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NCT Number: NCT03462511

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

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.

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Behavior Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Health Behavior Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Medication Adherence Patient Acceptance of Health Care Patient Compliance Treatment Adherence and Compliance

Age range

10 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Feinstein Institute for Medical Research, Manhasset, New York, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

- Youth:

  • 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

Inclusion criteria

- Parent:

  • Parent/guardian speaks/reads English or Spanish
  • Parent/ legal guardian willing to participate
  • Family expects to reside in community for ≥ 1.5 years

Exclusion criteria

- Youth:

  • 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
  • Cognitive impairment (>2 levels below expected grade)
  • Youth not residing with parent/legal guardian

Exclusion criteria

- Parent:

  • Parent/legal guardian does not reside with youth

Treatment and study plan

HABIT Intervention

Behavioral

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

Standard of care

Other

Standard of care used to treat patients with SCD

Education Materials

Other

Education materials provided to all patients enrolled in the trial

Primary outcomes

  1. Mean Change in Biomarker Fetal Hemoglobin (HbF)

    Time frame: Baseline, 6 months, 12 months

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

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

    Time frame: Baseline, 6 months, 12 months

    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.

Secondary outcomes

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

    Time frame: Baseline, 4 months, 9 months and 12 months

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

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

    Time frame: Baseline, 9 months and 12 months

    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.

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

    Time frame: Baseline, 6 months and 12 months

    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.

Sponsors and collaborators

Lead sponsor

Columbia University

Other

Collaborators

  • National Institute of Nursing Research (NINR)

Registry information

Important dates

Study start
2018
Primary completion
2021
Study completion
2021
First posted
Mar 12, 2018
Registry last updated
Jun 26, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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