Sickle Cell DiseaseSCD Mobile DotNCT02371720
Patient Centered Comprehensive Medication Adherence Management System in Patients With Sickle Cell Disease
A study testing Mobile DOT in people with Sickle Cell Disease, run by Emory University at 4 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 65 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Mobile DOT
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 164 participants
- Where
- 4 sitesUnited States
- Study dates
- 1 Jun 2014to 31 Dec 2018
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 2 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
University of Illinois at Chicago
Chicago, Illinois, 60607
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: 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.
Read the 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.
- Conditions
- Sickle Cell Disease
- Being tested
- Mobile DOT
- 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.
Mobile DOTBehavioural
Also called Comprehensive medication adherence management
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.
| Study group | Role | What they receive |
|---|---|---|
| Adults - Mobile DOT | Experimental | 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. Receives: Behavioral: Mobile DOT |
| Adults - standard of care then Mobile DOT | Active comparator | 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. Receives: Behavioral: Mobile DOT |
| Children - Mobile DOT | Experimental | 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. Receives: Behavioral: Mobile DOT |
| Children - standard of care then Mobile DOT | Active comparator | 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. Receives: Behavioral: Mobile DOT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Medication Possession Ratio (MPR)
Proportion of days the patient is in possession of the medication in the study period
Measured: 12 months
Secondary outcomes (7)
Change in Hemoglobin (Hb) levels
Change in hemoglobin levels from baseline to 24 months will be measured using the HemoCue® rapid test.
Measured: Baseline, 24 months
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.
Measured: Baseline, 24 months
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.
Measured: Baseline, 24 months
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.
Measured: Baseline, 24 months
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
Measured: Baseline, 24 months
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.
Measured: Baseline, 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.
Measured: Baseline, 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Feb 2015 (estimated)
- Study start1 Jun 2014
- Primary completion31 Dec 2018
- Study completion31 Dec 2018
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 Emory UniversityThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Health services research
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 164 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- Patient-Centered Outcomes Research Institute
- Responsible party
- Principal investigator, Lakshmanan Krishnamurti, Emory University
- Investigators
- Lakshmanan Krishnamurti, MD, Study chair, Emory University/Children's Healthcare of Atlanta
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02371720
- Sponsor's protocol ID
- IRB00074105
- Acronym
- SCD Mobile Dot
- Status last verified
- Mar 2021
- First submitted
- 19 Feb 2015
- Last updated
- 5 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Patient Centered Comprehensive Medication Adherence Management System to Improve Effectiveness of Disease Modifying Therapy With Hydroxyurea in Patients With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT02371720
- Acronym / sponsor ID
- SCD Mobile Dot / IRB00074105
- Conditions
- Sickle Cell Disease
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 164 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- Patient-Centered Outcomes Research Institute
- Study start
- 1 Jun 2014 (actual)
- Primary completion
- 31 Dec 2018 (actual)
- Study completion
- 31 Dec 2018 (actual)
- First posted
- 26 Feb 2015 (estimated)
- 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.
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.
Study design
- Primary purpose
- Health services research
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Adults - Mobile DOT | Experimental | Behavioral: Mobile DOTSubjects 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. |
| Adults - standard of care then Mobile DOT | Active comparator | Behavioral: Mobile DOTSubjects 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. |
| Children - Mobile DOT | Experimental | Behavioral: Mobile DOTSubjects 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. |
| Children - standard of care then Mobile DOT | Active comparator | Behavioral: Mobile DOTSubjects 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. |
Who may qualify
- Ages
- 2 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mobile DOTAlso: Comprehensive medication adherence managementDaily 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. | Behavioural | Adults - Mobile DOT, Adults - standard of care then Mobile DOT, Children - Mobile DOT, Children - standard of care then Mobile DOT |
Primary outcome
Medication Possession Ratio (MPR)
Time frame: 12 months
Proportion of days the patient is in possession of the medication in the study period
7 secondary outcomes
Change in Hemoglobin (Hb) levels
Time frame: Baseline, 24 months
Change in mean cell volume (MCV)
Time frame: Baseline, 24 months
Change in fetal hemoglobin (HbF) levels
Time frame: Baseline, 24 months
Impact of adherence on clinical outcomes and healthcare utilization
Time frame: Baseline, 24 months
Impact of adherence on patients' lives
Time frame: Baseline, 24 months
Change in adherence with using Mobile-DOT
Time frame: Baseline, 24 months
Acceptability of intervention and of Hydroxyurea
Time frame: Baseline, 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2021.
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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.
Lakshmanan Krishnamurti, MD study chair
Emory University/Children's Healthcare of Atlanta
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Mobile DOT in people with Sickle Cell Disease, run by Emory University at 4 sites 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 4 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: finished, as of the sponsor's last update on 5 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Medication Possession Ratio (MPR) (time frame: 12 months). 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 5 Mar 2021; AzTrials last refreshed its copy on 1 Nov 2026.