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

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ClinicalTrials.gov Identifier: NCT02371720
Recruitment Status : Recruiting
First Posted : February 26, 2015
Last Update Posted : July 23, 2018
Sponsor:
Collaborator:
Patient-Centered Outcomes Research Institute
Information provided by (Responsible Party):
Lakshmanan Krishnamurti, Emory University

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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Behavioral: Mobile DOT Not Applicable

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  (Clinical Trial)
Estimated Enrollment : 160 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Health Services Research
Official Title: Patient Centered Comprehensive Medication Adherence Management System to Improve Effectiveness of Disease Modifying Therapy With Hydroxyurea in Patients With Sickle Cell Disease
Actual Study Start Date : June 1, 2014
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine

Drug Information available for: Hydroxyurea

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.
Behavioral: Mobile DOT
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 Name: Comprehensive medication adherence management

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.
Behavioral: Mobile DOT
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 Name: Comprehensive medication adherence management

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.
Behavioral: Mobile DOT
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 Name: Comprehensive medication adherence management

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.
Behavioral: Mobile DOT
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 Name: Comprehensive medication adherence management




Primary Outcome Measures :
  1. Medication Possession Ratio (MPR) [ Time Frame: 12 months ]
    Proportion of days the patient is in possession of the medication in the study period


Secondary Outcome Measures :
  1. Change in Hemoglobin (Hb) levels [ Time Frame: Baseline, 24 months ]
    Change in hemoglobin levels from baseline to 24 months will be measured using the HemoCue® rapid test.

  2. Change in mean cell volume (MCV) [ Time Frame: Baseline, 24 months ]
    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 RBC count in millions per microliter of blood, then multiplying by 10.

  3. Change in fetal hemoglobin (HbF) levels [ Time Frame: Baseline, 24 months ]
    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.

  4. Impact of adherence on clinical outcomes and healthcare utilization [ Time Frame: Baseline, 24 months ]
    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.

  5. Impact of adherence on patients' lives [ Time Frame: Baseline, 24 months ]
    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

  6. Change in adherence with using Mobile-DOT [ Time Frame: Baseline, 24 months ]
    Retrospective chart review at baseline will be conducted to determine medication possession rate (MPR) and then compared to the MPR at 24 months.

  7. Acceptability of intervention and of Hydroxyurea [ Time Frame: Baseline, 24 months ]
    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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Ages Eligible for Study:   2 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 Hydroxyurea.
  • 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.
  • Patient is on liquid form of Hydroxyurea since MPR - calculations are difficult.
  • 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.

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): NCT02371720


Contacts
Contact: Leann Schilling 404-785-2025 leann.schilling@choa.org

Locations
United States, District of Columbia
Children's National Medical Center Recruiting
Washington, District of Columbia, United States, 20010
Contact: Deepika Darbari, MD    202-884-2140      
Principal Investigator: Deepikia Darbari, MD         
United States, Georgia
Children's Healthcare of Atlanta Recruiting
Atlanta, Georgia, United States, 30322
Contact: Leann Schilling    404-785-2025    leann.schilling@choa.org   
Principal Investigator: Robert C Brown, MD, PhD         
United States, Pennsylvania
Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Angela Martino, BSN    412-692-6467    angela.martino2@chp.edu   
Sponsors and Collaborators
Emory University
Patient-Centered Outcomes Research Institute
Investigators
Study Chair: Lakshmanan Krishnamurti, MD Emory University/Children's Healthcare of Atlanta

Responsible Party: Lakshmanan Krishnamurti, Professor, Emory University
ClinicalTrials.gov Identifier: NCT02371720     History of Changes
Other Study ID Numbers: IRB00074105
First Posted: February 26, 2015    Key Record Dates
Last Update Posted: July 23, 2018
Last Verified: July 2018

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Hydroxyurea
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors