Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Exploring Adherence Monitoring in Sickle Cell Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03708731
Recruitment Status : Recruiting
First Posted : October 17, 2018
Last Update Posted : March 22, 2019
Sponsor:
Information provided by (Responsible Party):
St. Jude Children's Research Hospital

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


Condition or disease
Sickle Cell Disease

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.


Layout table for study information
Study Type : Observational
Estimated Enrollment : 45 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Exploring Adherence Monitoring in Sickle Cell Disease
Actual Study Start Date : March 12, 2019
Estimated Primary Completion Date : January 2020
Estimated Study Completion Date : January 2024

Resource links provided by the National Library of Medicine


Group/Cohort
Sickle Cell Disease
All participants who meet eligibility requirements and consent to the study.



Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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 .

  5. 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).

  6. 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.


Secondary Outcome Measures :
  1. Estimate rate of consent to the study [ Time Frame: Once, at baseline ]
    We will descriptively examine rate of refusal and study participation: (Number participants consented ÷ Total families approached) x 100%

  2. Estimate rate of AdhereTech device use [ Time Frame: Continuous over two months, baseline to off study ]
    We will descriptively examine rate of AdhereTech device use: (Number participants keeping medication in device during entire trial ÷ Total participants) x 100%

  3. Estimate rate of AdhereTech device failure [ Time Frame: Continuous over two months, baseline to off study ]
    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%

  4. 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 ]
    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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   12 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Participants ages 12.0-17.99 with Sickle cell disease any genotype and their caregivers
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03708731


Contacts
Layout table for location contacts
Contact: Jerlym Porter, PhD 866-278-5833 referralinfo@stjude.org

Locations
Layout table for location information
United States, Tennessee
St. Jude Children's Research Hospital Recruiting
Memphis, Tennessee, United States, 38105
Contact: Jerlym Porter, PhD    866-278-5833    referralinfo@stjude.org   
Principal Investigator: Jerlym Porter, PhD         
Sponsors and Collaborators
St. Jude Children's Research Hospital
Investigators
Layout table for investigator information
Principal Investigator: Jerlym Porter, PhD St. Jude Children's Research Hospital

Additional Information:
Layout table for additonal information
Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT03708731     History of Changes
Other Study ID Numbers: ExAMS
First Posted: October 17, 2018    Key Record Dates
Last Update Posted: March 22, 2019
Last Verified: March 2019

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by St. Jude Children's Research Hospital:
Sickle Cell Disease
Youth
Hydroxyurea
Non-Adherence
Electronic Adherence Monitoring
AdhereTech smart bottle
Additional relevant MeSH terms:
Layout table for MeSH terms
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn