Cystic FibrosisPREDICT-CFNCT03894657
Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®.
A study testing Nasal brushing, Sputum sample and blood sample in people with Cystic Fibrosis and Homozygous F508del Mutation, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
The purpose of the study is to investigate whether the correction of CFTR function by Lumacaftor/Ivacaftor in a patient-derived primary nasal cell model is a surrogate biomarker for respiratory improvement in Orkambi® treated patients. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Cystic Fibrosis, Homozygous F508del Mutation
- What is being tested?
- Nasal brushing, Sputum sample and blood sample
- How many people
- 91 participants
- Where
- 1 siteFrance
- Study dates
- 18 Dec 2019to 12 May 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Homozygous F508del patient aged 5 years or older
- Patient with an indication for Orkambi® treatment according to the marketing authorization application
- Patient never received Orkambi® in the past
- Patient able to perform FEV1
- Signed Informed consent form by the patient (if aged ≥ 18 years), or by parents / legal guardian and patient's agreement (if aged < 18 years) Patient affiliated to the health insurance system
Who cannot join
- Homozygous F508del patients who do not meet the treatment indications according to the marketing authorization application
- Patients refusing Orkambi®
- CF patients not homozygous for the p.Phe508del mutation
- Active smoker
- Severe nasal mucosa disrepair
- Contraindications to xylocaine anesthesia,
- Participation with another interventional study with drug
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. Pathway to Personalized Therapy in Cystic Fibrosis
The purpose of the study is to investigate whether the correction of CFTR function by Lumacaftor/Ivacaftor in a patient-derived primary nasal cell model is a surrogate biomarker for respiratory improvement in Orkambi® treated patients.
Read the detailed description
Orkambi® is a combination of Ivacaftor (a CFTR channel potentiator) and Lumacaftor (a corrector partially rescuing the traffic of mutated CFTR). This treatment is now marketed in France for patients homozygotes for the mutation p.Phe508del, aged 12 and above. Systematic use of this product is a concern due to the lack of predictive markers of efficacy, the highly variable respiratory improvement in patients and potential serious side effects.
The purpose of this study is to investigate the predictive value for improvement of the respiratory function after 24 weeks of Orkambi treatment of an in vitro test. This test quantifies the correction of CFTR activity as assessed by the change of cyclic AMP (cAMP) dependant chloride (Cl-) secretion in patient derived Human Nasal Epithelial (HNE) derived primary culture after Lumacaftor/Ivacaftor 48 hours incubation.
- Conditions
- Cystic Fibrosis, Homozygous F508del Mutation
- Being tested
- Nasal brushing, Sputum sample, blood sample, Orkambi
- Keywords
- CYSTIC FIBROSIS, ORKAMBI
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nasal brushingDiagnostic test
Nasal scrapping at visit V0
Sputum sampleOther
Visit V0 : 1 Aliquot for Sputum biobank
Visit V1 : 2 Aliquots
- for Sputum biobank
- for pharmacokinetic (PK) study
Visit V2 : 2 Aliquots
- for Sputum biobank
- for pharmacokinetic (PK) study
blood sampleOther
Visit V0 : Additional 14 mL
- 5 mL in ethylenediaminetetraacetic acid (EDTA) tube for DNA Analysis
- 3 mL in Dry tube for Serum biobank
- 6 mL in acid citrate dextrose (ACD) tube for peripheral blood mononuclear cell (PBMC) biobank
Visit V1 : Additional 9 mL
- 2x3 mL in dry tube for pharmacokinetic (PK) study
- 3 mL in Dry tube for Serum biobank
Visit V2 : Additional 6 mL
- 3 mL in dry tube for pharmacokinetic (PK) study
- 3 mL in Dry tube for Serum biobank
OrkambiDrug
Study the predictive value for improvement of the respiratory function after 24 weeks of Orkambi treatment.
Orkambi treatment is part of usual care.
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of FEV1
Absolute change in the percentage of predicted forced expiratory volume in 1 second (%FEV1) from baseline to week 24 of Orkambi®
Measured: Baseline, Week 24
Secondary outcomes (14)
Z-score of FEV1
Absolute change in the Z-score of forced expiratory volume in 1 second (FEV1) from baseline to week 24 and to week 48
Measured: Baseline, Week 24, week 48
Percentage of FEV1
Absolute change in the percentage of predicted forced expiratory volume in 1 second (FEV1) from baseline through week 48
Measured: Week 48
% of FVC
Absolute change in percent predicted of forced vital capacity (%FVC) from baseline through week 24 and 48
Measured: Baseline, Week 24 and week 48
% of RFC
Absolute change in percent predicted of Functional Residual Capacity (%RFC) from baseline through week 24 and 48
Measured: Baseline, Week 24 and week 48
Lung clearance index
Absolute change in lung clearance index 2.5 (LCI2.5) from baseline through Week 48
Measured: Baseline, Week 48
Height
Absolute change in height-for-age-z-score from baseline to week 24 and 48
Measured: Baseline, Week 24 and week 48
Weight
Absolute change in weight-for-age-z-score from baseline to week 24 and 48
Measured: Baseline, Week 24 and week 48
colony forming unit (CFU)
Absolute change in colony forming unit (CFU) of sputum microorganisms from baseline to week 24 and 48
Measured: Baseline, Week 24 and week 48
Number of exacerbations
Number of exacerbations to week 48 in comparison to the year previous treatment with Orkambi®
Measured: Baseline, Week 48
Sweat Cl-
Absolute change in sweat Cl- from baseline to week 48
Measured: Baseline, Week 48
Level in Forskolin/IBMXdependant Short Circuit Current
Level in Forskolin/IBMXdependant Short Circuit Current change in patient nasal epithelial (HNE) cells incubated with Lumacaftor/Ivacaftor
Measured: Baseline
percentage of cells displaying apical staining
Correction of CFTR expression at the apical membrane in HNE cells incubated with Lumacaftor/Ivacaftor, assessed by the percentage of cells displaying apical staining.
Measured: baseline
Area under the curve (AUC) of Lumacaftor/Ivacaftor
Pharmacokinetic parameters of Lumacaftor, M28-lumacaftor, Ivacaftor, M1-ivacaftor, and M6-ivacaftor
Measured: Week 24, week 48
Drug concentrations of Lumacaftor/Ivacaftor
Pharmacokinetic parameters of Lumacaftor, M28-lumacaftor, Ivacaftor, M1-ivacaftor, and M6-ivacaftor
Measured: Week 24, week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Mar 2019
- Study start18 Dec 2019
- Primary completion25 Nov 2021
- Study completion12 May 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 91 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Responsible party
- Sponsor
- Investigators
- ISABELLE SERMET, PhD, Principal investigator, Hospital Necker Enfants Malades
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03894657
- Sponsor's protocol ID
- P170907J
- Other IDs
- 2018-002624-16, 2018
- Acronym
- PREDICT-CF
- Status last verified
- Mar 2026
- First submitted
- 12 Feb 2019
- Last updated
- 10 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Bouazza N, Urien S, Foissac F, Choupeaux L, Lui G, Froelicher Bournaud L, Rouillon S, Zheng Y, Bardin E, Stremler N, Bessaci K, Bihouee T, Coirier-Duet E, Marguet C, Deneuville E, Laurans M, Reix P, Gerardin M, Mittaine M, Epaud R, Thumerelle C, Weiss L, Berthaud R, Semeraro M, Treluyer JM, Benaboud S, Sermet-Gaudelus I. Lumacaftor/Ivacaftor Population Pharmacokinetics in Pediatric Patients with Cystic Fibrosis: A First Step Toward Personalized Therapy. Clin Pharmacokinet. 2024 Mar;63(3):333-342. doi: 10.1007/s40262-023-01342-3. Epub 2024 Feb 4.
PubMed 38310629
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. Pathway to Personalized Therapy in Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT03894657
- Acronym / sponsor ID
- PREDICT-CF / P170907J
- Conditions
- Cystic Fibrosis, Homozygous F508del Mutation
- Keywords
- CYSTIC FIBROSIS, ORKAMBI
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 91 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Study start
- 18 Dec 2019 (actual)
- Primary completion
- 25 Nov 2021 (actual)
- Study completion
- 12 May 2022 (actual)
- First posted
- 28 Mar 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to investigate whether the correction of CFTR function by Lumacaftor/Ivacaftor in a patient-derived primary nasal cell model is a surrogate biomarker for respiratory improvement in Orkambi® treated patients.
Detailed description
Orkambi® is a combination of Ivacaftor (a CFTR channel potentiator) and Lumacaftor (a corrector partially rescuing the traffic of mutated CFTR). This treatment is now marketed in France for patients homozygotes for the mutation p.Phe508del, aged 12 and above. Systematic use of this product is a concern due to the lack of predictive markers of efficacy, the highly variable respiratory improvement in patients and potential serious side effects.
The purpose of this study is to investigate the predictive value for improvement of the respiratory function after 24 weeks of Orkambi treatment of an in vitro test. This test quantifies the correction of CFTR activity as assessed by the change of cyclic AMP (cAMP) dependant chloride (Cl-) secretion in patient derived Human Nasal Epithelial (HNE) derived primary culture after Lumacaftor/Ivacaftor 48 hours incubation.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Homozygous F508del patient aged 5 years or older
- Patient with an indication for Orkambi® treatment according to the marketing authorization application
- Patient never received Orkambi® in the past
- Patient able to perform FEV1
- Signed Informed consent form by the patient (if aged ≥ 18 years), or by parents / legal guardian and patient's agreement (if aged < 18 years) Patient affiliated to the health insurance system
Key exclusion criteria (7 listed)
- Homozygous F508del patients who do not meet the treatment indications according to the marketing authorization application
- Patients refusing Orkambi®
- CF patients not homozygous for the p.Phe508del mutation
- Active smoker
- Severe nasal mucosa disrepair
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nasal brushingNasal scrapping at visit V0 | Diagnostic test | – |
| Sputum sampleVisit V0 : 1 Aliquot for Sputum biobank Visit V1 : 2 Aliquots * for Sputum biobank * for pharmacokinetic (PK) study Visit V2 : 2 Aliquots * for Sputum biobank * for pharmacokinetic (PK) study | Other | – |
| blood sampleVisit V0 : Additional 14 mL * 5 mL in ethylenediaminetetraacetic acid (EDTA) tube for DNA Analysis * 3 mL in Dry tube for Serum biobank * 6 mL in acid citrate dextrose (ACD) tube for peripheral blood mononuclear cell (PBMC) biobank Visit V1 : Additional 9 mL * 2x3 mL in dry tube for pharmacokinetic (PK) study * 3 mL in Dry tube for Serum biobank Visit V2 : Additional 6 mL * 3 mL in dry tube for pharmacokinetic (PK) study * 3 mL in Dry tube for Serum biobank | Other | – |
| OrkambiStudy the predictive value for improvement of the respiratory function after 24 weeks of Orkambi treatment. Orkambi treatment is part of usual care. | Drug | – |
Primary outcome
Percentage of FEV1
Time frame: Baseline, Week 24
Absolute change in the percentage of predicted forced expiratory volume in 1 second (%FEV1) from baseline to week 24 of Orkambi®
14 secondary outcomes
Z-score of FEV1
Time frame: Baseline, Week 24, week 48
Percentage of FEV1
Time frame: Week 48
% of FVC
Time frame: Baseline, Week 24 and week 48
% of RFC
Time frame: Baseline, Week 24 and week 48
Lung clearance index
Time frame: Baseline, Week 48
Height
Time frame: Baseline, Week 24 and week 48
Weight
Time frame: Baseline, Week 24 and week 48
colony forming unit (CFU)
Time frame: Baseline, Week 24 and week 48
Number of exacerbations
Time frame: Baseline, Week 48
Sweat Cl-
Time frame: Baseline, Week 48
Level in Forskolin/IBMXdependant Short Circuit Current
Time frame: Baseline
percentage of cells displaying apical staining
Time frame: baseline
Area under the curve (AUC) of Lumacaftor/Ivacaftor
Time frame: Week 24, week 48
Drug concentrations of Lumacaftor/Ivacaftor
Time frame: Week 24, week 48
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2026.
- Results publication: PubMed 38310629Bouazza N, Urien S, Foissac F, Choupeaux L, Lui G, Froelicher Bournaud L, Rouillon S, Zheng Y, Bardin E, Stremler N, Bessaci K, Bihouee T, Coirier-Duet E, Marguet C, Deneuville E, Laurans M, Reix P, Gerardin M, Mittaine M, Epaud R, Thumerelle C, Weiss L, Berthaud R, Semeraro M, Treluyer JM, Benaboud S, Sermet-Gaudelus I. Lumacaftor/Ivacaftor Population Pharmacokinetics in Pediatric Patients with Cystic Fibrosis: A First Step Toward Personalized Therapy. Clin Pharmacokinet. 2024 Mar;63(3):333-342. doi: 10.1007/s40262-023-01342-3. Epub 2024 Feb 4.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
ISABELLE SERMET, PhD principal investigator
Hospital Necker Enfants Malades
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Nasal brushing, Sputum sample and blood sample in people with Cystic Fibrosis and Homozygous F508del Mutation, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of FEV1 (time frame: Baseline, Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03894657, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Mar 2026.