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

Trial Summary

What is the purpose of this trial?

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.

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 data supports the effectiveness of the treatment Video Directly Observed Therapy (VDOT) for sickle cell disease?

Research shows that using video observation to monitor medication adherence, like in the case of hydroxyurea for sickle cell disease, can be a reliable method. Additionally, a mobile health application that included video recording of medication administration improved adherence and disease knowledge in pediatric patients, suggesting that VDOT could be effective in ensuring patients take their medications as prescribed. ^{1 2 3 4 5}

Is Video Directly Observed Therapy (VDOT) safe for use in humans?

The research does not provide specific safety data for Video Directly Observed Therapy (VDOT) itself, but it mentions that patients using a similar video-based method for medication adherence reported high satisfaction and ease of use, suggesting it is generally well-tolerated. ^{2 4 6 7 8}

How is Video Directly Observed Therapy (VDOT) unique for treating sickle cell disease?

Video Directly Observed Therapy (VDOT) is unique because it uses video technology to ensure patients take their medication correctly and consistently, which can improve adherence to treatment plans compared to traditional methods that rely on in-person supervision. ^{9 10 11 12 13}

Research Team

Susan Creary, MD, MSc

Principal Investigator

Nationwide Children's Hospital

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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 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]

References

1. United States 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. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Measuring hydroxyurea adherence by pharmacy and laboratory data compared with video observation in children with sickle cell disease. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

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

4. United States pubmed.ncbi.nlm.nih.gov

Utilizing a Novel Mobile Health "Selfie" Application to Improve Compliance to Iron Chelation in Pediatric Patients Receiving Chronic Transfusions. [2018]

5. United States pubmed.ncbi.nlm.nih.gov

A Scientific Renaissance: Novel Drugs in Sickle Cell Disease. [2018]

6. United States pubmed.ncbi.nlm.nih.gov

Accelerated approval of Oxbryta® (voxelotor): A case study on novel endpoint selection in sickle cell disease. [2021]

7. United States pubmed.ncbi.nlm.nih.gov

A retrospective study to assess the utility of frequent laboratory monitoring of pediatric patients with sickle cell disease on hydroxyurea. [2022]

8. England pubmed.ncbi.nlm.nih.gov

Patient perception of voxelotor treatment benefit in sickle cell disease. [2022]

9. England pubmed.ncbi.nlm.nih.gov

Sickle Cell Disease Treatment with Arginine Therapy (STArT): study protocol for a phase 3 randomized controlled trial. [2023]

10. United States pubmed.ncbi.nlm.nih.gov

Blood and marrow transplantation for sickle cell disease: overcoming barriers to success. [2021]

11. United States pubmed.ncbi.nlm.nih.gov

Intravenous fluid therapy and hospital outcomes for vaso-occlusive episodes in children, adolescents, and young adults with sickle cell disease. [2023]

12. England pubmed.ncbi.nlm.nih.gov

Real-world use of defibrotide for veno-occlusive disease/sinusoidal obstruction syndrome: the DEFIFrance Registry Study. [2023]

13. United States pubmed.ncbi.nlm.nih.gov

Virtual reality as complementary pain therapy in hospitalized patients with sickle cell disease. [2019]