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

Not Applicable

Completed

• Conditions: Sickle Cell Disease
• Interventions: Other: Standard of Care; Behavioral: HABIT Intervention; Other: Education materials

• Registration Number: NCT03462511
• Lead Sponsor: Columbia University

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.

Study Timeline

• Study First Submitted: 2018-03-06
• Study First Submitted QC: 2018-03-06
• Study First Posted: 2018-03-12
• Study Start: 2018-08-15
• Primary Completion: 2021-12-31
• Study Completion: 2021-12-31
• Disposition First Submitted: 2022-11-11
• Disposition First Submitted QC: 2022-12-01
• Disposition First Posted: 2022-12-06
• Results First Submitted: 2024-03-07
• Status Verified: 2024-06
• Results First Submitted QC: 2024-06-05
• Last Update Submitted: 2024-06-05
• Results First Posted: 2024-06-26
• Last Update Posted: 2024-06-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Control Group	Standard of Care	Dyads randomized to the control group will receive: Standard care and Education handouts.
Intervention Group	Standard of Care	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.
Intervention Group	Education materials	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.
Control Group	Education materials	Dyads randomized to the control group will receive: Standard care and Education handouts.
Intervention Group	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.

Primary Outcome Measures

Name	Time	Method
Mean Change in Biomarker Fetal Hemoglobin (HbF)	Baseline, 6 months, 12 months	A serum biomarker obtained from youth used to measure adherence to hydroxyurea
Mean Change in Proportion of Days Covered (PDC) by Hydroxyurea	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 Outcome Measures

Name	Time	Method
Mean Change in Youth Score on Peds Quality of Life (Generic Quality of Life)	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.
Mean Change in Parent Youth Concordance Regarding Self-management Responsibility	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.
Mean Change in Youth Score on PedsQL Sickle Cell Disease Module (Disease Specific Quality of Life)	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.

Trial Locations

Locations (4)

The Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Albert Einstein College of Medicine; 🇺🇸 Bronx, New York, United States

Columbia University Irving Medical Center; 🇺🇸 New York, New York, United States

Feinstein Institute for Medical Research; 🇺🇸 Manhasset, New York, United States