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

• ClinicalTrials.gov Identifier: NCT03462511
• Recruitment Status: Completed
• First Posted: March 12, 2018
• Last Update Posted: December 12, 2022
• Sponsor: Columbia University
• Collaborator: National Institute of Nursing Research (NINR)
• Information provided by (Responsible Party): Arlene Smaldone, Columbia University

Tracking Information

• First Submitted Date: March 6, 2018
• First Posted Date: March 12, 2018
• Last Update Posted Date: December 12, 2022
• Actual Study Start Date: August 15, 2018
• Actual Primary Completion Date: December 31, 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: March 6, 2018)

• Mean change in biomarker HbF [ Time Frame: 0 months, 6 months and 12 months ]
  Used to measure hydroxyurea adherence
• Mean change in proportion of days covered by hydroxyurea (using prescription refill data) [ Time Frame: Up to 12 months ]
  Used to measure hydroxyurea adherence

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: March 6, 2018)

• Mean change in score on Peds Quality of Life (generic quality of life) [ Time Frame: 0 months, 6 months and 12 months ]
  Used to measure health-related quality of life
• Mean change in score on PedsQL Sickle Cell Disease module (disease specific quality of life) [ Time Frame: 0 months, 6 months and 12 months ]
  Used to measure health-related quality of life
• Mean change in score on Sickle Cell Family Responsibility instrument [ Time Frame: 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

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial
• Official Title: Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial
• 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 Type: Interventional
• Study Phase: Not Applicable
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Single (Outcomes Assessor); Primary Purpose: Health Services Research
• Condition: 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.

Study Arms

• Active Comparator: Control Group

  Dyads randomized to the control group will receive:

  Standard care Education handouts.

  Intervention: Behavioral: HABIT Intervention
• 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.
  Intervention: Behavioral: HABIT Intervention

• Publications *: 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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: February 1, 2021): 100
• Original Estimated Enrollment (submitted: March 6, 2018): 208
• Actual Study Completion Date: December 31, 2021
• Actual Primary Completion Date: December 31, 2021 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 10 Years to 18 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT03462511
• Other Study ID Numbers: AAAR2908; 1R01NR017206-01 ( U.S. NIH Grant/Contract )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Arlene Smaldone, Columbia University
• Original Responsible Party: Same as current
• Current Study Sponsor: Columbia University
• Original Study Sponsor: Same as current
• 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

• PRS Account: Columbia University
• Verification Date: December 2022