A Proof-of-concept Study to Reduce Treatment Burden in Haemophilia Participants Receiving Factor VIII and Factor IX Infusions
- Conditions
- Hemophilia
- 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
- Primary Outcome Measures
Name Time Method 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 At Week 4 The anxiety of participants and caregivers before each Factor VIII or Factor IX infusion was measured with a 10-points VAS. The A-VAS is a 10-centimeter (cm) line with anchor statements on the left (0:no anxiety) and on the right (10: extreme anxiety). An increase in score from 0 to 10 indicated a worsening of anxiety.
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 Baseline up to Week 4 The anxiety of participants and caregivers before each Factor VIII or Factor IX infusion was measured with a 10-points VAS. The A-VAS is a 10 cm line with anchor statements on the left (0:no anxiety) and on the right (10: extreme anxiety). An increase in score from 0 to 10 indicated a worsening of anxiety. Participants are categorized based on their A-VAS score values at both Baseline (-5, -3, 0, and 9.1) and Week 4 (-5, -2.3, -1, 0 and 2). The A-VAS score reported within each category represent the mean difference between the score collected after and before infusion (after minus before). A negative value indicates an improvement in anxiety compared to the baseline score, while a positive value indicates a worsening of anxiety compared to the baseline score.
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 At Week 4 The anxiety of participants and caregivers before each Factor VIII or Factor IX infusion was measured with a 10-points VAS. The A-VAS is a 10 cm line with anchor statements on the left (0:no anxiety) and on the right (10: extreme anxiety). An increase in score from 0 to 10 indicated a worsening of anxiety.
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 At Week 4 The pain of participants after each Factor VIII or Factor IX infusion was measured with a 10-points VAS. The P-VAS is a 10 cm line with anchor statements on the left (0: no pain) and on the right (10: extreme pain). An increase in score from 0 to 10 indicated a worsening of pain.
Number of Participants With Absolute Variation of the Pain Measured by a P-VAS Scale 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 was measured with a 10-points VAS. The P-VAS is a 10 cm line with anchor statements on the left (0: no pain) and on the right (10: extreme pain). An increase in score from 0 to 10 indicated a worsening of pain. Participants are categorized based on P-VAS score values at both Baseline (0, 1.3, 2, 4 and 5) and Week 4 (0, 0.2, 0.4, 5 and 8). A positive value indicates worsening in pain compared to the baseline score.
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 Baseline up to Week 4 The pain of participants after each Factor VIII or Factor IX infusion was measured with a 10-points VAS. The P-VAS is a 10 cm line with anchor statements on the left (0: no pain) and on the right (10: extreme pain). An increase in score from 0 to 10 indicated a worsening of pain.
Mean Absolute Values of Anxiety Measured by an A-VAS Scale After Each Factor VIII or Factor IX Infusion in Participants and Caregivers At Week 4 The anxiety of participants and caregivers before each Factor VIII or Factor IX infusion was measured with a 10-points VAS. The A-VAS is a 10 cm line with anchor statements on the left (0:no anxiety) and on the right (10: extreme anxiety). An increase in score from 0 to 10 indicated a worsening of anxiety.
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 Baseline up to Week 4 The anxiety of participants and caregivers before each Factor VIII or Factor IX infusion was measured with a 10-points VAS. The A-VAS is a 10 cm line with anchor statements on the left (0:no anxiety) and on the right (10: extreme anxiety). An increase in score from 0 to 10 indicated a worsening of anxiety and negative value indicated worst health.
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 At Week 4 The EQ-5D-3L is a standardized instrument for use as a measure of health outcome and was administered to adult participants to assess the effect of the treatment on the participants' quality of life. Adult participants selected answer for each of the following 3-level dimensions: 1) mobility; 2) self-care; 3) usual activities; 4) pain/discomfort; 5) anxiety/depression used to compute an index score ranging from 0 (worst imaginable health state) to 1 (best imaginable health state). An increase in the EQ-5D-3L index score indicated improvement and negative value indicated worst health.
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 At Week 4 The EQ-5D-3L is a standardized instrument for use as a measure of health outcome and was administered to adult participants to assess the effect of the treatment on the participants' quality of life. Adult participants selected answer for each of the following 3-level dimensions: 1) mobility; 2) self-care; 3) usual activities; 4) pain/discomfort; 5) anxiety/depression used to compute an index score ranging from 0 (worst imaginable health state) to 1 (best imaginable health state). An increase in the EQ-5D-3L index score indicated improvement and negative value indicated worst health. Relative variation from baseline at visit (%) = 100 \* Absolute change from baseline at visit (in units) / value at baseline (in units).
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 At Week 4 The EQ-5D-Y is a standardized instrument for use as a measure of health outcome and was administered to youth participants to assess the effect of the treatment on the participants' quality of life. Youth participants selected answer for each of the following 3-level dimensions: 1) mobility; 2) self-care; 3) usual activities; 4) pain/discomfort; 5) anxiety/depression used to compute an index score ranging from 0 (worst imaginable health state) to 1 (best imaginable health state). An increase in the EQ-5D-Y index score indicated improvement and negative value indicated worst health.
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 At Week 4 The EQ-5D-Y is a standardized instrument for use as a measure of health outcome and was administered to youth participants to assess the effect of the treatment on the participants' quality of life. Youth participants selected answer for each of the following 3-level dimensions: 1) mobility; 2) self-care; 3) usual activities; 4) pain/discomfort; 5) anxiety/depression used to compute an index score ranging from 0 (worst imaginable health state) to 1 (best imaginable health state). An increase in the EQ-5D-Y index score indicated improvement and negative value indicated worst health. Relative variation from baseline at visit (%) = 100 \* Absolute change from baseline at visit (in units) / value at baseline (in units).
Number of Participants and Caregivers With a Decrease of 2 Points in the EQ-5D-3L VAS and EQ-5D-Y Score At Week 4 EQ-5D-3L health questionnaire is adult participant and caregivers and EQ-5D-Y is a youth participant 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 =extreme problems) in the relevant health dimension. Higher score indicated a worsening health condition.
Absolute Variation of All Participants Anxiety Measured by State-Trait Anxiety Inventory (STAI-Y) Score At Week 4 The STAI is a psychological inventory consisting of 40 self-report items on a 4-point Likert scale. The STAI measures two types of anxiety - state anxiety (STAI-Y1) and trait anxiety (STAI-Y2). Higher scores were positively correlated with higher levels of anxiety. Each type of anxiety has its own scale of 20 different questions and were rated on a 4-point scale (e.g. from 'Almost Never to 'Almost Always'). Low scores indicated a mild form of anxiety, and high scores indicated a severe form of anxiety. STAI score ranged from 20 to 80 where higher scores indicated greater anxiety and negative value indicated worst anxiety.
Relative Variation of All Participants Anxiety Measured by State-Trait Anxiety Inventory (STAI-Y) Score At Week 4 The STAI is a psychological inventory consisting of 40 self-report items on a 4-point Likert scale. The STAI measures two types of anxiety - state anxiety (STAI-Y1) and trait anxiety (STAI-Y2). Higher scores were positively correlated with higher levels of anxiety. Each type of anxiety has its own scale of 20 different questions and were rated on a 4-point scale (e.g. from 'Almost Never to 'Almost Always'). Low scores indicated a mild form of anxiety, and high scores indicated a severe form of anxiety. STAI score ranged from 20 to 80 where higher scores indicated greater anxiety and negative value indicated worst anxiety. Relative variation from baseline at visit (%) = 100 \* Absolute change from baseline at visit (in units) / value at baseline (in units).
Number of All Participants With Decrease of at Least One Level of State-Trait Anxiety Inventory Severity (STAI-S) At Week 4 The STAI-S is a psychological assessment tool used to measure the level of anxiety a person is experiencing at a particular moment in time that assesses both State anxiety (STAI-Y1) and Trait anxiety (STAI-Y2). Each type of anxiety has its own scale of 20 different questions each that are scored on a 4-point scale evaluating how respondents feel "right now, at this moment" (e.g. from 'Almost Never to 'Almost Always'). Total scores for each (Y1 and Y2) range from 20 to 80, with higher scores correlating with greater anxiety. Severity for each STAI-Y1 and STAI-Y2 is defined by the scores on STAI-S: 20-35 (very low) 36-45 (low) 46-55 (moderate) 56-65 (high) 66-80 (very high). Number of participants with decrease of at least one level of State-Trait Anxiety Inventory- Severity (STAI-S) are reported.
Absolute Variation of Participants Depression Measured by Patient Health Questionnaire (Module 9) (PHQ-9) Depression Severity At Week 4 The PHQ-9 measures self-reports of depressive symptoms within the prior 2 weeks and has been well-validated, shown high test-retest reliability, shown high internal consistency, and shown to be responsive to changes in treatment. Response options were on a 4-point scale (0 = not at all, 3 = nearly every day). The overall PHQ-9 score ranged from 0 to 27 where 0-5 (none) 5-10 (mild) 10-15 (moderate) 15-20 (moderately severe) 20-27 (severe). Higher values represented more severe depression status.
Relative Variation of Participants Depression Measured by Patient Health Questionnaire (Module 9) (PHQ-9) Depression Severity At Week 4 The PHQ-9 measures self-reports of depressive symptoms within the prior 2 weeks and has been well-validated, shown high test-retest reliability, shown high internal consistency, and shown to be responsive to changes in treatment. Response options were on a 4-point scale (0 = not at all, 3 = nearly every day). The overall PHQ-9 score ranged from 0 to 27 where 0-5 (none) 5-10 (mild) 10-15 (moderate) 15-20 (moderately severe) 20-27 (severe). Higher values represented more severe depression status. Relative variation from baseline at visit (%) = 100 \* Absolute change from baseline at visit (in units) / value at baseline (in units).
Number of Participants With Decrease of at Least One Level of Depression Severity Measured by PHQ-9 Scale Baseline up to Week 4 The PHQ-9 measures self-reports of depressive symptoms within the prior 2 weeks and has been well-validated, shown high test-retest reliability, shown high internal consistency, and shown to be responsive to changes in treatment. Response options were on a 4-point scale (0 = not at all, 3 = nearly every day). The overall PHQ-9 score ranged from 0 to 27 where 0-5 (none) 5-10 (mild) 10-15 (moderate) 15-20 (moderately severe) 20-27 (severe). Higher values represented more severe depression status.
Number of Participants With Absolute Adherence to Infusions Measured by 4-Item Morisky Medication Adherence Scale (MMAS-4) Scores Baseline up to Week 4 The Morisky Medication Adherence Scale in the original version (MMAS-4) is a structured self-report measure of medication-taking behaviour. It consisted of 4 items, with binary scoring (yes/no). Participants scored one point for every 'Yes' answer. A score of 0 to 1 indicated low adherence; a score of 2 to 3 indicated medium adherence, and a score of 4 indicated high adherence.
Number of Participants With Changes in Adherence to Infusions Measured by MMAS-4 by Classes Baseline up to Week 4 The Morisky Medication Adherence Scale in the original version (MMAS-4) is a structured self-report measure of medication-taking behaviour. It consisted of 4 items, with binary scoring (yes/no). Participants scored one point for every 'Yes' answer. A score of 0 to 1 indicated low adherence; a score of 2 to 3 indicated medium adherence, and a score of 4 indicated high adherence.
Number of Participants Who Scored 4 (High Adherence) Using MMAS-4 Scale at Week 4 At Week 4 The Morisky Medication Adherence Scale in the original version (MMAS-4) is a structured self-report measure of medication-taking behavior. It consisted of 4 items, with binary scoring (yes/no). Participants scored one point for every 'Yes' answer. A score of 0 to 1 indicated low adherence; a score of 2 to 3 indicated medium adherence, and a score of 4 indicated high adherence.
Number of Participants and Caregivers With Adherence to VR-Based Solutions at Week 4 At Week 4 Adherence to the VR-based solution was assessed at Week 4 using the MMAS. The Morisky Medication Adherence Scale in the original version (MMAS-4) is a structured self-report measure of medication-taking behaviour. It consisted of 4 items, with binary scoring (yes/no). Participants scored one point for every 'Yes' answer. A score of 0 to 1 indicated low adherence; a score of 2 to 3 indicated medium adherence, and a score of 4 indicated high adherence.
Number of Participants With a Patients' Global Impression of Change (PGIC) Score Greater Than (>) 3 Among Participants and Caregivers At Week 4 The PGIC is a 7-point Likert scale, a verbal rating scale that asked the respondent to best describe change in symptoms compared to the beginning of study. Participants/caregivers selected from scale range of 1 (very much worsened) to 7 (very much improved). Higher score indicated improved outcome.
- Secondary Outcome Measures
Name Time Method 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 At Week 4 A 4-point Likert scale was used to assess the number of participants, caregivers, and investigators with a score of 3 or 4 (satisfied or very satisfied) with the use of the VR- based system. The Likert scale is composed from 1=never satisfied to 4=very satisfied.
Number of Participants With Preference and No-preference for the VR-Based Solution Using a Binary Question Among Participants and Caregivers At Week 4 Preference for the VR-based solution in participants and caregivers was assessed using a binary question: Yes (Preference for the VR-based solution) and No (preference for VR-based solution) was assessed.
Number of Incidents With the VR-Based Solution Baseline up to 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 were mainly associated with misuse by the user of the solution or with a software and/or hardware failure.
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 From start of study 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 VR-based solution was 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
Centre de Reference Hemophilie Unite D'Hemostase Clinique
🇫🇷Bron, Auvergne-Rhône-Alpes, France
CHU Estaing
🇫🇷Clermont Ferrand, Auvergne-Rhône-Alpes, France
CHU Purpan
🇫🇷Toulouse, Haute-Garonne, France
CHU Pellegrin
🇫🇷Bordeaux, Nouvelle-Aquitaine, France
Hôpital Hôtel-Dieu
🇫🇷Nantes, Pays De La Loire, France
Hôpital de Hautepierre - Pôle MIRMED🇫🇷Strasbourg, Alsace-Champagne-Ardenne-Lorraine, France