Video Directly Observed Therapy for Sickle Cell Disease

(ADHERE Trial)

Recruiting at 2 trial locations

Overseen By Nate Goldfarb, MPH

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Nationwide Children's Hospital

Must be taking: Hydroxyurea

Must not be taking: Chronic transfusions, L-glutamine

Disqualifiers: Previous VDOT study, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 26 weeks

60 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores two methods to help young people with sickle cell disease adhere to their hydroxyurea medication schedule. One group will use video directly observed therapy (VDOT), sending videos to confirm medication intake. The other group will receive daily health tip alerts. This study targets English-speaking caregivers of children aged 1-10 and adolescents aged 11-25 with sickle cell disease who have taken hydroxyurea for at least six months. As an unphased trial, it offers participants the chance to contribute to innovative approaches in medication adherence.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it requires participants to continue taking hydroxyurea. If you are on other treatments for sickle cell disease, like chronic transfusions or L-glutamine, you may not be eligible to participate.

What prior data suggests that Video Directly Observed Therapy (VDOT) is safe for use in this study?

Research has shown that Video Directly Observed Therapy (VDOT) has been safely used in other areas to help people take their medication correctly. However, current studies lack specific safety information about using VDOT for sickle cell disease. VDOT primarily involves video check-ins to ensure patients take their medication as prescribed.

This trial is in a "Not Applicable" phase, focusing on gathering information about how well this method helps patients adhere to their medication schedules, rather than testing a new drug's safety. Therefore, the risks mainly involve privacy and technology use, not physical side effects.

For personalized information, discussing concerns with the study team or a healthcare provider is recommended. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about Video Directly Observed Therapy (VDOT) for sickle cell disease because it offers a new way to ensure patients stick to their medication routines. Unlike traditional treatments that rely on patient self-reporting, VDOT uses video technology to monitor medication adherence, providing a more accurate and engaging approach. This method can help improve the effectiveness of existing treatments like hydroxyurea by ensuring patients take their medication correctly and consistently. Additionally, the personalized follow-up through calls and texts aims to keep patients motivated and supported, which could lead to better health outcomes.

What evidence suggests that Video Directly Observed Therapy is effective for sickle cell disease?

Research has shown that Video Directly Observed Therapy (VDOT), which participants in this trial may receive, can help people take their medication as prescribed. For those with sickle cell disease, taking hydroxyurea regularly is crucial for its effectiveness. Studies comparing VDOT with regular check-ins found that VDOT helped patients adhere more closely to their medication schedule. Thus, VDOT could be a helpful method for people with sickle cell disease to manage their treatment effectively. ^{1 3 6 7 8}

Who Is on the Research Team?

Susan Creary, MD, MSc

Principal Investigator

Nationwide Children's Hospital

Are You a Good Fit for This Trial?

This trial is for caregivers of young children and adolescents with sickle cell disease who are prescribed hydroxyurea. Participants must be receiving care at a study site and willing to use an electronic monitor to track medication usage.

Inclusion Criteria

I am an English-speaking adult caregiver of a child aged 1-10 with SCD on hydroxyurea for 6+ months.

Exclusion Criteria

I am not on multiple treatments for sickle cell disease.

Adolescents and caregivers of younger children who participated in the previous VDOT study will be excluded.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Run-in Period

Participants undergo a run-in period before randomization

4 weeks

Treatment

Participants receive either video directly observed therapy (VDOT) or attention control for 180 days

26 weeks

Routine hematology visits

Ongoing Monitoring

Participants continue with electronic adherence monitoring and receive intermittent communication to encourage adherence

26 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Video Directly Observed Therapy (VDOT)

Trial Overview The study compares two methods: Video Directly Observed Therapy (VDOT) where medication intake is monitored via video, and Health Reminder Tip Alerts that prompt patients. It aims to assess how these affect adherence to taking hydroxyurea.

How Is the Trial Designed?

Group I: Video Directly Observed Therapy (VDOT) Experimental Treatment 1 Intervention

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 II: Attention Control Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Nationwide Children's Hospital

Lead Sponsor

Trials

354

Recruited

5,228,000+

National Heart, Lung, and Blood Institute (NHLBI)

Collaborator

Trials

3,987

Recruited

47,860,000+

Ann & Robert H Lurie Children's Hospital of Chicago

Collaborator

Trials

275

Recruited

5,182,000+

Hasbro Children's Hospital

Collaborator

Trials

5

Recruited

10,100+

National Institutes of Health (NIH)

Collaborator

Trials

2,896

Recruited

8,053,000+

Published Research Related to This Trial

Patients with sickle cell disease (SCD) experience significant quality of life impairments due to the disease's complications, highlighting the need for effective disease-modifying therapies.

Recent initiatives, such as the ASH Research Collaborative SCD Clinical Trials Network and the Cure Sickle Cell Initiative, aim to improve clinical trial designs and patient-centered outcomes, emphasizing the importance of patient-reported outcomes in evaluating treatment efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical trial considerations in sickle cell disease: patient-reported outcomes, data elements, and the stakeholder engagement framework. Badawy, SM.[2022]

A study involving 30 patients with sickle cell disease showed that a 15-minute immersive virtual reality (VR) session was safe and well-accepted, with no reported side effects and all patients wanting to use VR again.

The VR session significantly reduced pain intensity and the number of affected body areas, suggesting that VR could be a beneficial complementary therapy for managing vaso-occlusive pain episodes in sickle cell disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Virtual reality as complementary pain therapy in hospitalized patients with sickle cell disease. Agrawal, AK., Robertson, S., Litwin, L., et al.[2019]

Voxelotor, a first-in-class therapy for sickle cell disease (SCD), targets the root cause of the disease by preventing sickle hemoglobin polymerization, and has been shown to improve hemoglobin levels and reduce hemolysis in patients.

In a study of 27 patients treated for at least 8 weeks, both clinicians and patients reported significant improvements in health-related quality of life and disease symptoms, indicating voxelotor's positive impact on SCD outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Patient perception of voxelotor treatment benefit in sickle cell disease. Idowu, M., Haque, A., Williams, EM., et al.[2022]

A study involving 46 children with sickle cell disease showed that cognitive-behavioral therapy (CBT) delivered via smartphones significantly improved active coping skills over an 8-week period.

Using CBT skills on days with higher pain levels was associated with reduced next-day pain intensity, indicating that this intervention can effectively help manage pain in pediatric patients with sickle cell disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Changes in coping, pain, and activity after cognitive-behavioral training: a randomized clinical trial for pediatric sickle cell disease using smartphones. Schatz, J., Schlenz, AM., McClellan, CB., et al.[2022]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11198815/

Study protocol for ADHERE (Applying Directly observed ... Measuring hydroxyurea adherence by pharmacy and laboratory data compared with video observation in children with sickle cell disease.

2. withpower.com withpower.com/trial/phase-anemia-sickle-cell-2-2024-e5b42

Video Directly Observed Therapy for Sickle Cell Disease This study is for caregivers of young children with sickle cell disease and adolescents with sickle cell disease who are currently prescribed hydroxyurea ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9581185/

The use of remote video directly observed therapy to ... ICS therapy not only helps to control milder day-to-day symptoms but also reduces the risk of future asthma attacks. To be effective, ICS must be taken ...

4. nature.com nature.com/articles/s43856-024-00552-5

Hydroxyurea mobile directly observed therapy versus ... Hydroxyurea mobile directly observed therapy versus standard monitoring in patients with sickle cell anemia: a phase 2 randomized trial. Philip ...

5. trialx.com trialx.com/clinical-trials/listings/296251/applying-directly-observed-therapy-to-hydroxyurea-to-realize-effectiveness/?&radius=10&place=All%20Participating%20Sites&user_country=Germany

Applying Directly Observed Therapy to Hydroxyurea ... Sickle cell-related outcomes and healthcare utilization will be abstracted from patient electronic medical records throughout each study period.

6. journals.plos.org journals.plos.org/plosone/article?id=10.1371/journal.pone.0304644

Study protocol for ADHERE (Applying Directly observed ... Measuring hydroxyurea adherence by pharmacy and laboratory data compared with video observation in children with sickle cell disease.

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11315961/

Hydroxyurea mobile directly observed therapy versus ... Sickle cell anemia (SCA) prevalence remains high in sub-Saharan Africa. Long-term treatment with hydroxyurea (HU) increases survival, however, ...

8. mhealth.jmir.org mhealth.jmir.org/2019/8/e13452/

A Multidimensional Electronic Hydroxyurea Adherence ... A pilot study of electronic directly observed therapy to improve hydroxyurea adherence in pediatric patients with sickle-cell disease.