Sickle Cell DiseaseNCT04675645
Adherence to HU and HRQOL in Patients With Sickle Cell Disease: An Intervention Study Using HU-Go App
A study testing HU-Go app in people with Sickle Cell Disease and Sickle B+ Thalassemia, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
Trial summary
This project addresses three important research questions. First, adolescents and young adults (AYA) with sickle cell disease (SCD) and their parents/caregivers will be engaged to inform the (1) domains of health-related quality of life (HRQOL) most important to them, (2) frequency at which they are willing to… Read more about this study
- Who is it for?
- Ages 12 to 25 years
- Conditions the study is for
- Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia
- What is being tested?
- HU-Go app
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 1 siteUnited States
- Study dates
- 15 May 2018to 11 Jul 2020
- 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
- At least 12 years old at the time of study enrollment
- Diagnosis of sickle cell disease (hemoglobin SS or SC or S/B 0 thalassemia) confirmed by hemoglobin electrophoresis
- On hydroxyurea
- Own or have access to a smartphone
- Be able to speak and read English
Who cannot join
- Chronic monthly transfusion support
- Any hemoglobinopathy other than sickle cell disease
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: Adherence to Hydroxyurea and Health-related Quality of Life in Patients With Sickle Cell Disease: An Intervention Study Using a Smartphone App (HU-Go)
This project addresses three important research questions. First, adolescents and young adults (AYA) with sickle cell disease (SCD) and their parents/caregivers will be engaged to inform the (1) domains of health-related quality of life (HRQOL) most important to them, (2) frequency at which they are willing to complete them, and (3) other procedures related to the use, uptake and effect of the HU-Go app as a tool to improve hydroxyurea (HU) adherence. Second, this study seeks to utilize novel modern mobile technology using a multi-functional personalized platform to improve adherence to HU and measure HRQOL in youth with SCD, using NIH-endorsed PROMIS® measures, based on a conceptual model with predefined behavioral targets and mediators. Third, we plan to assess HRQOL changes and identify modifiable behavioral strategies that could serve as surrogates or predictors for HU adherence. This real-time feedback might empower self-directed changes in behavior that could improve adherence to HU.
Read the detailed description
Aim 1: Identify the needs, desires, concerns, and expectations of AYA patients with SCD and their parents/caregivers that will guide implementation of a mHealth tool, HU-Go, designed to improve adherence to HU. We will conduct semi-structured interviews with patients and their parents/caregivers. Transcripts will be independently coded and thematic analysis will be conducted.
Hypothesis 1: AYA patients with SCD and their parents/caregivers will identify specific features, content, and usability requirements of HU-Go.
Aim 2: Assess the longitudinal relationship of HU adherence to HRQoL domains, including fatigue and depression. HU adherence will be measured using electronic pill bottles, self-report measures, laboratory markers, and medication possession ratio (MPR). HRQOL will be assessed using PROMIS® measures.
Hypothesis 2: Low HU adherence is associated with impairment of HRQoL domains.
Aim 3: Conduct a single-arm, pilot study to determine the effect size associated with improvement in HU adherence after using HU-Go for 12 weeks. Adherence will be measured using self-report measures, laboratory markers and MPR.
Hypothesis 3: HU-Go will improve HU adherence by at least 20%.
- Conditions
- Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Hemoglobin C
- Being tested
- HU-Go 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.
HU-Go appOther
A novel multifunctional mobile app (HU-Go) to improve adherence to hydroxyurea in patients with sickle cell disease
| Study group | Role | What they receive |
|---|---|---|
| HU-Go app intervention arm | Other | Participants will use HU-Go app intervention arm for a total of 12 weeks. Receives: Other: HU-Go app |
What is being measured
The primary outcome is the main question the study is designed to answer.
Achieving feasibility based on the number of participants completed all study procedures within 6 months of study enrollment
Feasibility is defined as having 80% or more of study participants complete the study within 6 months of enrollment
Measured: 3 months of study enrollment
Secondary outcomes (4)
Adherence to Hydroxyurea using Modified Morisky Adherence Scale 8-items
Numerical value on a scale 0-8 (higher score indicating higher adherence to hydroxyurea)
Measured: 3 months of study enrollment
Adherence to Hydroxyurea using Visual Analogue Scale
Numerical value on a scale 0-100% (higher score indicating higher adherence to hydroxyurea)
Measured: 3 months of study enrollment
Patient satisfaction with the smartphone app intervention (HU-Go)
Customized patient satisfaction questionnaire, numerical value on a scale 0-10 (higher score indicating higher satisfaction with the app)
Measured: 3 months of study enrollment
HRQOL outcomes
Patient reported outcomes measurement information system (PROMIS) measures, numerical value on a scale of 0-100 points, normal average for the general population is 50 with standard deviation of 10 points. Each HRQOL outcome domain score will be reported separately (higher T scores indicating worse pain, fatigue, depression and anxiety, and lower scores indicating worse physical functioning and peer relationships).
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2020
- Study start15 May 2018
- Primary completion11 Jul 2020
- Study completion11 Jul 2020
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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 33 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
- NCT04675645
- Sponsor's protocol ID
- IRB 2015-761
- Status last verified
- Jul 2021
- First submitted
- 17 Aug 2016
- Last updated
- 29 Jul 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: All patients data will be deidentified.
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Adherence to Hydroxyurea and Health-related Quality of Life in Patients With Sickle Cell Disease: An Intervention Study Using a Smartphone App (HU-Go)
- ClinicalTrials.gov ID
- NCT04675645
- Acronym / sponsor ID
- IRB 2015-761
- Conditions
- Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Hemoglobin C
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of ChicagoUniversity, hospital or other
- Study start
- 15 May 2018 (actual)
- Primary completion
- 11 Jul 2020 (actual)
- Study completion
- 11 Jul 2020 (actual)
- First posted
- 19 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.
This project addresses three important research questions. First, adolescents and young adults (AYA) with sickle cell disease (SCD) and their parents/caregivers will be engaged to inform the (1) domains of health-related quality of life (HRQOL) most important to them, (2) frequency at which they are willing to complete them, and (3) other procedures related to the use, uptake and effect of the HU-Go app as a tool to improve hydroxyurea (HU) adherence. Second, this study seeks to utilize novel modern mobile technology using a multi-functional personalized platform to improve adherence to HU and measure HRQOL in youth with SCD, using NIH-endorsed PROMIS® measures, based on a conceptual model with predefined behavioral targets and mediators. Third, we plan to assess HRQOL changes and identify modifiable behavioral strategies that could serve as surrogates or predictors for HU adherence. This real-time feedback might empower self-directed changes in behavior that could improve adherence to HU.
Detailed description
Aim 1: Identify the needs, desires, concerns, and expectations of AYA patients with SCD and their parents/caregivers that will guide implementation of a mHealth tool, HU-Go, designed to improve adherence to HU. We will conduct semi-structured interviews with patients and their parents/caregivers. Transcripts will be independently coded and thematic analysis will be conducted.
Hypothesis 1: AYA patients with SCD and their parents/caregivers will identify specific features, content, and usability requirements of HU-Go.
Aim 2: Assess the longitudinal relationship of HU adherence to HRQoL domains, including fatigue and depression. HU adherence will be measured using electronic pill bottles, self-report measures, laboratory markers, and medication possession ratio (MPR). HRQOL will be assessed using PROMIS® measures.
Hypothesis 2: Low HU adherence is associated with impairment of HRQoL domains.
Aim 3: Conduct a single-arm, pilot study to determine the effect size associated with improvement in HU adherence after using HU-Go for 12 weeks. Adherence will be measured using self-report measures, laboratory markers and MPR.
Hypothesis 3: HU-Go will improve HU adherence by at least 20%.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HU-Go app intervention arm | Other | Other: HU-Go appParticipants will use HU-Go app intervention arm for a total of 12 weeks. |
Who may qualify
- Ages
- 12 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- At least 12 years old at the time of study enrollment
- Diagnosis of sickle cell disease (hemoglobin SS or SC or S/B 0 thalassemia) confirmed by hemoglobin electrophoresis
- On hydroxyurea
- Own or have access to a smartphone
- Be able to speak and read English
Key exclusion criteria (2 listed)
- Chronic monthly transfusion support
- Any hemoglobinopathy other than sickle cell disease
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 |
|---|---|---|
| HU-Go appA novel multifunctional mobile app (HU-Go) to improve adherence to hydroxyurea in patients with sickle cell disease | Other | HU-Go app intervention arm |
Primary outcome
Achieving feasibility based on the number of participants completed all study procedures within 6 months of study enrollment
Time frame: 3 months of study enrollment
Feasibility is defined as having 80% or more of study participants complete the study within 6 months of enrollment
4 secondary outcomes
Adherence to Hydroxyurea using Modified Morisky Adherence Scale 8-items
Time frame: 3 months of study enrollment
Adherence to Hydroxyurea using Visual Analogue Scale
Time frame: 3 months of study enrollment
Patient satisfaction with the smartphone app intervention (HU-Go)
Time frame: 3 months of study enrollment
HRQOL outcomes
Time frame: 3 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 29 Jul 2021.
- Individual participant dataSharing planned: No. All patients data will be deidentified.
Related trials for Sickle Cell Disease
Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
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 HU-Go app in people with Sickle Cell Disease and Sickle B+ Thalassemia, 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 5 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: finished, as of the sponsor's last update on 29 Jul 2021. 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?
Achieving feasibility based on the number of participants completed all study procedures within 6 months of study enrollment (time frame: 3 months of study enrollment). 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 29 Jul 2021; AzTrials last refreshed its copy on 7 Nov 2026.