Surrogate Markers of Response to New Therapies in Cystic Fibrosis Patients
ConditionCystic Fibrosis
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
AgeNot listed
SponsorHôpital Necker-Enfants Malades
About this trial
The purpose of this study is to determine which biological marker, or association of biological markers, best predict clinical response of cystic fibrosis patients to CFTR modulators.
Eligibility criteria
Qualifiers
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
Disqualifiers
pregnant or lactating women
contraindication to nasal swab
contraindication to rectal biopsy
Trial design
Treatments tested in this trial
- Nasal swab; rectal biopsy.
Treatment groups
75 Participants
are divided into 3 treatment groupsSponsors and collaborators
Hôpital Necker-Enfants Malades
Lead sponsor
Association Mucoviscidose-ABCF2
Collaborator
Vaincre la Mucoviscidose
Collaborator
Source ClinicalTrials.gov