N/A
Completed N=50
Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial
Source: ClinicalTrials.gov NCT03462511 ↗Enrolled (actual)
50
Serious AEs
0.0%
Results posted
Jun 2024
Primary outcomePrimary: Mean Change in Biomarker Fetal Hemoglobin (HbF) — 2.7; 0.3; -1.7; 1.8 Percent fetal hemoglobin — p=0.067
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Change in Biomarker Fetal Hemoglobin (HbF) |
2.7; 0.3; -1.7; 1.8 | 0.067 |
| PRIMARY Mean Change in Proportion of Days Covered (PDC) by Hydroxyurea |
17.9; 13.1; -1.6; -1.9 | 0.60 |
| SECONDARY Mean Change in Youth Score on Peds Quality of Life (Generic Quality of Life) |
10.3; 2.0; -2.5; 1.1 | <0.001 sig |
| SECONDARY Mean Change in Youth Score on PedsQL Sickle Cell Disease Module (Disease Specific Quality of Life) |
16.4; 9.0; -4.3; -0.7 | 0.47 |
| SECONDARY Mean Change in Parent Youth Concordance Regarding Self-management Responsibility |
1.6; 1.4; 0.3; 0.5 | 0.002 sig |
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 levels below expected grade)
- Youth not residing with parent/legal guardian
Exclusion Criteria - Parent:
- Parent/legal guardian does not reside with youth
Data sourced from ClinicalTrials.gov (NCT03462511). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.