Real-world Efficacy and Safety of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulator Therapy in Adult Patients With Cystic Fibrosis (CF)

NCT05526027 | Unknown

• 1 other identifier: BUN1432021000472
• Study Type: interventional
• Target: 85
• Locations: 0 countries
• Sites: N/A

Brief Summary

In this trial real-world data on the safety (side effects and medication interactions) and efficacy (evolution of lung function testing, chronic bacterial airway infection, quality of life and endo- and exocrine pancreatic function) will be collected in adult people with cystic fibrosis (pwCF) eligible for elexacaftor-tezacaftor-ivacaftor (ETI) up until 2 years after the start of this therapy.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (8)

• percent predicted forced expiratory volume in 1 second (ppFEV1)

  2 years

• Lung clearance index (LCI)

  2 years

• Fractional excretion of nitric oxide (FeNO)

  2 years

• CFQ-R questionnaire

  2 years

• fecal elastase (microgram per gram of feces)

  the concentration of elastase (a pancreatic enzyme) in feces before and 6 months after start of CFTR modulating treatment, as a surrogate measure for pancreatic function

  2 years

• the amount of participants experiencing a treatment-related adverse event

  2 years

• annual acute exacerbation rate

  2 years

• body mass index (BMI)

  2 years

Secondary Outcomes (5)

• SNOT-22 questionnaire

  2 years

• GAD-7 questionnaire

  2 years

• PHQ-9 questionnaire

  2 years

• aerobic culture on sputum or cough swab sample

  2 years

• dosage of pancreatic enzyme replacement therapy

  2 years

Study Arms (1)

ETI

OTHER

pwCF which are CFTR-modulator naive and pwCF previously treated with a CFTR-modulator (i.e. tezacaftor-ivacaftor or lumacaftor-ivacaftor) will undergo standard-of-care examinations as well as examinations in the context of this trial (i.e. CFQ-R, PHQ-9, GAD-7 and SNOT-22 questionnaires, fecal elastase measurement)

Diagnostic Test: CFQ-R questionnaire, SNOT-22 questionnaire, fecal elastase measurement

Interventions

CFQ-R questionnaire, SNOT-22 questionnaire, fecal elastase measurement DIAGNOSTIC_TEST

cf supra

ETI

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• eligible for ETI (i.e. age above 18 years and CFTR genotype F508del/any) based on reimbursement criteria in Belgium

You may not qualify if:

• inability to perform lung function testing

Sponsors & Collaborators

• Universitair Ziekenhuis Brussel: lead

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Central Study Contacts

Stefanie Vincken, M.D.

CONTACT

+32 2 477 68 41; stefanie.vincken@uzbrussel.be

Eef Vanderhelst, M.D. Ph.D.

CONTACT

+32 2 477 68 41; eef.vanderhelst@uzbrussel.be

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Medical doctor

Study Record Dates

• First Submitted: August 18, 2022
• First Posted: September 2, 2022
• Study Start: September 1, 2022
• Primary Completion: December 31, 2024
• Study Completion: December 31, 2024
• Last Updated: September 2, 2022

Record last verified: 2022-08

Data Sharing

• IPD Sharing: Will not share