Feasibility and Efficacy of Attentional-Control Training in Sickle Cell Disease (ACT)

Children with sickle cell disease (SCD) exhibit significantly reduced cognitive functioning (often difficulties with attention) compared to peers and siblings without SCD. EndeavorRx (Akili Interactive Labs: Boston, MA) is an FDA-approved home-based, electronic attentional-control training program designed to treat attention problems in youth. Users access EndeavorRx on a tablet device for 25-30 minutes each day, 5 days per week, for 4 weeks. The program involves training in a game-like environment that repeatedly challenges attentional-control abilities and adapts to user performance, becoming more difficult over time as performance improves. This pilot study is examining the feasibility, acceptability, and preliminary efficacy of EndeavorRx in a sample of 20 children with SCD ages 8-16 who are being treated with chronic blood transfusion therapy.

Study Overview

• Status: Recruiting
• Conditions: Sickle Cell Disease; Cognitive Deficit in Attention; Attention Deficit
• Intervention / Treatment: Device: EndeavorRx

Detailed Description

Children with sickle cell disease (SCD) exhibit significantly reduced cognitive functioning compared to peers and siblings without SCD. Cognitive deficits are greatest among those with severe SCD genotypes, persistent anemia, and strokes and these deficits worsen over time. These deficits often manifest as difficulties with executive functioning and attention due to the predominance of cerebral infarcts (>90%) that occur in the frontal cortex. Reduced capacity to engage such cognitive skills has significant real-world implications, leading to disruption to academic achievement and attainment, vocational outcomes, and quality of life. EndeavorRx (Akili Interactive Labs: Boston, MA) is an FDA-approved home-based, electronic attentional-control training program designed to treat attention problems in youth. Users access EndeavorRx on a tablet device for 25-30 minutes each day, 5 days per week, for 4 weeks. The program involves training in a game-like environment that repeatedly challenges attentional-control abilities and adapts to user performance, becoming more difficult over time as performance improves. Data support effects of EndeavorRx on performance-based and parent-reported measures of attention in youth with Attention-Deficit/Hyperactivity Disorder (ADHD). Given the similarities in attentional functioning among youth with ADHD and those with SCD, EndeavorRx holds promise as an intervention for patients experiencing SCD-related cognitive deficits. This pilot study is examining the feasibility, acceptability, and preliminary efficacy of EndeavorRx in a sample of 20 children with SCD ages 8-16 who are being treated with chronic blood transfusion therapy due to an identified high risk of stroke or as a preventive measure to protect against additional stroke events.

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Steven J Hardy, Ph.D.; Phone Number: 202-476-5000; Email: sjhardy@childrensnational.org

Study Locations

• United States
  • District of Columbia
    • Washington, District of Columbia, United States, 20010
      • Recruiting
      • Children's National Hospital
      • Contact: Steven Hardy, PhD; Phone Number: 202-476-5000; Email: sjhardy@childrensnational.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 8 years to 16 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• diagnosis of SCD
• ages 8-16 years
• maintained on monthly blood transfusions consistently for at least 3 months.
• patients will be proceed to the intervention phase only if they have a T-score > 75th percentile for Omission Errors on the Conners' Continuous Performance Test, 3rd Edition (CPT-3) or a T-score > 75th percentile for the Inattention subscale of the ADHD Rating Scale, Fifth Edition (ADHD-RS-V).

Exclusion Criteria:

• estimated Intelligence Quotient < 70
• motor, visual, or auditory impairment that prevents computer use
• known diagnosis of a mental health condition that precludes, or takes treatment precedence over, participation in cognitive training
• history of photosensitive seizures
• insufficient English fluency.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: EndeavorRx; Children will be asked to begin attentional control training at home within two weeks of baseline testing and to complete 6 training missions per day (25-30 minutes), 5 days per week, for 4 weeks (total = 120 training missions).

Device: EndeavorRx; EndeavorRx is a digital intervention delivered through an action video game that is indicated to improve attention function. EndeavorRx users first complete two discrete tasks: 1) a perceptual discrimination/attention task in which users respond to target stimuli and ignore distractor stimuli (e.g., tapping the screen quickly only when red aliens appear) and 2) a sensorimotor navigation task in which users tilt their device to steer a hovercraft down a river while targeting or avoiding certain objects. Users are subsequently presented with a multitask training session where they complete the perceptual discrimination/attention task and the sensorimotor navigation task simultaneously. Algorithms adapt the program in both real-time and between sessions to automatically adjust the difficulty level.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
EndeavorRx feasibility assessed by patient/family interest	Feasibility will be determined by examining the proportion of eligible patients/families approached about the study who enroll.	Approximately 10 months
EndeavorRx feasibility assessed by program completion rates	Feasibility will be evaluated by examining the proportion of the sample that completes at least 60 EndeavorRx missions (50%).	Approximately 4 weeks per participant
EndeavorRx feasibility assessed by reports of technical ease-of-use and satisfaction	Feasibility will be determined by examining parent and child reports of technical ease-of-use and satisfaction.	Approximately 4 weeks from start of intervention

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Inattention assessed by change in Omission Errors on the Conners' Continuous Performance Test, 3rd Edition (CPT-3)	EndeavorRx efficacy will be evaluated by examining change in Omission Errors on the CPT-3 computerized assessment after completing EndeavorRx.	Approximately 4 weeks from start of intervention
Inattention assessed by change in the Inattention subscale of the ADHD Rating Scale, Fifth Edition (ADHD-RS-V)	EndeavorRx efficacy will be evaluated by examining change in the Inattention subscale of the parent-reported ADHD-RS-V after completing EndeavorRx.	Approximately 4 weeks from start of intervention

Collaborators and Investigators

• Sponsor: Children's National Research Institute
• Investigators: Principal Investigator: Steven J Hardy, Ph.D., Children's National Research Institute

Study record dates

Study Major Dates

• Study Start (Actual): December 20, 2022
• Primary Completion (Estimated): December 1, 2025
• Study Completion (Estimated): December 1, 2025

Study Registration Dates

• First Submitted: October 19, 2021
• First Submitted That Met QC Criteria: October 19, 2021
• First Posted (Actual): October 29, 2021

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 8, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Genetic Diseases, Inborn; Neurocognitive Disorders; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Cognitive Dysfunction; Anemia, Sickle Cell; Cognition Disorders
• Other Study ID Numbers: Pro00016536

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes
• product manufactured in and exported from the U.S.: No