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

NCT02187445 | Completed Phase 1 DMC

• 1 other identifier: DDCF2014086
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 1

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).

Conditions

Sickle Cell Disease; Asthma; Acute Chest Syndrome

Keywords

Sickle Cell Disease; Budesonide inhalation suspension; Acute Chest Syndrome

Outcome Measures

Primary Outcomes (1)

• 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.

  6 Months

Secondary Outcomes (1)

• The impact of BIS on biological correlates of inflammation.

  12 weeks (or between 8-16 weeks) and at 24 weeks (or between 20-28 weeks)

Other Outcomes (2)

• Quality of life for guardians of children with sickle cell disease and ACS

  0 weeks, 12 weeks (or between 8-16 weeks) and at 24 weeks (or between 20-28 weeks)

• Respiratory symptoms

  6 months, monthly

Study Arms (1)

Budesonide inhalation suspension

EXPERIMENTAL

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

Interventions

Budesonide inhalation suspension DRUG

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).

Also known as: inhaled corticosteroids

Budesonide inhalation suspension

Eligibility Criteria

• Age: 1 Year - 4 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• \) confirmed diagnosis of sickle cell disease (SCD)
• \) age between 1 and 4 years (must have reached 1st but not yet 4th birthday)
• \) 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

You may not qualify if:

• \) patients already taking inhaled corticosteroids
• \) those receiving blood transfusions for elevated TCD or strokes
• \) presents over 2 weeks after discharge from hospital following initial ACS episode.
• Participants may be on hydroxyurea and participate in this trial.

Sponsors & Collaborators

• Vanderbilt University: lead
• Emory University: collaborator
• Children's National Research Institute: collaborator

Study Sites (1)

Vanderbilt University Medical Center

Nashville, Tennessee, 37232-9000, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Asthma; Acute Chest Syndrome

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Respiratory Hypersensitivity; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Respiration Disorders

Study Officials

• Michael R DeBaun, MD, MPH

  Vanderbilt University Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: 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

Study Record Dates

• First Submitted: June 27, 2014
• First Posted: July 11, 2014
• Study Start: June 1, 2014
• Primary Completion: December 1, 2016
• Study Completion: February 13, 2017
• Last Updated: February 17, 2017

Record last verified: 2017-02

Locations

US (1)