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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Personalized Treatment | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cholecalciferol | Drug | Oral administration |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| 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] | Baseline to 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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] |
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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:
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:
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| Name | Affiliation | Role |
|---|---|---|
| Theresa Guilbert, MD | Children's Hospital Medical Center, Cincinnati | Principal Investigator |
| Gurjit K Khurana Hershey, MD, PhD | Children's Hospital Medical Center, Cincinnati | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35123074 | Derived | 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Personalized Treatment | 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. Cholecalciferol: Oral administration antihistamine: Oral administration Azithromycin: Oral administration emollient cream: Topical Fluticasone Propionate: Nasal spray Asthma Controller Medication: Study participants asthma controller medication may be increased based off of their asthma control and the recommendation of the personalized plan. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
1 participant withdrew from the study prior to being assigned a personalized treatment.
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| ID | Title | Description |
|---|---|---|
| BG000 | Personalized Treatment | 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. Cholecalciferol: Oral administration antihistamine: Oral administration Azithromycin: Oral administration emollient cream: Topical Fluticasone Propionate: Nasal spray Asthma Controller Medication: Study participants asthma controller medication may be increased based off of their asthma control and the recommendation of the personalized plan. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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] | 17 participants whom had complete data sets were analyzed. 4 participants withdrew early from the study and were not included in this analysis due to incomplete data. | Posted | Median | Inter-Quartile Range | score on a scale | Baseline to 12 months |
|
1 year
Review for possible adverse events was completed at study visits and follow-up phone calls by the research coordinator. If adverse events were identified, they were documented on the Adverse Event Reporting Form and reviewed at the meeting of the Steering committee.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Personalized Treatment | 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. Cholecalciferol: Oral administration antihistamine: Oral administration Azithromycin: Oral administration emollient cream: Topical Fluticasone Propionate: Nasal spray Asthma Controller Medication: Study participants asthma controller medication may be increased based off of their asthma control and the recommendation of the personalized plan. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Major Depressive Disorder | Psychiatric disorders | Systematic Assessment | Major depressive disorder requiring hospitalization |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Asthma Exacerbation | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Theresa Guilbert, MD, MS | Cincinnati Children's Hospital Medical Center | (513) 636-6771 | Theresa.Guilbert@cchmc.org |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Mar 19, 2019 | Apr 2, 2021 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D002762 | Cholecalciferol |
| D014807 | Vitamin D |
| D006633 | Histamine Antagonists |
| D017332 | Cetirizine |
| D017336 | Loratadine |
| D017963 | Azithromycin |
| D000068298 | Fluticasone |
| D000068656 | Mometasone Furoate |
| D000068759 | Formoterol Fumarate |
| D000068297 | Fluticasone-Salmeterol Drug Combination |
| D001507 | Beclomethasone |
| D000069502 | Budesonide, Formoterol Fumarate Drug Combination |
| D000069447 | Tiotropium Bromide |
| ID | Term |
|---|---|
| D002782 | Cholestenes |
| D002776 | Cholestanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
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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.
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| antihistamine | Drug | Oral administration |
|
|
| Azithromycin | Drug | Oral administration |
|
|
| emollient cream | Drug | Topical |
|
| Fluticasone Propionate | Drug | Nasal spray |
|
|
| Asthma Controller Medication | Drug | Study participants asthma controller medication may be increased based off of their asthma control and the recommendation of the personalized plan. |
|
|
| Baseline to 12 months |
| 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] | Baseline to 12 months |
| 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). | Baseline to 12 months |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| Primary home within a 40 mile radius of Cincinnati Children's Base Location | Count of Participants | Participants |
|
| Health insurance coverage at enrollment | Count of Participants | Participants |
|
|
|
|
| Secondary | 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] | 17 participants whom had complete data sets were analyzed. 4 participants withdrew early from the study and were not included in this analysis due to incomplete data. | Posted | Median | Inter-Quartile Range | score on a scale | Baseline to 12 months |
|
|
|
|
| Secondary | 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] | 17 participants whom had complete data sets were analyzed. 4 participants withdrew early from the study and were not included in this analysis due to incomplete data. | Posted | Median | Inter-Quartile Range | Ratio | Baseline to 12 months |
|
|
|
|
| Secondary | 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). | 16 participants whom had complete data sets were analyzed. | Posted | Median | Inter-Quartile Range | percentage of medication taken | Baseline to 12 months |
|
|
|
|
| 0 |
| 21 |
| 1 |
| 21 |
| 12 |
| 21 |
|
| Psychiatric Evaluation | Psychiatric disorders | Systematic Assessment | Psychiatric Evaluation requiring hospitalization |
|
| Ankle Injury | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Influenza | Infections and infestations | Systematic Assessment |
|
| Streptococcal Pharyngitis | Infections and infestations | Systematic Assessment |
|
| Viral Gastroenteritis | Infections and infestations | Systematic Assessment |
|
| Sinusitis | Infections and infestations | Systematic Assessment |
|
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| D011083 |
| Polycyclic Compounds |
| D013261 | Sterols |
| D012632 | Secosteroids |
| D008563 | Membrane Lipids |
| D008055 | Lipids |
| D018494 | Histamine Agents |
| D018377 | Neurotransmitter Agents |
| D045504 | Molecular Mechanisms of Pharmacological Action |
| D020228 | Pharmacologic Actions |
| D020164 | Chemical Actions and Uses |
| D045505 | Physiological Effects of Drugs |
| D006919 | Hydroxyzine |
| D010879 | Piperazines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D003533 | Cyproheptadine |
| D003986 | Dibenzocycloheptenes |
| D001567 | Benzocycloheptenes |
| D011084 | Polycyclic Aromatic Hydrocarbons |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D010880 | Piperidines |
| D004917 | Erythromycin |
| D018942 | Macrolides |
| D061065 | Polyketides |
| D007783 | Lactones |
| D000730 | Androstadienes |
| D000736 | Androstenes |
| D000731 | Androstanes |
| D011244 | Pregnadienediols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D004983 | Ethanolamines |
| D000605 | Amino Alcohols |
| D000438 | Alcohols |
| D000588 | Amines |
| D000068299 | Salmeterol Xinafoate |
| D000420 | Albuterol |
| D010627 | Phenethylamines |
| D005021 | Ethylamines |
| D004338 | Drug Combinations |
| D004364 | Pharmaceutical Preparations |
| D011246 | Pregnadienetriols |
| D013258 | Steroids, Chlorinated |
| D019819 | Budesonide |
| D011282 | Pregnenediones |
| D011283 | Pregnenes |
| D012602 | Scopolamine Derivatives |
| D014326 | Tropanes |
| D053961 | Azabicyclo Compounds |
| D001372 | Aza Compounds |
| D000470 | Alkaloids |
| D019086 | Bridged Bicyclo Compounds, Heterocyclic |
| D006572 | Heterocyclic Compounds, Bridged-Ring |
| Title | Measurements |
|---|---|
|
| Wilcoxon (Mann-Whitney) | 0.01 | The change in ACT score from V1-V2 to V2-V3. | Equivalence | The Wilcoxon signed-rank test was employed for pairwise comparison between visits for continuous data. Data for the c-ACT/ACT was captured at clinical visits and from monthly phone calls. Because c-ACT/ACT could vary through time, we calculated the average c-ACT/ACT between V1-V2 and V2-V3. Data analysis was performed in SAS version 9.4 (SAS, Cary, NC). A level of statistical significance was established at < 0.05. |
| Title | Measurements |
|---|---|
|