Applying Directly Observed Therapy to Hydroxyurea to Realize Effectiveness

NCT06264700 | Recruiting DMC

• 2 other identifiers: STUDY00003303R44HL169085
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 3

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.

Conditions

Sickle Cell Disease

Keywords

Sickle Cell Disease; Hydroxyurea; Adherence

Outcome Measures

Primary Outcomes (3)

• Retention Rate

  The number of participants who are retained on both study arms during each study period will be reported.

  throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days

• Sustained Engagement

  The number of participants who continue to actively participate with the intervention (VDOT arm only) and who report being satisfied with the intervention on the VDOT satisfaction survey.

  Throughout the intervention period, which will take place from approximately 30 days after enrollment to 210 days after enrollment

• Electronic Adherence

  Electronic adherence data from the electronic monitors will be integrated with the prescribing and hospitalization data from participants' electronic medical record to account for days when participants may not open their electronic pill bottle but are still adherent to their prescribed regimen. Hospitalization days will be subtracted from the adherence calculation, since participants will receive hydroxyurea from the inpatient pharmacy.

  throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days

Secondary Outcomes (5)

• Pediatric SCD Medication Self-Management Questionnaire

  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

• Frequency of acute care visits (ACS and VOC), ICU admissions, and LOS for hospitalizations

  throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days

• Laboratory Studies (i.e. MCV, HbF)

  throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days

• Treatment Satisfaction

  throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days

• Participant Satisfaction

  At the end of the study, which will be approximately 390 days after enrollment

Study Arms (2)

Video Directly Observed Therapy (VDOT)

EXPERIMENTAL

Participants randomized to this arm will be connected with Scene Health by a study staff member (not the PI or Co-I's). After submitting and receiving approval of a test video submission, the participants' hydroxyurea dosing schedule will be entered into the VDOT app by the research staff and will be updated by these staff after their routine hematology visits and/or hospitalizations. Participants will receive VDOT for 180 days, beginning the day after randomization. After that time, they will start a 180-day ongoing monitoring period, during which VDOT participants will receive monthly telephone calls and intermittent text messages from Scene Health staff to encourage ongoing adherence. The Scene Health staff will access the electronic adherence platform and use this data to inform their communications during the ongoing monitoring period. Participants in both arms will be offered a smartphone with a data plan at enrollment to ensure equal opportunity for participation.

Behavioral: Video Directly Observed Therapy (VDOT)

Attention Control

OTHER

Participants randomized to this arm will receive an automated, daily, short health or safety tip alert.

Behavioral: Health Reminder Tip Alerts

Interventions

Video Directly Observed Therapy (VDOT) BEHAVIORAL

VDOT is an adherence-promoting intervention that involves partnering with an adolescent with SCD (or caregiver of a young patient with SCD) and observing the patient administer their hydroxyurea. In this study, VDOT will be delivered by a small business partner, Scene Health, via a smart phone app.

Video Directly Observed Therapy (VDOT)

Health Reminder Tip Alerts BEHAVIORAL

Participants randomized to this arm will receive an automated, daily, short health or safety tip alert (e.g. "Time to get moving! You should be active for at least 30 minutes each day") to receive during the 180-day intervention period on their smartphone. The daily tip will be unrelated to hydroxyurea adherence. After the 180-day intervention period, participants complete a 180-day ongoing monitoring period where they will no longer receive these messages, but they will be reminded to continue to use their electronic adherence monitoring device. All participants will be offered a smart phone with a data plan at enrollment to ensure equal opportunity for participation.

Attention Control

Eligibility Criteria

• Age: 11 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Nationwide Children's Hospital: lead
• Ann & Robert H Lurie Children's Hospital of Chicago: collaborator
• Hasbro Children's Hospital: collaborator
• National Institutes of Health (NIH): collaborator
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Sites (3)

Lurie Children's Hospital

Chicago, Illinois, 60611, United States

NOT YET RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

RECRUITING

Hasbro Children's Hospital

Providence, Rhode Island, 02903, United States

NOT YET RECRUITING

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Susan Creary, MD, MSc

  Nationwide Children's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Susan Creary, MD, MSc

CONTACT

(614) 722-3550; susan.creary@nationwidechildrens.org

Nate Goldfarb, MPH

CONTACT

(614) 965-4948; nate.goldfarb@nationwidechildrens.org

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: INVESTIGATOR
• Masking Details: The investigators will be blinded to prevent the possibility of any differential treatment that could affect the ability of the study team to evaluate the impact of VDOT on hydroxyurea adherence. As such, a statistician will create a randomization model and study staff will facilitate communication with participants as well as between participants and the VDOT team.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor of Pediatrics and Pediatric Hematologist, Division of Hematology/Oncology/BMT

Model Details: 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.

Study Record Dates

• First Submitted: January 12, 2024
• First Posted: February 20, 2024
• Study Start: July 11, 2024
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: April 8, 2025

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)