A

Phase 1

• Conditions: Homozygous F508del patient aged 12 years or older; MedDRA version: 20.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: EUCTR2018-002624-16-FR
• Lead Sponsor: ASSISTANC-PUBLIQUE-HOPITAUX DE PARIS (AP-HP)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-11-21
• Last Update Posted: 28 February 2019

Recruitment & Eligibility

• Status: A
• Sex: Not specified
• Target Recruitment: 104

Inclusion Criteria

- Homozygous F508del patient aged 12 years or older
- Patient with an indication for Orkambi® treatment according to the marketing authorization application
- Patient never received Orkambi® in the past
- 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

Are the trial subjects under 18? yes
Number of subjects for this age range: 52
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 50
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified