Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 2 for:    corticosteroids | Sickle Cell Disease
Previous Study | Return to List | Next Study

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02187445
Recruitment Status : Completed
First Posted : July 11, 2014
Last Update Posted : February 17, 2017
Sponsor:
Collaborators:
Emory University
Children's Research Institute
Information provided by (Responsible Party):
Michael DeBaun, Vanderbilt University

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).

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Asthma Acute Chest Syndrome Drug: Budesonide inhalation suspension Phase 1

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 36 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
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 Start Date : June 2014
Actual Primary Completion Date : December 2016
Actual Study Completion Date : February 13, 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Steroids
Drug Information available for: Budesonide

Arm Intervention/treatment
Experimental: Budesonide inhalation suspension
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).
Drug: Budesonide inhalation suspension
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).
Other Name: inhaled corticosteroids




Primary Outcome Measures :
  1. The acceptability of budesonide inhalation suspension [ Time Frame: 6 Months ]
    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 :
  1. The impact of BIS on biological correlates of inflammation. [ Time Frame: 12 weeks (or between 8-16 weeks) and at 24 weeks (or between 20-28 weeks) ]
    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).


Other Outcome Measures:
  1. Quality of life for guardians of children with sickle cell disease and ACS [ Time Frame: 0 weeks, 12 weeks (or between 8-16 weeks) and at 24 weeks (or between 20-28 weeks) ]
    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.

  2. Respiratory symptoms [ Time Frame: 6 months, monthly ]
    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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   1 Year to 4 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02187445


Locations
Layout table for location information
United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232-9000
Sponsors and Collaborators
Vanderbilt University
Emory University
Children's Research Institute
Investigators
Layout table for investigator information
Principal Investigator: Michael R DeBaun, MD, MPH Vanderbilt University Medical Center

Layout table for additonal information
Responsible Party: Michael DeBaun, Professor of Pediatrics and Medicine, Vice Chair of Clinical Research in Pediatrics, JC Peterson Endowed Chair in Pediatrics, Director, Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease, Vanderbilt University
ClinicalTrials.gov Identifier: NCT02187445     History of Changes
Other Study ID Numbers: DDCF2014086
First Posted: July 11, 2014    Key Record Dates
Last Update Posted: February 17, 2017
Last Verified: February 2017
Keywords provided by Michael DeBaun, Vanderbilt University:
Sickle Cell Disease
Budesonide inhalation suspension
Acute Chest Syndrome
Additional relevant MeSH terms:
Layout table for MeSH terms
Anemia, Sickle Cell
Disease
Disease Attributes
Hematologic Diseases
Genetic Diseases, Inborn
Lung Diseases
Respiratory Tract Diseases
Respiration Disorders
Acute Chest Syndrome
Syndrome
Recurrence
Pathologic Processes
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hemoglobinopathies
Budesonide
Anti-Inflammatory Agents
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Asthmatic Agents
Respiratory System Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists