A Study to Determine How a Virtual-reality Based Solution May Reduce the Treatment Burden of Children, Teenagers and Adults With Hemophilia A Who Receive Prophylactic Factor VIII Infusions

Overview

The main aim is to describe how the treatment burden and challenges of participants, relatives, and doctors during regular Factor VIII 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 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

Full Title of Study: “Ability of a Virtual-reality Based Solution Aiming to Reduce Patient Burden Related to Repetitive Intravenous Perfusions. A Proof-of-concept Study in Hemophilia Patients Receiving Prophylactic Factor VIII Infusions”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Supportive Care
    • Masking: None (Open Label)
  • Study Primary Completion Date: September 1, 2022

Interventions

  • Device: Virtual-reality (VR) Based Solution
    • VR-based solution comprises a mobile phone application including explanation on the different steps to be followed to perform infusions in satisfactory conditions, notably as regards safety, as well as VAS for pain and anxiety to be completed at each Factor VIII infusion between inclusion and the Week 4 visit. It also comprises a 3D mask (to be used before or during infusions) including simulation of the infusions themselves (before infusion) and a relaxing and distracting content (during and after infusions).

Arms, Groups and Cohorts

  • Experimental: VR-based Experimental Group
    • Participants will receive Factor VIII infusion using a VR-based solution. The VR-based solution will be provided in a medical device Conformitè EuropĂ«enne (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.

Clinical Trial Outcome Measures

Primary Measures

  • Change from Baseline in Anxiety of Participants and Relatives Using an Anxiety Visual Analogue Scale (A-VAS) Score Before Each Factor VIII Infusion
    • Time Frame: Baseline up to Week 4
    • The anxiety of participants and relatives before each Factor VIII 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.
  • Percent Change from Baseline in Anxiety of Participants and Relatives Using an A-VAS Score Before Each Factor VIII Infusion
    • Time Frame: Baseline up to Week 4
    • The anxiety of participants and relatives before each Factor VIII 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 Infusion
    • Time Frame: 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 Anxiety of Participants and Relatives Using an A-VAS Score After Each Factor VIII Infusion
    • Time Frame: Baseline up to Week 4
    • The anxiety of participants and relatives after each Factor VIII 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 Infusion
    • Time Frame: Baseline up to Week 4
    • The anxiety of participants and relatives after each Factor VIII 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 Infusion
    • Time Frame: 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 Pain of Participants Using Pain Visual Analogue Scale (P-VAS) Score After Each Factor VIII Infusion
    • Time Frame: Baseline up to Week 4
    • The pain of participants after each Factor VIII 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 Pain of Participants Using P-VAS Score After Each Factor VIII Infusion
    • Time Frame: Baseline up to Week 4
    • The pain of participants after each Factor VIII 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 With a Decrease of 2 Points out of 10 Points in the P-VAS Score After Each Factor VIII Infusion
    • Time Frame: 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 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
    • Time Frame: 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.
  • Percent Change from Baseline in Quality of Life of Participants and Relatives as Measured by EQ-5D-3L Questionnaire Score at Week 4
    • Time Frame: 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.
  • Number of Participants and Relatives with a Decrease of 2 Points in the EQ-5D-3L VAS Score
    • Time Frame: 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.
  • Change from Baseline in Quality of Life of Participants as Measured by EQ-5D-3L- Youth Version Questionnaire Score at Week 4
    • Time Frame: 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.
  • Percent Change from Baseline in Quality of Life of Participants as Measured by EQ-5D-3L- Youth Version Questionnaire Score at Week 4
    • Time Frame: 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 in the EQ-5D-Y VAS Score
    • Time Frame: 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.
  • Change from Baseline in Anxiety of Participants and Relatives Using State-Trait Anxiety Inventory (STAI-Y) Scale Scores at Week 4
    • Time Frame: 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 Anxiety of Participants and Relatives Using STAI-Y Scale Scores at Week 4
    • Time Frame: 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.
  • 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
    • Time Frame: 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 Depression of Participants and Relatives Using Patient Health Questionnaire (Module 9 for Depression) (PHQ-9) Depression Severity Scale Scores at Week 4
    • Time Frame: 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.
  • 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
    • Time Frame: 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
    • Time Frame: 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.
  • Change from Baseline in Adherence to Infusions Using 4-Item Morisky Medication Adherence Scale (MMAS-4) Scores
    • Time Frame: 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 indicates high adherence; a score of 1 or 2 indicates intermediate adherence, and a score of 3 or 4 indicates low adherence. The change in adherence to infusions of participants will be assessed.
  • Number of Participants with Low Adherence Scores (3 or 4) Using MMAS-4 Scale at Week 4
    • Time Frame: 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 indicates high adherence; a score of 1 or 2 indicates intermediate adherence, and a score of 3 or 4 indicates low adherence. Number of participants with scores of 3 or 4 on the scale will be assessed.
  • Number of Participants with Low Adherence to Virtual-reality Based Solution Using MMAS-4 Scale at Week 4
    • Time Frame: 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 indicates high adherence; a score of 1 or 2 indicates intermediate adherence, and a score of 3 or 4 indicates low adherence. Number of participants with scores 3 or 4 on the scale will be assessed.
  • Number of Participants, Relatives, and Caregivers with Points 3 or More in Patients’ Global Impression of Change (PGIC) Scale at Week 4
    • Time Frame: 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 Measures

  • Number of Participants, Caregivers and Relatives with Satisfaction, Willingness to Continue the Virtual-reality Based Solution Using a 4-point Likert Scale
    • Time Frame: 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
    • Time Frame: 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
    • Time Frame: 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 Virtual-reality Based Solution, and Serious Adverse Events (SAEs)
    • Time Frame: Up to Week 4
    • An AE is any untoward medical occurrence in a subject 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 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.

Participating in This Clinical Trial

Inclusion Criteria

1. Male participant over the age of 6 years, with diagnosed Congenital Hemophilia A, whatever the severity is 2. Participant under long-term prophylaxis with intravenous Factor VIII infusions 3. Participant with an available web connection for anxiety and pain self-evaluation during follow-up 4. 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 5. Participant affiliated to the national social security or beneficiary to such insurance Exclusion criteria:

1. Participant with known or suspected hypersensitivity to virtual-reality based tools 2. Participant with central venous line for the administration of Factor VIII 3. Participant (and the legal guardians if participant age <18 years) with history of unreliability or non-cooperation (including for completion of self-reported questionnaires) 4. Participant (and the legal guardians if participant age <18 years) with insufficient comprehension of French language 5. Participant taking part in another clinical trial 6. Participant deprived of his liberty by judicial or administrative order

Gender Eligibility: Male

Minimum Age: 6 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Takeda
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Study Director, Study Director, Takeda
  • Overall Contact(s)
    • Takeda Contact, +1-877-825-3327, medinfoUS@takeda.com

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