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

Primary Purpose

Sickle Cell Disease

Status

Completed

Phase

Not Applicable

Locations

United States

Study Type

Interventional

Intervention

HABIT Intervention

About this trial

This is an interventional health services research trial for Sickle Cell Disease focused on measuring Hydroxyurea, Community health worker (CHW), Text messages, Medication adherence

Eligibility Criteria

10 Years - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Albert Einstein College of Medicine
Feinstein Institute for Medical Research
Columbia University Irving Medical Center
The Children's Hospital of Philadelphia

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Experimental

Arm Label

Control Group

Intervention Group

Arm Description

Dyads randomized to the control group will receive: Standard care Education handouts.

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.

Outcomes

Primary Outcome Measures

Mean change in biomarker HbF

Used to measure hydroxyurea adherence

Mean change in proportion of days covered by hydroxyurea (using prescription refill data)

Used to measure hydroxyurea adherence

Secondary Outcome Measures

Mean change in score on Peds Quality of Life (generic quality of life)

Used to measure health-related quality of life

Mean change in score on PedsQL Sickle Cell Disease module (disease specific quality of life)

Used to measure health-related quality of life

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

Full Information

NCT ID

NCT03462511

First Posted

March 6, 2018

Last Updated

December 7, 2022

Sponsor

Columbia University

Collaborators

National Institute of Nursing Research (NINR)

1. Study Identification

Unique Protocol Identification Number

NCT03462511

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

Study Type

Interventional

2. Study Status

Record Verification Date

December 2022

Overall Recruitment Status

Completed

Study Start Date

August 15, 2018 (Actual)

Primary Completion Date

December 31, 2021 (Actual)

Study Completion Date

December 31, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Columbia University

Collaborators

National Institute of Nursing Research (NINR)

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Sickle Cell Disease

Keywords

Hydroxyurea, Community health worker (CHW), Text messages, Medication adherence

7. Study Design

Primary Purpose

Health Services Research

Study Phase

Not Applicable

Interventional Study Model

Parallel Assignment

Masking

Outcomes Assessor

Allocation

Randomized

Enrollment

100 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Control Group

Arm Type

Active Comparator

Arm Description

Dyads randomized to the control group will receive: Standard care Education handouts.

Arm Title

Intervention Group

Arm Type

Experimental

Arm Description

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 Type

Behavioral

Intervention Name(s)

HABIT Intervention

Intervention Description

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.

Primary Outcome Measure Information:

Title

Mean change in biomarker HbF

Description

Used to measure hydroxyurea adherence

Time Frame

0 months, 6 months and 12 months

Title

Mean change in proportion of days covered by hydroxyurea (using prescription refill data)

Description

Used to measure hydroxyurea adherence

Time Frame

Up to 12 months

Secondary Outcome Measure Information:

Title

Mean change in score on Peds Quality of Life (generic quality of life)

Description

Used to measure health-related quality of life

Time Frame

0 months, 6 months and 12 months

Title

Mean change in score on PedsQL Sickle Cell Disease module (disease specific quality of life)

Description

Used to measure health-related quality of life

Time Frame

0 months, 6 months and 12 months

Title

Mean change in score on Sickle Cell Family Responsibility instrument

Description

Used to measure parent/youth concordance regarding delegation of self-management responsibility by mean change in dyad concordance

Time Frame

0 months, 6 months and 12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

10 Years

Maximum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Arlene Smaldone, PhD, CPNP-PC

Organizational Affiliation

Columbia University School of Nursing

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Nancy S Green, MD

Organizational Affiliation

Columbia University

Official's Role

Principal Investigator

Facility Information:

Facility Name

Albert Einstein College of Medicine

City

Bronx

State/Province

New York

ZIP/Postal Code

10461

Country

United States

Facility Name

Feinstein Institute for Medical Research

City

Manhasset

State/Province

New York

ZIP/Postal Code

11030

Country

United States

Facility Name

Columbia University Irving Medical Center

City

New York

State/Province

New York

ZIP/Postal Code

10032

Country

United States

Facility Name

The Children's Hospital of Philadelphia

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Citations:

PubMed Identifier

31615480

Citation

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.

Results Reference

derived

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

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