- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02371720
Patient Centered Comprehensive Medication Adherence Management System in Patients With Sickle Cell Disease (SCD Mobile Dot)
March 3, 2021 updated by: Lakshmanan Krishnamurti, Emory University
Patient Centered Comprehensive Medication Adherence Management System to Improve Effectiveness of Disease Modifying Therapy With Hydroxyurea in Patients With Sickle Cell Disease
The purpose of this research study is to learn about ways to help children and adults with sickle cell disease who are taking the medication, hydroxyurea.
Study Overview
Detailed Description
Sickle cell disease (SCD) is an inherited chronic multi-organ system disorder that affects approximately 100,000 individuals in the United States, mostly belonging to minority, under-served populations.
SCD is associated with substantial morbidity, premature mortality, individual suffering, health care costs and loss of productivity.
Hydroxyurea (HU) the only disease modifying therapy for SCD is efficacious in reducing complications such as pain crisis and acute chest syndrome and improving survival.
It is however, vastly underutilized and poorly adhered to because of barriers at the health care system, provider, treatment, socioeconomic, and patient levels.
The investigator's overarching hypothesis is that barriers to acceptance and adherence to HU are multi-factorial and that a structured set of interventions can lead to improved adherence to medication and patient centered outcomes.
Study Type
Interventional
Enrollment (Actual)
164
Phase
- Not Applicable
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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District of Columbia
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Washington, District of Columbia, United States, 20010
- Children's National Medical Center
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Georgia
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Atlanta, Georgia, United States, 30322
- Children's Healthcare of Atlanta
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Illinois
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Chicago, Illinois, United States, 60607
- University of Illinois at Chicago
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Pennsylvania
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Pittsburgh, Pennsylvania, United States, 15224
- Children's Hospital of Pittsburgh
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
2 years to 65 years (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- be >2 years of age up to 65 years of age, inclusive
- have a diagnosis of SCD, with either βS/βS, βS/βC, βS/βD, βS/β0, βS/βO-Arab, or βS/β+ genotype
- prescribed Hydroxyurea for at least the 6 months prior to study entry
- have daily access to a smart phone, tablet, personal computer or other device capable of producing and transmitting videos over the internet
- be willing and able to record and transmit videos
Exclusion Criteria:
- patient or caregiver refuses to take Hydroxyurea as treatment for SCD
- diagnosis of significant psychiatric disorder of the subject that could seriously impede the ability to participate in the study
- an assessment by the investigator that the subject will not comply with the study procedures outlined in the study protocol
- patients receiving automatic home delivery of medications since medication possession ratio is reflective of the patient initiation the refill when they have exhausted the home supply of HU
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Health Services Research
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Adults - Mobile DOT
Subjects with SCD that are older than 21 years old will receive comprehensive medication adherence management (Mobile DOT) after 1 month assessment period.
The subjects will receive the Mobile DOT intervention for 24 months.
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Daily reminders via text or email to send a video of themselves taking their Hydroxyurea, positive feedback, and be encouraged to contact the research coordinator with any questions, concerns, etc.
Other Names:
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Active Comparator: Adults - standard of care then Mobile DOT
Subjects with SCD that are older than 21 years old will receive standard of care for the first 12 months.
They will then crossover to the comprehensive medication adherence management plan (Mobile DOT) after 1 month assessment period for the remaining 12 months.
|
Daily reminders via text or email to send a video of themselves taking their Hydroxyurea, positive feedback, and be encouraged to contact the research coordinator with any questions, concerns, etc.
Other Names:
|
Experimental: Children - Mobile DOT
Subjects with SCD that are younger than 21 years old will receive comprehensive medication adherence management (Mobile DOT) after 1 month assessment period.
The subjects will receive the Mobile DOT intervention for 24 months.
|
Daily reminders via text or email to send a video of themselves taking their Hydroxyurea, positive feedback, and be encouraged to contact the research coordinator with any questions, concerns, etc.
Other Names:
|
Active Comparator: Children - standard of care then Mobile DOT
Subjects with SCD that are younger than 21 years old will receive standard of care for the first 12 months.
They will then crossover to the comprehensive medication adherence management plan (Mobile DOT) after 1 month assessment period for the remaining 12 months.
|
Daily reminders via text or email to send a video of themselves taking their Hydroxyurea, positive feedback, and be encouraged to contact the research coordinator with any questions, concerns, etc.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Medication Possession Ratio (MPR)
Time Frame: 12 months
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Proportion of days the patient is in possession of the medication in the study period
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12 months
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in Hemoglobin (Hb) levels
Time Frame: Baseline, 24 months
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Change in hemoglobin levels from baseline to 24 months will be measured using the HemoCue® rapid test.
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Baseline, 24 months
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Change in mean cell volume (MCV)
Time Frame: Baseline, 24 months
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Change from baseline in MCV will be calculated as the value at 24 months minus the value at baseline.
MCV is the average size of the red blood cells expressed in femtoliters.
MCV is calculated by dividing the hematocrit (as percent) by the red blood cell (RBC) count in millions per microliter of blood, then multiplying by 10.
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Baseline, 24 months
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Change in fetal hemoglobin (HbF) levels
Time Frame: Baseline, 24 months
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Change from baseline in HbF will be calculated as the value at 24 months minus the value at baseline.
HbF is expressed as a percentage.
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Baseline, 24 months
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Impact of adherence on clinical outcomes and healthcare utilization
Time Frame: Baseline, 24 months
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Health care utilization in the emergency department and hospitalization due to sickle cell related complications such as vaso-occlusive crisis (VOC) or acute chest syndrome (ACS).
Retrospective chart review at baseline will be conducted to determine healthcare utilization.
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Baseline, 24 months
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Impact of adherence on patients' lives
Time Frame: Baseline, 24 months
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Impact of adherence on patients' lives measured using patient reported outcomes (PROMIS), surveys of school attendance, work absenteeism, out-of-pocket costs incurred by patients and their caregivers
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Baseline, 24 months
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Change in adherence with using Mobile-DOT
Time Frame: Baseline, 24 months
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Retrospective chart review at baseline will be conducted to determine medication possession rate (MPR) and then compared to the MPR at 24 months.
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Baseline, 24 months
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Acceptability of intervention and of Hydroxyurea
Time Frame: Baseline, 24 months
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Acceptability will be measured by Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) The TSQM is a 14-item subject-assessed evaluation of treatment medication including 4 factors, Effectiveness, Side Effects, Convenience, and Global Satisfaction, and it utilizes the following responses on a 7-point Likert scale: 1=Extremely Dissatisfied, 2=Very Dissatisfied, 3=Somewhat Dissatisfied, 4=Neither Satisfied Nor Dissatisfied, 5=Somewhat Satisfied, 6=Very Satisfied, 7=Extremely Satisfied.
Scores range from 0-100, with 0 as extremely dissatisfied and 100 as extremely satisfied.
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Baseline, 24 months
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Study Chair: Lakshmanan Krishnamurti, MD, Emory University/Children's Healthcare of Atlanta
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
June 1, 2014
Primary Completion (Actual)
December 31, 2018
Study Completion (Actual)
December 31, 2018
Study Registration Dates
First Submitted
February 19, 2015
First Submitted That Met QC Criteria
February 19, 2015
First Posted (Estimate)
February 26, 2015
Study Record Updates
Last Update Posted (Actual)
March 5, 2021
Last Update Submitted That Met QC Criteria
March 3, 2021
Last Verified
March 1, 2021
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- IRB00074105
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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