Personalized Treatment Algorithms for Difficult-to-treat Asthma

NCT04179461 | Completed Phase 2 Results FDA Drug

• 1 other identifier: ARC 7
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

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.

Conditions

Asthma in Children

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (3)

• Asthma Control Test (ACT)

  Baseline to 12 months

• Pulmonary Function Measured by Spirometry: Forced Expiratory Volume in 1 Second (FEV1) / Forced Vital Capacity (FVC)

  Baseline to 12 months

• Adherence of Asthma Controller Medication

  Baseline to 12 months

Study Arms (1)

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.

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

Interventions

Cholecalciferol DRUG

Oral administration

Also known as: Vitamin D

Personalized Treatment

antihistamine DRUG

Oral administration

Also known as: cetirizine, loratadine

Personalized Treatment

Azithromycin DRUG

Oral administration

Also known as: Zithromax

Personalized Treatment

emollient cream DRUG

Topical

Personalized Treatment

Fluticasone Propionate DRUG

Nasal spray

Also known as: Flonase Nasal Spray

Personalized Treatment

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.

Also known as: Mometasone/Formoterol, Fluticasone/Salmeterol, Mometasone Furoate, Fluticasone, Beclomethasone, Budesonide/Formoterol, Tiotropium

Personalized Treatment

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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
• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Related Publications (1)

• 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.

  PMID: 35123074 DERIVED

MeSH Terms

Interventions

Cholecalciferol; Vitamin D; Histamine Antagonists; Cetirizine; Loratadine; Azithromycin; Fluticasone; Mometasone Furoate; Formoterol Fumarate; Fluticasone-Salmeterol Drug Combination; Beclomethasone; Budesonide, Formoterol Fumarate Drug Combination; Tiotropium Bromide

Intervention Hierarchy (Ancestors)

Cholestenes; Cholestanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Sterols; Secosteroids; Membrane Lipids; Lipids; Histamine Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Pharmacologic Actions; Chemical Actions and Uses; Physiological Effects of Drugs; Hydroxyzine; Piperazines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Cyproheptadine; Dibenzocycloheptenes; Benzocycloheptenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Piperidines; Erythromycin; Macrolides; Polyketides; Lactones; Androstadienes; Androstenes; Androstanes; Pregnadienediols; Pregnadienes; Pregnanes; Ethanolamines; Amino Alcohols; Alcohols; Amines; Salmeterol Xinafoate; Albuterol; Phenethylamines; Ethylamines; Drug Combinations; Pharmaceutical Preparations; Pregnadienetriols; Steroids, Chlorinated; Budesonide; Pregnenediones; Pregnenes; Scopolamine Derivatives; Tropanes; Azabicyclo Compounds; Aza Compounds; Alkaloids; Bridged Bicyclo Compounds, Heterocyclic; Heterocyclic Compounds, Bridged-Ring

Results Point of Contact

• Title: Theresa Guilbert, MD, MS
• Organization: Cincinnati Children's Hospital Medical Center

Theresa.Guilbert@cchmc.org

(513) 636-6771

Study Officials

• 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

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Model Details: 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.

Study Record Dates

• First Submitted: November 14, 2019
• First Posted: November 27, 2019
• Study Start: March 16, 2018
• Primary Completion: October 3, 2019
• Study Completion: October 3, 2019
• Last Updated: August 20, 2021
• Results First Posted: August 20, 2021

Record last verified: 2021-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)