Identifying markers to predict response to cystic fibrosis treatments
Personalized Therapy of Cystic Fibrosis: Set-up of Response Markers
This study is trying to find out if certain biological markers can help predict how well cystic fibrosis patients will respond to new CFTR medications.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 75 (estimated) |
| Sex | All |
| Sponsor | Hôpital Necker-Enfants Malades Academic / other |
| Locations | 1 site (Paris) |
| Trial ID | NCT02965326 on ClinicalTrials.gov |
What this trial studies
This study aims to identify biological markers that can predict how cystic fibrosis patients will respond to CFTR modulators. It hypothesizes that the clinical response to these drugs correlates with in vitro responses from epithelial cells derived from the patients. The study will utilize nasal and rectal epithelial cells, including cultured cells and organoids, to assess responses to drugs like Ivacaftor and Lumacaftor/Ivacaftor. The results will be compared to clinical responses observed at 6 and 12 months, focusing on CFTR function.
Who should consider this trial
Good fit: Ideal candidates include cystic fibrosis patients currently treated with CFTR modulators or those not treated, as well as individuals suspected of having cystic fibrosis but previously excluded by tests.
Not a fit: Patients who are pregnant or lactating, or those with contraindications to nasal swabs or rectal biopsies may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to more personalized treatment approaches for cystic fibrosis patients, improving their clinical outcomes.
How similar studies have performed: Other studies have explored similar approaches in identifying biomarkers for treatment response in cystic fibrosis, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Cystic fibrosis patients treated by CFTR modulators (Ivacaftor or the association Ivacaftor-Lumacaftor) * Cystic fibrosis patients non treated by CFTR modulators * Patients in whom cystic fibrosis diagnosis has been suspected, but excluded by physiological and genetic investigations Exclusion Criteria: * pregnant or lactating women * contraindication to nasal swab * contraindication to rectal biopsy
Where this trial is running
Paris
- Necker Hospital — Paris, France (Recruiting)
Study contacts
- Study coordinator: Isabelle Sermet, MD, PhD
- Email: isabelle.sermet@aphp.fr
- Phone: 33 1 44 49 48 87
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.