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

June 5, 2024 updated by: Arlene Smaldone, Columbia University
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

50

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New York
      • Bronx, New York, United States, 10461
        • Albert Einstein College of Medicine
      • Manhasset, New York, United States, 11030
        • Feinstein Institute for Medical Research
      • New York, New York, United States, 10032
        • Columbia University Irving Medical Center
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • The Children's Hospital of Philadelphia

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

10 years to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Health Services Research
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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.
Other: Standard of Care
Standard of care used to treat patients with SCD
Other: Education materials
Education materials provided to all patients enrolled in the trial
Behavioral: HABIT Intervention
Dyads randomized to the intervention group will receive the HABIT intervention, which includes CHW support and tailored text messages.
Active Comparator: Control Group

Dyads randomized to the control group will receive:

Standard care and Education handouts.
Other: Standard of Care
Standard of care used to treat patients with SCD
Other: Education materials
Education materials provided to all patients enrolled in the trial

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Baseline, 6 months, 12 months
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.
Baseline, 6 months, 12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Baseline, 4 months, 9 months and 12 months
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.
Baseline, 9 months and 12 months
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.
Baseline, 6 months and 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Columbia University

Collaborators

National Institute of Nursing Research (NINR)

Investigators

  • Principal Investigator: Arlene Smaldone, PhD, CPNP-PC, Columbia University School of Nursing
  • Principal Investigator: Nancy S Green, MD, Columbia University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 15, 2018

Primary Completion (Actual)

December 31, 2021

Study Completion (Actual)

December 31, 2021

Study Registration Dates

First Submitted

March 6, 2018

First Submitted That Met QC Criteria

March 6, 2018

First Posted (Actual)

March 12, 2018

Study Record Updates

Last Update Posted (Actual)

June 26, 2024

Last Update Submitted That Met QC Criteria

June 5, 2024

Last Verified

June 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AAAR2908
  • 1R01NR017206-01 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Sickle Cell Disease

Clinical Trials on Standard of Care

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Iran

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe