Clinical trial NCT03708731: Exploring Adherence Monitoring in Sickle Cell Disease
Official title: Exploring Adherence Monitoring in Sickle Cell Disease
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Sickle Cell Disease
Keywords: Sickle Cell Disease
Youth
Hydroxyurea
Non-Adherence
Electronic Adherence Monitoring
AdhereTech smart bottle
Lead sponsor: St. Jude Children's Research Hospital
Sponsor class: OTHER
First posted: 2018-10-17
Enrollment: 36
Sex: ALL
Minimum age (years): 12
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants ages 12.0-17.99 with Sickle cell disease any genotype and their caregivers

Brief 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

Detailed description: The AdhereTech smart bottle is an electronic medicine dispenser with a tracking and reminder system connected wirelessly. The device provides real time adherence aids to the patient and this information is relayed to the provider. The device can sense the amount of pills remaining inside the bottle. This information is measured periodically and concurrently sent to a secure database using cellular data technology. Thus, AdhereTech bottles can be used to determine whether medication was removed from the bottle, the amount of medication removed, and the timing of removal.

Participants ages 12.0-17.99 who are receiving HU prescribed in pill form will use the AdhereTech smart bottle for a two-month period. Participants and their caregivers will complete self-report measures of adherence at the baseline, 1 month and 2 month clinic visits, a Demographic questionnaire at the baseline clinic visit and an Acceptability questionnaire at the 1 month and 2 month clinic visits using Audio Computer-Assisted Self-Interviews (ACASI) on a password protected laptop.

Lab values and clinical information will be obtained through medical records review and adherence information from pill counts and pharmacy records.

Eligibility criteria: 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.

Primary outcomes:
measure: Estimate the association between AdhereTech device HU adherence to caregiver-report of adherence
timeFrame: Continuous over two months, baseline to off study
measure: Estimate the association between AdhereTech device HU adherence to youth-reported adherence
timeFrame: Continuous over two months, baseline to off study
measure: Estimate the association between AdhereTech device HU adherence to fetal hemoglobin (HbF) lab values
timeFrame: Continuous over two months, baseline to off study
measure: Estimate the association between AdhereTech device HU adherence to mean corpuscular volume (MCV) lab values
timeFrame: Continuous over two months, baseline to off study
measure: Estimate the association between AdhereTech device HU adherence to the Medication Possession Ratio (MPR)
timeFrame: Continuous over two months, baseline to off study
measure: Estimate the association between AdhereTech device HU adherence to pill count
timeFrame: Continuous over two months, baseline to off study
Secondary outcomes:
measure: Estimate rate of consent to the study
timeFrame: Once, at baseline
measure: Estimate rate of AdhereTech device use
timeFrame: Continuous over two months, baseline to off study
measure: Estimate rate of AdhereTech device failure
timeFrame: Continuous over two months, baseline to off study
measure: Estimate the perceived acceptability of using the AdhereTech device, as reported by caregivers and youth
timeFrame: Continuous over two months, baseline to off study
Other outcomes:
Locations:
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
Study officials:
affiliation: St. Jude Children's Research Hospital
name: Jerlym Porter, PhD
role: PRINCIPAL_INVESTIGATOR
References: