Personalized Treatment Algorithms for Difficult-to-treat Asthma

Phase 2

Completed

Conditions: Asthma in Children

Interventions: Drug: Cholecalciferol
Drug: antihistamine
Drug: Azithromycin
Drug: emollient cream
Drug: Fluticasone Propionate
Drug: Asthma Controller Medication

Registration Number: NCT04179461

Lead Sponsor: Children's Hospital Medical Center, Cincinnati

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.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 21

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

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Personalized Treatment	antihistamine	Personalized asthma treatment plan based off of individual's asthma severity/control, personal and family medical history, history of environmental exposures, adherence, medical visits, biomarker assays, and home trigger assessment. Study participants were prescribed recommended medications for the treatment of their asthma. These medications were prescribed through their insurance based of the of personalized treatment plan recommendation. Asthma controller medications may be increased based off of the participant's asthma control and the recommendation of the personalized plan. They would receive one of the asthma controller medications listed in the intervention.
Personalized Treatment	emollient cream	Personalized asthma treatment plan based off of individual's asthma severity/control, personal and family medical history, history of environmental exposures, adherence, medical visits, biomarker assays, and home trigger assessment. Study participants were prescribed recommended medications for the treatment of their asthma. These medications were prescribed through their insurance based of the of personalized treatment plan recommendation. Asthma controller medications may be increased based off of the participant's asthma control and the recommendation of the personalized plan. They would receive one of the asthma controller medications listed in the intervention.
Personalized Treatment	Asthma Controller Medication	Personalized asthma treatment plan based off of individual's asthma severity/control, personal and family medical history, history of environmental exposures, adherence, medical visits, biomarker assays, and home trigger assessment. Study participants were prescribed recommended medications for the treatment of their asthma. These medications were prescribed through their insurance based of the of personalized treatment plan recommendation. Asthma controller medications may be increased based off of the participant's asthma control and the recommendation of the personalized plan. They would receive one of the asthma controller medications listed in the intervention.
Personalized Treatment	Azithromycin	Personalized asthma treatment plan based off of individual's asthma severity/control, personal and family medical history, history of environmental exposures, adherence, medical visits, biomarker assays, and home trigger assessment. Study participants were prescribed recommended medications for the treatment of their asthma. These medications were prescribed through their insurance based of the of personalized treatment plan recommendation. Asthma controller medications may be increased based off of the participant's asthma control and the recommendation of the personalized plan. They would receive one of the asthma controller medications listed in the intervention.
Personalized Treatment	Cholecalciferol	Personalized asthma treatment plan based off of individual's asthma severity/control, personal and family medical history, history of environmental exposures, adherence, medical visits, biomarker assays, and home trigger assessment. Study participants were prescribed recommended medications for the treatment of their asthma. These medications were prescribed through their insurance based of the of personalized treatment plan recommendation. Asthma controller medications may be increased based off of the participant's asthma control and the recommendation of the personalized plan. They would receive one of the asthma controller medications listed in the intervention.
Personalized Treatment	Fluticasone Propionate	Personalized asthma treatment plan based off of individual's asthma severity/control, personal and family medical history, history of environmental exposures, adherence, medical visits, biomarker assays, and home trigger assessment. Study participants were prescribed recommended medications for the treatment of their asthma. These medications were prescribed through their insurance based of the of personalized treatment plan recommendation. Asthma controller medications may be increased based off of the participant's asthma control and the recommendation of the personalized plan. They would receive one of the asthma controller medications listed in the intervention.

Primary Outcome Measures

Name	Time	Method
Change in Composite Asthma Severity Index (CASI)	Baseline to 12 months	CASI was measured by questionnaire and is a severity score of symptom burden, exacerbations, healthcare utilization, lung function and dose of inhaled corticosteroids. The change in CASI score was calculated between V1 (Baseline), V2 (Guideline Care - before intervention), and V3 (12 Months). \[The CASI score has a minimum value = 0, maximum value = 20, a higher score indicates greater asthma severity\]

Secondary Outcome Measures

Name	Time	Method
Asthma Control Test (ACT)	Baseline to 12 months	ACT was measured by questionnaire, assessing frequency of reported asthma symptoms, rescue medication use, the effect of asthma on daily functioning, and overall asthma control. The change in ACT score was calculated between V1 (Baseline), V2 (Guideline Care - before intervention), and V3 (12 Months). \[The ACT score has a minimum value = 5, maximum value = 25, a score 19 indicates well-controlled asthma\]
Adherence of Asthma Controller Medication	Baseline to 12 months	Adherence was measured using the Propeller Health Inhaler monitor and web-based software management platform that tracks adherence of asthma medications. The change in adherence was calculated between V1 (baseline) to V3 (12 Months).
Pulmonary Function Measured by Spirometry: Forced Expiratory Volume in 1 Second (FEV1) / Forced Vital Capacity (FVC)	Baseline to 12 months	FEV1 is air volume exhaled in 1 second during spirometry. Forced vital capacity is the amount of air that can be forcibly exhaled from the lungs after taking the deepest breath possible. The change in FEV1/FVC was calculated between V1 (Baseline), V2 (Guideline Care - before intervention), and V3 (12 Months). This will be used as a measurement in asthma severity. \[A lower FEV1/FVC ratio indicates more severe asthma\]