Functional Respiratory Imaging and Orkambi in CF

NCT03956589 | Terminated Phase 4

• 1 other identifier: 2018-001573-24
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (2)

• Change in specific image-based airway resistance (siRaw)

  Change in CFD-based airway resistance normalized by the lung volume

  baseline and after three months of therapy

• Change in specific image-based airway volumes (siVaw)

  change in CT-based airway volumes normalized by the lung volume

  baseline and after three months of therapy

Secondary Outcomes (13)

• Internal Airflow Distribution

  baseline and after three months of therapy

• Air Trapping

  baseline and after three months of therapy

• Airway Wall Volume

  baseline and after three months of therapy

• Aerosol Deposition

  baseline and after three months of therapy

• Dynamic lung volumes

  baseline and after three months of therapy

Study Arms (1)

Orkambi open-label arm

EXPERIMENTAL

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

Drug: Orkambi

Interventions

Orkambi DRUG

Open label of Orkambi treatment during 3 months

Orkambi open-label arm

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University Hospital, Antwerp: lead

Study Sites (1)

Antwerp University Hospital

Edegem, 2650, Belgium

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

lumacaftor, ivacaftor drug combination

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Stijn Verhulst, MD, PhD

  University Hospital, Antwerp

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director Department of Pediatrics

Study Record Dates

• First Submitted: May 2, 2019
• First Posted: May 20, 2019
• Study Start: October 1, 2018
• Primary Completion: April 1, 2020
• Study Completion: April 1, 2020
• Last Updated: April 8, 2020

Record last verified: 2020-04

Data Sharing

• IPD Sharing: Will not share

Locations

BE (1)