Sickle Cell DiseaseNCT03577691
Efficacy of a Decision Aid for Hydroxyurea in Sickle Cell Disease
A study testing Training for use of web based Decision Aid, No training for use of web based Decision Aid and Pretest surveys and posttest surveys in people with Sickle Cell Disease, run by Emory University at 3 sites in United States.
Trial summary
The trial is a prospective, randomized study to determine the efficacy of training for use of a web based Decision Aid for Hydroxyurea(HU) usage among subjects with Sickle Cell Disease(SCD). Subjects are randomized primarily either to using a web based decision aid with training versus without training. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Training for use of web based Decision Aid, No training for use of web based Decision Aid and Pretest surveys and posttest surveys
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 2 participants
- Where
- 3 sitesUnited States
- Study dates
- 3 Aug 2018to 3 Jul 2019
- 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 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Individuals with sickle cell disease ages 18 to 80 years, inclusive Or parents of children (ages 9 months to <18 years) whose child has not started Hydroxyurea
- Patients who have made a past decision to not obtain treatment with Hydroxyurea or who have not obtained treatment with Hydroxyurea in past 12 months. Or parents of children who have made a past decision to not obtain treatment with Hydroxyurea or who have not obtained treatment with Hydroxyurea in the past 12 months.
- Patients or parents of children who's doctor identifies the patient/child as someone who should be on Hydroxyurea
- All participants will be able to comprehend English
- Subjects will have access to the internet from iPad, smart phone, or personal computer
- For the cognitive interviews participants will not be eligible to consider hydroxyurea as a treatment option
Who cannot join
- Patient who has already made a decision to begin and has started Hydroxyurea. Or parent who has already made a decision for their child to begin and the child has started Hydroxyurea.
- Participated in prior Decision Aid study
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Grady Health System
Atlanta, Georgia, 30303
Hughes Spalding Children's Hospital
Atlanta, Georgia, 30303
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
About this study
Official title: Efficacy of a Decision Aid for Hydroxyurea(HU) in Sickle Cell Disease(SCD)
The trial is a prospective, randomized study to determine the efficacy of training for use of a web based Decision Aid for Hydroxyurea(HU) usage among subjects with Sickle Cell Disease(SCD). Subjects are randomized primarily either to using a web based decision aid with training versus without training. Subjects in each group will be further randomized to 1) subjects receiving pretest surveys; and 2) subjects that do not receive pretest surveys.
Read the detailed description
Hydroxyurea (HU) has been demonstrated to be efficacious in reducing complications such as vasocclusive pain crises and acute chest syndrome in children and adults with Sickle Cell Disease (SCD) and in improving survival in adults. It is also the only disease-modifying therapy and the only drug approved by the FDA for use in patients with SCD. However significant barriers remain in prescription and utilization of HU. Some of the barriers are under prescription and when prescribed, underutilization by patients. The majority of the reasons for underutilization by patients being fear of cancer or other side effects, concern about lack of efficacy, and unwillingness to take the medicine or come to clinic or pharmacy.
Decision aids are designed to help patients improve their knowledge of the options, achieve a more accurate perception of risk and benefits of treatment, participate actively in decision making, and make choices more in keeping with their values and preferences. Decision aids have been shown to be effective in providing greater knowledge; lower decisional conflict among the subjects. The web based decision aid is also likely to serve as a tool for clarification of patient values to themselves and to their healthcare providers. The web based decision aid, sickleoptions.org is a rather extensive and detailed website with information regarding sickle cell disease(SCD) complications and standard of care monitoring and management as well as the treatment options of hydroxyurea, Endari, chronic blood transfusion, and bone marrow transplant. It is necessary to assure user ability to navigate all of the elements of the site. The study wants to determine the efficacy of training for a web based Decision Aid for Hydroxyurea(HU) usage among subjects with Sickle Cell Disease(SCD) that are randomized to using a web based decision aid with training versus without training.
In the first phase of the study cognitive interviews will be conducted with 10-20 parents of patients with SCD or patients with SCD age > 18 years for development of training to navigate website. Interviews will be conducted in 5-subject iterative cycles to identify ease of usability and issues regarding navigation and usability to develop a training guide for website.
After consent, subjects will be asked to complete demographic questionnaires. Subjects will be randomized to either receive training or to not receive training to use the website. All subjects will be asked to spend about 30mts navigating the website after consent. Those who are randomized to receive training will be trained to use it during this time. Those who do not receive training will be asked to navigate through the site on their own. They may be randomized to complete 4 question surveys during this time. Not everyone will need to complete these surveys. This will take about 15 minutes. They will then be contacted each week for the next 3 weeks with a reminder to use the website. At week 4 they will complete 5 surveys over the phone. They may be randomized to complete a telephone interview. This interview will take about 30 to 45 minutes and will be audio recorded for transcription and analysis. Subjects will then be contacted every 3 months for 1 year to see if they made a decision about whether or not to start hydroxyurea for themselves (>18 yr-old subjects) or their child. These phone calls will take less than 5 minutes.
- Conditions
- Sickle Cell Disease
- Being tested
- Training for use of web based Decision Aid, No training for use of web based Decision Aid, Pretest surveys and posttest surveys, Only posttest surveys
- Keywords
- Blood Disorders, Hydroxyurea (HU), Decision Aid
- Medical subject headings
- Anemia, Sickle Cell, Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Training for use of web based Decision AidOther
Subjects will be guided during a 30 minutes training session to use the website
No training for use of web based Decision AidOther
Subjects will not be guided during a 30 minutes training session to use the website
Pretest surveys and posttest surveysOther
Subjects will be randomized to one of two groups initially: Those who do not receive training for the decision aid and those who are trained to navigate the decision aid. subjects in each group will be randomized to complete pre test and posttest surveys or only posttest surveys that will take 15 mts of time
Only posttest surveysOther
Subjects will be randomized to one of two groups initially: Those who do not receive training for the decision aid and those who are trained to navigate the decision aid. subjects in each group will be randomized to complete pretest and posttest surveys or only posttest surveys.
| Study group | Role | What they receive |
|---|---|---|
| Training for use of web based Decision Aid | Experimental | Subjects will be provided access to the decision aid website and will receive a log-in identification, user password and url at time of consent and will be guided during a 30 minutes training session to use the website. They will ben be asked to continue to peruse the website at home to learn more about hydroxyurea. Participants in each group will be further randomized to 1) pretest surveys and posttest surveys; and 2)only posttest surveys Receives: Other: Training for use of web based Decision Aid; Other: Pretest surveys and posttest surveys; Other: Only posttest surveys |
| No training for use of web based Decision Aid | Placebo comparator | Subjects will receive a log-in identification, user password and url at time of scheduled appointment for web access. They will not receive training but will be instructed to maneuver through the website and access the information pertaining to hydroxyurea and access the videos for the purposes of learning. Participants will be asked to peruse the website for 30 minutes at time of consent then to continue to access the website at home to learn about hydroxyurea treatment. Participants in each group will be further randomized to 1) pretest surveys and posttest surveys; and 2)only posttest surveys Receives: Other: No training for use of web based Decision Aid; Other: Pretest surveys and posttest surveys; Other: Only posttest surveys |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Knowledge Survey score to estimate the impact of use of the Decision Aid with training versus without training in improving knowledge regarding HydroxyUrea for Sickle Cell Disease
Subjects knowledge will be tested by using a knowledge Survey that was given at baseline and at 4 weeks follow up phone call. It is a 27 True, False or Unsure questionnaire that tests subjects knowledge of understanding risk for severe lung disease (pulmonary hypertension). This test is administered to both groups that are in turn randomized to to receive the pretest AND posttest or just the posttest surveys.
Measured: Baseline and 4 week follow up
Secondary outcomes (3)
Change in decisional conflict scale to estimate the impact of use of the Decision Aid with training versus without training aid on decisional conflict
The decisional conflict scale (DCS) measures personal perceptions of: uncertainty in choosing options; modifiable factors contributing to uncertainty such as feeling uninformed, unclear about personal values and unsupported in decision making; and effective decision making such as feeling the choice is informed, values-based, likely to be implemented and expressing satisfaction with the choice. There are 16 questions with score value from 0=strongly agree to 4=strongly disagree. Scores range from 0(no decisional conflict) to 100 (extremely high decisional conflict)
Measured: Baseline and 4 week follow up
Change in decision self efficacy scale to estimate the impact of use of the Decision Aid with training versus without training aid on decisional conflict
The decision self efficacy scale measures self confidence or belief in one's abilities in decision making, including shared decision making. There are 11 questions with score value from 0=not confident at all to 4=very confident. Scores range from 0(extremely low self efficacy) to 100 (extremely high self efficacy)
Measured: Baseline and 4 week follow up
Change in Preparation for Decision Making Scale to estimate the impact of use of the Decision Aid with training versus without training aid on decisional conflict
The 'Preparation for Decision Making' scale assesses a patient's perception of how useful a decision aid or other decision support intervention is in preparing the respondent to communicate with their practitioner at a consultation focused on making a health decision.There are 10 questions with score value from 0=not at all to 5=a great deal. High scores indicate higher perceived level of preparation for decision making
Measured: Baseline and 4 week follow up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jul 2018
- Study start3 Aug 2018
- Primary completion3 Jul 2019
- Study completion3 Jul 2019
Why it stopped: Poor enrollment
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
- Supportive care
- Allocation
- Randomised
- Intervention model
- Factorial
- Model description
- Prospective, randomized,controlled, unblinded study
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Lakshmanan Krishnamurti, Emory University
- Investigators
- Lakshmanan Krishnamurti, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03577691
- Sponsor's protocol ID
- IRB00101669
- Status last verified
- Jul 2019
- First submitted
- 28 Jun 2018
- Last updated
- 23 Jul 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 23 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of a Decision Aid for Hydroxyurea(HU) in Sickle Cell Disease(SCD)
- ClinicalTrials.gov ID
- NCT03577691
- Acronym / sponsor ID
- IRB00101669
- Conditions
- Sickle Cell Disease
- Keywords
- Blood Disorders, Hydroxyurea (HU), Decision Aid
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Poor enrollment
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Study start
- 3 Aug 2018 (actual)
- Primary completion
- 3 Jul 2019 (actual)
- Study completion
- 3 Jul 2019 (actual)
- First posted
- 5 Jul 2018 (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.
The trial is a prospective, randomized study to determine the efficacy of training for use of a web based Decision Aid for Hydroxyurea(HU) usage among subjects with Sickle Cell Disease(SCD). Subjects are randomized primarily either to using a web based decision aid with training versus without training. Subjects in each group will be further randomized to 1) subjects receiving pretest surveys; and 2) subjects that do not receive pretest surveys.
Detailed description
Hydroxyurea (HU) has been demonstrated to be efficacious in reducing complications such as vasocclusive pain crises and acute chest syndrome in children and adults with Sickle Cell Disease (SCD) and in improving survival in adults. It is also the only disease-modifying therapy and the only drug approved by the FDA for use in patients with SCD. However significant barriers remain in prescription and utilization of HU. Some of the barriers are under prescription and when prescribed, underutilization by patients. The majority of the reasons for underutilization by patients being fear of cancer or other side effects, concern about lack of efficacy, and unwillingness to take the medicine or come to clinic or pharmacy.
Decision aids are designed to help patients improve their knowledge of the options, achieve a more accurate perception of risk and benefits of treatment, participate actively in decision making, and make choices more in keeping with their values and preferences. Decision aids have been shown to be effective in providing greater knowledge; lower decisional conflict among the subjects. The web based decision aid is also likely to serve as a tool for clarification of patient values to themselves and to their healthcare providers. The web based decision aid, sickleoptions.org is a rather extensive and detailed website with information regarding sickle cell disease(SCD) complications and standard of care monitoring and management as well as the treatment options of hydroxyurea, Endari, chronic blood transfusion, and bone marrow transplant. It is necessary to assure user ability to navigate all of the elements of the site. The study wants to determine the efficacy of training for a web based Decision Aid for Hydroxyurea(HU) usage among subjects with Sickle Cell Disease(SCD) that are randomized to using a web based decision aid with training versus without training.
In the first phase of the study cognitive interviews will be conducted with 10-20 parents of patients with SCD or patients with SCD age > 18 years for development of training to navigate website. Interviews will be conducted in 5-subject iterative cycles to identify ease of usability and issues regarding navigation and usability to develop a training guide for website.
After consent, subjects will be asked to complete demographic questionnaires. Subjects will be randomized to either receive training or to not receive training to use the website. All subjects will be asked to spend about 30mts navigating the website after consent. Those who are randomized to receive training will be trained to use it during this time. Those who do not receive training will be asked to navigate through the site on their own. They may be randomized to complete 4 question surveys during this time. Not everyone will need to complete these surveys. This will take about 15 minutes. They will then be contacted each week for the next 3 weeks with a reminder to use the website. At week 4 they will complete 5 surveys over the phone. They may be randomized to complete a telephone interview. This interview will take about 30 to 45 minutes and will be audio recorded for transcription and analysis. Subjects will then be contacted every 3 months for 1 year to see if they made a decision about whether or not to start hydroxyurea for themselves (>18 yr-old subjects) or their child. These phone calls will take less than 5 minutes.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Prospective, randomized,controlled, unblinded study
| Arm | Type | Receives |
|---|---|---|
| Training for use of web based Decision Aid | Experimental | Other: Training for use of web based Decision Aid, Other: Pretest surveys and posttest surveys, Other: Only posttest surveysSubjects will be provided access to the decision aid website and will receive a log-in identification, user password and url at time of consent and will be guided during a 30 minutes training session to use the website. They will ben be asked to continue to peruse the website at home to learn more about hydroxyurea. Participants in each group will be further randomized to 1) pretest surveys and posttest surveys; and 2)only posttest surveys |
| No training for use of web based Decision Aid | Placebo comparator | Other: No training for use of web based Decision Aid, Other: Pretest surveys and posttest surveys, Other: Only posttest surveysSubjects will receive a log-in identification, user password and url at time of scheduled appointment for web access. They will not receive training but will be instructed to maneuver through the website and access the information pertaining to hydroxyurea and access the videos for the purposes of learning. Participants will be asked to peruse the website for 30 minutes at time of consent then to continue to access the website at home to learn about hydroxyurea treatment. Participants in each group will be further randomized to 1) pretest surveys and posttest surveys; and 2)only posttest surveys |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Individuals with sickle cell disease ages 18 to 80 years, inclusive Or parents of children (ages 9 months to <18 years) whose child has not started Hydroxyurea
- Patients who have made a past decision to not obtain treatment with Hydroxyurea or who have not obtained treatment with Hydroxyurea in past 12 months. Or parents of children who have made a past decision to not obtain treatment with Hydroxyurea or who have not obtained treatment with Hydroxyurea in the past 12 months.
- Patients or parents of children who's doctor identifies the patient/child as someone who should be on Hydroxyurea
- All participants will be able to comprehend English
- Subjects will have access to the internet from iPad, smart phone, or personal computer
Key exclusion criteria (2 listed)
- Patient who has already made a decision to begin and has started Hydroxyurea. Or parent who has already made a decision for their child to begin and the child has started Hydroxyurea.
- Participated in prior Decision Aid study
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 |
|---|---|---|
| Training for use of web based Decision AidSubjects will be guided during a 30 minutes training session to use the website | Other | Training for use of web based Decision Aid |
| No training for use of web based Decision AidSubjects will not be guided during a 30 minutes training session to use the website | Other | No training for use of web based Decision Aid |
| Pretest surveys and posttest surveysSubjects will be randomized to one of two groups initially: Those who do not receive training for the decision aid and those who are trained to navigate the decision aid. subjects in each group will be randomized to complete pre test and posttest surveys or only posttest surveys that will take 15 mts of time | Other | No training for use of web based Decision Aid, Training for use of web based Decision Aid |
| Only posttest surveysSubjects will be randomized to one of two groups initially: Those who do not receive training for the decision aid and those who are trained to navigate the decision aid. subjects in each group will be randomized to complete pretest and posttest surveys or only posttest surveys. | Other | No training for use of web based Decision Aid, Training for use of web based Decision Aid |
Primary outcome
Change in Knowledge Survey score to estimate the impact of use of the Decision Aid with training versus without training in improving knowledge regarding HydroxyUrea for Sickle Cell Disease
Time frame: Baseline and 4 week follow up
Subjects knowledge will be tested by using a knowledge Survey that was given at baseline and at 4 weeks follow up phone call. It is a 27 True, False or Unsure questionnaire that tests subjects knowledge of understanding risk for severe lung disease (pulmonary hypertension). This test is administered to both groups that are in turn randomized to to receive the pretest AND posttest or just the posttest surveys.
3 secondary outcomes
Change in decisional conflict scale to estimate the impact of use of the Decision Aid with training versus without training aid on decisional conflict
Time frame: Baseline and 4 week follow up
Change in decision self efficacy scale to estimate the impact of use of the Decision Aid with training versus without training aid on decisional conflict
Time frame: Baseline and 4 week follow up
Change in Preparation for Decision Making Scale to estimate the impact of use of the Decision Aid with training versus without training aid on decisional conflict
Time frame: Baseline and 4 week follow up
Results and safety
The study was stopped early (reason given: Poor enrollment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jul 2019.
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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 principal investigator
Emory University
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Training for use of web based Decision Aid, No training for use of web based Decision Aid and Pretest surveys and posttest surveys in people with Sickle Cell Disease, run by Emory University at 3 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 6 inclusion and 2 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: stopped early, as of the sponsor's last update on 23 Jul 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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. Some participants in this study may receive a placebo. 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 was stopped early (reason given: Poor enrollment). Results are not available in the source record.
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?
Change in Knowledge Survey score to estimate the impact of use of the Decision Aid with training versus without training in improving knowledge regarding HydroxyUrea for Sickle Cell Disease (time frame: Baseline and 4 week follow up). 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 23 Jul 2019.