Clinical trial NCT04179461: Personalized Treatment Algorithms for Difficult-to-treat Asthma
Official title: Personalized Treatment Algorithms for Difficult-to-treat Asthma: Bench to Community
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Asthma in Children
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
First posted: 2019-11-27
Enrollment: 21
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: The study has 3 study phases: baseline, intervention, and observation. Each participant will independently transition through these study phases.

Baseline period: Each participant will be given a treatment recommendation according to their asthma severity/control using the National Asthma Education and Prevention Program (NAEPP asthma guidelines).

Intervention period: Each participant will be given a personalized treatment plan based on their asthma severity/control during the baseline period, biologic, and environmental factors.

Observation period: All participants will be observed for at least 6 months. Investigators will track the participant's personal outcomes (comparing to baseline) for reutilization, symptom-free days, and quality of life.
Primary purpose: TREATMENT

Brief summary: Asthma is a common, complex and costly chronic condition. Moreover, asthma is heterogeneous in terms of treatment response. This heterogeneity contributes to the difficulty in both studying and treating asthma. This is a pilot study to improve health outcomes in youths with difficult to treat asthma with ongoing symptoms and healthcare utilization despite medium to high doses of inhaled corticosteroids. Asthma heterogeneity in both disease pathophysiology and treatment response contributes to the difficulty in both studying and managing asthma. In order to begin to develop personalized algorithms for patients, investigators need to model novel biomarkers and other factors that contribute to individual differences in asthma outcome and test other factors that contribute to individual differences in asthma outcome and test personalized treatment strategies.

Detailed description: With this study investigators will conduct study visits to determine and quantify known molecular, genetic, genomic, epigenetics, immunologic, and exposure biomarkers that will help elucidate molecular disease endotype within the difficult-to-treat phenotype. This information will be used along with clinical, psychosocial, and adherence data to develop a personalized treatment plan. Following the personalized treatment plan, study clinicians will prescribe the medications through a home delivery or routine pharmacy depending on the family's preference and covered by the participant's health insurance. Investigators will track disease outcome metrics including exacerbations, symptom-free days, and asthma symptom scores to determine the effectiveness of this personalized approach.

Eligibility criteria: Inclusion Criteria:

* History of provider-diagnosed asthma
* Meets one of the following definition for NAEPP guidelines uncontrolled moderate persistent asthma or severe persistent asthma in the past 12 months:

  * NAEPP step 3-4 with one of the following criteria in the past 12 months:

    * Two Asthma Control Test (ACT) scores \<20
    * 1 Urgent Care or Emergency Department visit or hospitalization for asthma
    * \>2 prednisone bursts
  * NAEPP step 5-6
* Current health insurance coverage at enrollment. This will be verified at V1 at the registration desk. If the family loses insurance during the study, the patient will be referred to the pulmonary social worker and financial office for assistance
* Reside at a primary home on average 5 out of 7 days a week.
* Primary home is within a 40 mile radius of Cincinnati Children's Base location or PI's discretion.

Exclusion Criteria:

* Received biologic therapy 6 months prior to enrollment
* Received systemic steroids 6 weeks prior to enrollment
* Active chronic disease apart from asthma or allergic disease
* Co-morbid lung disease
* Dependence on immunosuppressive drugs for a condition other than asthma
* Participant is pregnant
* Has a severe bleeding disorder
* Has significant developmental disability
* Share a bedroom with a currently enrolled Breath Warriors study participant

Primary outcomes:
measure: Change in Composite Asthma Severity Index (CASI)
timeFrame: Baseline to 12 months
Secondary outcomes:
measure: Asthma Control Test (ACT)
timeFrame: Baseline to 12 months
measure: Pulmonary Function Measured by Spirometry: Forced Expiratory Volume in 1 Second (FEV1) / Forced Vital Capacity (FVC)
timeFrame: Baseline to 12 months
measure: Adherence of Asthma Controller Medication
timeFrame: Baseline to 12 months
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Children's Hospital Medical Center, Cincinnati
name: Theresa Guilbert, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital Medical Center, Cincinnati
name: Gurjit K Khurana Hershey, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Guilbert TW, Biagini JM, Ramsey RR, Keidel K, Curtsinger K, Kroner JW, Durrani SR, Stevens M, Pilipenko V, Martin LJ, Kercsmar CM, Hommel K, Hershey GKK. Treatment by biomarker-informed endotype vs guideline care in children with difficult-to-treat asthma. Ann Allergy Asthma Immunol. 2022 May;128(5):535-543.e6. doi: 10.1016/j.anai.2022.01.030. Epub 2022 Feb 3.