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Functional Respiratory Imaging and Orkambi in CF

Primary Purpose

Cystic Fibrosis

Status
Terminated
Phase
Phase 4
Locations
Belgium
Study Type
Interventional
Intervention
Orkambi
Sponsored by
University Hospital, Antwerp
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Antwerp University Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Orkambi open-label arm

Arm Description

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

Outcomes

Primary Outcome Measures

Change in specific image-based airway resistance (siRaw)
Change in CFD-based airway resistance normalized by the lung volume
Change in specific image-based airway volumes (siVaw)
change in CT-based airway volumes normalized by the lung volume

Secondary Outcome Measures

Internal Airflow Distribution
calculated using thorax HRCT
Air Trapping
calculated using thorax HRCT
Airway Wall Volume
calculated using thorax HRCT
Aerosol Deposition
calculated using thorax HRCT
Dynamic lung volumes
calculated using spirometry
Static lung volumes
calculated using body plethysmography
airway resistances
calculated using body plethysmography
Lung clearance index
Marker of lung ventilation inhomogeneity
6-minute walking test
Marker of fitness for daily activities
Sweat chloride test
Chloride values in sweat chloride test
CFQ-R
standardized measures of quality of life will be administered to subject and to parents of subjects under 18 years of age. Disease-specific instrument designed to measure impact on overall health, daily life, perceived well-being and symptoms. Developed specifically for use in patients with a diagnosis of cystic fibrosis. Scores range from 0 to 100, with higher scores indicating better health.
Digital lung auscultation
Digital analysis of digital lung sounds obtained by a digital stethoscope
Exacerbation frequency
Number of cystic fibrosis exacerbations

Full Information

First Posted
May 2, 2019
Last Updated
April 7, 2020
Sponsor
University Hospital, Antwerp
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1. Study Identification

Unique Protocol Identification Number
NCT03956589
Brief Title
Functional Respiratory Imaging and Orkambi in CF
Official Title
Functional Respiratory Imaging (FRI) to Assess the Short-term Effect of the Product ORKAMBI (Lumacaftor/ Ivacaftor) on Lung Function in ORKAMBInaive Patients With Cystic Fibrosis Homozygous for Phe508del
Study Type
Interventional

2. Study Status

Record Verification Date
April 2020
Overall Recruitment Status
Terminated
Why Stopped
Patient inclusion has stopped after interim analysis.
Study Start Date
October 1, 2018 (Actual)
Primary Completion Date
April 1, 2020 (Actual)
Study Completion Date
April 1, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University Hospital, Antwerp

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Orkambi open-label arm
Arm Type
Experimental
Arm Description
Open-label study: all subjects will receive Orkambi during 3 months.
Intervention Type
Drug
Intervention Name(s)
Orkambi
Intervention Description
Open label of Orkambi treatment during 3 months
Primary Outcome Measure Information:
Title
Change in specific image-based airway resistance (siRaw)
Description
Change in CFD-based airway resistance normalized by the lung volume
Time Frame
baseline and after three months of therapy
Title
Change in specific image-based airway volumes (siVaw)
Description
change in CT-based airway volumes normalized by the lung volume
Time Frame
baseline and after three months of therapy
Secondary Outcome Measure Information:
Title
Internal Airflow Distribution
Description
calculated using thorax HRCT
Time Frame
baseline and after three months of therapy
Title
Air Trapping
Description
calculated using thorax HRCT
Time Frame
baseline and after three months of therapy
Title
Airway Wall Volume
Description
calculated using thorax HRCT
Time Frame
baseline and after three months of therapy
Title
Aerosol Deposition
Description
calculated using thorax HRCT
Time Frame
baseline and after three months of therapy
Title
Dynamic lung volumes
Description
calculated using spirometry
Time Frame
baseline and after three months of therapy
Title
Static lung volumes
Description
calculated using body plethysmography
Time Frame
baseline and after three months of therapy
Title
airway resistances
Description
calculated using body plethysmography
Time Frame
baseline and after three months of therapy
Title
Lung clearance index
Description
Marker of lung ventilation inhomogeneity
Time Frame
baseline and after three months of therapy
Title
6-minute walking test
Description
Marker of fitness for daily activities
Time Frame
baseline and after three months of therapy
Title
Sweat chloride test
Description
Chloride values in sweat chloride test
Time Frame
baseline and after three months of therapy
Title
CFQ-R
Description
standardized measures of quality of life will be administered to subject and to parents of subjects under 18 years of age. Disease-specific instrument designed to measure impact on overall health, daily life, perceived well-being and symptoms. Developed specifically for use in patients with a diagnosis of cystic fibrosis. Scores range from 0 to 100, with higher scores indicating better health.
Time Frame
baseline and after three months of therapy
Title
Digital lung auscultation
Description
Digital analysis of digital lung sounds obtained by a digital stethoscope
Time Frame
baseline and after three months of therapy
Title
Exacerbation frequency
Description
Number of cystic fibrosis exacerbations
Time Frame
baseline and after three months of therapy

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Stijn Verhulst, MD, PhD
Organizational Affiliation
University Hospital, Antwerp
Official's Role
Principal Investigator
Facility Information:
Facility Name
Antwerp University Hospital
City
Edegem
ZIP/Postal Code
2650
Country
Belgium

12. IPD Sharing Statement

Plan to Share IPD
No

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Functional Respiratory Imaging and Orkambi in CF

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