Sickle Cell DiseaseNCT03344900
Re-Aiming at Hydroxyurea Adherence for Sickle Cell With mHealth
An observational study of Sickle Cell Disease, run by St. Jude Children's Research Hospital at 2 sites in United States.
Trial summary
National Institutes of Health (NIH)/National Heart, Lung, and Blood Institute (NHLBI) guidelines recommend that hydroxyurea be offered to symptomatic adults and all children with sickle cell disease (SCD) (HbSS and HbSβ0-thal genotypes) age ≥9 months. Read more about this study
- Who is it for?
- Ages 15 to 44 years
- Conditions the study is for
- Sickle Cell Disease
- How many people
- 100 participants
- Where
- 2 sitesUnited States
- Study dates
- 5 Dec 2017to 28 Jun 2018
- Phase
- Observational
- 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 15 to 44 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Participants will reside in the Memphis, Tennessee, area and meet the inclusion criteria.
Who may be able to join
- Diagnosis of sickle cell disease (any genotype)
- Between the ages of 15 and 44.9 years
- English-speaking
Who cannot join
- Inability or unwillingness of research participant or legal guardian/representative to give written informed consent
Trial locations and contacts
2 sites in 1 country.
United States2 sites
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Baptist Memorial Hospital, Baptist Clinical Research Institute
Memphis, Tennessee, 38120
About this study
National Institutes of Health (NIH)/National Heart, Lung, and Blood Institute (NHLBI) guidelines recommend that hydroxyurea be offered to symptomatic adults and all children with sickle cell disease (SCD) (HbSS and HbSβ0-thal genotypes) age ≥9 months. Research has shown that hydroxyurea reduces hospitalizations and mortality, supporting its effectiveness outside of clinical trials. Hydroxyurea is given as a once-daily oral dose that costs <$1 per day. Despite overwhelming evidence for positive effects, hydroxyurea is vastly underutilized. Given the relative ease of its administration, low cost, and safety profile, barriers to hydroxyurea utilization are primarily constrained to the health system and patient determinants. System-level barriers include insufficient access to SCD-specific care, limited access to medication (due to lack of health coverage), and providers' reluctance in prescribing it; while patient-level barriers include low acceptance (due to insufficient knowledge or misconceptions regarding risks and benefits), and forgetfulness leading to poor adherence.
Mobile health (mHealth) refers to the practice of medicine and public health supported by mobile devices. Short message service (SMS) text messaging (through cell phones) is a widespread means of communication, particularly among adolescents and young adults and is an emerging intervention modality to improve medication adherence. Its low cost, simplicity, and prevalence allow for relatively easy adoption and dissemination in medical practices.
Read more
This protocol seeks to examine barriers to hydroxyurea adherence among SCD patients between 15 and 45 years of age who are living in the Memphis region by conducting a needs assessment. In addition to examining these barriers, the needs assessment will provide data that will inform the development of an mHealth application (e.g. mobile phone application) for assisting patients in increasing their medication adherence. The developed mHealth intervention will then undergo a pilot trial to test its acceptability, satisfaction, and feasibility among 56 patients living with SCD.
- To conduct multi-level needs assessment of hydroxyurea utilization barriers and facilitators, in Memphis, Tennessee (Phase I).
- To test the feasibility and acceptance of a patient-informed smart phone application aimed at improving hydroxyurea adherence in the Memphis, Tennessee region, and to estimate the efficacy parameters needed to design a definitive large phase III trial (Phase II).
Read the detailed description
Data for the needs assessment and pilot trial will be collected from the patients via validated surveys, qualitative methods (e.g., interviews and focus groups), and medical record abstraction.
The study will utilize a Hybrid Effectiveness-Implementation design that will blend both a new intervention to impact the effectiveness of hydroxyurea, text messaging, and study its potential for adoption and dissemination in "real-world" practice settings. In Phase I (formative process evaluation), actionable barriers of hydroxyurea adherence in Memphis, Tennessee, will be identified. The investigators will begin by conducting a broad evaluation of hydroxyurea utilization, impediments and facilitators of hydroxyurea adherence and care management in general for patients with SCD. Next, this information will be merged with patients' preferences for the content of an mHealth intervention, and test it in a local cohort (Phase II). If successful and approved by the Sickle Cell Disease Implementation Consortium (SCDIC) executive committee, this intervention will advance to the next phase, namely, the dissemination and implementation in a larger geographical area (the other SCDIC participating sites in Phase III). We will utilize the RE-AIM framework to evaluate the intervention representativeness and impact during all phases.
- Conditions
- Sickle Cell Disease
- Keywords
- mHealth, Medication adherence, Barriers to adherence
- Medical subject headings
- Anemia, Sickle Cell, Medication Adherence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Phase I | It is estimated that 100 participants with SCD will be enrolled for the Phase I portion which will identify barriers to hydroxyurea utilization. | |
| Phase II | It is estimated that 72 participants with SCD will be enrolled for the Phase II portion of the study which will evaluate the degree of feasibility and acceptance of mHealth intervention on hydroxyurea adherence. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Compare the number of patients with hydroxyurea barriers or facilitators in hydroxyurea maintenance users and hydroxyurea former users/non-users (Phase I)
Multi-level needs assessment of hydroxyurea utilization barriers and facilitators, in Memphis, Tennessee, will be conducted to identify potential factors that affect the barriers or facilitators of hydroxyurea acceptance and that affect the adherence/maintenance of hydroxyurea. Enrollment for Phase I is estimated to be 100. The numbers of patients with barriers or facilitators in hydroxyurea maintenance users and hydroxyurea former users/non-users will be provided and will be compared using Fisher's exact text. This will be done via questionnaires with individuals living with sickle cell disease.
Measured: From on-study date up to 12 months later
Number of participants who are enrolled and randomized on the study (Phase II)
In terms of feasibility of enrollment and randomization, that is, we will assess if we can enroll and randomize 40% (versus an unacceptable rate of 30%) of the approached eligible individuals (56 patients out of 150 approached) in the 24-month accrual duration.
Measured: From study start date through up to 24 months later
Number of randomized patients completing baseline and 24-week evaluations for both arms
In terms of the feasibility of conducting a randomized trial, that is, after randomization, per each arm, we will assess if we can have 75% (versus an unacceptable rate of 50%) of randomized individuals (19 patients out of 28 randomized per arm) will complete at least baseline and 24-week evaluations.
Measured: From on-study date up to 24 weeks later
Compare the number of participants who report themHealth intervention is useful
Measure the patients' perceived usefulness of mHealth intervention as an aid to hydroxyurea adherence. The proportions of patients who reported the mHealth intervention is useful at weeks 12 and 24 will be provided and will be tested to see if it is significantly different from random at a significance level of 0.05.
Measured: Weeks 12 and 24
The mean and standard deviation of change in satisfaction with mHealth intervention
Measure satisfaction of the intervention by comparing the change in satisfaction measured by the Treatment Satisfaction Questionnaire for Medication (TSQM-9) from baseline to 24 weeks. The change in satisfaction from baseline to 24 weeks will be summarized using mean and standard deviation in the text messaging arm and will be tested using t-test or Wilcoxon signed rank test depending on the normality of the data tested by Shapiro Wilk test at a significance level of 0.05.
Measured: From on-study date up to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Nov 2017
- Study start5 Dec 2017
- Primary completion28 Jun 2018
- Study completion28 Jun 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 St. Jude Children's Research HospitalThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- University of Memphis, National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Jane S. Hankins, MD, MS, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03344900
- Sponsor's protocol ID
- REHASH
- Other IDs
- U01HL133996
- Status last verified
- Nov 2019
- First submitted
- 6 Nov 2017
- Last updated
- 11 Dec 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Re-Aiming at Hydroxyurea Adherence for Sickle Cell With mHealth
- ClinicalTrials.gov ID
- NCT03344900
- Acronym / sponsor ID
- REHASH
- Conditions
- Sickle Cell Disease
- Keywords
- mHealth, Medication adherence, Barriers to adherence
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Collaborators
- University of Memphis, National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 5 Dec 2017 (actual)
- Primary completion
- 28 Jun 2018 (actual)
- Study completion
- 28 Jun 2018 (actual)
- First posted
- 17 Nov 2017 (actual)
- 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.
National Institutes of Health (NIH)/National Heart, Lung, and Blood Institute (NHLBI) guidelines recommend that hydroxyurea be offered to symptomatic adults and all children with sickle cell disease (SCD) (HbSS and HbSβ0-thal genotypes) age ≥9 months. Research has shown that hydroxyurea reduces hospitalizations and mortality, supporting its effectiveness outside of clinical trials. Hydroxyurea is given as a once-daily oral dose that costs <$1 per day. Despite overwhelming evidence for positive effects, hydroxyurea is vastly underutilized. Given the relative ease of its administration, low cost, and safety profile, barriers to hydroxyurea utilization are primarily constrained to the health system and patient determinants. System-level barriers include insufficient access to SCD-specific care, limited access to medication (due to lack of health coverage), and providers' reluctance in prescribing it; while patient-level barriers include low acceptance (due to insufficient knowledge or misconceptions regarding risks and benefits), and forgetfulness leading to poor adherence.
Mobile health (mHealth) refers to the practice of medicine and public health supported by mobile devices. Short message service (SMS) text messaging (through cell phones) is a widespread means of communication, particularly among adolescents and young adults and is an emerging intervention modality to improve medication adherence. Its low cost, simplicity, and prevalence allow for relatively easy adoption and dissemination in medical practices.
This protocol seeks to examine barriers to hydroxyurea adherence among SCD patients between 15 and 45 years of age who are living in the Memphis region by conducting a needs assessment. In addition to examining these barriers, the needs assessment will provide data that will inform the development of an mHealth application (e.g. mobile phone application) for assisting patients in increasing their medication adherence. The developed mHealth intervention will then undergo a pilot trial to test its acceptability, satisfaction, and feasibility among 56 patients living with SCD.
- To conduct multi-level needs assessment of hydroxyurea utilization barriers and facilitators, in Memphis, Tennessee (Phase I).
- To test the feasibility and acceptance of a patient-informed smart phone application aimed at improving hydroxyurea adherence in the Memphis, Tennessee region, and to estimate the efficacy parameters needed to design a definitive large phase III trial (Phase II).
Detailed description
Data for the needs assessment and pilot trial will be collected from the patients via validated surveys, qualitative methods (e.g., interviews and focus groups), and medical record abstraction.
The study will utilize a Hybrid Effectiveness-Implementation design that will blend both a new intervention to impact the effectiveness of hydroxyurea, text messaging, and study its potential for adoption and dissemination in "real-world" practice settings. In Phase I (formative process evaluation), actionable barriers of hydroxyurea adherence in Memphis, Tennessee, will be identified. The investigators will begin by conducting a broad evaluation of hydroxyurea utilization, impediments and facilitators of hydroxyurea adherence and care management in general for patients with SCD. Next, this information will be merged with patients' preferences for the content of an mHealth intervention, and test it in a local cohort (Phase II). If successful and approved by the Sickle Cell Disease Implementation Consortium (SCDIC) executive committee, this intervention will advance to the next phase, namely, the dissemination and implementation in a larger geographical area (the other SCDIC participating sites in Phase III). We will utilize the RE-AIM framework to evaluate the intervention representativeness and impact during all phases.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Phase I | – | –It is estimated that 100 participants with SCD will be enrolled for the Phase I portion which will identify barriers to hydroxyurea utilization. |
| Phase II | – | –It is estimated that 72 participants with SCD will be enrolled for the Phase II portion of the study which will evaluate the degree of feasibility and acceptance of mHealth intervention on hydroxyurea adherence. |
Who may qualify
- Ages
- 15 to 44 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Participants will reside in the Memphis, Tennessee, area and meet the inclusion criteria.
Key inclusion criteria (3 listed)
- Diagnosis of sickle cell disease (any genotype)
- Between the ages of 15 and 44.9 years
- English-speaking
Key exclusion criteria (1 listed)
- Inability or unwillingness of research participant or legal guardian/representative to give written informed consent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Compare the number of patients with hydroxyurea barriers or facilitators in hydroxyurea maintenance users and hydroxyurea former users/non-users (Phase I)
Time frame: From on-study date up to 12 months later
Multi-level needs assessment of hydroxyurea utilization barriers and facilitators, in Memphis, Tennessee, will be conducted to identify potential factors that affect the barriers or facilitators of hydroxyurea acceptance and that affect the adherence/maintenance of hydroxyurea. Enrollment for Phase I is estimated to be 100. The numbers of patients with barriers or facilitators in hydroxyurea maintenance users and hydroxyurea former users/non-users will be provided and will be compared using Fisher's exact text. This will be done via questionnaires with individuals living with sickle cell disease.
Number of participants who are enrolled and randomized on the study (Phase II)
Time frame: From study start date through up to 24 months later
In terms of feasibility of enrollment and randomization, that is, we will assess if we can enroll and randomize 40% (versus an unacceptable rate of 30%) of the approached eligible individuals (56 patients out of 150 approached) in the 24-month accrual duration.
Number of randomized patients completing baseline and 24-week evaluations for both arms
Time frame: From on-study date up to 24 weeks later
In terms of the feasibility of conducting a randomized trial, that is, after randomization, per each arm, we will assess if we can have 75% (versus an unacceptable rate of 50%) of randomized individuals (19 patients out of 28 randomized per arm) will complete at least baseline and 24-week evaluations.
Compare the number of participants who report themHealth intervention is useful
Time frame: Weeks 12 and 24
Measure the patients' perceived usefulness of mHealth intervention as an aid to hydroxyurea adherence. The proportions of patients who reported the mHealth intervention is useful at weeks 12 and 24 will be provided and will be tested to see if it is significantly different from random at a significance level of 0.05.
The mean and standard deviation of change in satisfaction with mHealth intervention
Time frame: From on-study date up to 24 weeks
Measure satisfaction of the intervention by comparing the change in satisfaction measured by the Treatment Satisfaction Questionnaire for Medication (TSQM-9) from baseline to 24 weeks. The change in satisfaction from baseline to 24 weeks will be summarized using mean and standard deviation in the text messaging arm and will be tested using t-test or Wilcoxon signed rank test depending on the normality of the data tested by Shapiro Wilk test at a significance level of 0.05.
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 11 Dec 2019.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- ClinicalTrials Open at St. JudeLink provided by the sponsor.
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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.
Jane S. Hankins, MD, MS principal investigator
St. Jude Children's Research Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease, run by St. Jude Children's Research Hospital at 2 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 3 inclusion and 1 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 11 Dec 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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?
Compare the number of patients with hydroxyurea barriers or facilitators in hydroxyurea maintenance users and hydroxyurea former users/non-users (Phase I) (time frame: From on-study date up to 12 months later). The study lists 5 primary outcomes in all. 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 11 Dec 2019.