Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®.

• Register: ClinicalTrials.gov
• Last refreshed on: 30 May 2022
• Main ID: NCT03894657
• Date of registration: 12/02/2019
• Prospective Registration: Yes
• Primary sponsor: Assistance Publique - Hôpitaux de Paris
• Public title: Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. PREDICT-CF
• Scientific title: Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. Pathway to Personalized Therapy in Cystic Fibrosis
• Date of first enrolment: December 18, 2019
• Target sample size: 91
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03894657
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Other. Masking: None (Open Label).
• Phase: N/A

Countries of recruitment

France

Contacts

• Name: ISABELLE SERMET, PhD
• Affiliation: Hospital Necker Enfants Malades

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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

Exclusion Criteria:

- 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



Age minimum: 5 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Homozygous F508del Mutation

Cystic Fibrosis

Intervention(s)

Drug: Orkambi

Other: Sputum sample

Diagnostic Test: Nasal brushing

Other: blood sample

Primary Outcome(s)

Percentage of FEV1 [Time Frame: Baseline, Week 24]

Secondary Outcome(s)

colony forming unit (CFU) [Time Frame: Baseline, Week 24 and week 48]

% of RFC [Time Frame: Baseline, Week 24 and week 48]

% of FVC [Time Frame: Baseline, Week 24 and week 48]

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]

Percentage of FEV1 [Time Frame: Week 48]

Height [Time Frame: Baseline, Week 24 and week 48]

Level in Forskolin/IBMXdependant Short Circuit Current [Time Frame: Baseline]

Weight [Time Frame: Baseline, Week 24 and week 48]

Number of exacerbations [Time Frame: Baseline, Week 48]

Z-score of FEV1 [Time Frame: Baseline, Week 24, week 48]

Lung clearance index [Time Frame: Baseline, Week 48]

percentage of cells displaying apical staining [Time Frame: baseline]

Sweat Cl- [Time Frame: Baseline, Week 48]

Secondary ID(s)

2018-002624-16

P170907J

Source(s) of Monetary Support

Please refer to primary and secondary sponsors