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Inhaled Corticosteroid Use to Prevent Acute Chest Syndrome Recurrence in Children Between 1 and 4 With Sickle Cell Disease: a Feasibility Trial

Primary Purpose

Sickle Cell Disease, Asthma, Acute Chest Syndrome

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Budesonide inhalation suspension
Sponsored by
Vanderbilt University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Sickle Cell Disease focused on measuring Sickle Cell Disease, Budesonide inhalation suspension, Acute Chest Syndrome

Eligibility Criteria

1 Year - 4 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • 1) confirmed diagnosis of sickle cell disease (SCD)
  • 2) age between 1 and 4 years (must have reached 1st but not yet 4th birthday)
  • 3) a prior diagnosis of ACS, defined as acute respiratory illness with a new radiodensity on CXR, and one of the following: fever (temperature > 38.50C), decrease in oxygen saturation more than 3% from baseline, or increase in respiratory rate above baseline

Exclusion Criteria:

  • 1) patients already taking inhaled corticosteroids
  • 2) those receiving blood transfusions for elevated TCD or strokes
  • 3) presents over 2 weeks after discharge from hospital following initial ACS episode.

Participants may be on hydroxyurea and participate in this trial.

Sites / Locations

  • Vanderbilt University Medical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Budesonide inhalation suspension

Arm Description

To determine the acceptability of budesonide inhalation suspension (BIS) 0.5 QD for 6 months for children with SCD that develop ACS between 1 and 4 years of age (n=10).

Outcomes

Primary Outcome Measures

The acceptability of budesonide inhalation suspension
Specific Aim 1: To determine the acceptability of budesonide inhalation suspension (BIS) 0.5 QD for 6 months for children with SCD that develop ACS between 1 and 4 years of age (n=10). We will determine the proportions of eligible families who were willing to participate and families that enrolled and elected to stay throughout the six months of the trial. We will also assess adherence to BIS using the Morisky scale; this will be our primary outcome. If the participation rate for the trial is less than 60%, the dropout rate is greater than 20%, or if our adherence rate is poor as measured by the Morisky scale, then alternative strategies for recruitment, retention and adherence must be considered.

Secondary Outcome Measures

The impact of BIS on biological correlates of inflammation.
Specific Aim 2: To explore the impact of BIS on biological correlates of inflammation. For this purpose, a blood sample measurement will be taken at baseline, at 12 weeks (between 8-16 weeks) and at 24 weeks (between 20-28 weeks), as per routine clinic visits. The research visit will be coordinated with the standard care visits and phlebotomy. Soluble vascular cell adhesion molecule-1 (sVCAM-1), a marker of chronic vasculopathy, will be the primary measure of vascular injury. Secondary outcome measures will include additional inflammatory markers (sP-selectin, sE-selectin, IL-1B, IL-6, TNFα, IFN-y, leukotrienes).

Full Information

First Posted
June 27, 2014
Last Updated
February 16, 2017
Sponsor
Vanderbilt University
Collaborators
Emory University, Children's National Research Institute
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1. Study Identification

Unique Protocol Identification Number
NCT02187445
Brief Title
Inhaled Corticosteroid Use to Prevent Acute Chest Syndrome Recurrence in Children Between 1 and 4 With Sickle Cell Disease: a Feasibility Trial
Official Title
Inhaled Corticosteroid Use to Prevent Acute Chest Syndrome Recurrence in Children Between 1 and 4 With Sickle Cell Disease: a Feasibility Trial
Study Type
Interventional

2. Study Status

Record Verification Date
February 2017
Overall Recruitment Status
Completed
Study Start Date
June 2014 (undefined)
Primary Completion Date
December 2016 (Actual)
Study Completion Date
February 13, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Vanderbilt University
Collaborators
Emory University, Children's National Research Institute

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
Acute and chronic pulmonary complications with concomitant inflammatory response are a leading cause of morbidity and mortality in children with sickle cell disease (SCD). Acute chest syndrome (ACS), defined broadly as an increase in respiratory effort, fever and new radiodensity on chest x-ray, is a major cause of death in children and adults with SCD. There is a high rate of ACS in children between 1 and 4 years of age that is associated with an asthma diagnosis, and children with ACS events before 4 years of age have a 50% rate of being hospitalized for either ACS or pain within 1 year of admission. For children with SCD that develop ACS, the investigators propose that the use of budesonide inhalation suspension (BIS) will attenuate pulmonary inflammation after an ACS episode and will decrease future vaso-occlusive pain and ACS episodes. Through a single-arm prospective feasibility trial and in preparation for a limited-institution randomized trial, the investigators plan to test the following primary hypothesis for a phase III definitive trial: In children with SCD admitted to the hospital for an ACS episode between 1 and 4 years of age, low dose BIS for 6 months will result in a 50% reduction in the recurrent incidence rate of ACS or pain requiring hospitalization. Through this trial, the investigators will determine the acceptability of and adherence to BIS in the study population. The investigators will track respiratory symptoms in cases versus controls over 6 months. Finally, the investigators will explore the impact of BIS on biological correlates (sVCAM-1).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease, Asthma, Acute Chest Syndrome
Keywords
Sickle Cell Disease, Budesonide inhalation suspension, Acute Chest Syndrome

7. Study Design

Primary Purpose
Prevention
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
36 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Budesonide inhalation suspension
Arm Type
Experimental
Arm Description
To determine the acceptability of budesonide inhalation suspension (BIS) 0.5 QD for 6 months for children with SCD that develop ACS between 1 and 4 years of age (n=10).
Intervention Type
Drug
Intervention Name(s)
Budesonide inhalation suspension
Other Intervention Name(s)
inhaled corticosteroids
Intervention Description
To determine the acceptability of budesonide inhalation suspension (BIS) 0.5 QD for 6 months for children with SCD that develop ACS between 1 and 4 years of age (n=10).
Primary Outcome Measure Information:
Title
The acceptability of budesonide inhalation suspension
Description
Specific Aim 1: To determine the acceptability of budesonide inhalation suspension (BIS) 0.5 QD for 6 months for children with SCD that develop ACS between 1 and 4 years of age (n=10). We will determine the proportions of eligible families who were willing to participate and families that enrolled and elected to stay throughout the six months of the trial. We will also assess adherence to BIS using the Morisky scale; this will be our primary outcome. If the participation rate for the trial is less than 60%, the dropout rate is greater than 20%, or if our adherence rate is poor as measured by the Morisky scale, then alternative strategies for recruitment, retention and adherence must be considered.
Time Frame
6 Months
Secondary Outcome Measure Information:
Title
The impact of BIS on biological correlates of inflammation.
Description
Specific Aim 2: To explore the impact of BIS on biological correlates of inflammation. For this purpose, a blood sample measurement will be taken at baseline, at 12 weeks (between 8-16 weeks) and at 24 weeks (between 20-28 weeks), as per routine clinic visits. The research visit will be coordinated with the standard care visits and phlebotomy. Soluble vascular cell adhesion molecule-1 (sVCAM-1), a marker of chronic vasculopathy, will be the primary measure of vascular injury. Secondary outcome measures will include additional inflammatory markers (sP-selectin, sE-selectin, IL-1B, IL-6, TNFα, IFN-y, leukotrienes).
Time Frame
12 weeks (or between 8-16 weeks) and at 24 weeks (or between 20-28 weeks)
Other Pre-specified Outcome Measures:
Title
Quality of life for guardians of children with sickle cell disease and ACS
Description
At each clinic visit, we will also collect patient-centered outcomes, assessing caregiver burden. Data will be collected using the Pediatric Asthma Caregiver's Quality of Life Questionnaire (PACQLQ), validated for parents of children with asthma.
Time Frame
0 weeks, 12 weeks (or between 8-16 weeks) and at 24 weeks (or between 20-28 weeks)
Title
Respiratory symptoms
Description
Using the TRACK survey, validated for guardians of children under the age of 5 years, we will call families monthly to collect data on respiratory symptoms over the course of the study.
Time Frame
6 months, monthly

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
4 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 1) confirmed diagnosis of sickle cell disease (SCD) 2) age between 1 and 4 years (must have reached 1st but not yet 4th birthday) 3) a prior diagnosis of ACS, defined as acute respiratory illness with a new radiodensity on CXR, and one of the following: fever (temperature > 38.50C), decrease in oxygen saturation more than 3% from baseline, or increase in respiratory rate above baseline Exclusion Criteria: 1) patients already taking inhaled corticosteroids 2) those receiving blood transfusions for elevated TCD or strokes 3) presents over 2 weeks after discharge from hospital following initial ACS episode. Participants may be on hydroxyurea and participate in this trial.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Michael R DeBaun, MD, MPH
Organizational Affiliation
Vanderbilt University Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232-9000
Country
United States

12. IPD Sharing Statement

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Inhaled Corticosteroid Use to Prevent Acute Chest Syndrome Recurrence in Children Between 1 and 4 With Sickle Cell Disease: a Feasibility Trial

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