Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators (SHiP HU)

• ClinicalTrials.gov Identifier: NCT02197845
• Recruitment Status: Completed
• First Posted: July 23, 2014
• Last Update Posted: June 26, 2019
• Sponsor: Virginia Commonwealth University
• Collaborators: Eastern Virginia Medical School; Children's Hospital of The King's Daughters; Virginia Department of Health; James Madison University; National Heart, Lung, and Blood Institute (NHLBI)
• Information provided by (Responsible Party): Virginia Commonwealth University

Tracking Information

• First Submitted Date: July 21, 2014
• First Posted Date: July 23, 2014
• Last Update Posted Date: June 26, 2019
• Actual Study Start Date: October 2012
• Actual Primary Completion Date: July 7, 2018 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: December 16, 2014)

• Phase I: Percent of enrolled Phase I subjects who complete a provider visit by 3 months post enrollment [ Time Frame: 3 months ]
• Phase II: Increase in fetal hemoglobin (HbF) as measured by hemoglobin electrophoresis [ Time Frame: Baseline, 6 months, 1 year ]

• Original Primary Outcome Measures (submitted: July 22, 2014): Phase II: Increase in the hemoglobin electrophoresis (HbF) level post-randomization [ Time Frame: 6 months, 1 year ]

Current Secondary Outcome Measures (submitted: June 20, 2016)

• Phase II: Measures of adherence to HU [ Time Frame: Baseline, 6 months, 1 year ]
  For patients prescribed HU, clinical research coordinators will assess HU prescription refills from pill counts, pharmacy records, and self-report at baseline, 6 months, and 1 year. Patient navigators will regularly assess their patients' HU adherence by conducting pill counts at home visits.
• Phase II: Percent of patients achieving either maximum tolerated dose (MTD) or maximum dose [ Time Frame: Baseline, 6 months, 1 year ]
  Maximum tolerated dose (MTD) is the daily single oral dose that can be maintained for at least 16 weeks without toxicity (< 3 x l09 neutrophils/L, <100 x l09 platelets/L, < 125 x l09 reticulocytes/L, 20% drop in [Hb] or an absolute value of <4.5 g/dL, 50% rise in creatinine or absolute increase of >0.4 mg/dL, 100% rise in ALT,GI disturbance, or rash or hair loss not attributable to other causes). Maximum dose is 35 mg/kg.
• Phase II: Number of emergency department and hospital visits [ Time Frame: Baseline, 6 months, 1 year ]
• Phase II: Mean corpuscular volume [ Time Frame: Baseline, 6 months, 1 year ]
• Phase II: Total hemoglobin [ Time Frame: Baseline, 6 months, 1 year ]
• Phase II: White blood cell count [ Time Frame: Baseline, 6 months, 1 year ]
• Phase II: Reticulocyte count [ Time Frame: Baseline, 6 months, 1 year ]
• Phase II: Quality of life measures [ Time Frame: Baseline, 6 months, 1 year ]
  Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me), Patient Reported Outcomes Measurement Information System (PROMIS)
• Phase II: Patient activation measures [ Time Frame: Baseline, 6 months, 1 year ]
  Patient Activation Measure
• Phase II: Patient knowledge measures [ Time Frame: 1 year ]
  Assessment of Sickle Cell Knowledge - University of Florida (UF-ASCK) (unpublished)
• Phase II: Health care knowledge and skills, self-efficacy, sickle cell stress measures [ Time Frame: Baseline, 6 months, 1 year ]
  Sickle Cell Transition intervention Program (TIP) Survey (non-validated)
• Phase II: Social support measures [ Time Frame: Baseline, 6 months, 1 year ]
  Sickle Cell Transition intervention Program (TIP) Survey (non-validated) and Multidimensional Scale of Perceived Social Support
• Phase II: Coping strategies [ Time Frame: Baseline, 6 months, 1 year ]
  Coping Strategies Questionnaire for SCD
• Phase II: Associated pain conditions and comorbidities [ Time Frame: Baseline, 6 months, 1 year ]
  Chart Review, Self Report and Surveys: Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me), Patient Reported Outcomes Measurement Information System (PROMIS)
• Phase II: Blood transfusion measures (if applicable) [ Time Frame: 6 months, 1 year ]
  Chart review
• Phase II: Patient Navigator Satisfaction (if applicable) [ Time Frame: 1 year ]
  Patient Navigator Satisfaction Surveys, 12 month follow up, Patient Study Experience Review for Patient Navigators

Original Secondary Outcome Measures (submitted: July 22, 2014)

• Phase II: Measures of adherence to HU [ Time Frame: 6 months, 1 year ]
  For patients prescribed HU, clinical research coordinators will assess HU prescription refills from pill counts, pharmacy records, and self-report at six months and one year. Patient navigators will regularly assess their patients prescribed HU by pill counts at home visits.
• Phase II: Percent of patients achieving either maximum tolerated dose, a clinical endpoint, or maximum dose [ Time Frame: 6 months, 1 year ]
• Phase II: Number of emergency department and hospital visits [ Time Frame: 6 months, 1 year ]
• Phase II: Mean corpuscular volume [ Time Frame: 6 months, 1 year ]
• Phase II: Total hemoglobin [ Time Frame: 6 months, 1 year ]
• Phase II: White blood cell count [ Time Frame: 6 months, 1 year ]
• Phase II: Reticulocyte count [ Time Frame: 6 months, 1 year ]
• Phase II: Quality of life measures [ Time Frame: 6 months, 1 year ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators
• Official Title: Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators

Brief Summary

Multi-phase, patient navigator-based program in the Richmond and Tidewater regions of Virginia to demonstrate:

1. the feasibility of using patient navigators to improve the percentage of children and adult (age 15 and older) patients with sickle cell disease (SCD) in SCD specialty care
2. the efficacy of using patient navigators to improve hydroxyurea (HU) (re-)initiation and adherence among adult patients with SCD eligible for HU

(Patient navigators may also be known as public health workers.)

Detailed Description

The state of Virginia, including the Virginia Department of Health and three academic medical centers and one federally qualified health center, plans a two-phase demonstration, first of improvement in the percentage of adults with SCD who are in SCD specialty care (Phase I), then of improvement in adherence to HU of eligible SCD adults (Phase II). Both phases will use existing academic SCD providers, and an innovative, multimodal strategy, featuring specially trained SCD patient navigators (PNs), that addresses barriers to care and to HU use. In Phase I we will demonstrate the feasibility of utilizing PNs to overcome patient access barriers to SCD care. In Phase II we will test the efficacy of PNs for overcoming barriers to acceptance of and adherence with HU therapy. Patients will be randomized to a PN arm or to a usual care arm. Providers will implement NIH guidelines for HU eligibility and prescribing in both arms. All HU eligible patients will be offered HU at each clinical visit. Among patients prescribed HU, if a maximum tolerated dose (MTD, defined in end points) has not been reached, providers will dose escalate every 8-12 weeks to MTD, rather than to clinical effect, before declaring treatment success or failure.

This project will be critically important and impactful by demonstrating the feasibility of a statewide community-based strategy to assist vulnerable SCD adults in obtaining SCD specialty care and likely prolonging life, a model that other states could adopt.

• Study Type: Interventional
• Study Phase: Not Applicable
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Single (Investigator); Primary Purpose: Health Services Research
• Condition: Sickle Cell Disease

Intervention

• Behavioral: Patient Navigator
  A specially trained (SCD knowledgable) Patient Navigator will act as a liaison who assists participants in increasing adherence to HU through disease education, addressing barriers to care and improving adherence to HU. PN utilize various techniques geared toward the individual patients needs. Interventions utilized by a PN include improving disease management skills, educational materials about disease, HU adherence, motivational interviewing, care coordination and social support.
  Other Names:

  • Public Health Worker
  • Community Health Worker
• Behavioral: Recruitment into Specialty Care
  A specially trained (SCD knowledgeable) Patient Navigator will act as a liaison to participants in increasing adherence to Hydroxyurea. Interventions utilized by a PN include teaching disease management skills, addressing barriers to care, disease education, HU management, motivational interviewing, care coordination and social support.
  Other Name: Sickle Cell Disease Specialty Care

Study Arms

• Experimental: Phase I: Recruitment into Specialty Care
  Participants in the Phase I Experimental Arm are enrolled into SCD specialty care. PN's will contact patient up to 3 times to assure patients have had an initial visit by 3 months time.
  Intervention: Behavioral: Recruitment into Specialty Care
• Experimental: Phase II: Patient Navigator Arm
  Participants in the Phase II Experimental Arm follow routine clinical care and are assigned a Patient Navigator. A specially trained (SCD specefic)PN will work with participants for one year. Participants will be contacted by their Navigator weekly for the first 6 months, then biweekly for the second 6 months.
  Intervention: Behavioral: Patient Navigator
• No Intervention: Phase II: Passenger Arm
  No Intervention. Participants in the Phase II Passenger Arm follow routine clinical care.

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: June 25, 2019): 353
• Original Estimated Enrollment (submitted: July 22, 2014): 270
• Actual Study Completion Date: December 31, 2018
• Actual Primary Completion Date: July 7, 2018 (Final data collection date for primary outcome measure)

Eligibility Criteria

PHASE I:

Inclusion Criteria:

• Patient Self Report of Sickle Cell Disease (Genotypes: Hb SS, SC, SBoThal, SB+Thal)
• 15 years or older
• Virginia resident

Exclusion Criteria:

-Visited one of a pre-selected list of sickle cell specialists in Virginia within the last 6 months

PHASE II:

Inclusion Criteria:

• Sickle Cell Disease (SCD) patient (Genotypes: SS or SBoThal)
• Eligible for Hydroxyurea (according to NIH guidelines)
• 15 years or older
• Virginia resident

Exclusion Criteria:

• Pregnancy
• Enrollment in scheduled chronic transfusion program
• SCD Genotype: Hb SC and SB+Thal)

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 15 Years and older (Child, Adult, Older 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: NCT02197845
• Other Study ID Numbers: HM14641; 1R18HL112737-01 ( U.S. NIH Grant/Contract )
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Virginia Commonwealth University
• Original Responsible Party: Same as current
• Current Study Sponsor: Virginia Commonwealth University
• Original Study Sponsor: Same as current

Collaborators

• Eastern Virginia Medical School
• Children's Hospital of The King's Daughters
• Virginia Department of Health
• James Madison University
• National Heart, Lung, and Blood Institute (NHLBI)

Investigators

• Principal Investigator: Wally R Smith, MD; Virginia Commonwealth University

• PRS Account: Virginia Commonwealth University
• Verification Date: June 2019