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Completed

NCT Number: NCT04865575

Systems Pharmacology Approach to Uncontrolled Pediatric Asthma

Background:

Asthma is a heterogeneous respiratory disease and the most common chronic disease in children. A small subset of children has continuous poor asthma control despite appropriate adherence to asthma medication. There is a clinical need to identify these children as early as possible to optimize treatment and/or to find therapeutic alternatives. Therefore, the "Systems Pharmacology approach to uncontrolled Pediatric Asthma" (SysPharmPediA) study was set up.

Objective:

To establish a cohort of pediatric moderate-to-severe uncontrolled and controlled patients with asthma in order to investigate pathophysiological mechanisms underlying uncontrolled moderate-to-severe asthma in children on maintenance treatment, using a multi-omics systems medicine approach.

Methods:

In this multicenter observational case-control study, moderate-to-severe asthmatic children (n=145, age 6-17 years), were included in specialized hospitals in four European countries (Netherlands, Germany, Spain and Slovenia). Recruited subjects were selected based on good asthma control (controlled asthmatics, n=54) or poor asthma control / recurrent exacerbations (uncontrolled asthmatics, n=91). Comprehensive details concerning demographics, current and past patient/family history and clinical characteristics were collected. In addition, systems-wide omics layers, including epi(genomics), transcriptomics, microbiome, proteomics and metabolomics will be evaluated from multiple collected, relatively non-invasive, samples of from the recruited individuals, such as: blood, feces, saliva, nasal swabs and exhaled breath. Follow-up visits were performed 6 and 12 months after inclusion.

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Key information

Conditions

Pediatric Asthma Uncontrolled Asthma

Age range

6 year–18 year

Sex eligibility

All sexes

Study type

Observational

Primary location

University Regensburg, Regensburg, Germany

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

General:

  • Between 6-17 years of age
  • Doctor diagnosis of asthma

In addition to the general criteria, a case with uncontrolled asthma (case group A) should meet:

  • Asthma treatment Global INitiative of Asthma (GINA) step 3 or higher

≥ 1of the following criteria (based on the guideline 'Childhood Asthma' of the section paediatric pulmonology of the Dutch Society of Paediatricians, 2007):

  • Frequent exacerbations requiring OCS use (≥1 in the past year) and/or
  • Severe exacerbations requiring hospitalization or ER visits in the past year and/or
  • ACQ/ACT scores indicating uncontrolled asthma

In addition to the general criteria, a case with an acute exacerbation (case group B) should meet:

  • Asthma treatment Global INitiative of Asthma (GINA) step 2 or higher
  • Current severe asthma exacerbation requiring hospitalization

In addition to the general inclusion criteria, a control (controlled asthma) should meet the following criteria:

  • Asthma treatment Global INitiative of Asthma (GINA) step 3 or higher
  • Lack of severe exacerbations requiring OCS use or hospitalizations or ER visits in the past year
  • ACQ/ACT scores indicating well controlled asthma or few reported asthma symptoms during asthma checks in the past year.

Exclusion criteria

  • Recent use of a course of antibiotics (< 1 month). This will affect the microbiome analyses.

A patient can be re-screened 1 month after the use of the antibiotic treatment has finished.

Treatment and study plan

Primary outcomes

  1. Saliva and stool microbiome

    Time frame: Baseline

    Differences in 16S rRNA microbial α- and ß- diversities between cases and controls

  2. Exhaled breath metabolome

    Time frame: Baseline

    Differences in presence and levels of volatile organic compounds between cases and controls

Other outcomes

  1. Age

    Time frame: Baseline (+ follow-up at 6 and 12 months after baseline inclusion)

    Age in years

  2. Sex

    Time frame: Baseline (+ follow-up at 6 and 12 months after baseline inclusion)

    Female/Male

  3. BMI

    Time frame: Baseline (+ follow-up at 6 and 12 months after baseline inclusion)

    kg/m2

  4. Uncontrolled asthma

    Time frame: Baseline (+ follow-up at 6 and 12 months after baseline inclusion)

    according to the Asthma Control Test (ACT) questionnaire (<19 score) and frequent exacerbation within the past 12 months (>n of exacerbation in the past 12 months

  5. Exacerbations

    Time frame: Baseline (+ follow-up at 6 and 12 months after baseline inclusion)

    Exacerbation requiring OCS use, hospitalization or ER visit

  6. Spirometry

    Time frame: Baseline

    FEV1 (L, percent predicted value)

  7. Pediatric Asthma Quality of Life Questionnaire (PAQLQ)

    Time frame: Baseline (+ follow-up at 6 and 12 months after baseline inclusion)

    Questionnaire including 23 questions in 3 domains (symptoms, activity limitation, emotional function) and a 7-point scale (7 = not bothered at all; 1 = extremely bothered)

  8. Atopy

    Time frame: Baseline (+ follow-up at 6 and 12 months after baseline inclusion)

    Questionnaire on history of allergic diseases

  9. Current asthma medication intake

    Time frame: Baseline (+ follow-up at 6 and 12 months after baseline inclusion)

    ICS: inhaled corticosteroids, LTRA: leukotriene antagonist, SABA: short-acting beta agonist, LABA: long-acting beta agonist, OCS: oral corticosteroids, Omalizumab, Mepolizumab

  10. Asthma severity

    Time frame: Baseline (+ follow-up at 6 and 12 months after baseline inclusion)

    Asthma Control Test (ACT)

Sponsors and collaborators

Lead sponsor

Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

Other

Collaborators

  • Hospital Universitario Donostia, San Sebastián, Spain.
  • Instituto de Salud Carlos III
  • Karolinska Institutet
  • Universidad de La Laguna, San Cristóbal de La Laguna, Santa Cruz de Tenerife, Spain.
  • University of Maribor, Maribor, Slovenia.
  • University of Regensburg
  • University of the Basque Country (UPV/EHU)
  • Utrecht University, Utrecht, the Netherlands.

Registry information

Official study title

Systems Pharmacology Approach to Uncontrolled Pediatric Asthma (SysPharmPediA)

Important dates

Study start
2018
Primary completion
2019
Study completion
2020
First posted
Apr 29, 2021
Registry last updated
Apr 29, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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