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

Sponsor
Columbia University (Other)
Overall Status
Completed
CT.gov ID
NCT03462511
Collaborator
National Institute of Nursing Research (NINR) (NIH)
100
Enrollment
4
Locations
2
Arms
39.7
Actual Duration (Months)
25
Patients Per Site
0.6
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Behavioral: HABIT Intervention
 N/A

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 Design

Study Type:
Interventional
Actual Enrollment :
100 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Single (Outcomes Assessor)
Primary Purpose:
Health Services Research
Official Title:
Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial
Actual Study Start Date :
Sep 10, 2018
Actual Primary Completion Date :
Dec 31, 2021
Actual Study Completion Date :
Dec 31, 2021

Arms and Interventions

Arm Intervention/Treatment
No Intervention: Control Group

Dyads randomized to the control group will receive standard care and education handouts.
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.

Behavioral: HABIT Intervention
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.

Outcome Measures

Primary Outcome Measures

  1. Mean change in biomarker HbF [0 months, 6 months and 12 months]

    Used to measure hydroxyurea adherence

  2. Mean change in proportion of days covered by hydroxyurea (using prescription refill data) [Up to 12 months]

    Used to measure hydroxyurea adherence

Secondary Outcome Measures

  1. Mean change in score on Peds Quality of Life (generic quality of life) [0 months, 6 months and 12 months]

    Used to measure health-related quality of life

  2. Mean change in score on PedsQL Sickle Cell Disease module (disease specific quality of life) [0 months, 6 months and 12 months]

    Used to measure health-related quality of life

  3. Mean change in score on Sickle Cell Family Responsibility instrument [0 months, 6 months and 12 months]

    Used to measure parent/youth concordance regarding delegation of self-management responsibility by mean change in dyad concordance

Eligibility Criteria

Criteria

Ages Eligible for Study:
10 Years to 18 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 Albert Einstein College of Medicine Bronx New York United States 10461
2 Feinstein Institute for Medical Research Manhasset New York United States 11030
3 Columbia University Irving Medical Center New York New York United States 10032
4 The Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104

Sponsors and Collaborators

  • Columbia University
  • 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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Arlene Smaldone, Professor of Nursing, Columbia University
ClinicalTrials.gov Identifier:
NCT03462511
Other Study ID Numbers:
  • AAAR2908
  • 1R01NR017206-01
First Posted:
Mar 12, 2018
Last Update Posted:
Mar 9, 2022
Last Verified:
Mar 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by Arlene Smaldone, Professor of Nursing, Columbia University
Hydroxyurea
Community health worker (CHW)
Text messages
Medication adherence
Additional relevant MeSH terms:
Anemia, Sickle Cell

Study Results

No Results Posted as of Mar 9, 2022