Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy

Study Overview
=================
Brief Summary
-----------------
The primary objective of the study is to evaluate anxiety level during intrathecal administration (IT) under standard of care (SOC) and virtual reality (VR) conditions using a reliable self-rating scale.

Official Title
-----------------
A Randomized Crossover Pragmatic Study to Evaluate Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Patients With Spinal Muscular Atrophy. REALITY Study.

Conditions
-----------------
Spinal Muscular Atrophy (SMA)

Intervention / Treatment
-----------------
* Device: Virtual reality
* Procedure: Standard of Care


Participation Criteria
=================
Eligibility Criteria
-----------------
Key Inclusion Criteria: Participants aged 7 years and greater Genetically documented spinal muscular atrophy linked to chromosome 5q (5q-SMA) Loading nusinersen dose period is completed, and ongoing treatment with nusinersen Ability of the candidate and/or their legally authorized representatives (e.g., parent, spouse, or legal guardian), as appropriate and applicable, to understand the purpose and risks of the study, to provide informed consent, and to authorize the use of confidential health information in accordance with national and local privacy regulations Signed written informed consent from adult participants, or from legal authorized representatives for minors Key Exclusion Criteria: History of any clinically significant abnormalities that would render the candidate unsuitable for VR (e.g., visual or hearing impairment, presence of eye, face or sclap injuries) or for inclusion (e.g. cognitive impairment), as determined by the Investigator Conditions that could be exacerbated by the VR environment, such as: (i) current symptomatic nausea, vomiting, dizziness, migraine; (ii) history of psychosis, hallucinations, epilepsy Ongoing medical conditions or treatments that according to the Investigator would interfere with the conduct and assessments of the study (e.g. general anesthesia) Participants wearing a pacemaker and pregnant woman NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Ages Eligible for Study
-----------------
Minimum Age: 7 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Supportive Care
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: VR followed by SOC<br>Participants will receive a 20-minute (min) VR session at the first nusinersen IT (IT1), followed by SOC (local anesthesia - lidocaine or intravenous sedation) at the subsequent nusinersen IT (IT2) during the main study, later followed by 20-min VR sessions at two subsequent nusinersen ITs (IT3 and IT4) during the extension period, for up to 450 days. | Device: Virtual reality<br>* Administered as specified in the treatment arm.<br>Procedure: Standard of Care<br>* Administered as specified in the treatment arm.<br>|
| Experimental: SOC followed by VR<br>Participants will receive SOC (local anesthesia - lidocaine or intravenous sedation) at nusinersen IT1, followed by a 20-min VR session at the subsequent nusinersen IT (IT2) during the main study, later followed by 20-min VR sessions at two subsequent nusinersen ITs (IT3 and IT4) during the extension period, for up to 450 days. | Device: Virtual reality<br>* Administered as specified in the treatment arm.<br>Procedure: Standard of Care<br>* Administered as specified in the treatment arm.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Visual Analog Scale for Anxiety (VAS-A) Score During IT as Evaluated by the Participant Just After IT | The VAS-A scale is used in the assessment of anxiety. The 101-point scale (0 to 100) is a horizontal 100 millimeter (mm) line that starts with no anxiety on the left and ends with maximum anxiety on the right. The VAS score is determined by measuring in mm from the left-hand end of the line to the point that the participant marks. High scores on the scale indicate that anxiety is high. | Up to 450 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Blood Pressure Before and After IT | Systolic and diastolic blood pressures will be assessed. | Up to 450 days | 
| Heart Rate Before and After IT |  | Up to 450 days | 
| Short State-Trait Anxiety Inventory (Short STAI) Score During IT as Evaluated by the Participant Just After IT | The 6-item short form of the Spielberger STAI is used to measure self-reported symptoms of state anxiety. It consists of 6 questions each having scale 1 (Almost Never) to 4 (Almost Always). The score range for the short STAI is 6 to 24 points, with 6 points signifying no anxiety and 24 points signifying the highest level of anxiety. | Up to 450 days | 
| Number of Participants With Use of Medication/Intervention for Anxiety and Pain Management Before, During and up to 72 hour (h) After IT |  | Up to 450 days | 
| Visual Analog Scale for Pain (VAS-P) Score Just After IT | The VAS-P scale is used in the assessment of pain. The 101 point scale (0 to 100) is a horizontal 100 mm line that starts with no pain on the left and ends with maximum pain on the right. The VAS score is determined by measuring in mm from the left hand end of the line to the point that the participant marks. High scores indicate worse pain. | Up to 450 days | 
| Maximal VAS-P Score Within 72h of IT | The VAS-P scale is used in the assessment of pain. The 101 point scale (0 to 100) is a horizontal 100 mm line that starts with no pain on the left and ends with maximum pain on the right. The VAS score is determined by measuring in mm from the left hand end of the line to the point that the participant marks. High scores indicate worse pain. | Up to 450 days | 
| Number of Participants With Satisfactory Experience of Using VR, Desire to Continue IT, Desire to Continue VR | Participants will be asked to complete a satisfaction survey to evaluate his/her experience. It is a questionnaire consisting of 6 (for age >12 years) to 7 (for age <12 years) questions. | Up to 150 days | 
| Number of Participants With Satisfactory Experience of Using VR, as Assessed by Custom Care Team | The custom care team will be asked to complete a satisfaction survey to evaluate participant's experience. It is a questionnaire consisting of 5 questions. | Up to 150 days |