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

Cystic Fibrosis Clinical Trial

Official title:

Real-world Efficacy and Safety of CFTR Modulator Therapy in Adult Patients With Cystic Fibrosis

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05526027
• Other study ID #: BUN1432021000472
• Status: Not yet recruiting
• Phase: N/A
• Start date: September 1, 2022
• Est. completion date: December 31, 2024

Study information

• Verified date: August 2022
• Source: Universitair Ziekenhuis Brussel
• Contact: Stefanie Vincken, M.D.
• Phone: +32 2 477 68 41
• Email: stefanie.vincken[@]uzbrussel.be
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

PwCF group A: CFTR-modulator-naive pwCF eligible for ETI (based on age and CFTR genotype)

PwCF group B: pwCF already on CFTR modulating therapy (i.e. ivacaftor-lumacaftor or ivacaftor-tezacaftor) and switching to ETI.

Patients in both groups will undergo these examinations:

• Physical examination (including weight)

• Anamnesis for current and recent medication use (including dosage of pancreatic enzymes) and for acute respiratory exacerbations

• Lung function testing including spiometry, multiple breath washout testing and fractional exhaled nitric oxide

• Blood sampling: liver function tests, creatine kinase, albumin, PT, red and white blood cell count, platelet count

• Sputum/cough swab sampling

• fecal elastase measurement

• Cystic fibrosis questionnaire-revised (CFQ-R) questionnaire

• Patient health questionnaire-9 (PHQ-9) questionnaire

• General anxiety disorder-7 (GAD-7) questionnaire

• Sino-nasal outcome test-22 (SNOT-22) questionnaire

These will be performed at baseline (prior to the start of ETI, on the same day of start of ETI), and every 3 months (+/- 7 days) thereafter. A blood sample will also be performed 14 (+/- 7 days) days after start of ETI (for safety). Fecal elastase measurement will only be performed at baseline if not available in the patient's medical record, and only 6 months after start of ETI.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 85
• Est. completion date: December 31, 2024
• Est. primary completion date: December 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

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

Exclusion Criteria:

• inability to perform lung function testing

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Universitair Ziekenhuis Brussel

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	percent predicted forced expiratory volume in 1 second (ppFEV1)		2 years
Primary	Lung clearance index (LCI)		2 years
Primary	Fractional excretion of nitric oxide (FeNO)		2 years
Primary	CFQ-R questionnaire		2 years
Primary	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
Primary	the amount of participants experiencing a treatment-related adverse event		2 years
Primary	annual acute exacerbation rate		2 years
Primary	body mass index (BMI)		2 years
Secondary	SNOT-22 questionnaire		2 years
Secondary	GAD-7 questionnaire		2 years
Secondary	PHQ-9 questionnaire		2 years
Secondary	aerobic culture on sputum or cough swab sample		2 years
Secondary	dosage of pancreatic enzyme replacement therapy		2 years