Functional Respiratory Imaging and Orkambi in CF

NCT ID: NCT03956589

Last Updated: 2020-04-08

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE4
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-10-01
• Study Completion Date: 2020-04-01

Brief Summary

Open-label study to investigate the effects of Orkambi in CF patients homozygous for the F508del mutation by functional respiratory imaging. Primary endpoints in this study are the changes in Specific airway volumes (siVaw) and Specific Airway resistance (siRaw). A total of 20 ORKAMBI-naive patients with Cystic Fibrosis, homozygous for the F508del mutation will be included in this open label study and will be followed through 3 months of treatment. The treatment will be started after all assessments are performed at visit 1. After the start of the treatment some baseline measurements will be repeated throughout the 3-month treatment period. The patient will be asked to visit the hospital monthly. All study visits should be scheduled around the same time.

Conditions

Cystic Fibrosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Orkambi open-label arm

Open-label study: all subjects will receive Orkambi during 3 months.

Group Type: EXPERIMENTAL

Orkambi

Intervention Type: DRUG

Open label of Orkambi treatment during 3 months

Interventions

Orkambi

Open label of Orkambi treatment during 3 months

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Documented diagnosis of CF (homozygous for the F508del mutation must be present, this should be documented in the medical history).
• Age ≥ 12 years
• FEV1 > 50%
• Signed informed consent. If patient is a minor, parents/guardians must give written informed consent
• Patient must be on a stable regimen of CF medication for 4 weeks prior to Visit

Exclusion Criteria

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

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University Hospital, Antwerp

OTHER

Sponsor Role: lead

Responsible Party

Stijn Verhulst

Director Department of Pediatrics

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Stijn Verhulst, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

University Hospital, Antwerp

Locations

Antwerp University Hospital

Edegem, , Belgium

Countries

Belgium

Other Identifiers

2018-001573-24

Identifier Type: -

Identifier Source: org_study_id