NCT05437211

A Proof-of-concept Study to Reduce Treatment Burden in Haemophilia Participants Receiving Factor VIII and Factor IX Infusions

Completed NA Results posted Last updated 5 June 2025

What this trial tests

NA trial testing Virtual-reality (VR) Based Solution in Hemophilia in 24 participants. Completed in 21 February 2024.

Timeline

23 June 2023

Primary endpoint
21 February 2024

21 February 2024

Quick facts

Lead sponsor	Takeda
Phase	NA
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	supportive care
Enrollment	24
Start date	23 June 2023
Primary completion	21 February 2024
Estimated completion	21 February 2024
Sites	6 locations across France

Drugs / interventions tested

• Virtual-reality (VR) Based Solution

Conditions studied

• Hemophilia — all drugs for Hemophilia →

Sponsor

Takeda — full company profile →

Who can join

6 and older, male only, with Hemophilia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

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 Primary · 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.

A-VAS before infusion For Participants

Group	Value	95% CI
VR-based Experimental Group: With Autonomy	0.2	± 0.4

A-VAS before infusion For Caregivers

Group	Value	95% CI
VR-based Experimental Group: With Autonomy	1.5	± 2.0
VR-based Experimental Group: Without Autonomy	0.0	± 0.0

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 Primary · 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 n

Participants with variation of the anxiety VAS values between Baseline (-5) and Week 4 (-5)

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	0

Participants with variation of the anxiety VAS values between Baseline (-5) and Week 4 (-2.3)

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	1

Participants with variation of the anxiety VAS values between Baseline (-5) and Week 4 (-1)

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	1

Participants with variation of the anxiety VAS values between Baseline (-5) and Week 4 (0)

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	2

Participants with variation of the anxiety VAS values between Baseline (-5) and Week 4 (2)

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	0

Participants with variation of the anxiety VAS values between Baseline (-3) and Week 4 (-5)

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	0

Participants with variation of the anxiety VAS values between Baseline (-3) and Week 4 (-2.3)

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	0

Participants with variation of the anxiety VAS values between Baseline (-3) and Week 4 (-1)

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	0

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 Primary · 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.

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	5

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 Primary · 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.

Group	Value	95% CI
VR-based Experimental Group: With Autonomy	1.4	± 2.7
VR-based Experimental Group: Without Autonomy	0.6	± 1.7

Number of Participants With Absolute Variation of the Pain Measured by a P-VAS Scale After Each Factor VIII or Factor IX Infusion Primary · 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.

Participants with absolute variation of the P-VAS values between Baseline (0) and Week 4 (0)

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	13

Participants with absolute variation of the P-VAS values between Baseline (0) and Week 4 (0.2)

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	0

Participants with absolute variation of the P-VAS values between Baseline (0) and Week 4 (0.4)

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	0

Participants with absolute variation of the P-VAS values between Baseline (0) and Week 4 (5)

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	1

Participants with absolute variation of the P-VAS values Baseline (0) and Week 4 (8)

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	1

Participants with absolute variation of the P-VAS values between Baseline (1.3) and Week 4 (0)

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	1

Participants with absolute variation of the P-VAS values between Baseline (1.3) and Week 4 (0.2)

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	0

Participants with absolute variation of the P-VAS values between Baseline (1.3) and Week 4 (0.4)

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	0

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 Primary · 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.

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	4

Mean Absolute Values of Anxiety Measured by an A-VAS Scale After Each Factor VIII or Factor IX Infusion in Participants and Caregivers Primary · 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.

A-VAS after infusion for participants

Group	Value	95% CI
VR-based Experimental Group: With Autonomy	0.2	± 0.4

A-VAS after infusion for caregivers

Group	Value	95% CI
VR-based Experimental Group: With Autonomy	0.8	± 2.0
VR-based Experimental Group: Without Autonomy	0.0	± 0.0

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 Primary · 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.

Group	Value	95% CI
Pooled Arm: VR-based Experimental Group	2

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 Primary · 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.

Absolute Variation for adult participants

Group	Value	95% CI
VR-based Experimental Group: With Autonomy	0.0	± 0.2
VR-based Experimental Group: Without Autonomy	-0.0	± 0.1

Absolute Variation for caregivers

Group	Value	95% CI
VR-based Experimental Group: With Autonomy	0.3	± 0.1
VR-based Experimental Group: Without Autonomy	-0.0	± 0.1

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 Primary · 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

Relative Variation for adult participants

Group	Value	95% CI
VR-based Experimental Group: With Autonomy	7.1	± 32.7
VR-based Experimental Group: Without Autonomy	-5.1	± 7.2

Relative Variation for caregivers

Group	Value	95% CI
VR-based Experimental Group: With Autonomy	61.0	± 34.8
VR-based Experimental Group: Without Autonomy	-2.0	± 11.8

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 Primary · 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.

Group	Value	95% CI
VR-based Experimental Group: With Autonomy	-0.1	± 0.2
VR-based Experimental Group: Without Autonomy	-0.1	± 0.3

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 Primary · 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 va

Group	Value	95% CI
VR-based Experimental Group: With Autonomy	-21.2	± 35.3
VR-based Experimental Group: Without Autonomy	-12.8	± 32.8

Adverse events — posted to ClinicalTrials.gov

Time frame: From start of study up to Week 4. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

VR-based Experimental Group: With Autonomy

Serious: 0/14 (0%)

Deaths: 0/14

VR-based Experimental Group: Without Autonomy

Serious: 0/10 (0%)

Deaths: 0/10

Other adverse events (2 terms — click to expand)

Reaction	System	VR-based Experimental Grou…	VR-based Experimental Grou…
Headache	Nervous system disorders	—	—
Neck pain	Musculoskeletal and connective tissue disorders	—	—

Data from ClinicalTrials.gov NCT05437211 adverse events section.

Sponsor's own description

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

Publications & conference data

No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.