Exploring Adherence Monitoring in Sickle Cell Disease

NCT03708731 | Active Not Recruiting

• 1 other identifier: ExAMS
• Study Type: observational
• Target: 36
• Locations: 1 country
• Sites: 1

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

Conditions

Sickle Cell Disease

Keywords

Sickle Cell Disease; Youth; Hydroxyurea; Non-Adherence; Electronic Adherence Monitoring; AdhereTech smart bottle

Outcome Measures

Primary Outcomes (6)

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

  For each subject, we will calculate the proportion of days of opening the AdhereTech device across the two month study period (days with missing information will not be counted). Caregiver-report of their child's HU adherence will be assessed by the Medical Adherence Measure (MAM), a measure of 7-day self-reported adherence. Each subject's caregiver-reported adherence will be calculated as: \[(medication reported taken \[(medication reported taken ÷ medication prescribed) x 100\]. We will estimate the Spearman's rank-based correlation, with 95% confidence interval, of the AdhereTech device adherence with the caregiver-report of their child's adherence.

  Continuous over two months, baseline to off study

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

  For each subject, we will calculate the proportion of days of opening the AdhereTech device across the two month study period (days with missing information will not be counted). Youth-report of their HU adherence will be assessed by the Medical Adherence Measure (MAM), a measure of 7-day self-reported adherence. Each subject's self-reported adherence will be calculated as: \[(medication reported taken \[(medication reported taken ÷ medication prescribed) x 100\]. We will estimate the Spearman's rank-based correlation, with 95% confidence interval, of the AdhereTech device adherence with the youth-reported adherence.

  Continuous over two months, baseline to off study

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

  For each subject, we will calculate the proportion of days of opening the AdhereTech device across the two month study period (days with missing information will not be counted). We will extract fetal hemoglobin (HbF; g/dl) lab value from each subject's medical record. We will estimate the Spearman's rank-based correlation, with 95% confidence interval, of the AdhereTech device adherence with the fetal hemoglobin (HbF) lab value.

  Continuous over two months, baseline to off study

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

  For each subject, we will calculate the proportion of days of opening the AdhereTech device across the two month study period (days with missing information will not be counted). We will extract mean corpuscular volume (MCV; L) lab value from each subject's medical record. We will estimate the Spearman's rank-based correlation, with 95% confidence interval, of the AdhereTech device adherence with the mean corpuscular volume (MCV) lab value .

  Continuous over two months, baseline to off study

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

  For each subject, we will calculate the proportion of days of opening the AdhereTech device across the two month study period (days with missing information will not be counted). Medication Possession Ratio (MPR) will be calculated as: \[(number of days medication in family's possession ÷ number of days for which medication prescribed) x 100\]. We will estimate the Spearman's rank-based correlation, with 95% confidence interval, of the AdhereTech device adherence with the Medication Possession Ratio (MPR).

  Continuous over two months, baseline to off study

• Estimate the association between AdhereTech device HU adherence to pill count

  For each subject, we will calculate the proportion of days of opening the AdhereTech device across the two month study period (days with missing information will not be counted). We will calculate each subject's pill count adherence as: \[(number of pills removed ÷ medication prescribed) x 100\]. We will estimate the Spearman's rank-based correlation, with 95% confidence interval, of the AdhereTech device adherence with pill count adherence.

  Continuous over two months, baseline to off study

Secondary Outcomes (4)

• Estimate rate of consent to the study

  Once, at baseline

• Estimate rate of AdhereTech device use

  Continuous over two months, baseline to off study

• Estimate rate of AdhereTech device failure

  Continuous over two months, baseline to off study

• Estimate the perceived acceptability of using the AdhereTech device, as reported by caregivers and youth

  Continuous over two months, baseline to off study

Study Arms (1)

Sickle Cell Disease

All participants who meet eligibility requirements and consent to the study.

Eligibility Criteria

• Age: 12 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Participants ages 12.0-17.99 with Sickle cell disease any genotype and their caregivers

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Jerlym Porter, PhD

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 4, 2018
• First Posted: October 17, 2018
• Study Start: March 12, 2019
• Primary Completion: August 10, 2022
• Study Completion (Estimated): September 30, 2026
• Last Updated: April 23, 2026

Record last verified: 2026-04

Locations

US (1)