Sickle Cell DiseaseNCT04678037
Home-based Assessment of PRO Measures in SCD Using A Smartphone App Platform: A Feasibility Study
A study testing PROs assessment using sickle cell disease mobile app (SCD-app) in people with Sickle Cell Disease and Sickle Cell Hemoglobin C, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
Trial summary
The overarching goal of this proposal is to identify modifiable behavioral strategies based on patient-reported outcomes (PROs) and health-related quality of life (HRQOL) that will improve hydroxyurea (HU) adherence among adolescents and young adults with sickle cell disease (SCD). Read more about this study
- Who is it for?
- Ages 12 to 25 years
- Conditions the study is for
- Sickle Cell Disease, Sickle Cell Hemoglobin C, Sickle Beta Zero Thalassemia
- What is being tested?
- PROs assessment using sickle cell disease mobile app (SCD-app)
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Apr 2017to 21 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 12 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 12 years or older at the time of study enrollment;
- SCD diagnosis (all genotypes) confirmed by hemoglobin electrophoresis;
- Own or have access to a smartphone or a tablet; and
- Speak and read English.
Who cannot join
- Patients or caregivers with cognitive impairment;
- Patients or caregivers physical impairment; and
- Patients or caregivers who will not be able to complete study assessments.
Trial locations and contacts
1 site in 1 country.
United States1 site
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
About this study
Official title: Home-based Assessment of Patient Reported Outcome (PROs) Measures in Sickle Cell Disease (SCD) Using A Smartphone App Platform: A Feasibility Study
The overarching goal of this proposal is to identify modifiable behavioral strategies based on patient-reported outcomes (PROs) and health-related quality of life (HRQOL) that will improve hydroxyurea (HU) adherence among adolescents and young adults with sickle cell disease (SCD). In this proposed study, we intend to test the functionality of a PROs-toolbox feature, which will be integrated into our existing smartphone application platform (SCD-app), over a 24-week period in a cohort of SCD patients and their caregivers.
Read the detailed description
Adherence to hydroxyurea is a multi-factorial dynamic process, and, to date, predictors for changes in adherence levels over time remain unclear. Changes in HRQOL data could serve as surrogate markers for changes in hydroxyurea adherence over time, and could suggest when patients might or might not need or benefit from an intervention based on worse or better HRQOL scores, respectively.
The main hypothesis of this study is that the assessment of patients' HRQOL at home using a SCD smartphone application platform (app) is feasible and acceptable, and that less frequent assessments of HRQOL at home will have an overall higher completion rate when compared to more frequent ones. We plan to assess HRQOL using the NIH-developed patient reported outcomes measurement information system (PROMIS®) with computerized adaptive testing (CAT) approach. PROMIS®-CAT approach is a reliable and valid PROs assessment platform that has been used in limited studies in SCD.
The study will include the following specific aims:
- To evaluate the feasibility and acceptability of the assessment of patients HRQOL at home using smartphones with PROMIS®-CAT measures integrated into a SCD-app.
- To examine the effect of the frequency of required HRQOL assessments on participants' completion rate over 24-week period with HRQOL evaluated every 2 weeks (Group 1) versus every 4 weeks (Group 2).
- To explore participants' experience and preferences with the process and the frequency of HRQOL assessment at home using their smartphones with PROMIS®-CAT measures integrated into a SCD-app.
- Conditions
- Sickle Cell Disease, Sickle Cell Hemoglobin C, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia
- Being tested
- PROs assessment using sickle cell disease mobile app (SCD-app)
- Medical subject headings
- Anemia, Sickle Cell, Hemoglobin SC Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PROs assessment using sickle cell disease mobile app (SCD-app)Behavioural
A smartphone app platform designed for patients with sickle cell disease. The SCD-app is programmed to send notification to patients to complete PROs assessment. The SCD-app is able to collect PROs data as patients complete the assessment using the app platform.
| Study group | Role | What they receive |
|---|---|---|
| Group A (PROs assessment every 2 weeks) | Active comparator | Not described Receives: Behavioral: PROs assessment using sickle cell disease mobile app (SCD-app) |
| Group A (PROs assessment every 4 weeks) | Active comparator | Not described Receives: Behavioral: PROs assessment using sickle cell disease mobile app (SCD-app) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Feasibility of completing PRO assessments at home using SCD-app (HU-Go)
Having an overall completion rate of ≥ 50% for all study participants (Group A and B). This will be reported as a dichotomous outcome, either yes or no. The percentage (%) completion rate will be calculated as the number of PROs assessments completed divided by the number of PROs assessments expected over the study period.
Measured: 6 months
Secondary outcomes (1)
Completion rate of PRO assessments at home using SCD-app (HU-Go)
Completion rate of different PRO assessments at home using PROMIS®-CAT measures among patients with SCD and/or their parents. PRO assessments are planned to be every 2 weeks in Group 1 and every 4 weeks in Group 2. This outcome will be calculated and reported as percent (%) completion rate.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2020
- Study start4 Apr 2017
- Primary completion21 Jul 2019
- Study completion21 Jul 2019
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.
- 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
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sherif Badawy, MD, Ann & Robert H Lurie Children's Hospital of Chicago
- Investigators
- Sherif M Badawy, MD, MS, Principal investigator, Ann & Robert H Lurie Children's Hospital of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04678037
- Sponsor's protocol ID
- IRB 2017-757
- Status last verified
- Dec 2020
- First submitted
- 3 Dec 2020
- Last updated
- 21 Dec 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: All study data are de-identified and will be analysed as aggregate.
Study at a glance
From the registry record, last updated by the sponsor on 21 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Home-based Assessment of Patient Reported Outcome (PROs) Measures in Sickle Cell Disease (SCD) Using A Smartphone App Platform: A Feasibility Study
- ClinicalTrials.gov ID
- NCT04678037
- Acronym / sponsor ID
- IRB 2017-757
- Conditions
- Sickle Cell Disease, Sickle Cell Hemoglobin C, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of ChicagoUniversity, hospital or other
- Study start
- 4 Apr 2017 (actual)
- Primary completion
- 21 Jul 2019 (actual)
- Study completion
- 21 Jul 2019 (actual)
- First posted
- 21 Dec 2020 (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 overarching goal of this proposal is to identify modifiable behavioral strategies based on patient-reported outcomes (PROs) and health-related quality of life (HRQOL) that will improve hydroxyurea (HU) adherence among adolescents and young adults with sickle cell disease (SCD). In this proposed study, we intend to test the functionality of a PROs-toolbox feature, which will be integrated into our existing smartphone application platform (SCD-app), over a 24-week period in a cohort of SCD patients and their caregivers.
Detailed description
Adherence to hydroxyurea is a multi-factorial dynamic process, and, to date, predictors for changes in adherence levels over time remain unclear. Changes in HRQOL data could serve as surrogate markers for changes in hydroxyurea adherence over time, and could suggest when patients might or might not need or benefit from an intervention based on worse or better HRQOL scores, respectively.
The main hypothesis of this study is that the assessment of patients' HRQOL at home using a SCD smartphone application platform (app) is feasible and acceptable, and that less frequent assessments of HRQOL at home will have an overall higher completion rate when compared to more frequent ones. We plan to assess HRQOL using the NIH-developed patient reported outcomes measurement information system (PROMIS®) with computerized adaptive testing (CAT) approach. PROMIS®-CAT approach is a reliable and valid PROs assessment platform that has been used in limited studies in SCD.
The study will include the following specific aims:
- To evaluate the feasibility and acceptability of the assessment of patients HRQOL at home using smartphones with PROMIS®-CAT measures integrated into a SCD-app.
- To examine the effect of the frequency of required HRQOL assessments on participants' completion rate over 24-week period with HRQOL evaluated every 2 weeks (Group 1) versus every 4 weeks (Group 2).
- To explore participants' experience and preferences with the process and the frequency of HRQOL assessment at home using their smartphones with PROMIS®-CAT measures integrated into a SCD-app.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group A (PROs assessment every 2 weeks) | Active comparator | Behavioral: PROs assessment using sickle cell disease mobile app (SCD-app) |
| Group A (PROs assessment every 4 weeks) | Active comparator | Behavioral: PROs assessment using sickle cell disease mobile app (SCD-app) |
Who may qualify
- Ages
- 12 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Age 12 years or older at the time of study enrollment;
- SCD diagnosis (all genotypes) confirmed by hemoglobin electrophoresis;
- Own or have access to a smartphone or a tablet; and
- Speak and read English.
Key exclusion criteria (3 listed)
- Patients or caregivers with cognitive impairment;
- Patients or caregivers physical impairment; and
- Patients or caregivers who will not be able to complete study assessments.
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 |
|---|---|---|
| PROs assessment using sickle cell disease mobile app (SCD-app)A smartphone app platform designed for patients with sickle cell disease. The SCD-app is programmed to send notification to patients to complete PROs assessment. The SCD-app is able to collect PROs data as patients complete the assessment using the app platform. | Behavioural | Group A (PROs assessment every 2 weeks), Group A (PROs assessment every 4 weeks) |
Primary outcome
Feasibility of completing PRO assessments at home using SCD-app (HU-Go)
Time frame: 6 months
Having an overall completion rate of ≥ 50% for all study participants (Group A and B). This will be reported as a dichotomous outcome, either yes or no. The percentage (%) completion rate will be calculated as the number of PROs assessments completed divided by the number of PROs assessments expected over the study period.
1 secondary outcome
Completion rate of PRO assessments at home using SCD-app (HU-Go)
Time frame: 6 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 21 Dec 2020.
- Individual participant dataSharing planned: No. All study data are de-identified and will be analysed as aggregate.
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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.
Sherif M Badawy, MD, MS principal investigator
Ann & Robert H Lurie Children's Hospital of Chicago
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing PROs assessment using sickle cell disease mobile app (SCD-app) in people with Sickle Cell Disease and Sickle Cell Hemoglobin C, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site 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 4 inclusion and 3 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 21 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Feasibility of completing PRO assessments at home using SCD-app (HU-Go) (time frame: 6 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 21 Dec 2020.