Sickle Cell DiseaseNCT03708731
Exploring Adherence Monitoring in Sickle Cell Disease
An observational study of Sickle Cell Disease, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial 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%. Read more about this study
- Who is it for?
- Ages 12 to 17 years
- Conditions the study is for
- Sickle Cell Disease
- How many people
- 36 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Mar 2019to 30 Sep 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 12 to 17 years
- All sexes
- No healthy volunteers
- Child
Study population: Participants ages 12.0-17.99 with Sickle cell disease any genotype and their caregivers
Who may be able to join
- 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
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.
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
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.
Read more
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
Read the 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.
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle Cell Disease, Youth, Hydroxyurea, Non-Adherence, Electronic Adherence Monitoring, AdhereTech smart bottle
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Sickle Cell Disease | All participants who meet eligibility requirements and consent to the study. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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 .
Measured: 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).
Measured: 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.
Measured: Continuous over two months, baseline to off study
Secondary outcomes (4)
Estimate rate of consent to the study
We will descriptively examine rate of refusal and study participation: (Number participants consented ÷ Total families approached) x 100%
Measured: Once, at baseline
Estimate rate of AdhereTech device use
We will descriptively examine rate of AdhereTech device use: (Number participants keeping medication in device during entire trial ÷ Total participants) x 100%
Measured: Continuous over two months, baseline to off study
Estimate rate of AdhereTech device failure
We will descriptively examine AdhereTech device failure (i.e., a device does not register openings as verified by study staff at the time device is returned to study team): (Number devices failing ÷ Total devices) x 100%
Measured: Continuous over two months, baseline to off study
Estimate the perceived acceptability of using the AdhereTech device, as reported by caregivers and youth
We will descriptively examine reported acceptability of using the AdhereTech device: Point estimation and interval estimations for the counts and frequencies of favorable responses of acceptability will be calculated.
Measured: Continuous over two months, baseline to off study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Oct 2018
- Study start12 Mar 2019
- Primary completion10 Aug 2022
- Study completion30 Sep 2026 (estimated)
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
- Jerlym Porter, PhD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03708731
- Sponsor's protocol ID
- ExAMS
- Status last verified
- Jun 2026
- First submitted
- 4 Oct 2018
- Last updated
- 17 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Exploring Adherence Monitoring in Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT03708731
- Acronym / sponsor ID
- ExAMS
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle Cell Disease, Youth, Hydroxyurea, Non-Adherence, Electronic Adherence Monitoring, AdhereTech smart bottle
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 36 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- 12 Mar 2019 (actual)
- Primary completion
- 10 Aug 2022 (actual)
- Study completion
- 30 Sep 2026 (estimated)
- First posted
- 17 Oct 2018 (actual)
- 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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sickle Cell Disease | – | –All participants who meet eligibility requirements and consent to the study. |
Who may qualify
- Ages
- 12 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Participants ages 12.0-17.99 with Sickle cell disease any genotype and their caregivers
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (3 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.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Estimate the association between AdhereTech device HU adherence to caregiver-report of adherence
Time frame: Continuous over two months, baseline to off study
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.
Estimate the association between AdhereTech device HU adherence to youth-reported adherence
Time frame: Continuous over two months, baseline to off study
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.
Estimate the association between AdhereTech device HU adherence to fetal hemoglobin (HbF) lab values
Time frame: Continuous over two months, baseline to off study
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.
Estimate the association between AdhereTech device HU adherence to mean corpuscular volume (MCV) lab values
Time frame: Continuous over two months, baseline to off study
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 .
Estimate the association between AdhereTech device HU adherence to the Medication Possession Ratio (MPR)
Time frame: Continuous over two months, baseline to off study
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).
Estimate the association between AdhereTech device HU adherence to pill count
Time frame: Continuous over two months, baseline to off study
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.
4 secondary outcomes
Estimate rate of consent to the study
Time frame: Once, at baseline
Estimate rate of AdhereTech device use
Time frame: Continuous over two months, baseline to off study
Estimate rate of AdhereTech device failure
Time frame: Continuous over two months, baseline to off study
Estimate the perceived acceptability of using the AdhereTech device, as reported by caregivers and youth
Time frame: Continuous over two months, baseline to off study
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jun 2026.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
Related trials for Sickle Cell Disease
Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
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.
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 5 inclusion and 3 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: under way, not looking for participants, as of the sponsor's last update on 17 Jun 2026. 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?
This study is still under way. Results are not available in the source record yet.
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?
Estimate the association between AdhereTech device HU adherence to caregiver-report of adherence (time frame: Continuous over two months, baseline to off study). The study lists 6 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 17 Jun 2026.