Sickle Cell DiseaseENHANCENCT02578017
ElectroNic Hydroxyurea AdhereNCE: A Strategy to Improve Hydroxyurea Adherence in Patients With Sickle Cell Disease
A study testing Mobile DOT in people with Sickle Cell Disease, run by Nationwide Children's Hospital at 1 site in United States.
Trial summary
HU is an FDA approved medication for the treatment of SCD. Many studies have shown that HU can reduce SCD related symptoms, but only 50% of patients take it as often as they should. This limits how much HU can help reduce SCD symptoms. Read more about this study
- Who is it for?
- Ages Up to 19 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Mobile DOT
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 71 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2014to Jun 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 Up to 19 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of Sickle Cell Disease (SCD), any genotype
- Prescribed HU for at least the previous 6 months
- Plans to receive SCD-related care at Nationwide Children's Hospital for the study duration
- For participants ≥18 years: participant must have access to a smart-phone or computer capable of recording and submitting videos to Mobile DOT
- For participants <18 years: consenting adult must have access to a smart-phone or computer capable of recording and submitting videos to Mobile DOT AND agrees to enter into a mutual agreement to participate in the daily medication administration routine
- Patient and/or consenting adult must speak English
- Access to a working phone (smart phone or landline)
Who cannot join
- Current chronic transfusion therapy or apheresis
Trial locations and contacts
1 site in 1 country.
United States1 site
Nationwide Children's Hospital
Columbus, Ohio, 43205
About this study
Official title: ENHANCE Study (ElectroNic Hydroxyurea AdhereNCE): A Strategy to Improve Hydroxyurea Adherence in Patients With Sickle Cell Disease
HU is an FDA approved medication for the treatment of SCD. Many studies have shown that HU can reduce SCD related symptoms, but only 50% of patients take it as often as they should. This limits how much HU can help reduce SCD symptoms.
Researchers are interested to see if electronic directly observed therapy (Mobile DOT), a program that uses cell phone reminder messages, videos, feedback messages, and incentives will help patients with SCD take HU as prescribed.
Read the detailed description
This is a 12-month, single-arm, cross-over study for pediatric and adolescent patients with SCD who are prescribed HU at Nationwide Children's Hospital in order to compare HU adherence prior to the study, during Mobile Dot use and after using Mobile DOT.
Hydroxyurea (HU) is the only disease-modifying medication for patients with sickle cell disease (SCD). Multiple clinical trials show that HU can reduce SCD-related complications but only 50% of pediatric patients adhere to HU at the rates achieved in clinical trials. This poor adherence limits its clinical effectiveness and results in increased costs and in lower patient-reported quality of life.
An innovative, practical, inexpensive, and efficient strategy is needed to improve HU adherence in pediatric patients with SCD. Electronic directly observed therapy (Mobile DOT) is a pilot-tested, multi-dimensional tool that is a feasible and acceptable strategy and can achieve >90% HU adherence rates in a small cohort of pediatric patients with SCD. This study will provide further testing to confirm if Mobile DOT can improve adherence and improve clinical outcomes in patients with SCD.
Mobile DOT uses patients' smart phones and computers to provide electronic reminder alerts and to video-record patients' daily HU administrations. Patients are provided with text (SMS) messages, e-mails, and phone call communications to encourage adherence and they receive monetary incentives when they reach adherence goals.
Researchers will determine if video adherence correlates with self-report, biomarker, and refill adherence. Also, surveys will be completed by participants to determine if their self-management skills improve with Mobile DOT.
- Conditions
- Sickle Cell Disease
- Being tested
- Mobile DOT
- Keywords
- Sickle Cell Disease, Hydroxyurea, Adherence
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mobile DOTBehavioural
Mobile DOT is a multi-dimensional tool that uses automated reminders, adherence feedback messages, incentives, and video observation of medication administration.
| Study group | Role | What they receive |
|---|---|---|
| Mobile DOT | Experimental | All Participants will utilize Mobile DOT for 6 months. The Mobile DOT intervention includes: reminder alerts, participant videos, research staff feedback on adherence, and contingency management. Receives: Behavioral: Mobile DOT |
| Post-intervention observation | No intervention | All participants will not receive any additional adherenece intervention after completing the Mobile DOT arm. Participants will be observed for 6 months. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Achievement of ≥80% HU adherence
Compare the number of participants who achieve ≥80% HU adherence when patients receive Mobile DOT to the 6 months prior to study entry.
Measured: 12 months
Secondary outcomes (2)
Correlation of video observation adherence with other measures of adherence.
Determine if video observation adherence correlations with biomarker adherence (MCV, HbF, and urine assay), self-report, and refill estimated adherence.
Measured: 12 months
Changes in self-managment skills
To explore adolescent and young adults' self-managment skills using the transition readiness questionnaire before and after mobile DOT.
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 posted16 Oct 2015 (estimated)
- Study startJul 2014
- Primary completionJun 2019
- Study completionJun 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- 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
- Other
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 71 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nationwide Children's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Susan Creary, Nationwide Children's Hospital
- Investigators
- Susan Creary, MD, Principal investigator, Nationwide Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02578017
- Sponsor's protocol ID
- NCH14-00163
- Acronym
- ENHANCE
- Status last verified
- Jul 2019
- First submitted
- 23 Sep 2015
- Last updated
- 10 Jul 2019
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Creary S, Chisolm D, Stanek J, Neville K, Garg U, Hankins JS, O'Brien SH. Measuring hydroxyurea adherence by pharmacy and laboratory data compared with video observation in children with sickle cell disease. Pediatr Blood Cancer. 2020 Aug;67(8):e28250. doi: 10.1002/pbc.28250. Epub 2020 May 9.
PubMed 32386106Creary S, Chisolm D, Stanek J, Hankins J, O'Brien SH. A Multidimensional Electronic Hydroxyurea Adherence Intervention for Children With Sickle Cell Disease: Single-Arm Before-After Study. JMIR Mhealth Uhealth. 2019 Aug 8;7(8):e13452. doi: 10.2196/13452.
PubMed 31397291
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ENHANCE Study (ElectroNic Hydroxyurea AdhereNCE): A Strategy to Improve Hydroxyurea Adherence in Patients With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT02578017
- Acronym / sponsor ID
- ENHANCE / NCH14-00163
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle Cell Disease, Hydroxyurea, Adherence
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 71 participants (actual)
- Lead sponsor
- Nationwide Children's HospitalUniversity, hospital or other
- Study start
- Jul 2014
- Primary completion
- Jun 2019 (actual)
- Study completion
- Jun 2019 (actual)
- First posted
- 16 Oct 2015 (estimated)
- 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.
HU is an FDA approved medication for the treatment of SCD. Many studies have shown that HU can reduce SCD related symptoms, but only 50% of patients take it as often as they should. This limits how much HU can help reduce SCD symptoms.
Researchers are interested to see if electronic directly observed therapy (Mobile DOT), a program that uses cell phone reminder messages, videos, feedback messages, and incentives will help patients with SCD take HU as prescribed.
Detailed description
This is a 12-month, single-arm, cross-over study for pediatric and adolescent patients with SCD who are prescribed HU at Nationwide Children's Hospital in order to compare HU adherence prior to the study, during Mobile Dot use and after using Mobile DOT.
Hydroxyurea (HU) is the only disease-modifying medication for patients with sickle cell disease (SCD). Multiple clinical trials show that HU can reduce SCD-related complications but only 50% of pediatric patients adhere to HU at the rates achieved in clinical trials. This poor adherence limits its clinical effectiveness and results in increased costs and in lower patient-reported quality of life.
An innovative, practical, inexpensive, and efficient strategy is needed to improve HU adherence in pediatric patients with SCD. Electronic directly observed therapy (Mobile DOT) is a pilot-tested, multi-dimensional tool that is a feasible and acceptable strategy and can achieve >90% HU adherence rates in a small cohort of pediatric patients with SCD. This study will provide further testing to confirm if Mobile DOT can improve adherence and improve clinical outcomes in patients with SCD.
Mobile DOT uses patients' smart phones and computers to provide electronic reminder alerts and to video-record patients' daily HU administrations. Patients are provided with text (SMS) messages, e-mails, and phone call communications to encourage adherence and they receive monetary incentives when they reach adherence goals.
Researchers will determine if video adherence correlates with self-report, biomarker, and refill adherence. Also, surveys will be completed by participants to determine if their self-management skills improve with Mobile DOT.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Mobile DOT | Experimental | Behavioral: Mobile DOTAll Participants will utilize Mobile DOT for 6 months. The Mobile DOT intervention includes: reminder alerts, participant videos, research staff feedback on adherence, and contingency management. |
| Post-intervention observation | No intervention | –All participants will not receive any additional adherenece intervention after completing the Mobile DOT arm. Participants will be observed for 6 months. |
Who may qualify
- Ages
- Up to 19 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Diagnosis of Sickle Cell Disease (SCD), any genotype
- Prescribed HU for at least the previous 6 months
- Plans to receive SCD-related care at Nationwide Children's Hospital for the study duration
- For participants ≥18 years: participant must have access to a smart-phone or computer capable of recording and submitting videos to Mobile DOT
- For participants <18 years: consenting adult must have access to a smart-phone or computer capable of recording and submitting videos to Mobile DOT AND agrees to enter into a mutual agreement to participate in the daily medication administration routine
Key exclusion criteria (1 listed)
- Current chronic transfusion therapy or apheresis
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 |
|---|---|---|
| Mobile DOTMobile DOT is a multi-dimensional tool that uses automated reminders, adherence feedback messages, incentives, and video observation of medication administration. | Behavioural | Mobile DOT |
Primary outcome
Achievement of ≥80% HU adherence
Time frame: 12 months
Compare the number of participants who achieve ≥80% HU adherence when patients receive Mobile DOT to the 6 months prior to study entry.
2 secondary outcomes
Correlation of video observation adherence with other measures of adherence.
Time frame: 12 months
Changes in self-managment skills
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 10 Jul 2019.
- Derived from this study: PubMed 32386106Creary S, Chisolm D, Stanek J, Neville K, Garg U, Hankins JS, O'Brien SH. Measuring hydroxyurea adherence by pharmacy and laboratory data compared with video observation in children with sickle cell disease. Pediatr Blood Cancer. 2020 Aug;67(8):e28250. doi: 10.1002/pbc.28250. Epub 2020 May 9.
- Derived from this study: PubMed 31397291Creary S, Chisolm D, Stanek J, Hankins J, O'Brien SH. A Multidimensional Electronic Hydroxyurea Adherence Intervention for Children With Sickle Cell Disease: Single-Arm Before-After Study. JMIR Mhealth Uhealth. 2019 Aug 8;7(8):e13452. doi: 10.2196/13452.
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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.
Susan Creary, MD principal investigator
Nationwide Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Mobile DOT in people with Sickle Cell Disease, run by Nationwide Children's Hospital 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 7 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 10 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 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?
Achievement of ≥80% HU adherence (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02578017, which also lists 2 linked publications.
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 10 Jul 2019.