Clinical trial NCT05437211: A Proof-of-concept Study to Reduce Treatment Burden in Haemophilia Participants Receiving Factor VIII and Factor IX 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 or Factor IX Infusions
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Hemophilia
Keywords: Drug Therapy
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2022-06-29
Enrollment: 24
Sex: MALE
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

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


Eligibility criteria: Inclusion criteria:

1. Male participant over the age of 6 years, with diagnosed Congenital Hemophilia A or B, whatever the severity is
2. Participant under long-term prophylaxis with intravenous Factor VIII or Factor IX infusions
3. 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
4. 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 or Factor IX
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

NB: participants with haemophilia A having participated in the test phase of the study could be included in the clinical study itself after a period of 4 months minimum with no use of the virtual-reality based solution.

Primary outcomes:
measure: 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
timeFrame: At Week 4
measure: 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
timeFrame: Baseline up to Week 4
measure: 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
timeFrame: At Week 4
measure: 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
timeFrame: At Week 4
measure: Number of Participants With Absolute Variation of the Pain Measured by a P-VAS Scale After Each Factor VIII or Factor IX Infusion
timeFrame: Baseline up to Week 4
measure: 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
timeFrame: Baseline up to Week 4
measure: Mean Absolute Values of Anxiety Measured by an A-VAS Scale After Each Factor VIII or Factor IX Infusion in Participants and Caregivers
timeFrame: At Week 4
measure: 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
timeFrame: Baseline up to Week 4
measure: 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
timeFrame: At Week 4
measure: 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
timeFrame: At Week 4
measure: 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
timeFrame: At Week 4
measure: 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
timeFrame: At Week 4
measure: Number of Participants and Caregivers With a Decrease of 2 Points in the EQ-5D-3L VAS and EQ-5D-Y Score
timeFrame: At Week 4
measure: Absolute Variation of All Participants Anxiety Measured by State-Trait Anxiety Inventory (STAI-Y) Score
timeFrame: At Week 4
measure: Relative Variation of All Participants Anxiety Measured by State-Trait Anxiety Inventory (STAI-Y) Score
timeFrame: At Week 4
measure: Number of All Participants With Decrease of at Least One Level of State-Trait Anxiety Inventory Severity (STAI-S)
timeFrame: At Week 4
measure: Absolute Variation of Participants Depression Measured by Patient Health Questionnaire (Module 9) (PHQ-9) Depression Severity
timeFrame: At Week 4
measure: Relative Variation of Participants Depression Measured by Patient Health Questionnaire (Module 9) (PHQ-9) Depression Severity
timeFrame: At Week 4
measure: Number of Participants With Decrease of at Least One Level of Depression Severity Measured by PHQ-9 Scale
timeFrame: Baseline up to Week 4
measure: Number of Participants With Absolute Adherence to Infusions Measured by 4-Item Morisky Medication Adherence Scale (MMAS-4) Scores
timeFrame: Baseline up to Week 4
measure: Number of Participants With Changes in Adherence to Infusions Measured by MMAS-4 by Classes
timeFrame: Baseline up to Week 4
measure: Number of Participants Who Scored 4 (High Adherence) Using MMAS-4 Scale at Week 4
timeFrame: At Week 4
measure: Number of Participants and Caregivers With Adherence to VR-Based Solutions at Week 4
timeFrame: At Week 4
measure: Number of Participants With a Patients' Global Impression of Change (PGIC) Score Greater Than (>) 3 Among Participants and Caregivers
timeFrame: At Week 4
Secondary outcomes:
measure: 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
timeFrame: At Week 4
measure: Number of Participants With Preference and No-preference for the VR-Based Solution Using a Binary Question Among Participants and Caregivers
timeFrame: At Week 4
measure: Number of Incidents With the VR-Based Solution
timeFrame: Baseline up to Week 4
measure: 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
timeFrame: From start of study up to Week 4
Other outcomes:
Locations:
city: Strasbourg
country: France
facility: Hôpital de Hautepierre - Pôle MIRMED
state: Alsace-Champagne-Ardenne-Lorraine
city: Bron
country: France
facility: Centre de Reference Hemophilie Unite D'Hemostase Clinique
state: Auvergne-Rhône-Alpes
city: Clermont Ferrand
country: France
facility: CHU Estaing
state: Auvergne-Rhône-Alpes
city: Toulouse
country: France
facility: CHU Purpan
state: Haute-Garonne
city: Bordeaux
country: France
facility: CHU Pellegrin
state: Nouvelle-Aquitaine
city: Nantes
country: France
facility: Hôpital Hôtel-Dieu
state: Pays De La Loire
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: