Study of Urinary Predictors of Exacerbations by Biomarkers in Cystic Fibrosis (SUPERB-CF)

Study of Urinary Predictors of Exacerbations by Biomarkers in Cystic Fibrosis (SUPERB-CF)

Study of Urinary Predictors of Exacerbations by Biomarkers in Cystic Fibrosis

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis

Detailed Description

People with cystic fibrosis (CF) are prone to chest infections (pulmonary exacerbations) and suffer premature death due to respiratory failure. Patients that experience more frequent pulmonary exacerbations have worse prognosis and early antibiotic treatment of pulmonary exacerbations is therefore one of the major goals of CF care. Antibiotic treatment is often currently delayed, since we rely on patients contacting the CF team when they develop worsening symptoms. We hypothesise that if we could allow patients to detect and receive treatment for early pulmonary exacerbations by measuring urinary biomarkers, this would minimise lung damage and result in improved clinical outcomes.

In phase 1, 40 patients will be asked to collect daily urine samples, in addition to recording daily spirometry and a daily symptom score for 4 months. Phase 1 aims to identify the urinary biomarkers that are associated with CF pulmonary exacerbations. In phase 2, the same 40 patients will be asked to collect and test a daily urine sample using a novel testing device in addition to recording daily spirometry and a daily symptom score for 4 months. Phase 2 aims to validate the use of the urine testing device as a method of diagnosing early pulmonary exacerbations.

In summary, this study aims to develop and validate a novel noninvasive point of care (near-patient) diagnostic testing system, to allow people with CF to diagnose early pulmonary exacerbations by measuring urinary biomarkers. If successful, we hope that this will provide patients with an easy to use device, which will empower patients and their caregivers to treat exacerbations at an earlier stage, with potential health and economic benefits.

• Study Type: Observational
• Enrollment (Actual): 29

Contacts and Locations

Study Locations

• United Kingdom
  • Birmingham, United Kingdom, B9 5SS
    • Birmingham Heartlands Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: N/A
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients with CF

Description

Inclusion Criteria:

• Confirmed CF diagnosis
• Aged >18 years of age
• Patients able and willing to give informed consent
• Requirement for at least one course of intravenous antibiotics for a pulmonary exacerbation within the previous 3 months.

Exclusion Criteria:

• Patients not able or not willing to give informed consent
• Patients who are currently participating in another clinical trial (excluding observational studies)
• Pneumothorax or lung surgery within the previous 3 months, eye surgery (e.g. cataract operation) in the previous 4 weeks, or any other factor that prevents safe measurement of spirometry.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Measurement of biomarkers in the urine of CF patients to assess their potential for predicting exacerbations	Signalling molecules/acute phase proteins e.g. Interleukin 6 (IL-6), Interleukin 8 (IL-8), N-Formyl methionine Leucyl phenylalanine (fMLP), Fibrinogen, C-reactive protein (CRP). Proteases & proteolytic activity, e.g. Matrix metalloproteases (MMP's), Myeloperoxidase (MPO), Human neutrophil elastase (HNE), Cathepsins. Protease inhibitors, e.g. Tissue inhibitor of Metalloproteinase (TIMP), Alpha-1 anti-Trypsin (A1AT), Cystatin C, Secretory leukocyte peptidase inhibitor (SLPI). Degradation products e.g. Desmosine and Elastin Fragments, Acetyl PGP (Ac-PGP). Metabolites and other urinary markers, e.g. Creatinine, Human Serum Albumin (HSA), Retinol Binding Protein 4 (RBP4), Beta 2 Microglobulin (B2M). Other markers of interest, e.g. Siglec 8, Chitinase 3 like 1 protein, Club cell protein 16 (CC16). Note. Concentrations of all these biomarkers are expressed in microgramme/ml	4 months

Collaborators and Investigators

• Sponsor: Mologic Ltd
• Collaborators: Heart of England NHS Foundation Trust
• Investigators: Principal Investigator: Edward F Nash, BSc, Heart of England NHS Foundation Trust

Study record dates

Study Major Dates

• Study Start (Actual): February 1, 2018
• Primary Completion (Actual): July 31, 2019
• Study Completion (Actual): July 31, 2019

Study Registration Dates

• First Submitted: March 3, 2020
• First Submitted That Met QC Criteria: March 4, 2020
• First Posted (Actual): March 5, 2020

Study Record Updates

• Last Update Posted (Actual): March 11, 2020
• Last Update Submitted That Met QC Criteria: March 9, 2020
• Last Verified: March 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: SUPERB-CF

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No