Points out of 10 Points in the A-VAS Score After Each Factor VIII or Factor IX Infusion Up to Week 4 The A-VAS is a 10 cm line with anchor statements on the left (no anxiety) and on the right (extreme anxiety). An increase in score from 0 to 10 indicates a worsening of anxiety. The number of participants and relatives with a decrease of 2 points out of 10 points will be assessed.
Change from Baseline in Quality of Life of Participants and Relatives as Measured by European Quality of Life 5 Dimensions 3-levels (EQ-5D-3L) Questionnaire Score at Week 4 Baseline, Week 4 EQ-5D-3L health questionnaire is a participant/relative answered questionnaire scoring 5 dimensions - mobility, self-care, usual activities, pain/discomfort and anxiety/depression. The status of each dimension had 3 possible responses (1 =no problem, 2 =some problem 3 =severe problems) in the relevant health dimension. Higher score indicated a worsening health condition. The change in quality of life of participants and relatives will be assessed.
Number of Participants and Relatives with a Decrease of at Least one Level of Anxiety State Severity in the State-Trait Anxiety Inventory-State Type (STAI-S) Scale Week 4 Severity is defined as a score on STAI-S 20-35 (very low) 36-45 (low) 46-55 (moderate) 56-65 (high) 66-80 (very high). The STAI is a commonly used measure of trait and state anxiety. STAI is a psychological inventory based on a 4-point Likert scale and consists of 40 questions on a self-report basis. The STAI measures two types of anxiety - state anxiety or anxiety about an event, and trait anxiety or anxiety level as a personal characteristic. Scores range from 20 to 80, with higher scores correlating with greater anxiety. The 4-point scale for S-anxiety is as follows: 1) not at all, 2) somewhat, 3) moderately so, 4) very much so. The number of participants and relatives with a decrease of at least one level of anxiety state severity will be assessed.
Change from Baseline in Pain of Participants Using Pain Visual Analogue Scale (P-VAS) Score After Each Factor VIII or Factor IX Infusion Baseline up to Week 4 The pain of participants after each Factor VIII or Factor IX infusion will be measured with a 10-points VAS. The P-VAS is a 10 cm line with anchor statements on the left (no pain) and on the right (extreme pain). An increase in score from 0 to 10 indicates a worsening of pain.
Number of Participants and Relatives with a Decrease of 2 Points in the EQ-5D-3L VAS Score Week 4 The EQ-5D-3L VAS score is a self-assigned rating of overall health using a visual, vertical scale, with a score of 0 as the worst and 100 as the best possible health. The decrease of 2 points in the EQ-5D VAS score represents a worsening in the quality of life for participants and relatives.
Percent Change from Baseline in Quality of Life of Participants as Measured by EQ-5D-3L- Youth Version Questionnaire Score at Week 4 Baseline, Week 4 The EQ-5D-3L-Y contains one question for each of the five dimensions: mobility, looking after myself, doing usual activities, having pain or discomfort, and feeling worried, sad, or unhappy. Participants select from three response levels (no problems, some problems, a lot of problems) for each dimension. A health state profile score can be calculated from the responses on these five questions. The reference to a high score indicates a better outcome of quality of life.
Number of Participants with High Adherence Score (At Score 4) Using MMAS-4 Scale at Week 4 Week 4 The Morisky Medication Adherence Scale in the original version (MMAS-4) is a structured self-report measure of medication-taking behavior. It consists of 4 items, with binary scoring (yes/no). Participants score one point for every 'Yes' answer. A score of 0 to 1 indicates low adherence; a score of 2 to 3 indicates medium adherence, and a score of 4 indicates high adherence. Number of participants with score at 4 on the scale will be assessed.
Number of Participants with a Decrease of 2 Points in the EQ-5D-Y VAS Score Week 4 The EQ-5D-Y VAS score is a self-assigned rating of overall health using a visual, vertical scale, with a score of 0 as the worst and 100 as the best possible health. The decrease of 2 points in the EQ-5D VAS score represents a worsening in the quality of life for participants.
Percent Change from Baseline in Anxiety of Participants and Relatives Using an A-VAS Score Before Each Factor VIII or Factor IX Infusion Baseline up to Week 4 The anxiety of participants and relatives before each Factor VIII or Factor IX infusion will be measured with a 10-points VAS. The A-VAS is a 10 cm line with anchor statements on the left (no anxiety) and on the right (extreme anxiety). An increase in score from 0 to 10 indicates a worsening of anxiety.
Number of Participants and Relatives with a Decrease of 2 Points out of 10 Points in the A-VAS Score Before Each Factor VIII or Factor IX Infusion Up to Week 4 The A-VAS is a 10 cm line with anchor statements on the left (no anxiety) and on the right (extreme anxiety). An increase in score from 0 to 10 indicates a worsening of anxiety. The number of participants and relatives with a decrease of 2 points out of 10 points will be assessed.
Percent Change from Baseline in Pain of Participants Using P-VAS Score After Each Factor VIII or Factor IX Infusion Baseline up to Week 4 The pain of participants after each Factor VIII or Factor IX infusion will be measured with a 10-points VAS. The P-VAS is a 10 cm line with anchor statements on the left (no pain) and on the right (extreme pain). An increase in score from 0 to 10 indicates a worsening of pain.
Percent Change from Baseline in Quality of Life of Participants and Relatives as Measured by EQ-5D-3L Questionnaire Score at Week 4 Baseline, Week 4 EQ-5D-3L health questionnaire is a participant/relative answered questionnaire scoring 5 dimensions - mobility, self-care, usual activities, pain/discomfort and anxiety/depression. The status of each dimension had 3 possible responses (1 =no problem, 2 =some problem 3 =severe problems) in the relevant health dimension. Higher score indicated a worsening health condition. The percent change in quality of life of participants and relatives will be assessed.
Change from Baseline in Quality of Life of Participants as Measured by EQ-5D-3L- Youth Version Questionnaire Score at Week 4 Baseline, Week 4 The EQ-5D-3L-Y contains one question for each of the five dimensions: mobility, looking after myself, doing usual activities, having pain or discomfort, and feeling worried, sad, or unhappy. Participants select from three response levels (no problems, some problems, a lot of problems) for each dimension. A health state profile score can be calculated from the responses on these five questions. The reference to a high score indicates a better outcome of quality of life.
Number of Participants With a Decrease of 2 Points out of 10 Points in the P-VAS Score After Each Factor VIII or Factor IX Infusion Up to Week 4 The P-VAS is a 10 cm line with anchor statements on the left (no pain) and on the right (extreme pain). An increase in score from 0 to 10 indicates a worsening of pain. The number of participants with a decrease of 2 points out of 10 points will be assessed.
Change from Baseline in Anxiety of Participants and Relatives Using State-Trait Anxiety Inventory (STAI-Y) Scale Scores at Week 4 Baseline, Week 4 The STAI is a commonly used measure of trait and state anxiety. STAI is a psychological inventory based on a 4-point Likert scale and consists of 40 questions on a self-report basis. The STAI measures two types of anxiety - state anxiety or anxiety about an event, and trait anxiety or anxiety level as a personal characteristic. Scores range from 20 to 80, with higher scores correlating with greater anxiety. Each measure has a different rating scale. The 4-point scale for S-anxiety is as follows: 1) not at all, 2) somewhat, 3) moderately so, 4) very much so. The 4-point scale for T-anxiety is as follows: 1) almost never, 2) sometimes, 3.) often, 4) almost always. The change in the anxiety of participants and relatives will be assessed.
Percent Change from Baseline in Depression of Participants and Relatives Using Patient Health Questionnaire (Module 9 for Depression) (PHQ-9) Depression Severity Scale Scores at Week 4 Baseline, Week 4 Level of depression severity will be assessed at Week 4 in participants and relatives after inclusion with the PHQ-9 scale. PHQ-9 consists of scores from the nine-item questionnaire ranging from 0 - 27, where 0-5 (none) 5-10 (mild) 10-15 (moderate) 15-20 (moderately severe) 20-27 (severe). Higher scores indicate greater severity of depression. The percent change in depression of participants and relatives will be assessed.
Number of Participants and Relatives with a Decrease of at Least one level of Depression Severity in the PHQ-9 Scale Scores Week 4 Severity is defined as a score on PHQ-9: 0-5 (none) 5-10 (mild) 10-15 (moderate) 15-20 (moderately severe) 20-27 (severe). PHQ-9 consists of scores from the nine-item questionnaire ranging from 0 - 27. Higher scores indicate greater severity of depression. Participants and relatives with a decrease of at least one level of depression severity will be assessed.
Percent Change from Baseline in Anxiety of Participants and Relatives Using STAI-Y Scale Scores at Week 4 Baseline, Week 4 The STAI is a commonly used measure of trait and state anxiety. STAI is a psychological inventory based on a 4-point Likert scale and consists of 40 questions on a self-report basis. The STAI measures two types of anxiety - state anxiety or anxiety about an event, and trait anxiety or anxiety level as a personal characteristic. Scores range from 20 to 80, with higher scores correlating with greater anxiety. Each measure has a different rating scale. The 4-point scale for S-anxiety is as follows: 1) not at all, 2) somewhat, 3) moderately so, 4) very much so. The 4-point scale for T-anxiety is as follows: 1) almost never, 2) sometimes, 3.) often, 4) almost always. The percent change in anxiety of participants and relatives will be assessed.
Change from Baseline in Depression of Participants and Relatives Using Patient Health Questionnaire (Module 9 for Depression) (PHQ-9) Depression Severity Scale Scores at Week 4 Baseline, Week 4 Level of depression severity will be assessed at Week 4 in participants and relatives after inclusion with the PHQ-9 scale. PHQ-9 consists of scores from the nine-item questionnaire ranging from 0 - 27, where 0-5 (none) 5-10 (mild) 10-15 (moderate) 15-20 (moderately severe) 20-27 (severe). Higher scores indicate greater severity of depression. The change in depression of participants and relatives will be assessed.
Change from Baseline in Adherence to Infusions Using 4-Item Morisky Medication Adherence Scale (MMAS-4) Scores Baseline, Week 4 The Morisky Medication Adherence Scale in the original version (MMAS-4) is a structured self-report measure of medication-taking behavior. It consists of 4 items, with binary scoring (yes/no). Participants score one point for every 'Yes' answer. A score of 0 to 1 indicates low adherence; a score of 2 to 3 indicates medium adherence, and a score of 4 indicates high adherence. The change in adherence to infusions of participants will be assessed.
Change from Baseline in Class of Adherence to Infusions Using MMAS-4 Scale at Week 4 Baseline, Week 4 The MMAS-4 is a structured self-report measure of medication-taking behavior. It consists of 4 items, with binary scoring (yes/no). Participants score one point for every 'Yes' answer. A score of 0 to 1 indicates low adherence; a score of 2 to 3 indicates medium adherence, and a score of 4 indicates high adherence.
Number of Participants with Low, Medium and High Adherence to Virtual-reality Based Solution Using MMAS-4 Scale at Week 4 Week 4 The Morisky Medication Adherence Scale in the original version (MMAS-4) is a structured self-report measure of medication-taking behavior. It consists of 4 items, with binary scoring (yes/no). Participants score one point for every 'Yes' answer. A score of 0 to 1 indicates low adherence; a score of 2 to 3 indicates medium adherence, and a score of 4 indicates high adherence.
Number of Participants, Relatives, and Caregivers with Points 3 or More in Patients' Global Impression of Change (PGIC) Scale at Week 4 Week 4 The PGIC is a 7-point Likert scale, a verbal rating scale that asks the respondent to best describe change in symptoms compared to the beginning of study. Participants/relatives/caregivers select from scale range of 1-7: very much improved (1); much improved (2); minimally improved (3); no change (4); minimally worse (5); much worse (6); very much worse (7). Number of participants, relatives, and caregivers with points 3 or more on the scale will be assessed.
- Secondary Outcome Measures
Name Time Method Number of Participants, Caregivers and Relatives with Satisfaction, Willingness to Continue the Virtual-reality Based Solution Using a 4-point Likert Scale Week 4 A 4-point Likert scale was used to assess the number of participants, caregivers, and relatives with a score of 3 or 4 (satisfied or very satisfied) with the use of the virtual-reality-based system. The Likert scale is composed from 1=never satisfied to 4=very satisfied.
Number of Participants, Caregivers and Relatives with Preference for the Virtual-reality Based Solution Using a Binary Question Week 4 Preference for the virtual-reality based solution in participants, caregivers and relatives will be assessed using a binary question: Preference for the virtual-reality based solution versus no virtual-reality based solution.
Incidents with the Virtual-reality Based Solution Week 4 An incident is a minor hardware and/or software event that prevents the correct use of the solution in its context. The incidents are mainly associated with misuse by the user of the solution or with a software and/or hardware failure. A total number of incidents reported throughout the clinical study will be assessed.
Incidents of Adverse Events (AEs) Related to the Infusions of Factor VIII or Factor IX or Virtual-reality Based Solution, and Serious Adverse Events (SAEs) Up to Week 4 An AE is any untoward medical occurrence in a participant administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. AEs related to infusions of Factor VIII or Factor IX or use of virtual-reality based solution will be assessed. An SAE is any untoward medical occurrence that at any dose: results in death, is a life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect or any other medically important event that, in the opinion of the healthcare provider, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition above.
Trial Locations
- Locations (6)
Hôpital de Hautepierre - Pôle MIRMED
🇫🇷Strasbourg, Alsace-Champagne-Ardenne-Lorraine, France
Hôpital Hôtel-Dieu
🇫🇷Nantes, Pays De La Loire, France
CHU Pellegrin
🇫🇷Bordeaux, Nouvelle-Aquitaine, France
CHU Estaing
🇫🇷Clermont Ferrand, Auvergne-Rhône-Alpes, France
Centre de Reference Hemophilie Unite D'Hemostase Clinique
🇫🇷Bron, Auvergne-Rhône-Alpes, France
CHU Purpan
🇫🇷Toulouse, Haute-Garonne, France