Clinical trial NCT06264700: Applying Directly Observed Therapy to Hydroxyurea to Realize Effectiveness
Official title: Applying Directly Observed Therapy to Hydroxyurea to Realize Effectiveness
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Sickle Cell Disease
Keywords: Sickle Cell Disease
Hydroxyurea
Adherence
Lead sponsor: Nationwide Children's Hospital
Sponsor class: OTHER
Collaborators: name: Ann & Robert H Lurie Children's Hospital of Chicago
name: Hasbro Children's Hospital
name: National Institutes of Health (NIH)
name: National Heart, Lung, and Blood Institute (NHLBI)
First posted: 2024-02-20
Enrollment: 60
Sex: ALL
Minimum age (years): 11
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: After completing a one to three-month run-in period, participants will be randomized to receive six months of either VDOT or attention control. All participants will then complete six months of ongoing monitoring during which VDOT patients will receive intermittent communication to encourage adherence and both groups will continue to use their electronic adherence monitors.
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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 criteria: 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.

Primary outcomes:
measure: Retention Rate
timeFrame: throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days
measure: Sustained Engagement
timeFrame: Throughout the intervention period, which will take place from approximately 30 days after enrollment to 210 days after enrollment
measure: Electronic Adherence
timeFrame: throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days
Secondary outcomes:
measure: Pediatric SCD Medication Self-Management Questionnaire
timeFrame: At multiple points throughout the study, including at enrollment, at approximately 90 days after randomization, at approximately 180 days months after randomization, and at approximately 360 days after randomization
measure: Frequency of acute care visits (ACS and VOC), ICU admissions, and LOS for hospitalizations
timeFrame: throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days
measure: Laboratory Studies (i.e. MCV, HbF)
timeFrame: throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days
measure: Treatment Satisfaction
timeFrame: throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days
measure: Participant Satisfaction
timeFrame: At the end of the study, which will be approximately 390 days after enrollment
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Lurie Children's Hospital
state: Illinois
city: Columbus
country: United States
facility: Nationwide Children&#39;s Hospital
state: Ohio
city: Providence
country: United States
facility: Hasbro Children's Hospital
state: Rhode Island
Study officials:
affiliation: Nationwide Children&#39;s Hospital
name: Susan Creary, MD, MSc
role: PRINCIPAL_INVESTIGATOR
References: