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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

Primary Purpose

Hemophilia A

Status
Recruiting
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
Virtual-reality (VR) Based Solution
Sponsored by
Takeda
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional supportive care trial for Hemophilia A

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • Centre de Reference Hemophilie Unite D'Hemostase CliniqueRecruiting
  • CHU Estaing
  • CHU Purpan
  • CHU Pellegrin
  • CHU Saint Eloi

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

VR-based Experimental Group

Arm Description

Participants will receive Factor VIII 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.

Outcomes

Primary Outcome Measures

Change from Baseline in Anxiety of Participants and Relatives Using an Anxiety Visual Analogue Scale (A-VAS) Score Before Each Factor VIII Infusion
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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

Number of Participants, Caregivers and Relatives with Satisfaction, Willingness to Continue the Virtual-reality Based Solution Using a 4-point Likert Scale
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
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
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)
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.

Full Information

First Posted
June 24, 2022
Last Updated
August 10, 2023
Sponsor
Takeda
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1. Study Identification

Unique Protocol Identification Number
NCT05437211
Brief Title
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
Official Title
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
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
November 24, 2022 (Actual)
Primary Completion Date
November 30, 2023 (Anticipated)
Study Completion Date
November 30, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Takeda

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A

7. Study Design

Primary Purpose
Supportive Care
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
34 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
VR-based Experimental Group
Arm Type
Experimental
Arm Description
Participants will receive Factor VIII 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.
Intervention Type
Device
Intervention Name(s)
Virtual-reality (VR) Based Solution
Intervention Description
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).
Primary Outcome Measure Information:
Title
Change from Baseline in Anxiety of Participants and Relatives Using an Anxiety Visual Analogue Scale (A-VAS) Score Before Each Factor VIII Infusion
Description
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.
Time Frame
Baseline up to Week 4
Title
Percent Change from Baseline in Anxiety of Participants and Relatives Using an A-VAS Score Before Each Factor VIII Infusion
Description
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.
Time Frame
Baseline up to Week 4
Title
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
Description
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.
Time Frame
Up to Week 4
Title
Change from Baseline in Anxiety of Participants and Relatives Using an A-VAS Score After Each Factor VIII Infusion
Description
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.
Time Frame
Baseline up to Week 4
Title
Percent Change from Baseline in Anxiety of Participants and Relatives Using an A-VAS Score After Each Factor VIII Infusion
Description
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.
Time Frame
Baseline up to Week 4
Title
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
Description
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.
Time Frame
Up to Week 4
Title
Change from Baseline in Pain of Participants Using Pain Visual Analogue Scale (P-VAS) Score After Each Factor VIII Infusion
Description
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.
Time Frame
Baseline up to Week 4
Title
Percent Change from Baseline in Pain of Participants Using P-VAS Score After Each Factor VIII Infusion
Description
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.
Time Frame
Baseline up to Week 4
Title
Number of Participants With a Decrease of 2 Points out of 10 Points in the P-VAS Score After Each Factor VIII Infusion
Description
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.
Time Frame
Up to Week 4
Title
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
Description
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.
Time Frame
Baseline, Week 4
Title
Percent Change from Baseline in Quality of Life of Participants and Relatives as Measured by EQ-5D-3L Questionnaire Score at Week 4
Description
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.
Time Frame
Baseline, Week 4
Title
Number of Participants and Relatives with a Decrease of 2 Points in the EQ-5D-3L VAS Score
Description
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.
Time Frame
Week 4
Title
Change from Baseline in Quality of Life of Participants as Measured by EQ-5D-3L- Youth Version Questionnaire Score at Week 4
Description
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.
Time Frame
Baseline, Week 4
Title
Percent Change from Baseline in Quality of Life of Participants as Measured by EQ-5D-3L- Youth Version Questionnaire Score at Week 4
Description
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.
Time Frame
Baseline, Week 4
Title
Number of Participants with a Decrease of 2 Points in the EQ-5D-Y VAS Score
Description
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.
Time Frame
Week 4
Title
Change from Baseline in Anxiety of Participants and Relatives Using State-Trait Anxiety Inventory (STAI-Y) Scale Scores at Week 4
Description
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.
Time Frame
Baseline, Week 4
Title
Percent Change from Baseline in Anxiety of Participants and Relatives Using STAI-Y Scale Scores at Week 4
Description
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.
Time Frame
Baseline, Week 4
Title
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
Description
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.
Time Frame
Week 4
Title
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
Description
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.
Time Frame
Baseline, Week 4
Title
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
Description
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.
Time Frame
Baseline, Week 4
Title
Number of Participants and Relatives with a Decrease of at Least one level of Depression Severity in the PHQ-9 Scale Scores
Description
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.
Time Frame
Week 4
Title
Change from Baseline in Adherence to Infusions Using 4-Item Morisky Medication Adherence Scale (MMAS-4) Scores
Description
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.
Time Frame
Baseline, Week 4
Title
Number of Participants with Low Adherence Scores (3 or 4) Using MMAS-4 Scale at Week 4
Description
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.
Time Frame
Week 4
Title
Number of Participants with Low Adherence to Virtual-reality Based Solution Using MMAS-4 Scale at Week 4
Description
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.
Time Frame
Week 4
Title
Number of Participants, Relatives, and Caregivers with Points 3 or More in Patients' Global Impression of Change (PGIC) Scale at Week 4
Description
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.
Time Frame
Week 4
Secondary Outcome Measure Information:
Title
Number of Participants, Caregivers and Relatives with Satisfaction, Willingness to Continue the Virtual-reality Based Solution Using a 4-point Likert Scale
Description
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.
Time Frame
Week 4
Title
Number of Participants, Caregivers and Relatives with Preference for the Virtual-reality Based Solution Using a Binary Question
Description
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.
Time Frame
Week 4
Title
Incidents with the Virtual-reality Based Solution
Description
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.
Time Frame
Week 4
Title
Incidents of Adverse Events (AEs) Related to the Infusions of Factor VIII or Virtual-reality Based Solution, and Serious Adverse Events (SAEs)
Description
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.
Time Frame
Up to Week 4

10. Eligibility

Sex
Male
Gender Based
Yes
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion criteria: Male participant over the age of 6 years, with diagnosed Congenital Hemophilia A, whatever the severity is Participant under long-term prophylaxis with intravenous Factor VIII infusions Participant with an available web connection for anxiety and pain self-evaluation during follow-up 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 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Takeda Contact
Phone
+1-877-825-3327
Email
medinfoUS@takeda.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
Centre de Reference Hemophilie Unite D'Hemostase Clinique
City
Bron
State/Province
Auvergne-Rhône-Alpes
ZIP/Postal Code
69677
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sandrine Meunier, MD
Phone
(0)4 72 11 88 10
Ext
+33
Email
Sandrine.meunier@chu-lyon.fr
Facility Name
CHU Estaing
City
Clermont Ferrand
State/Province
Auvergne-Rhône-Alpes
ZIP/Postal Code
69003
Country
France
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Aurelien Lebreton, MD
Phone
(0)4 73 75 00 81
Ext
+33
Email
alebreton@chu-clermontferrand.fr
Facility Name
CHU Purpan
City
Toulouse
State/Province
Haute-Garonne
ZIP/Postal Code
31059
Country
France
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ségolène Claeyssens, MD
Phone
(0)5 61 77 68 03
Ext
+33
Email
Claeyssens.s@chu-toulouse.fr
Facility Name
CHU Pellegrin
City
Bordeaux
State/Province
Nouvelle-Aquitaine
ZIP/Postal Code
33076
Country
France
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sabine Castet, MD
Phone
(0)5 56 79 48 79
Ext
+33
Email
Sabine-marie.castet@chu-bordeaux.fr
Facility Name
CHU Saint Eloi
City
Montpellier cedex 5
State/Province
Occitanie
ZIP/Postal Code
34295
Country
France
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Christine Biron, MD
Phone
(0)4 67 33 77 71
Ext
+33
Email
c-biron@chu-montpellier.fr

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
IPD Sharing Access Criteria
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/ For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
IPD Sharing URL
https://vivli.org/ourmember/takeda/
Links:
URL
https://clinicaltrials.takeda.com/study-detail/dad25f4820fb4885?idFilter=%5B%22TAK-660-4013%22%5D
Description
To obtain more information on the study, click here/on this link.

Learn more about this trial

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

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