Symptom Clusters in Children With Exacerbation-prone Asthma

Pediatric participants with exacerbation-prone asthma will receive an intramuscular injection of triamcinolone acetonide and will be followed for 48 weeks. The study visit 2 weeks after the injection will assess the response to the study medication, while the remaining study visits will examine the temporal stability of the symptom clusters.

Study Overview

• Status: Recruiting
• Conditions: Asthma in Children
• Intervention / Treatment: Drug: Triamcinolone Acetonide

Detailed Description

Asthma symptom control is suboptimal in the majority of children in the United States, despite widespread availability of asthma controller medications and standardized treatment guidelines. While deaths from asthma have declined, 53.7% of children with asthma continue to experience an exacerbation each year and the associated public health burden is substantial.

While the factors responsible for poor asthma symptom control are complex and include limited access to care, poor adherence to preventative asthma medications, and exposures to environmental allergens and irritants such as tobacco smoke, it is also recognized that children with exacerbation-prone asthma are a heterogeneous group with differing clinical outcomes and longitudinal disease trajectories. Symptoms (defined as subjective sensations) can also be quite varied within and among affected children. Whereas some children have persistent, troublesome respiratory symptoms, others have respiratory symptoms only with upper respiratory infections. Mental health symptoms and social health symptoms have been inadequately characterized in this population, but some children with asthma also report depression and anxiety and impaired family functioning and relationships that may further worsen asthma outcomes. However, prior studies are limited by a narrow focus on individual symptoms in isolation. To date, there has been no attempt to identify symptom clusters (defined as two or more concurrent symptoms independent of other clusters) in children with exacerbation-prone asthma.

Poor understanding of symptom clusters is a major shortcoming in asthma symptom science. In other chronic disorders such as cancer, compared with a single symptom, symptom clusters of pain, fatigue, sleep disturbance and mood disturbance significantly worsen patient-reported outcomes of functional status and quality of life. There is also emerging evidence that interventions for one symptom within a cluster (i.e., cognitive-behavioral therapy for pain) reduce the severity of other symptoms within that cluster (i.e., fatigue and sleep disturbance). Because children with exacerbation-prone asthma rarely report a single symptom, greater knowledge of the assessment (and ultimately management) of symptom clusters in these children has the potential to significantly improve individualized treatment and clinical outcomes.

The researchers here propose a 48-week cohort study (N=173) to test the overarching hypothesis that symptom clusters and their associated inflammatory and metabolic pathways predict corticosteroid treatment responsiveness (primary objective outcome) and quality of life (patient-reported secondary outcome) in children 8-17 years with exacerbation-prone asthma.

• Study Type: Interventional
• Enrollment (Anticipated): 173
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Anne Fitzpatrick, PhD; Phone Number: 404-727-9112; Email: anne.fitzpatrick@emory.edu

Study Locations

• United States
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Recruiting
      • Emory Children's Center
    • Atlanta, Georgia, United States, 30322
      • Recruiting
      • Children's Healthcare of Altanta

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years to 19 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Age 6 to less than 21 years at the enrollment visit
• Physician diagnosis of asthma

• History of an asthma exacerbation in the previous 12 months, defined as either:

  • Treatment with systemic corticosteroids, or
  • Increase in rescue medication use (i.e., albuterol or inhaled corticosteroid) for 24 hours or more, or
  • One or more missed school days due to asthma symptoms, or
  • An unscheduled visit for asthma at either a physician's office, urgent care, hospital emergency room, or
  • Hospitalization for asthma.
  • In the event of a recent exacerbation treated with systemic corticosteroids or requiring hospitalization, the first study visit will be postponed until two weeks after the last dose of systemic corticosteroids (i.e., prednisone or prednisolone).

Exclusion Criteria:

• Previous allergic reaction to systemic corticosteroids
• Hepatic, biliary, or renal disease that can interfere with drug metabolism/excretion
• Chronic medical disorders that may increase the risk of drug-related injury, including osteogenesis imperfecta (increased risk of fracture with corticosteroids), or Crohn's disease, ulcerative colitis, juvenile rheumatoid arthritis, clotting disorders, or Factor deficiency (increased risk of bleeding with corticosteroid therapy)
• Pregnancy
• Current smoking
• Congenital disorders or deformities of the chest wall, lungs or airways
• History of premature birth <35 weeks gestation
• Unwillingness to receive triamcinolone
• Planning to relocate before study completion

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Children receiving triamcinolone acetonide; Pediatric participants with exacerbation-prone asthma will receive an intramuscular injection of triamcinolone acetonide and will be followed for 48 weeks.	Drug: Triamcinolone Acetonide; An intramuscular injection of triamcinolone acetonide (1 mg/kg, up to 40 mg maximum) will be administered deep in the gluteal muscle by a trained registered nurse.; Other Names: Kenalog

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Asthma Control Questionnaire (ACQ) Score	Responsiveness to the study treatment will be assessed with the ACQ. This 7-item questionnaire includes questions related to daytime and nocturnal symptoms, short-acting bronchodilator use, and lung function during the clinic visit on that day. Participants report how difficult their asthma was to control on a scale from 0 (no impairment) to 6 (maximum impairment). Total raw scores range from 0 to 42, with higher scores indicating poorer asthma control.	Baseline, Week 2

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Asthma Impact Scale (PAIS)	Quality of life will be assessed with the 8-item PAIS instrument. As with all PROMIS measures, the PAIS is scored on the T-score metric, with higher scores reflecting more of the concept being measured. On the T-score metric, 50 is the mean of the reference population and 10 is the standard deviation; thus scores of 40 and 60 are one standard deviation lower and higher than the mean of the reference population, respectively.	Weeks 16, 32, and 48

Collaborators and Investigators

• Sponsor: Emory University
• Collaborators: National Institute of Nursing Research (NINR)
• Investigators: Principal Investigator: Anne Fitzpatrick, PhD, Emory University

Study record dates

Study Major Dates

• Study Start (Actual): November 13, 2019
• Primary Completion (Anticipated): June 30, 2024
• Study Completion (Anticipated): June 30, 2024

Study Registration Dates

• First Submitted: June 27, 2019
• First Submitted That Met QC Criteria: June 27, 2019
• First Posted (Actual): June 28, 2019

Study Record Updates

• Last Update Posted (Actual): May 9, 2023
• Last Update Submitted That Met QC Criteria: May 8, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: Asthma; Pediatrics
• Additional Relevant MeSH Terms: Pathologic Processes; Respiratory Tract Diseases; Immune System Diseases; Lung Diseases; Hypersensitivity, Immediate; Disease; Bronchial Diseases; Lung Diseases, Obstructive; Respiratory Hypersensitivity; Hypersensitivity; Syndrome; Asthma; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Anti-Inflammatory Agents; Immunosuppressive Agents; Immunologic Factors; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Triamcinolone; Triamcinolone Acetonide; Triamcinolone hexacetonide; Triamcinolone diacetate
• Other Study ID Numbers: IRB00111087; R01NR018666 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: All of the individual participant data collected during the trial will be available for sharing, after deidentification.
• IPD Sharing Time Frame: Data will be made available for sharing three months following publication, with no end date.
• IPD Sharing Access Criteria: Data will be available for sharing with researchers who provide a methodologically sound proposal, in order to achieve the aims in the proposal. Proposals should be directed to anne.fitzpatrick@emory.edu. To gain access, data requestors will need to sign a data access agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No