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

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03462511
• Other study ID #: AAAR2908
• Secondary ID: 1R01NR017206-01
• Status: Completed
• Phase: N/A
• Start date: August 15, 2018
• Est. completion date: December 31, 2021

Study information

• Verified date: December 2022
• Source: Columbia University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 100
• Est. completion date: December 31, 2021
• Est. primary completion date: December 31, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 10 Years to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

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.

Locations

Country	Name	City	State
United States	Albert Einstein College of Medicine	Bronx	New York
United States	Feinstein Institute for Medical Research	Manhasset	New York
United States	Columbia University Irving Medical Center	New York	New York
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania

Sponsors (2)

Lead Sponsor	Collaborator
Columbia University	National Institute of Nursing Research (NINR)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Mean change in biomarker HbF	Used to measure hydroxyurea adherence	0 months, 6 months and 12 months
Primary	Mean change in proportion of days covered by hydroxyurea (using prescription refill data)	Used to measure hydroxyurea adherence	Up to 12 months
Secondary	Mean change in score on Peds Quality of Life (generic quality of life)	Used to measure health-related quality of life	0 months, 6 months and 12 months
Secondary	Mean change in score on PedsQL Sickle Cell Disease module (disease specific quality of life)	Used to measure health-related quality of life	0 months, 6 months and 12 months
Secondary	Mean change in score on Sickle Cell Family Responsibility instrument	Used to measure parent/youth concordance regarding delegation of self-management responsibility by mean change in dyad concordance	0 months, 6 months and 12 months