Modulate-CF: Cystic Fibrosis Transmembrane Regulator (CFTR) Biomarker Study to Evaluate the Rescue of Mutant CFTR in Patients With Cystic Fibrosis Treated With CFTR-modulators

NCT04732910 · tracked via the Priya Life Science Germany tracker

Sponsor

Charite University, Berlin, Germany

Phase

Observational

Started

2018-07-01

Last updated

2026-02-24

Condition(s) studied

Cystic Fibrosis

Investigational drug(s) / intervention(s)

Treatment with cystic fibrosis transmembrane regulator (CFTR) modualtors Ivacaftor, Lumacaftor-Ivacaftor, Tezacaftor-Ivacaftor, Elexacaftor-Tezacaftor-Ivacaftor, Vanzacaftor-Tezacaftor-Deutivacaftor

Treatment with cystic fibrosis transmembrane regulator (CFTR) modualtors Ivacaftor, Lumacaftor-Ivacaftor, Tezacaftor-Ivacaftor, Elexacaftor-Tezacaftor-Ivacaftor, Vanzacaftor-Tezacaftor-Deutivacaftor: observational

Study summary

This observational study evaluates the effect of therapy with cystic fibrosis transmembrane regulator (CFTR) modulators on CFTR function measured by the CFTR biomarker intestinal current measurement (ICM), nasal potential difference (NPD) and sweat chloride in a post-approval setting in patients with cystic fibrosis (CF).

Eligibility

Sex

ALL

Min age

6 Months

Max age

—

Healthy volunteers

Inclusion Criteria: * Decision for cystic fibrosis (CF) transmembrane regulator (CFTR)-modulator therapy by the patient and the caring CF physician * Signed informed consent form (ICF) and, where appropriate, signed assent form. Exclusion Criteria: * Ongoing participation in an investigational drug study (including studies investigating lumacaftor, tezacaftor or ivacaftor)

Primary outcome measure(s)

Intestinal current measurement (ICM) — 12 weeks
Absolute change from baseline of the chloride secretory ion current induced by cyclic adenosine monophosphate (cAMP) stimulation (forskolin/3-isobutyl-1-methylxanthine (IBMX)) in rectal tissue determined by intestinal current measurement (ICM) as a cystic fibrosis transmembrane conductance regulator (CFTR) biomarker

Trial sites (4)

Facility	City	Region	Status
Charité - Universitätsmedizin Berlin	Berlin	State of Berlin	Recruiting
Justus-Liebig-University Giessen	Giessen	Germany	Recruiting
Hannover Medical School	Hanover	Germany	Recruiting
University of Heidelberg	Heidelberg	Germany	Recruiting

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Other trials for the same condition

A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy Active, not recruiting

Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del Active, not recruiting

Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older Enrolling by invitation

Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age Active, not recruiting

Development and Validation of a Disease Specific PROM to Assess Abdominal Involvement in Patients With CF (CFAbd-Score) Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04732910 on ClinicalTrials.gov ↗ ← All trials in Germany