Assessing CFTR Modulator Response in Children with Cystic Fibrosis
Evaluation of the Response to CFTR Modulators in Patients With Cystic Fibrosis Less Than 18 Years of Age
This study is testing how well CFTR modulators work and their safety in children with cystic fibrosis by looking at their lung health and overall well-being over several years.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 600 (estimated) |
| Ages | N/A to 18 Years |
| Sex | All |
| Sponsor | Societe Francaise de la Mucoviscidose Academic / other |
| Locations | 1 site (Paris) |
| Trial ID | NCT04301856 on ClinicalTrials.gov |
What this trial studies
This observational cohort study aims to evaluate the effectiveness and safety of CFTR modulators in children under 18 years with cystic fibrosis. It focuses on monitoring pulmonary structural impairment using low-dose CT scans at the end of the first year of treatment, as well as at 3 and 5 years. Secondary objectives include assessing respiratory function, growth, quality of life, and potential side effects of the treatment. The study is conducted as part of routine care, emphasizing the need for sensitive outcome measures in this pediatric population.
Who should consider this trial
Good fit: Ideal candidates are children under 18 years old diagnosed with cystic fibrosis who are currently undergoing CFTR modulator therapy.
Not a fit: Patients over the age of 18 or those without an indication for CFTR modulator therapy will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved management and outcomes for children with cystic fibrosis receiving CFTR modulators.
How similar studies have performed: While CFTR modulators have been studied in adults, this specific focus on pediatric patients and the use of low-dose CT scans for monitoring is a novel approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: Children with cystic fibrosis under the age of 18 under CFTR modulator therapy Exclusion Criteria: * Patients with cystic fibrosis without indication for CFTR modulator therapy * Patients over the age of 18 * Pregnant or lactating women
Where this trial is running
Paris
- Sermet-Gaudelus Isabelle — Paris, France (Recruiting)
Study contacts
- Principal investigator: Isabelle Sermet-Gaudelus, MD PhD — Société Francaise de la Mucoviscidose
- Study coordinator: Stéphane Mazur, PhD
- Email: stephane.mazur@chu-lyon.fr
- Phone: 0033427855043
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.