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Patient Centered Comprehensive Medication Adherence Management System in Patients With Sickle Cell Disease (SCD Mobile Dot)

Primary Purpose

Sickle Cell Disease

Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Mobile DOT
Sponsored by
Emory University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional health services research trial for Sickle Cell Disease

Eligibility Criteria

2 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Children's National Medical Center
  • Children's Healthcare of Atlanta
  • University of Illinois at Chicago
  • Children's Hospital of Pittsburgh

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Active Comparator

Experimental

Active Comparator

Arm Label

Adults - Mobile DOT

Adults - standard of care then Mobile DOT

Children - Mobile DOT

Children - standard of care then Mobile DOT

Arm Description

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.

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.

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.

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.

Outcomes

Primary Outcome Measures

Medication Possession Ratio (MPR)
Proportion of days the patient is in possession of the medication in the study period

Secondary Outcome Measures

Change in Hemoglobin (Hb) levels
Change in hemoglobin levels from baseline to 24 months will be measured using the HemoCue® rapid test.
Change in mean cell volume (MCV)
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.
Change in fetal hemoglobin (HbF) levels
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.
Impact of adherence on clinical outcomes and healthcare utilization
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.
Impact of adherence on patients' lives
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
Change in adherence with using Mobile-DOT
Retrospective chart review at baseline will be conducted to determine medication possession rate (MPR) and then compared to the MPR at 24 months.
Acceptability of intervention and of Hydroxyurea
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.

Full Information

First Posted
February 19, 2015
Last Updated
March 3, 2021
Sponsor
Emory University
Collaborators
Patient-Centered Outcomes Research Institute
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1. Study Identification

Unique Protocol Identification Number
NCT02371720
Brief Title
Patient Centered Comprehensive Medication Adherence Management System in Patients With Sickle Cell Disease
Acronym
SCD Mobile Dot
Official Title
Patient Centered Comprehensive Medication Adherence Management System to Improve Effectiveness of Disease Modifying Therapy With Hydroxyurea in Patients With Sickle Cell Disease
Study Type
Interventional

2. Study Status

Record Verification Date
March 2021
Overall Recruitment Status
Completed
Study Start Date
June 1, 2014 (Actual)
Primary Completion Date
December 31, 2018 (Actual)
Study Completion Date
December 31, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Emory University
Collaborators
Patient-Centered Outcomes Research Institute

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease

7. Study Design

Primary Purpose
Health Services Research
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
164 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Adults - Mobile DOT
Arm Type
Experimental
Arm Description
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.
Arm Title
Adults - standard of care then Mobile DOT
Arm Type
Active Comparator
Arm Description
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.
Arm Title
Children - Mobile DOT
Arm Type
Experimental
Arm Description
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.
Arm Title
Children - standard of care then Mobile DOT
Arm Type
Active Comparator
Arm Description
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.
Intervention Type
Behavioral
Intervention Name(s)
Mobile DOT
Other Intervention Name(s)
Comprehensive medication adherence management
Intervention Description
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.
Primary Outcome Measure Information:
Title
Medication Possession Ratio (MPR)
Description
Proportion of days the patient is in possession of the medication in the study period
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Change in Hemoglobin (Hb) levels
Description
Change in hemoglobin levels from baseline to 24 months will be measured using the HemoCue® rapid test.
Time Frame
Baseline, 24 months
Title
Change in mean cell volume (MCV)
Description
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.
Time Frame
Baseline, 24 months
Title
Change in fetal hemoglobin (HbF) levels
Description
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.
Time Frame
Baseline, 24 months
Title
Impact of adherence on clinical outcomes and healthcare utilization
Description
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.
Time Frame
Baseline, 24 months
Title
Impact of adherence on patients' lives
Description
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
Time Frame
Baseline, 24 months
Title
Change in adherence with using Mobile-DOT
Description
Retrospective chart review at baseline will be conducted to determine medication possession rate (MPR) and then compared to the MPR at 24 months.
Time Frame
Baseline, 24 months
Title
Acceptability of intervention and of Hydroxyurea
Description
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.
Time Frame
Baseline, 24 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Lakshmanan Krishnamurti, MD
Organizational Affiliation
Emory University/Children's Healthcare of Atlanta
Official's Role
Study Chair
Facility Information:
Facility Name
Children's National Medical Center
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Children's Healthcare of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
University of Illinois at Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60607
Country
United States
Facility Name
Children's Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Patient Centered Comprehensive Medication Adherence Management System in Patients With Sickle Cell Disease

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