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Applying Directly Observed Therapy to Hydroxyurea to Realize Effectiveness

Applying Directly Observed Therapy to Hydroxyurea to Realize Effectiveness

Recruiting
11 years and older
All
Phase N/A

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Overview

This study is for caregivers of young children with sickle cell disease and adolescents with sickle cell disease who are currently prescribed hydroxyurea and are receiving care at one of the study sites. The study will assess retention and engagement during a pilot randomized control trial comparing video directly observed therapy (VDOT) to attention control. We also hope to understand more about patient and family preferences longer-term adherence monitoring and intervention.

Participants will use an electronic adherence monitor (provided by the study team) to measure how often they are taking their hydroxyurea. Participants will also be asked to complete questionnaires throughout the study period to provide information about their expectations for, experience with, and satisfaction with the study materials.

Description

This is an investigator-blinded, multi-center pilot randomized controlled trial (RCT) of adolescents with sickle cell disease (SCD) and caregivers of young children with SCD. After completion of a run-in period, subjects will undergo randomization to either six months of video directly observed therapy (VDOT) or attention control. Electronic adherence monitors will be used to measure adherence. After the intervention period, all participants will then complete a six-month ongoing monitoring period where VDOT participants will continue to use electronic adherence monitors and receive intermittent communication to encourage adherence and attention controls will continue to use their electronic adherence monitors.

The investigators are partnering with a small business, Scene Health, to administer VDOT. Outcomes such as engagement, retention, and satisfaction will be measured through survey data collected at routine study visits. Sickle cell-related outcomes and healthcare utilization will be abstracted from patient electronic medical records throughout each study period. Adherence data will be recorded by the electronic adherence devices.

Eligibility

Inclusion

Adult caregivers will be eligible if they:

  • Are English speaking.
  • Have a child who is 1-10 years of age with SCD (any genotype) who has been prescribed hydroxyurea for at least 180 days prior to enrollment.
  • Note: Caregivers who have multiple children meeting criteria will only be able to enroll once.

Adolescents (>11 years at enrollment) are eligible if they:

  • Are English speaking.
  • Are 11-25 years of age.
  • Have a diagnosis of SCD (any genotype) and have been prescribed hydroxyurea for at least 180 days prior to enrollment.
    Exclusion
  • Adolescents and caregivers of younger children who participated in the previous VDOT

    study will be excluded.

  • Adolescents and caregivers of younger children receiving multiple SCD modifying treatments (e.g., chronic transfusions or L-glutamine) will be excluded.

Randomization: enrolled subjects will be eligible for randomization if they open their electronic adherence monitoring device at home at least once during the run-in period.

Study details
    Sickle Cell Disease

NCT06264700

Nationwide Children's Hospital

14 April 2025

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