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Personalized Treatment Algorithms for Difficult-to-treat Asthma

Primary Purpose

Asthma in Children

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Cholecalciferol
antihistamine
Azithromycin
emollient cream
Fluticasone Propionate
Asthma Controller Medication
Sponsored by
Children's Hospital Medical Center, Cincinnati
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Asthma in Children

Eligibility Criteria

6 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Cincinnati Children's Hospital Medical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Personalized Treatment

Arm Description

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.

Outcomes

Primary Outcome Measures

Change in Composite Asthma Severity Index (CASI)
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

Asthma Control Test (ACT)
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]
Pulmonary Function Measured by Spirometry: Forced Expiratory Volume in 1 Second (FEV1) / Forced Vital Capacity (FVC)
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]
Adherence of Asthma Controller Medication
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).

Full Information

First Posted
November 14, 2019
Last Updated
July 28, 2021
Sponsor
Children's Hospital Medical Center, Cincinnati
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1. Study Identification

Unique Protocol Identification Number
NCT04179461
Brief Title
Personalized Treatment Algorithms for Difficult-to-treat Asthma
Official Title
Personalized Treatment Algorithms for Difficult-to-treat Asthma: Bench to Community
Study Type
Interventional

2. Study Status

Record Verification Date
July 2021
Overall Recruitment Status
Completed
Study Start Date
March 16, 2018 (Actual)
Primary Completion Date
October 3, 2019 (Actual)
Study Completion Date
October 3, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Children's Hospital Medical Center, Cincinnati

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Asthma in Children

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
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.
Masking
None (Open Label)
Allocation
N/A
Enrollment
21 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Personalized Treatment
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Cholecalciferol
Other Intervention Name(s)
Vitamin D
Intervention Description
Oral administration
Intervention Type
Drug
Intervention Name(s)
antihistamine
Other Intervention Name(s)
cetirizine, loratadine
Intervention Description
Oral administration
Intervention Type
Drug
Intervention Name(s)
Azithromycin
Other Intervention Name(s)
Zithromax
Intervention Description
Oral administration
Intervention Type
Drug
Intervention Name(s)
emollient cream
Intervention Description
Topical
Intervention Type
Drug
Intervention Name(s)
Fluticasone Propionate
Other Intervention Name(s)
Flonase Nasal Spray
Intervention Description
Nasal spray
Intervention Type
Drug
Intervention Name(s)
Asthma Controller Medication
Other Intervention Name(s)
Mometasone/Formoterol, Fluticasone/Salmeterol, Mometasone Furoate, Fluticasone, Beclomethasone, Budesonide/Formoterol, Tiotropium
Intervention Description
Study participants asthma controller medication may be increased based off of their asthma control and the recommendation of the personalized plan.
Primary Outcome Measure Information:
Title
Change in Composite Asthma Severity Index (CASI)
Description
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]
Time Frame
Baseline to 12 months
Secondary Outcome Measure Information:
Title
Asthma Control Test (ACT)
Description
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]
Time Frame
Baseline to 12 months
Title
Pulmonary Function Measured by Spirometry: Forced Expiratory Volume in 1 Second (FEV1) / Forced Vital Capacity (FVC)
Description
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]
Time Frame
Baseline to 12 months
Title
Adherence of Asthma Controller Medication
Description
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).
Time Frame
Baseline to 12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Theresa Guilbert, MD
Organizational Affiliation
Children's Hospital Medical Center, Cincinnati
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Gurjit K Khurana Hershey, MD, PhD
Organizational Affiliation
Children's Hospital Medical Center, Cincinnati
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
35123074
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.
Results Reference
derived

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Personalized Treatment Algorithms for Difficult-to-treat Asthma

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