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
This trial does not accept healthy volunteersQualifiers
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
Design model
Parallel
Treatments tested in this trial
Nasal swab; rectal biopsy.
Procedure/SurgeryNasal epithelial cells will be obtained by nasal swabs from patients of the three arms; intestinal epithelial cells will be obtained, by rectal biopsy, only from patients treated by CFTR modulators.
Treatment groups
75 Participants
are divided into 3 treatment groupsGroup A: Cystic fibrosis, treatedOther 1 intervention
Group B: Cystic fibrosis, non treatedOther 1 intervention
Group C: Non-Cystic fibrosisOther 1 intervention
Trial outcomes
Primary outcomes
1
Forced Expiratory Volume in 1 second
Respiratory Function test
Time frame
initiation, 7 days, 1 month, 6 months, 1 year and every 6 monthsSecondary outcomes
1
Forced Vital Capacity
Respiratory Function test
Time frame
initiation, 7 days, 1 month, 6 months, 1 year and every 6 months2
Forced Expiratory Flow 25-75
Respiratory Function test
Time frame
initiation, 7 days, 1 month, 6 months, 1 year and every 6 months3
Residual Volume
Respiratory Function test
Time frame
initiation, 7 days, 1 month, 6 months, 1 year and every 6 months4
elastase in sputum
elastase activity in UI/g of sputum
Time frame
initiation, 7 days, 1 month, 6 months, 1 year and every 6 monthsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Hôpital Necker-Enfants Malades
Lead sponsor
Association Mucoviscidose-ABCF2
Collaborator
Vaincre la Mucoviscidose
Collaborator
Source ClinicalTrials.gov