Evaluation of the short-term effect of ORKAMBI on lung function in patients with cystic fibrosis who have two copies of the F508del mutation using functional respiratory imaging

Phase 1

• Conditions: Cystic Fibrosis Homozygous (homozygous for the F508del mutation); MedDRA version: 20.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2018-001573-24-BE
• Lead Sponsor: Antwerp University Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-05-08
• Last Update Posted: 6 April 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

1. Documented diagnosis of CF (homozygous for the F508del mutation must be present, this should be documented in the medical history).
2. Age = 12 years
3. FEV1 ³ 50%
4. Signed informed consent. If patient is a minor, parents/guardians must give written informed consent
5. Patient must be on a stable regimen of CF medication for 4 weeks prior to Visit 1
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 10
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. FEV1 < 50%
2. Anticipated requirement for hospitalization within the next three weeks
3. History of pneumothorax within the past 6 months prior to Visit 1
4. History of haemoptysis requiring embolization within the past 12 months prior to Visit 1
5. Unable or unwilling to complete study visits or provide follow-up data as required per the study protocol
6. Has taken Intravenous (IV) antibiotics within the past 4 weeks prior to Visit 1
7. Has ongoing exacerbation or Allergic bronchopulmonary aspergillosis (ABPA)
8. Pregnant or lactating female
9. Posttransplant patients
10. Patients with severe hepatic impairment

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of this study is to assess the efficacy of ORKAMBI on lung functionality across multiple FRI parameters.;Secondary Objective: Secondary outcome variables are Patient Reported Outcome, lung function tests, digital lung auscultation, exercise tolerance and exacerbation frequencies.;Primary end point(s): FRI parameters:<br>• Specific airway resistance (siRaw)<br>• Specific Airway volumes (siVaw);Timepoint(s) of evaluation of this end point: HRCT scans will be taken at baseline (visit 1) and after 3 months of treatment (Visit 4).