Clinical trial NCT04002362: Symptom Clusters in Children With Exacerbation-prone Asthma
Official title: Symptom Clusters in Children With Exacerbation-prone Asthma
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Asthma in Children
Keywords: Asthma
Pediatrics
Lead sponsor: Emory University
Sponsor class: OTHER
Collaborators: name: National Institute of Nursing Research (NINR)
First posted: 2019-06-28
Enrollment: 173
Sex: ALL
Minimum age (years): 6
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

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 propose a 48-week cohort study 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 6- 21years of age with exacerbation-prone asthma. Participants will be given the option to either: 1) complete a single study visit, with telephone/email contacts and a review of their electronic medical record for up to 48 weeks after enrollment, or 2) complete the 48-week cohort study with scheduled study visits. Participants who select option 2 with follow-up over 48 weeks will also have the option to complete visits 3, 4 and 5 by telephone.

Eligibility criteria: 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

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

Primary outcomes:
measure: Change in Asthma Control Questionnaire (ACQ) Score
timeFrame: Baseline, Week 2
Secondary outcomes:
measure: Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Asthma Impact Scale (PAIS)
timeFrame: Weeks 16, 32, and 48
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Children's Healthcare of Altanta
state: Georgia
city: Atlanta
country: United States
facility: Emory Children's Center
state: Georgia
Study officials:
affiliation: Emory University
name: Anne Fitzpatrick, PhD
role: PRINCIPAL_INVESTIGATOR
References: