Sickle Cell DiseaseNCT04691323
The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit in SCD.
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
The primary objective of this study is to better understand factors contributing to variations in hydroxyurea (HU) adherence behavior in adolescents and young adults (AYA) with sickle cell disease (SCD). Read more about this study
- Who is it for?
- Ages 12 to 21 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
- 68 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Mar 2021to 29 May 2025
- 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 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 12-21 years old
- Any sickle cell disease genotype
- On steady state of hydroxyurea for 2 months
- Own or have access to a smartphone
- Parents of patients that meet the eligibility criteria and are enrolled in the study will be included
Who cannot join
- Patients with recent hospitalizations within the past 7 days
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: The Longitudinal Relationship of Hydroxyurea Adherence Behavior to Health-related Quality of Life, Barriers to Adherence and Habit Formation in Patients With Sickle Cell Disease.
The primary objective of this study is to better understand factors contributing to variations in hydroxyurea (HU) adherence behavior in adolescents and young adults (AYA) with sickle cell disease (SCD). To meet this objective, the researchers will conduct a prospective cohort study to determine the longitudinal relationship between HU adherence and health-related quality of life (HRQOL) overtime among AYA with SCD. The long-term goal of this research is to promote medication adherence behavior and improve health outcomes in AYA with SCD.
Read the detailed description
Sickle cell disease is the most common genetic disorder in the US, affecting about 100,000 Americans, and about 1 in 400 African American live births, incurring annual health care costs of $335 million. SCD can lead to serious complications including unpredictable, debilitating pain episodes, cardiopulmonary disease, stroke, and long-term end organ damage.These complications lead to significant declines in health-related quality of life (HRQOL) and other patient-reported outcomes (PROs), culminating in early mortality, particularly among AYA. Hydroxyurea, at present, is the main FDA approved medication for SCD that reduces morbidity and mortality, improves HRQoL and lowers healthcare utilization.However, adherence to HU remains suboptimal with only 35-50% of patients achieving high adherence (≥90%), particularly among AYA with SCD. Low HU adherence has been associated with worse health outcomes, poor HRQOL and increased healthcare utilization. Low HU adherence is multifactorial, especially in AYA with other competing priorities and vulnerability in developmental and psychological factors contributing to adherence behavior. The specific aim for this study is to determine the longitudinal relationship of HU adherence behavior to health-related quality of life, barriers to adherence and habit formation among AYA with SCD.
- Conditions
- Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Hemoglobin C
- Being tested
- HU-Go app
- Keywords
- sickle cell disease, hydroxyurea
- 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 the HU-Go app intervention arm for 12 months. Receives: Other: HU-Go app |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hydroxyurea adherence rate
Adherence rate is defined as number of given HU doses given, as captured by HU-Go app, divided by total number of doses expected during study period.
Measured: 12 months
Secondary outcomes (9)
Adherence to Hydroxyurea using PROMIS Medication Adherence Scale (PMAS)
Higher score indicating higher adherence to hydroxyurea
Measured: 12 months
Adherence to Hydroxyurea using Visual Analogue Scale
Numerical value on a scale 0-100% (higher score indicating higher adherence to hydroxyurea)
Measured: 12 months
Pain score using Patient reported outcomes measurement information system (PROMIS) measure
Patient reported outcomes measurement information system (PROMIS) measure for pain intensity, numerical value on a scale of 0-100 points, normal average for the general population is 50 with standard deviation of 10 points (higher T scores indicating worse pain).
Measured: 12 months
Fatigue score using Patient reported outcomes measurement information system (PROMIS) measure
Patient reported outcomes measurement information system (PROMIS) measure for fatigue, numerical value on a scale of 0-100 points, normal average for the general population is 50 with standard deviation of 10 points (higher T scores indicating worse fatigue).
Measured: 12 months
Physical function score using Patient reported outcomes measurement information system (PROMIS) measure
Patient reported outcomes measurement information system (PROMIS) measure for physical function, numerical value on a scale of 0-100 points, normal average for the general population is 50 with standard deviation of 10 points (lower T scores indicating worse physical function).
Measured: 12 months
Depression score using Patient reported outcomes measurement information system (PROMIS) measure
Patient reported outcomes measurement information system (PROMIS) measure for depression, numerical value on a scale of 0-100 points, normal average for the general population is 50 with standard deviation of 10 points (higher T scores indicating worse depression).
Measured: 12 months
Anxiety score using Patient reported outcomes measurement information system (PROMIS) measure
Patient reported outcomes measurement information system (PROMIS) measure for anxiety, numerical value on a scale of 0-100 points, normal average for the general population is 50 with standard deviation of 10 points (higher T scores indicating worse anxiety).
Measured: 12 months
Peer relationships score using Patient reported outcomes measurement information system (PROMIS) measure
Patient reported outcomes measurement information system (PROMIS) measure for peer relationships, numerical value on a scale of 0-100 points, normal average for the general population is 50 with standard deviation of 10 points (lower T scores indicating worse peer relationships).
Measured: 12 months
Codes and themes from qualitative data analysis
Semi-structured interviews focused on HU taking behavior, barriers to adherence and habit formation
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Dec 2020
- Study start2 Mar 2021
- Primary completion18 Jun 2024
- Study completion29 May 2025
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
- 68 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- 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
- NCT04691323
- Sponsor's protocol ID
- IRB 2020-3366
- Other IDs
- 1K23HL150232
- Status last verified
- Jul 2026
- First submitted
- 24 Dec 2020
- Last updated
- 31 Jul 2026
- 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 31 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Longitudinal Relationship of Hydroxyurea Adherence Behavior to Health-related Quality of Life, Barriers to Adherence and Habit Formation in Patients With Sickle Cell Disease.
- ClinicalTrials.gov ID
- NCT04691323
- Acronym / sponsor ID
- IRB 2020-3366
- Conditions
- Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Hemoglobin C
- Keywords
- sickle cell disease, hydroxyurea
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 68 participants (actual)
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of ChicagoUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 2 Mar 2021 (actual)
- Primary completion
- 18 Jun 2024 (actual)
- Study completion
- 29 May 2025 (actual)
- First posted
- 31 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 primary objective of this study is to better understand factors contributing to variations in hydroxyurea (HU) adherence behavior in adolescents and young adults (AYA) with sickle cell disease (SCD). To meet this objective, the researchers will conduct a prospective cohort study to determine the longitudinal relationship between HU adherence and health-related quality of life (HRQOL) overtime among AYA with SCD. The long-term goal of this research is to promote medication adherence behavior and improve health outcomes in AYA with SCD.
Detailed description
Sickle cell disease is the most common genetic disorder in the US, affecting about 100,000 Americans, and about 1 in 400 African American live births, incurring annual health care costs of $335 million. SCD can lead to serious complications including unpredictable, debilitating pain episodes, cardiopulmonary disease, stroke, and long-term end organ damage.These complications lead to significant declines in health-related quality of life (HRQOL) and other patient-reported outcomes (PROs), culminating in early mortality, particularly among AYA. Hydroxyurea, at present, is the main FDA approved medication for SCD that reduces morbidity and mortality, improves HRQoL and lowers healthcare utilization.However, adherence to HU remains suboptimal with only 35-50% of patients achieving high adherence (≥90%), particularly among AYA with SCD. Low HU adherence has been associated with worse health outcomes, poor HRQOL and increased healthcare utilization. Low HU adherence is multifactorial, especially in AYA with other competing priorities and vulnerability in developmental and psychological factors contributing to adherence behavior. The specific aim for this study is to determine the longitudinal relationship of HU adherence behavior to health-related quality of life, barriers to adherence and habit formation among AYA with SCD.
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 the HU-Go app intervention arm for 12 months. |
Who may qualify
- Ages
- 12 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Age 12-21 years old
- Any sickle cell disease genotype
- On steady state of hydroxyurea for 2 months
- Own or have access to a smartphone
- Parents of patients that meet the eligibility criteria and are enrolled in the study will be included
Key exclusion criteria (1 listed)
- Patients with recent hospitalizations within the past 7 days
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
Hydroxyurea adherence rate
Time frame: 12 months
Adherence rate is defined as number of given HU doses given, as captured by HU-Go app, divided by total number of doses expected during study period.
9 secondary outcomes
Adherence to Hydroxyurea using PROMIS Medication Adherence Scale (PMAS)
Time frame: 12 months
Adherence to Hydroxyurea using Visual Analogue Scale
Time frame: 12 months
Pain score using Patient reported outcomes measurement information system (PROMIS) measure
Time frame: 12 months
Fatigue score using Patient reported outcomes measurement information system (PROMIS) measure
Time frame: 12 months
Physical function score using Patient reported outcomes measurement information system (PROMIS) measure
Time frame: 12 months
Depression score using Patient reported outcomes measurement information system (PROMIS) measure
Time frame: 12 months
Anxiety score using Patient reported outcomes measurement information system (PROMIS) measure
Time frame: 12 months
Peer relationships score using Patient reported outcomes measurement information system (PROMIS) measure
Time frame: 12 months
Codes and themes from qualitative data analysis
Time frame: 12 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 31 Jul 2026.
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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 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 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 31 Jul 2026. 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?
Hydroxyurea adherence rate (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 31 Jul 2026.