Active — Not RecruitingNot Applicable

Exploring Adherence Monitoring in Sickle Cell Disease

United States36 participantsStarted 2019-03-12

Plain-language summary

Despite the well-documented benefits of hydroxyurea (HU) therapy in decreasing morbidity and mortality in youth with Sickle cell disease (SCD), pediatric HU adherence rates range as low as 49% and lead to discontinuation of HU regimens in 8-20%. In addition, treatment non-adherence may lead to unnecessary increases in medication dosage resulting from erroneous assumption that a patient is non-responsive to treatment (versus non-adherent to the regimen as prescribed). Given the detrimental effects of non-adherence, assessment of and intervention for HU non-adherence is essential to improving health outcomes in the pediatric SCD population. Electronic adherence monitoring is widely considered the "gold standard" in objective adherence measurement. These monitors provide continuous, real- time records of medication adherence and reveal problematic behavior patterns, including underdosing, overdosing, delayed dosing, "drug holidays," and "white coat" adherence. Overall, electronic adherence measures are considered valid, reliable, and accurate, with clear advantages over pharmacy refill records, physician estimates and self-report measures. The primary purpose of this pilot study is to determine the use of the AdhereTech as a feasible and valid measure of HU adherence in pediatric SCD. Primary Objective Estimate the association between HU adherence as measured by the AdhereTech device to a) caregiver-report, b) youth-report, c) lab values, d) pill- count, and e) Medication Possession Ratio (MPR) adherence measures Secondary Objectives Estimate the rate of consent to the study, the rate of AdhereTech device use, the rate of AdhereTech device failure, and the perceived acceptability of using the AdhereTech device, as reported by caregivers and youth

Who can participate

Age range

12 Years – 17 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Confirmed diagnosis of SCD (any genotype) * Ages 12.0 - 17.99 at time of study enrollment * Stable HU dose composed of only one capsule strength prescribed in pill formulation for ≥ 6 months without documented hematological toxicity (excluding dose adjustments for weight gain) * Lives with their legal guardian * Anticipated to return to clinic at proposed 4-week intervals Exclusion Criteria: * Primary caregiver and/or youth unable to understand English and/or youth not cognitively intact (known IQ \< 70) such that the study questionnaire cannot be understood and completed. * Participant unable to complete the questionnaires due to refusal or current acute illness (e.g., pain crisis). * Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Estimate the association between AdhereTech device HU adherence to caregiver-report of adherence

Timeframe: Continuous over two months, baseline to off study

Estimate the association between AdhereTech device HU adherence to youth-reported adherence

Timeframe: Continuous over two months, baseline to off study

Estimate the association between AdhereTech device HU adherence to fetal hemoglobin (HbF) lab values

Timeframe: Continuous over two months, baseline to off study

Estimate the association between AdhereTech device HU adherence to mean corpuscular volume (MCV) lab values

Timeframe: Continuous over two months, baseline to off study

Estimate the association between AdhereTech device HU adherence to the Medication Possession Ratio (MPR)

Timeframe: Continuous over two months, baseline to off study

Estimate the association between AdhereTech device HU adherence to pill count

Timeframe: Continuous over two months, baseline to off study

Trial details

NCT IDNCT03708731

SponsorSt. Jude Children's Research Hospital

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2022-08-10

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