Clinical trial NCT04784390: Proof of Concept Study of Binocular Videogames Versus Patching for Amblyopia
Official title: A 16 Week Randomized, Single-masked, Multicenter Proof of Concept Study of Binocular Videogames Versus Patching for Amblyopia in Children 4-7 Years of Age With an Open-label Substudy of Binocular Videogames in Children 8-12 Years of Age
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Amblyopia
Keywords: DDO001
amblyopia
binocular video games
patching
eye disorder
lazy eye
unable to focus in one eye
eye muscle disorder
wandering eye
abnormal vision development
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2021-03-05
Enrollment: 27
Sex: ALL
Minimum age (years): 4
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this PoC study was to evaluate the potential therapeutic efficacy of binocular video games played on a tablet and to compare the efficacy of binocular video games versus patching in amblyopic patients 4 to 7 years old (Part A) as well as to gain experience with binocular video games in older children population of 8 to12 years old (Part B).

Part A and Part B was designed to provide long term data on durability of binocular video games treatment.

The study consisted of two parts, Part A: randomized, single masked PoC study in children 4 to 7 years old at Screening, and Part B: open-label substudy in children 8 to 12 year old at Screening.

Detailed description: The clinical investigation consisted of 2 parts:

* Part A: a single-masked, randomized clinical investigation in subjects 4 to 7 years of age.
* Part B: an open-label, non-randomized sub-investigation in subjects 8 to 12 years of age.

Part A was a 16-week, prospective, randomized, single-masked, multicenter, controlled, 2 arm, parallel-group clinical investigation in subjects 4 to 7 years of age with amblyopia. Subjects were randomly assigned in a 1:1 ratio to either binocular videogame treatment for 8 to 12 weeks or patching treatment for 16 weeks. Randomization was stratified by severity of amblyopia in eligible subjects (moderate amblyopia with best-corrected visual acuity (BCVA) of the amblyopic eye of 20/100 or better, or severe amblyopia with BCVA of the amblyopic eye worse than 20/100).

Part B was a 16-week, open-label, single arm sub investigation in subjects 8 to 12 years of age with amblyopia, in which selected sites could participate. Subjects received binocular videogame treatment for 8 weeks, followed by 8 weeks of follow-up.

Eligibility criteria: Inclusion Criteria:

1. Written informed consent must be signed by the parent(s) or legal guardian(s) prior to participation in the study.
2. Male or female children 4 to 7 years old at Screening (Part A) or 8 to 12 years old at Screening (Part B).
3. Diagnosis of amblyopia due to strabismus, anisometropia, or both.
4. Best corrected visual acuity (BCVA) of amblyopic eye (study eye) between 0.3 to 1.0 Logarithm of the Minimum Angle of Resolution (logMAR) (20/40 to 20/200 Snellen inclusive, 33 to 72 ETDRS letters inclusive) at Screening and Baseline.
5. BCVA of the sound eye (fellow eye) 0.1 logMAR (20/25 Snellen, 80 Early Treatment Diabetic Retinopathy Study (ETDRS) letters) or better in children 5 years of age and older or 0.2 logMAR (20/30 Snellen, 75 ETDRS letters) or better in children 4 years of age at Screening and Baseline.
6. Interocular difference of BCVA at least 0.3 logMAR (≥3 lines; ETDRS≥15 letters) at Baseline.
7. Patient is able to play the binocular game (Dig Rush and Monster Burner) on at least level 3 on the study tablet under binocular conditions (with red-green glasses).

Exclusion Criteria:

1. Treatment for amblyopia with patching, Bangerter filter, vision therapy, or binocular treatment in the past 1 week prior to Screening, or atropine in the past 4 weeks prior to Screening.
2. Treatment for amblyopia with patching, Bangerter filter, vision therapy, binocular treatment or atropine for more than 1 year prior to screening cumulatively.
3. Myopia ≥ -6.00D spheric equivalent in either eye at Screening or Baseline.
4. Prior amblyopia treatment (patching, Bangerter filter, vision therapy, binocular treatment or atropine) for more than a year prior to screening cumulatively.

Primary outcomes:
measure: Change in best corrected visual acuity (BCVA) in the amblyopic eye.
timeFrame: from Baseline to Week 8
Secondary outcomes:
measure: Attain BCVA of 0.1 logMAR or better in the amblyopic eye
timeFrame: at Week 4, Week 8, Week 12, and Week 16.
measure: Change in stereoacuity or binocular video game relative to patching.
timeFrame: from Baseline to Week 4, Week 8, Week 12, and Week 16.
measure: Attain a 2-line or better improvement in BCVA in the amblyopic eye
timeFrame: at Week 4, Week 8, Week 12, and Week 16.
measure: Change in BCVA in the amblyopic eye
timeFrame: from Baseline to Week 4, Week 12, and Week 16.
measure: Frequency of treatment-emergent Adverse Events
timeFrame: from Baseline to week 16
measure: Time played as recorded in the diary
timeFrame: from Baseline to week 12
measure: Time patched as recorded in the diary
timeFrame: from Baseline to week 16
measure: Time played as recorded in the video game system
timeFrame: from Baseline to week 12
measure: Percentage of participants who complete at least 75% of prescribed game play
timeFrame: from Baseline to Week 12
Other outcomes:
Locations:
city: Gainesville
country: United States
facility: Novartis Investigative Site
state: Florida
city: Maitland
country: United States
facility: Novartis Investigative Site
state: Florida
city: Chicago
country: United States
facility: Novartis Investigative Site
state: Illinois
city: Frederick
country: United States
facility: Novartis Investigative Site
state: Maryland
city: Las Vegas
country: United States
facility: Novartis Investigative Site
state: Nevada
city: North Ryde
country: Australia
facility: Novartis Investigative Site
state: New South Wales
city: Kitchener
country: Canada
facility: Novartis Investigative Site
state: Ontario
city: Waterloo
country: Canada
facility: Novartis Investigative Site
state: Ontario
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: