Sickle Cell DiseaseNCT02143076
Medication Adherence in Youth With Sickle Cell Disease (SCD)
An observational study of Sickle Cell Disease, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 17 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Demographic Questionnaire, Medical Adherence Measure Questionnaire and Acceptability Questionnaire
- Type of treatment
- Lifestyle or therapy programme
- How many people
- 36 participants
- Where
- 1 siteUnited States
- Study dates
- May 2014to May 2015
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 17 years
- All sexes
- No healthy volunteers
- Child
Study population: Participants will have a diagnosis of sickle cell disease,be receiving treatment with hydroxyurea, and meet eligibility criteria.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
Official title: Use of WISE Device Technology to Measure Medication Adherence in Youth With Sickle Cell Disease (SCD): A Pilot Study
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.
Read the 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.
- Conditions
- Sickle Cell Disease
- Being tested
- Demographic Questionnaire, Medical Adherence Measure Questionnaire, Acceptability Questionnaire
- Keywords
- Medication Adherence, Hydroxyurea
- Medical subject headings
- Anemia, Sickle Cell, Medication Adherence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Demographic QuestionnaireBehavioural
At study enrollment, participants will complete a questionnaire to collect demographic information about the participant's family background, education and living situation.
Medical Adherence Measure QuestionnaireBehavioural
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.
Acceptability QuestionnaireBehavioural
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 group | Role | What they receive |
|---|---|---|
| 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. Receives: Behavioral: Demographic Questionnaire; Behavioral: Medical Adherence Measure Questionnaire; Behavioral: Acceptability Questionnaire |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Rate of Consent to Study Participation
Of the total number of eligible participants, the percent of participants who agree to participate.
Measured: Baseline
Percent Rate of Device Use
The percent of the total participants who keep medicine in the device during the entire trial period.
Measured: Two months after baseline
Percent Rate of Device Failure
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.
Measured: Two months after baseline
Acceptability of Using the Wise Device
Responses by participants and/or caregivers to the Acceptability Questionnaire based on the Likert scale from strongly disagree to strongly agree.
Measured: Two months after baseline
Secondary outcomes (1)
Medication Adherence as Measured by Wise Device Compared to Other Measures of Medication Adherence
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.
Measured: Two months after baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2014 (estimated)
- Study startMay 2014
- Primary completionMay 2015
- Study completionMay 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Lisa Ingerski, PhD, Principal investigator, St. Jude Children's Research HospitalJerlym Porter, PhD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02143076
- Sponsor's protocol ID
- WISEP
- Status last verified
- May 2015
- First submitted
- 16 May 2014
- Last updated
- 27 May 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Use of WISE Device Technology to Measure Medication Adherence in Youth With Sickle Cell Disease (SCD): A Pilot Study
- ClinicalTrials.gov ID
- NCT02143076
- Acronym / sponsor ID
- WISEP
- Conditions
- Sickle Cell Disease
- Keywords
- Medication Adherence, Hydroxyurea
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- May 2014
- Primary completion
- May 2015 (actual)
- Study completion
- May 2015 (actual)
- First posted
- 20 May 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sickle Cell Disease | – | Behavioral: Demographic Questionnaire, Behavioral: Medical Adherence Measure Questionnaire, Behavioral: Acceptability QuestionnaireParticipants 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. |
Who may qualify
- Ages
- Up to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Participants will have a diagnosis of sickle cell disease,be receiving treatment with hydroxyurea, and meet eligibility criteria.
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Demographic QuestionnaireAt study enrollment, participants will complete a questionnaire to collect demographic information about the participant's family background, education and living situation. | Behavioural | Sickle Cell Disease |
| Medical Adherence Measure QuestionnaireAt 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. | Behavioural | Sickle Cell Disease |
| Acceptability QuestionnaireAt 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. | Behavioural | Sickle Cell Disease |
Primary outcome
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.
1 secondary outcome
Medication Adherence as Measured by Wise Device Compared to Other Measures of Medication Adherence
Time frame: Two months after baseline
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 May 2015.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lisa Ingerski, PhD principal investigator
St. Jude Children's Research Hospital
Jerlym Porter, PhD principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease, run by St. Jude Children's Research Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 May 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Rate of Consent to Study Participation (time frame: Baseline). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 May 2015.