Medication Adherence in Youth With Sickle Cell Disease (SCD)

• ClinicalTrials.gov Identifier: NCT02143076
• Recruitment Status: Completed
• First Posted: May 20, 2014
• Last Update Posted: May 27, 2015
• Sponsor: St. Jude Children's Research Hospital
• Information provided by (Responsible Party): St. Jude Children's Research Hospital

Tracking Information

• First Submitted Date: May 16, 2014
• First Posted Date: May 20, 2014
• Last Update Posted Date: May 27, 2015
• Study Start Date: May 2014
• Actual Primary Completion Date: May 2015 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: May 16, 2014)

• Percent Rate of Consent to Study Participation [ Time Frame: Baseline ]
  Of the total number of eligible participants, the percent of participants who agree to participate.
• Percent Rate of Device Use [ Time Frame: Two months after baseline ]
  The percent of the total participants who keep medicine in the device during the entire trial period.
• Percent Rate of Device Failure [ Time Frame: Two months after baseline ]
  Percent of devices that failed defined as a device does not register opening as verified by study staff at the time device is returned to the study team.
• Acceptability of Using the Wise Device [ Time Frame: Two months after baseline ]
  Responses by participants and/or caregivers to the Acceptability Questionnaire based on the Likert scale from strongly disagree to strongly agree.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: May 16, 2014)

Medication Adherence as Measured by Wise Device Compared to Other Measures of Medication Adherence [ Time Frame: Two months after baseline ]

Adherence will be determined and compared between: a) adherence indicated by Wise device, b) adherence indicated by caregiver and youth-report, c) adherence indicated by lab values, d) adherence indicated by pill count/bottle volume, and e) adherence indication by medication possession ratio (MPR). Each participant's Wise adherence will be calculated as: [(number of device openings ÷ medication prescribed) x 100] where larger percentages suggest better adherence (range: 0-100%). Descriptive statistics for Wise device adherence and other adherence measures, including mean and standard deviation, median and interquartile range, will be estimated. Spearman's rank correlation coefficients and intraclass correlations between different adherence measures will be calculated to measure the associations between HU adherence measures for the entire sample as well as for each age group.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Medication Adherence in Youth With Sickle Cell Disease (SCD)
• Official Title: Use of WISE Device Technology to Measure Medication Adherence in Youth With Sickle Cell Disease (SCD): A Pilot Study

Brief Summary

Youth diagnosed with sickle cell disease (SCD) may have difficulty taking medication as prescribed (adherence). Hydroxyurea (HU) is one medication that youth may take to help manage SCD. 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" (i.e. where individuals do not take medications for a specified interval of time), and "white coat" adherence (i.e., a pattern of drug adherence as a function of time where individuals display good adherence immediately before and after clinic attendance with worsening adherence in the period between).

Overall, electronic adherence measures are considered valid, reliable, and accurate, with clear advantages over pharmacy refill records, physician estimates and self-report measures. Currently, only one electronic measure capable of monitoring medications in both pill and liquid form is being manufactured: WisePill and WiseBag. While data are limited regarding its validity and reliability, preliminary data support the use of Wise technology to measure adherence to medication. The current study will determine the Wise device's ability to feasibly measure adherence to liquid and solid form HU medication in a pediatric SCD population.

Detailed Description

This research study will evaluate HU adherence using an electronic storage device (Wise device) compared to caregiver report, youth report, lab values, and pill-count adherence measures. The investigators will ask participants to store their medication in the Wise device and answer questions about their use of the device during normal clinical care visits.

PRIMARY OBJECTIVE

• To examine feasibility of the WISE device by estimating a) rate of consent to the study, b) rate of WISE device use, c) rate of WISE device failure, and d) the perceived acceptability of using the WISE device, as reported by caregivers and youth.

SECONDARY OBJECTIVE

• To estimate the association between HU adherence as measured by the Wise device, to a) caregiver-report, b) youth-report, c) lab values, d) pill-count, and e) Medication Possession Ratio (MPR) adherence measures.

Evaluation of study outcomes will be stratified by age group: (1) infants/toddlers, ages 0-7 years; (2) school age, ages 8-12 years; and (3) teen, ages 13-17 years.

At study enrollment, participants will be asked to complete one questionnaire and will receive instructions on how to use the Wise device. They will be asked to use the Wise device over the following two months. A study team member will contact each participant by phone within seven days of study enrollment to monitor participant use of the Wise device and to answer any questions. Participants will be asked to return the Wise device in two months at their normal clinic care visit. At this visit, participants will be asked to complete two more questionnaires and, as part of standard clinical care, a pill count/bottle weight will be completed by the pharmacy.

• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Participants will have a diagnosis of sickle cell disease,be receiving treatment with hydroxyurea, and meet eligibility criteria.
• Condition: Sickle Cell Disease

Intervention

• Behavioral: Demographic Questionnaire
  At study enrollment, participants will complete a questionnaire to collect demographic information about the participant's family background, education and living situation.
• Behavioral: Medical Adherence Measure Questionnaire

  At the two-month follow-up visit, participants will be asked to complete a questionnaire describing all medications prescribed for them, when they take their medications, how much medication they take each time, how many times during the week they missed taking or were late taking each medication, reasons why they missed or were late, and how they manage their medications.

  In the case that a participant's clinic schedule is delayed greater than two months from the time of consent (e.g., missed appointment, change in clinic appointment frequency), procedures scheduled for the anticipated 2-month visit will take place at the participant's next clinic appointment.

• Behavioral: Acceptability Questionnaire

  At the two-month follow-up visit, participants will be asked to complete a questionnaire about their perceptions of using the Wise electronic medication container.

  In the case that a participant's clinic schedule is delayed greater than two months from the time of consent (e.g., missed appointment, change in clinic appointment frequency), procedures scheduled for the anticipated 2-month visit will take place at the participant's next clinic appointment.

Study Groups/Cohorts

Sickle Cell Disease

Participants will complete three questionnaires during the course of the study: Demographic Questionnaire, Medical Adherence Measure Questionnaire, and Acceptability Questionnaire. All questionnaires will be completed in a private clinic room.

Interventions:

• Behavioral: Demographic Questionnaire
• Behavioral: Medical Adherence Measure Questionnaire
• Behavioral: Acceptability Questionnaire

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: May 26, 2015): 36
• Original Estimated Enrollment (submitted: May 16, 2014): 60
• Actual Study Completion Date: May 2015
• Actual Primary Completion Date: May 2015 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Confirmed diagnosis of SCD (any genotype).
• Less than 18 years of age at time of study entry.
• Currently followed in the St. Jude Children's Research Hospital (SJCRH) SCD clinic.
• Stable HU dose prescribed (in pill or liquid formulation) for ≥ 6 months without documented hematological toxicity (excluding dose adjustments for weight gain).
• Lives with their primary caregiver.
• Anticipated to return to clinic at proposed 4-week or 8-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.
• Any condition or chronic illness that in the opinion of the PI or Co-I makes participation on the study ill-advised.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: up to 17 Years (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT02143076
• Other Study ID Numbers: WISEP
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: St. Jude Children's Research Hospital
• Original Responsible Party: Same as current
• Current Study Sponsor: St. Jude Children's Research Hospital
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Lisa Ingerski, PhD; St. Jude Children's Research Hospital
• Principal Investigator: Jerlym Porter, PhD; St. Jude Children's Research Hospital

• PRS Account: St. Jude Children's Research Hospital
• Verification Date: May 2015