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Functional Respiratory Imaging and Orkambi in CF

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ClinicalTrials.gov Identifier: NCT03956589
Recruitment Status : Recruiting
First Posted : May 20, 2019
Last Update Posted : May 21, 2019
Sponsor:
Information provided by (Responsible Party):
Stijn Verhulst, University Hospital, Antwerp

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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: Orkambi Phase 4

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : October 1, 2018
Estimated Primary Completion Date : December 31, 2019
Estimated Study Completion Date : December 31, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: Orkambi open-label arm
Open-label study: all subjects will receive Orkambi during 3 months.
Drug: Orkambi
Open label of Orkambi treatment during 3 months




Primary Outcome Measures :
  1. Change in specific image-based airway resistance (siRaw) [ Time Frame: baseline and after three months of therapy ]
    Change in CFD-based airway resistance normalized by the lung volume

  2. Change in specific image-based airway volumes (siVaw) [ Time Frame: baseline and after three months of therapy ]
    change in CT-based airway volumes normalized by the lung volume


Secondary Outcome Measures :
  1. Internal Airflow Distribution [ Time Frame: baseline and after three months of therapy ]
    calculated using thorax HRCT

  2. Air Trapping [ Time Frame: baseline and after three months of therapy ]
    calculated using thorax HRCT

  3. Airway Wall Volume [ Time Frame: baseline and after three months of therapy ]
    calculated using thorax HRCT

  4. Aerosol Deposition [ Time Frame: baseline and after three months of therapy ]
    calculated using thorax HRCT

  5. Dynamic lung volumes [ Time Frame: baseline and after three months of therapy ]
    calculated using spirometry

  6. Static lung volumes [ Time Frame: baseline and after three months of therapy ]
    calculated using body plethysmography

  7. airway resistances [ Time Frame: baseline and after three months of therapy ]
    calculated using body plethysmography

  8. Lung clearance index [ Time Frame: baseline and after three months of therapy ]
    Marker of lung ventilation inhomogeneity

  9. 6-minute walking test [ Time Frame: baseline and after three months of therapy ]
    Marker of fitness for daily activities

  10. Sweat chloride test [ Time Frame: baseline and after three months of therapy ]
    Chloride values in sweat chloride test

  11. CFQ-R [ Time Frame: baseline and after three months of therapy ]
    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.

  12. Digital lung auscultation [ Time Frame: baseline and after three months of therapy ]
    Digital analysis of digital lung sounds obtained by a digital stethoscope

  13. Exacerbation frequency [ Time Frame: baseline and after three months of therapy ]
    Number of cystic fibrosis exacerbations



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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03956589


Contacts
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Contact: Stijn Verhulst, MD, PhD +3238213251 stijn.verhulst@uza.be
Contact: Pediatric Pulmonology +3238213251 kinderpneumologie@uza.be

Locations
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Belgium
Antwerp University Hospital Recruiting
Edegem, Belgium, 2650
Contact: Stijn Verhulst, MD, PhD    +3238213251    stijn.verhulst@uza.be   
Sponsors and Collaborators
University Hospital, Antwerp
Investigators
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Principal Investigator: Stijn Verhulst, MD, PhD University Hospital, Antwerp

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Responsible Party: Stijn Verhulst, Director Department of Pediatrics, University Hospital, Antwerp
ClinicalTrials.gov Identifier: NCT03956589     History of Changes
Other Study ID Numbers: 2018-001573-24
First Posted: May 20, 2019    Key Record Dates
Last Update Posted: May 21, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases