Sickle Cell DiseaseADHERENCT06264700
Applying Directly Observed Therapy to Hydroxyurea to Realize Effectiveness
A study testing Video Directly Observed Therapy (VDOT) and Health Reminder Tip Alerts in people with Sickle Cell Disease, run by Nationwide Children's Hospital at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 11 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Video Directly Observed Therapy (VDOT) and Health Reminder Tip Alerts
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 3 sitesUnited States
- Study dates
- 11 Jul 2024to 23 Jul 2026
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 11 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Lurie Children's Hospital
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
Hasbro Children's Hospital
Providence, Rhode Island, 02903
About this study
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.
Read the 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.
- Conditions
- Sickle Cell Disease
- Being tested
- Video Directly Observed Therapy (VDOT), Health Reminder Tip Alerts
- Keywords
- Sickle Cell Disease, Hydroxyurea, Adherence
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Video Directly Observed Therapy (VDOT)Behavioural
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.
Health Reminder Tip AlertsBehavioural
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Behavioral: Health Reminder Tip Alerts |
What is being measured
The primary outcome is the main question the study is designed to answer.
Retention Rate
The number of participants who are retained on both study arms during each study period will be reported.
Measured: 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.
Measured: 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.
Measured: 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
: An adapted questionnaire to assess caregivers' hydroxyurea knowledge and expectations, perception of their adherence, their barriers to adherence, and their beliefs about hydroxyurea's efficacy.
Measured: 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
Any acute visit (e.g., emergency, day hospital, and/or hospitalization visits) that is documented in the electronic medical record during the study. Emergency and day hospital visits that result in hospitalization will be categorized as one visit. Details from each of these visits will be abstracted.
Measured: 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)
Routine hematologic labs that are collected on patients receiving hydroxyurea will be collected from the electronic medical record (EMR).
Measured: throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days
Treatment Satisfaction
An 11-item survey to assess hydroxyurea treatment satisfaction over four domains, side effects, effectiveness, convenience and global satisfaction.
Measured: throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days
Participant Satisfaction
A Likert-scale survey to assess participant motivation for participating, understanding of the study and its procedures, experience interacting with research staff and completing study visits and activities.
Measured: At the end of the study, which will be approximately 390 days after enrollment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2024
- Study start11 Jul 2024
- Primary completion23 Jul 2026
- Study completion23 Jul 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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.
- Masking
- Single blind
- Who is masked
- Investigator
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nationwide Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Ann & Robert H Lurie Children's Hospital of Chicago, Hasbro Children's Hospital, National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Principal investigator, Susan Creary, Nationwide Children's Hospital
- Investigators
- Susan Creary, MD, MSc, Principal investigator, Nationwide Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06264700
- Sponsor's protocol ID
- STUDY00003303
- Other IDs
- R44HL169085
- Acronym
- ADHERE
- Status last verified
- Aug 2026
- First submitted
- 12 Jan 2024
- Last updated
- 21 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: The majority of the data generated in this project will be shared by conference presentation and direct publication in the scientific literature. After acceptance for publication of the main findings, other investigators may make a written request to MPI Dr. Creary to access the data from the project. All requests for data sharing will be reviewed by the PI. Data and associated documentation will be released under a data-use agreement which specifies that the data will be used for research purposes, stored securely, and destroyed when the project is completed. All identifiers will be removed from the dataset prior to release for sharing and the investigator provides assurance that they will not attempt to identify any participant.
Publications
Walden J, Brown L, Seiguer S, Munshaw K, Rausch J, Badawy S, McGann P, Winkler S, Gonzalez L, Creary S. Study protocol for ADHERE (Applying Directly observed therapy to HydroxyurEa to Realize Effectiveness): Using small business partnerships to deliver a scalable and novel hydroxyurea adherence solution to youth with sickle cell disease. PLoS One. 2024 Jun 25;19(6):e0304644. doi: 10.1371/journal.pone.0304644. eCollection 2024.
PubMed 38917111
Study at a glance
From the registry record, last updated by the sponsor on 21 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Applying Directly Observed Therapy to Hydroxyurea to Realize Effectiveness
- ClinicalTrials.gov ID
- NCT06264700
- Acronym / sponsor ID
- ADHERE / STUDY00003303
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle Cell Disease, Hydroxyurea, Adherence
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Nationwide Children's HospitalUniversity, hospital or other
- Collaborators
- Ann & Robert H Lurie Children's Hospital of Chicago, Hasbro Children's Hospital, National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 11 Jul 2024 (actual)
- Primary completion
- 23 Jul 2026 (actual)
- Study completion
- 23 Jul 2026 (actual)
- First posted
- 20 Feb 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (investigator)
- Number of arms
- 2
- 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.
| Arm | Type | Receives |
|---|---|---|
| Video Directly Observed Therapy (VDOT) | Experimental | Behavioral: 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. |
| Attention Control | Other | Behavioral: Health Reminder Tip AlertsParticipants randomized to this arm will receive an automated, daily, short health or safety tip alert. |
Who may qualify
- Ages
- 11 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
- Are English speaking.
- Are 11-25 years of age.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Behavioural | Video Directly Observed Therapy (VDOT) |
| Health Reminder Tip AlertsParticipants 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. | Behavioural | Attention Control |
Primary outcome
Retention Rate
Time frame: throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days
The number of participants who are retained on both study arms during each study period will be reported.
Sustained Engagement
Time frame: Throughout the intervention period, which will take place from approximately 30 days after enrollment to 210 days after enrollment
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.
Electronic Adherence
Time frame: throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days
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.
5 secondary outcomes
Pediatric SCD Medication Self-Management Questionnaire
Time frame: 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
Time frame: 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)
Time frame: throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days
Treatment Satisfaction
Time frame: throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days
Participant Satisfaction
Time frame: At the end of the study, which will be approximately 390 days after enrollment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Aug 2026.
- Derived from this study: PubMed 38917111Walden J, Brown L, Seiguer S, Munshaw K, Rausch J, Badawy S, McGann P, Winkler S, Gonzalez L, Creary S. Study protocol for ADHERE (Applying Directly observed therapy to HydroxyurEa to Realize Effectiveness): Using small business partnerships to deliver a scalable and novel hydroxyurea adherence solution to youth with sickle cell disease. PLoS One. 2024 Jun 25;19(6):e0304644. doi: 10.1371/journal.pone.0304644. eCollection 2024.
- Individual participant dataSharing planned: No. The majority of the data generated in this project will be shared by conference presentation and direct publication in the scientific literature. After acceptance for publication of the main findings, other investigators may make a written request to MPI Dr. Creary to access the data from the project. All requests for data sharing will be reviewed by the PI. Data and associated documentation will be released under a data-use agreement which specifies that the data will be used for research purposes, stored securely, and destroyed when the project is completed. All identifiers will be removed from the dataset prior to release for sharing and the investigator provides assurance that they will not attempt to identify any participant.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Susan Creary, MD, MSc principal investigator
Nationwide Children's Hospital
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Video Directly Observed Therapy (VDOT) and Health Reminder Tip Alerts in people with Sickle Cell Disease, run by Nationwide Children's Hospital at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Retention Rate (time frame: throughout the study at various timepoints after enrollment including: after approximately 30-90 days, 210 days, and 390 days). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06264700, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Aug 2026.