Applying Directly Observed Therapy to Hydroxyurea to Realize Effectiveness

NCT ID: NCT06264700

Last Updated: 2025-04-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

NA

Total Enrollment

60 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-07-11

Study Completion Date

2025-12-31

Brief Summary

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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

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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.

Conditions

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Sickle Cell Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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 Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Investigators
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.

Study Groups

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Video Directly Observed Therapy (VDOT)

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.

Group Type EXPERIMENTAL

Video Directly Observed Therapy (VDOT)

Intervention Type 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.

Attention Control

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

Group Type OTHER

Health Reminder Tip Alerts

Intervention Type 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.

Interventions

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Video Directly Observed Therapy (VDOT)

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.

Intervention Type BEHAVIORAL

Health Reminder Tip Alerts

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.

Intervention Type BEHAVIORAL

Eligibility Criteria

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Inclusion Criteria

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.
Minimum Eligible Age

11 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ann & Robert H Lurie Children's Hospital of Chicago

OTHER

Sponsor Role collaborator

Hasbro Children's Hospital

OTHER

Sponsor Role collaborator

National Institutes of Health (NIH)

NIH

Sponsor Role collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role collaborator

Nationwide Children's Hospital

OTHER

Sponsor Role lead

Responsible Party

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Susan Creary

Associate Professor of Pediatrics and Pediatric Hematologist, Division of Hematology/Oncology/BMT

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Susan Creary, MD, MSc

Role: PRINCIPAL_INVESTIGATOR

Nationwide Children's Hospital

Locations

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Lurie Children's Hospital

Chicago, Illinois, United States

Site Status NOT_YET_RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status RECRUITING

Hasbro Children's Hospital

Providence, Rhode Island, United States

Site Status NOT_YET_RECRUITING

Countries

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United States

Central Contacts

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Susan Creary, MD, MSc

Role: CONTACT

(614) 722-3550

Nate Goldfarb, MPH

Role: CONTACT

(614) 965-4948

Facility Contacts

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Sherif Badawy, MD

Role: primary

312-227-4836

Susan Creary, MD, MSc

Role: primary

614-722-3563

Patrick McGann, MD, PhD

Role: primary

617-519-9555

Other Identifiers

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R44HL169085

Identifier Type: NIH

Identifier Source: secondary_id

View Link

STUDY00003303

Identifier Type: -

Identifier Source: org_study_id

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