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.
Brief Title
Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial
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.
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.
Categories
Completion Date
Completion Date Type
Actual
Conditions
Sickle Cell Disease
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
* 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
Inclusion 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
* 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
Gender
All
Gender Based
false
Keywords
Hydroxyurea
Community health worker (CHW)
Text messages
Medication adherence
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
18 Years
Minimum Age
10 Years
NCT Id
NCT03462511
Org Class
Other
Org Full Name
Columbia University
Org Study Id
AAAR2908
Overall Status
Completed
Phases
Not Applicable
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial
Primary Outcomes
Outcome Description
A serum biomarker obtained from youth used to measure adherence to hydroxyurea
Outcome Measure
Mean Change in Biomarker Fetal Hemoglobin (HbF)
Outcome Time Frame
Baseline, 6 months, 12 months
Outcome Description
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.
Outcome Measure
Mean Change in Proportion of Days Covered (PDC) by Hydroxyurea
Outcome Time Frame
Baseline, 6 months, 12 months
Secondary Ids
Secondary Id
1R01NR017206-01
Secondary Outcomes
Outcome Description
Used to measure health-related quality of life. Scores range from 0-100 with a higher score indicating a better quality of life.
Outcome Time Frame
Baseline, 4 months, 9 months and 12 months
Outcome Measure
Mean Change in Youth Score on Peds Quality of Life (Generic Quality of Life)
Outcome Description
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.
Outcome Time Frame
Baseline, 9 months and 12 months
Outcome Measure
Mean Change in Youth Score on PedsQL Sickle Cell Disease Module (Disease Specific Quality of Life)
Outcome Description
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.
Outcome Time Frame
Baseline, 6 months and 12 months
Outcome Measure
Mean Change in Parent Youth Concordance Regarding Self-management Responsibility
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Child
Adult
Maximum Age Number (converted to Years and rounded down)
18
Minimum Age Number (converted to Years and rounded down)
10
Investigators
Investigator Type
Principal Investigator
Investigator Name
Deepa Manwani
Investigator Email
dmanwani@montefiore.org
Investigator Phone
718-741-2342