AzTrials
AzTrials

Sickle Cell DiseaseNCT03462511

Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial

A study testing HABIT Intervention and Education materials in people with Sickle Cell Disease, run by Columbia University at 4 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

Many youth with chronic disease have difficulty taking medication every day and therefore do not receive full benefit from treatment. Sickle Cell Disease (SCD) is an inherited blood disease that affects African Americans and other underserved communities. Read more about this study

Who is it for?
Ages 10 to 18 years
Conditions the study is for
Sickle Cell Disease
What is being tested?
HABIT Intervention and Education materials
Could I receive a placebo?
No, everyone receives an active treatment
How many people
50 participants
Where
4 sitesUnited States
Study dates
15 Aug 2018to 31 Dec 2021
Phase
Interventional
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 10 to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • One of the two most common sickle cell disease variants (HbSS or HbS-B0 thalassemia)
  • Age 10 through18 years (inclusive)
  • Currently prescribed hydroxyurea (HU) ≥18 months (for identifying historical Personal best HbF)
  • Current HU dose is within 5% of dose at Personal Best HbF
  • Pre-enrollment HbF ≥15% below historical Personal best, based on mean of ≥2 HbF assessments over preceding 12 months
  • Youth able to speak/read English or Spanish
Show all 9Show fewer
  • Parent/guardian speaks/reads English or Spanish
  • Parent/ legal guardian willing to participate
  • Family expects to reside in community for ≥ 1.5 years

Who cannot join

  • Youth not prescribed HU
  • <2 HbF assessments over past 12 months
  • Transfusion within 3 months preceding enrollment
  • Final screen HbF (visit 0) of ≤15% decrease below Personal best HbF
  • Sexually active female 10 years or older and not using reliable contraception (due to HU teratogenic risk)
  • Pregnancy
Show all 9Show fewer
  • Cognitive impairment (>2 levels below expected grade)
  • Youth not residing with parent/legal guardian
  • Parent/legal guardian does not reside with youth

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • Feinstein Institute for Medical Research

    Manhasset, New York, 11030

  • Columbia University Irving Medical Center

    New York, New York, 10032

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

  • Albert Einstein College of Medicine

    The Bronx, New York, 10461

Other trials in United States

About this study

Many youth with chronic disease have difficulty taking medication every day and therefore do not receive full benefit from treatment. Sickle Cell Disease (SCD) is an inherited blood disease that affects African Americans and other underserved communities. Hydroxyurea (HU) is the sole FDA-approved drug therapy for SCD and is highly effective and improves quality of life. The proposed study, a 5-site four-year randomized control trial (RCT), builds upon the investigators' recent feasibility study of the same title. Overall goals are reducing barriers to HU use and improving adherence for youth 10-18 years through creation of a daily medication habit. The goal of the proposed multi-site study is to test the efficacy of the HABIT intervention at 6 months and sustainability of the effect at 12 months.

Read the detailed description

Barriers to medication adherence are common in youth with chronic illness and are a source of racial/ethnic disparities in underserved communities. An inherited blood disease, Sickle Cell Disease (SCD) is characterized by chronic and acute illness and reduced quality of life (QOL). It affects African Americans and other underserved communities. Hydroxyurea (HU) is the sole FDA-approved drug therapy for SCD and is highly effective and improves QOL. Poor adherence is common among youth and young adults with SCD.

The importance of poor medication adherence, use of community-based health workers (CHWs) to bridge the gap between health services and underserved parent-youth dyads affected by SCD, the strength of the science, the success of the investigators' multi-ethnic feasibility study, and the potential application of study findings to youth with other serious chronic illnesses speak to the importance of this trial.

Being tested
HABIT Intervention, Standard of Care, Education materials
Keywords
Hydroxyurea, Community health worker (CHW), Text messages, Medication adherence
Medical subject headings
Anemia, Sickle Cell, Medication Adherence

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • HABIT InterventionBehavioural

    Dyads randomized to the intervention group will receive the HABIT intervention, which includes CHW support and tailored text messages.

  • Standard of CareOther

    Standard of care used to treat patients with SCD

  • Education materialsOther

    Education materials provided to all patients enrolled in the trial

Study groups in NCT03462511
Study groupRoleWhat they receive
Control GroupActive comparatorDyads randomized to the control group will receive: Standard care and Education handouts.
Receives: Other: Standard of Care; Other: Education materials
Intervention GroupExperimentalIn addition to standard care and education handouts, dyads randomized to the intervention group will receive the HABIT intervention, which includes CHW support and tailored text messages.
Receives: Behavioral: HABIT Intervention; Other: Standard of Care; Other: Education materials

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Mean Change in Biomarker Fetal Hemoglobin (HbF)

    A serum biomarker obtained from youth used to measure adherence to hydroxyurea

    Measured: Baseline, 6 months, 12 months

  2. Mean Change in Proportion of Days Covered (PDC) by Hydroxyurea

    The days covered by hydroxyurea was assessed using youth prescription refill data and was used to measure hydroxyurea adherence. The baseline measure is the proportion of days covered by hydroxyurea in the year prior to study enrollment, using prescription refill data.

    Measured: Baseline, 6 months, 12 months

Secondary outcomes (3)

  1. Mean Change in Youth Score on Peds Quality of Life (Generic Quality of Life)

    Used to measure health-related quality of life. Scores range from 0-100 with a higher score indicating a better quality of life.

    Measured: Baseline, 4 months, 9 months and 12 months

  2. Mean Change in Youth Score on PedsQL Sickle Cell Disease Module (Disease Specific Quality of Life)

    Used to measure sickle cell disease specific health-related quality of life. Scores range from 0-100 with a higher score indicating a better quality of life.

    Measured: Baseline, 9 months and 12 months

  3. Mean Change in Parent Youth Concordance Regarding Self-management Responsibility

    Concordance between parent and youth scores Sickle Cell Family Responsibility scores for 11 items measuring self-management tasks. Scores range from 0-11 with a higher score indicating better concordance.

    Measured: Baseline, 6 months and 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Mar 2018
  2. Study start15 Aug 2018
  3. Primary completion31 Dec 2021
  4. Study completion31 Dec 2021

Resources

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
Health services research
Allocation
Randomised
Intervention model
Parallel groups
Masking
Single blind
Who is masked
Outcomes assessor
Enrolment
50 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03462511
Sponsor's protocol ID
AAAR2908
Other IDs
1R01NR017206-01
Status last verified
Jun 2024
First submitted
6 Mar 2018
Last updated
26 Jun 2024
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Bruzzese JM, Usseglio J, Iannacci-Manasia L, Diggs KA, Smaldone AM, Green NS. Mental and Emotional Health of Caregivers of Youth with Sickle Cell Disease: A Systematic Review. J Health Care Poor Underserved. 2023;34(3):1070-1104.

    PubMed 38015138

  • Askew MA, Smaldone AM, Gold MA, Smith-Whitley K, Strouse JJ, Jin Z, Green NS. Pediatric hematology providers' contraceptive practices for female adolescents and young adults with sickle cell disease: A national survey. Pediatr Blood Cancer. 2022 Oct;69(10):e29877. doi: 10.1002/pbc.29877. Epub 2022 Jul 20.

    PubMed 35856776

  • Smaldone A, Manwani D, Aygun B, Smith-Whitley K, Jia H, Bruzzese JM, Findley S, Massei J, Green NS. HABIT efficacy and sustainability trial, a multi-center randomized controlled trial to improve hydroxyurea adherence in youth with sickle cell disease: a study protocol. BMC Pediatr. 2019 Oct 15;19(1):354. doi: 10.1186/s12887-019-1746-6.

    PubMed 31615480

  • Green NS, Manwani D, Aygun B, Appiah-Kubi A, Smith-Whitley K, Castillo Y, Soriano L, Jia H, Smaldone AM. Hydroxyurea Adherence for Personal Best in Sickle Cell Treatment (HABIT) efficacy trial: Community health worker support may increase hydroxyurea adherence of youth with sickle cell disease. Pediatr Blood Cancer. 2024 Apr;71(4):e30878. doi: 10.1002/pbc.30878. Epub 2024 Feb 6.

    PubMed 38321562

  • Green NS, Manwani D, Smith-Whitley K, Aygun B, Appiah-Kubi A, Smaldone AM. Mental health assessment of youth with sickle cell disease and their primary caregivers during the COVID-19 pandemic. Pediatr Blood Cancer. 2022 Sep;69(9):e29797. doi: 10.1002/pbc.29797. Epub 2022 May 25.

    PubMed 35614571

  • Lalji R, Koh L, Francis A, Khalid R, Guha C, Johnson DW, Wong G. Patient navigator programmes for children and adolescents with chronic diseases. Cochrane Database Syst Rev. 2024 Oct 9;10(10):CD014688. doi: 10.1002/14651858.CD014688.pub2.

    PubMed 39382077

Questions about this trial

What is this clinical trial studying?

A study testing HABIT Intervention and Education materials in people with Sickle Cell Disease, run by Columbia University at 4 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 9 inclusion and 9 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 26 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 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. 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?

Yes. Results are posted in the registry (first posted 26 Jun 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Mean Change in Biomarker Fetal Hemoglobin (HbF) (time frame: Baseline, 6 months, 12 months). The study lists 2 primary outcomes in all. 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 NCT03462511, which also lists 6 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 26 Jun 2024.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in