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

NCT ID: NCT03462511

Last Updated: 2024-06-26

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-08-15
• Study Completion Date: 2021-12-31

Brief 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. 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.

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.

Conditions

Sickle Cell Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: HEALTH_SERVICES_RESEARCH
• Blinding Strategy: SINGLE

Study Groups

Control Group

Dyads randomized to the control group will receive:

Standard care and Education handouts.

Group Type: ACTIVE_COMPARATOR

Standard of Care

Intervention Type: OTHER

Standard of care used to treat patients with SCD

Education materials

Intervention Type: OTHER

Education materials provided to all patients enrolled in the trial

Intervention Group

In 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.

Group Type: EXPERIMENTAL

HABIT Intervention

Intervention Type: BEHAVIORAL

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

Standard of Care

Intervention Type: OTHER

Standard of care used to treat patients with SCD

Education materials

Intervention Type: OTHER

Education materials provided to all patients enrolled in the trial

Interventions

HABIT Intervention

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

Intervention Type: BEHAVIORAL

Standard of Care

Standard of care used to treat patients with SCD

Intervention Type: OTHER

Education materials

Education materials provided to all patients enrolled in the trial

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• 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

• 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 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

• Parent/legal guardian does not reside with youth

• Minimum Eligible Age: 10 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Institute of Nursing Research (NINR)

NIH

Sponsor Role: collaborator

Columbia University

OTHER

Sponsor Role: lead

Responsible Party

Arlene Smaldone

Professor of Nursing

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Arlene Smaldone, PhD, CPNP-PC

Role: PRINCIPAL_INVESTIGATOR

Columbia University School of Nursing

Nancy S Green, MD

Role: PRINCIPAL_INVESTIGATOR

Columbia University

Locations

Feinstein Institute for Medical Research

Manhasset, New York, United States

Columbia University Irving Medical Center

New York, New York, United States

Albert Einstein College of Medicine

The Bronx, New York, United States

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Countries

United States

References

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.

Reference Type: BACKGROUND

PMID: 38015138 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 35856776 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 31615480 (View on PubMed)

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.

Reference Type: RESULT

PMID: 38321562 (View on PubMed)

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.

Reference Type: RESULT

PMID: 35614571 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 39382077 (View on PubMed)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

1R01NR017206-01

Identifier Type: NIH

Identifier Source: secondary_id

View Link

AAAR2908

Identifier Type: -

Identifier Source: org_study_id