Trial Outcomes & Findings for Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial (NCT NCT03462511)
NCT ID: NCT03462511
Last Updated: 2024-06-26
Results Overview
A serum biomarker obtained from youth used to measure adherence to hydroxyurea
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
50 participants
Primary outcome timeframe
Baseline, 6 months, 12 months
Results posted on
2024-06-26
Participant Flow
24 Dyads were randomized to the intervention (24 parents + 24 youth) 26 Dyads were randomized to the control (26 parents + 26 youth)
Participant milestones
| Measure |
Intervention Group
In addition to standard care and education handouts, dyads randomized to the intervention group will receive the HABIT intervention, which includes community health workers (CHW) support and tailored text messages.
|
Control Group
Dyads randomized to the control group will receive:
Standard care and Education handouts.
|
|---|---|---|
|
Overall Study
STARTED
|
24
|
26
|
|
Overall Study
COMPLETED
|
22
|
23
|
|
Overall Study
NOT COMPLETED
|
2
|
3
|
Reasons for withdrawal
| Measure |
Intervention Group
In addition to standard care and education handouts, dyads randomized to the intervention group will receive the HABIT intervention, which includes community health workers (CHW) support and tailored text messages.
|
Control Group
Dyads randomized to the control group will receive:
Standard care and Education handouts.
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
2
|
3
|
Baseline Characteristics
Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial
Baseline characteristics by cohort
| Measure |
Intervention Group
n=24 Participants
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
n=26 Participants
Dyads randomized to the control group will receive:
Standard care and Education handouts.
|
Total
n=50 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
14.1 years
STANDARD_DEVIATION 1.9 • n=5 Participants
|
13.0 years
STANDARD_DEVIATION 1.7 • n=7 Participants
|
13.3 years
STANDARD_DEVIATION 1.9 • n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
21 Participants
n=5 Participants
|
21 Participants
n=7 Participants
|
42 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
21 Participants
n=5 Participants
|
20 Participants
n=7 Participants
|
41 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
3 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
24 participants
n=5 Participants
|
26 participants
n=7 Participants
|
50 participants
n=5 Participants
|
|
No other chronic condition besides Sickle Cell Disease (SCD)
|
12 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
25 Participants
n=5 Participants
|
|
Had 1 or more ED visits in past year
|
11 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
|
Had 1 more hospitalizations in past year
|
11 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
22 Participants
n=5 Participants
|
|
Parent status: Single, separated or divorced
|
16 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
32 Participants
n=5 Participants
|
|
Parent education high school or less
|
7 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
|
Caregiver affected by SCD
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Other people in home affected by SCD
|
4 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Biomarker Fetal Hemoglobin (HbF)
|
12.2 Percent
STANDARD_DEVIATION 5.9 • n=5 Participants
|
12.4 Percent
STANDARD_DEVIATION 5.3 • n=7 Participants
|
12.3 Percent
STANDARD_DEVIATION 5.5 • n=5 Participants
|
|
Proportion of days covered by hydroxyurea
|
55.2 Percent of days
STANDARD_DEVIATION 26.7 • n=5 Participants
|
55.9 Percent of days
STANDARD_DEVIATION 26.1 • n=7 Participants
|
55.6 Percent of days
STANDARD_DEVIATION 26.1 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, 6 months, 12 monthsPopulation: Change between 6-12 months: 22 of 24 intervention group participants analyzed as blood samples were collected at both timepoints for 22 of 24 participants, due to difficulties coming into clinic during the COVID-19 pandemic.
A serum biomarker obtained from youth used to measure adherence to hydroxyurea
Outcome measures
| Measure |
Intervention Group
n=24 Participants
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
n=26 Participants
Dyads randomized to the control group will receive:
Standard care and Education handouts.
|
|---|---|---|
|
Mean Change in Biomarker Fetal Hemoglobin (HbF)
Baseline to Month 6
|
2.7 Percent fetal hemoglobin
Standard Deviation 1.3
|
0.3 Percent fetal hemoglobin
Standard Deviation 1.3
|
|
Mean Change in Biomarker Fetal Hemoglobin (HbF)
Month 6 to Month 12
|
-1.7 Percent fetal hemoglobin
Standard Deviation 1.2
|
1.8 Percent fetal hemoglobin
Standard Deviation 1.3
|
PRIMARY outcome
Timeframe: Baseline, 6 months, 12 monthsPopulation: 21 of 24 intervention group participants and 22 of 26 control group participants analyzed as data could not be collected from pharmacies due to COVID-19 difficulties and loss of data resulting from fire.
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 measures
| Measure |
Intervention Group
n=21 Participants
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
n=22 Participants
Dyads randomized to the control group will receive:
Standard care and Education handouts.
|
|---|---|---|
|
Mean Change in Proportion of Days Covered (PDC) by Hydroxyurea
Baseline and 6 months
|
17.9 change in percentage of PDC
Standard Deviation 27.3
|
13.1 change in percentage of PDC
Standard Deviation 29.7
|
|
Mean Change in Proportion of Days Covered (PDC) by Hydroxyurea
6 months and 12 months
|
-1.6 change in percentage of PDC
Standard Deviation 22.8
|
-1.9 change in percentage of PDC
Standard Deviation 33.7
|
SECONDARY outcome
Timeframe: Baseline, 4 months, 9 months and 12 monthsUsed to measure health-related quality of life. Scores range from 0-100 with a higher score indicating a better quality of life.
Outcome measures
| Measure |
Intervention Group
n=24 Participants
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
n=26 Participants
Dyads randomized to the control group will receive:
Standard care and Education handouts.
|
|---|---|---|
|
Mean Change in Youth Score on Peds Quality of Life (Generic Quality of Life)
Baseline to 9 months
|
10.3 score on a scale
Standard Deviation 12.6
|
2.0 score on a scale
Standard Deviation 12.3
|
|
Mean Change in Youth Score on Peds Quality of Life (Generic Quality of Life)
9 months to 12 months
|
-2.5 score on a scale
Standard Deviation 11.6
|
1.1 score on a scale
Standard Deviation 16.2
|
SECONDARY outcome
Timeframe: Baseline, 9 months and 12 monthsUsed 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 measures
| Measure |
Intervention Group
n=24 Participants
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
n=26 Participants
Dyads randomized to the control group will receive:
Standard care and Education handouts.
|
|---|---|---|
|
Mean Change in Youth Score on PedsQL Sickle Cell Disease Module (Disease Specific Quality of Life)
Baseline to 9 months
|
16.4 score on a scale
Standard Deviation 23.3
|
9.0 score on a scale
Standard Deviation 12.0
|
|
Mean Change in Youth Score on PedsQL Sickle Cell Disease Module (Disease Specific Quality of Life)
9 months to 12 months
|
-4.3 score on a scale
Standard Deviation 21.9
|
-0.7 score on a scale
Standard Deviation 15.7
|
SECONDARY outcome
Timeframe: Baseline, 6 months and 12 monthsConcordance 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 measures
| Measure |
Intervention Group
n=24 Participants
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
n=26 Participants
Dyads randomized to the control group will receive:
Standard care and Education handouts.
|
|---|---|---|
|
Mean Change in Parent Youth Concordance Regarding Self-management Responsibility
Baseline to 6 months
|
1.6 units on a scale
Standard Deviation 2.4
|
1.4 units on a scale
Standard Deviation 2.5
|
|
Mean Change in Parent Youth Concordance Regarding Self-management Responsibility
6 months to 12 months
|
0.3 units on a scale
Standard Deviation 2.1
|
0.5 units on a scale
Standard Deviation 2.3
|
Adverse Events
Control Group
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Intervention Group
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Arlene Smaldone, PhD, RN
Columbia University School of Nursing
Phone: 212-342-3048
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place