Cystic FibrosisPhase 4NCT03956589
Functional Respiratory Imaging and Orkambi in CF
A Phase 4 study testing Orkambi in people with Cystic Fibrosis, run by University Hospital, Antwerp at 1 site in Belgium.
Trial 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). Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Orkambi
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteBelgium
- Study dates
- 1 Oct 2018to 1 Apr 2020
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 10Show fewer
- Has ongoing exacerbation or Allergic bronchopulmonary aspergillosis (ABPA)
- Pregnant or lactating female
- Posttransplant patients
- Patients with severe hepatic impairment
Trial locations and contacts
1 site in 1 country.
About this study
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
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
- Being tested
- Orkambi
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OrkambiDrug
Open label of Orkambi treatment during 3 months
| Study group | Role | What they receive |
|---|---|---|
| Orkambi open-label arm | Experimental | Open-label study: all subjects will receive Orkambi during 3 months. Receives: Drug: Orkambi |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in specific image-based airway resistance (siRaw)
Change in CFD-based airway resistance normalized by the lung volume
Measured: 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
Measured: baseline and after three months of therapy
Secondary outcomes (13)
Internal Airflow Distribution
calculated using thorax HRCT
Measured: baseline and after three months of therapy
Air Trapping
calculated using thorax HRCT
Measured: baseline and after three months of therapy
Airway Wall Volume
calculated using thorax HRCT
Measured: baseline and after three months of therapy
Aerosol Deposition
calculated using thorax HRCT
Measured: baseline and after three months of therapy
Dynamic lung volumes
calculated using spirometry
Measured: baseline and after three months of therapy
Static lung volumes
calculated using body plethysmography
Measured: baseline and after three months of therapy
airway resistances
calculated using body plethysmography
Measured: baseline and after three months of therapy
Lung clearance index
Marker of lung ventilation inhomogeneity
Measured: baseline and after three months of therapy
6-minute walking test
Marker of fitness for daily activities
Measured: baseline and after three months of therapy
Sweat chloride test
Chloride values in sweat chloride test
Measured: baseline and after three months of therapy
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.
Measured: baseline and after three months of therapy
Digital lung auscultation
Digital analysis of digital lung sounds obtained by a digital stethoscope
Measured: baseline and after three months of therapy
Exacerbation frequency
Number of cystic fibrosis exacerbations
Measured: baseline and after three months of therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2019
- Study start1 Oct 2018
- Primary completion1 Apr 2020
- Study completion1 Apr 2020
Why it stopped: Patient inclusion has stopped after interim analysis.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Antwerp
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Stijn Verhulst, University Hospital, Antwerp
- Investigators
- Stijn Verhulst, MD, PhD, Principal investigator, University Hospital, Antwerp
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03956589
- Sponsor's protocol ID
- 2018-001573-24
- Other IDs
- 2018
- Status last verified
- Apr 2020
- First submitted
- 2 May 2019
- Last updated
- 8 Apr 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03956589
- Acronym / sponsor ID
- 2018-001573-24
- Conditions
- Cystic Fibrosis
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Patient inclusion has stopped after interim analysis.
- Enrolment
- 12 participants (actual)
- Lead sponsor
- University Hospital, AntwerpUniversity, hospital or other
- Study start
- 1 Oct 2018 (actual)
- Primary completion
- 1 Apr 2020 (actual)
- Study completion
- 1 Apr 2020 (actual)
- First posted
- 20 May 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Orkambi open-label arm | Experimental | Drug: OrkambiOpen-label study: all subjects will receive Orkambi during 3 months. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OrkambiOpen label of Orkambi treatment during 3 months | Drug | Orkambi open-label arm |
Primary outcome
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
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
13 secondary outcomes
Internal Airflow Distribution
Time frame: baseline and after three months of therapy
Air Trapping
Time frame: baseline and after three months of therapy
Airway Wall Volume
Time frame: baseline and after three months of therapy
Aerosol Deposition
Time frame: baseline and after three months of therapy
Dynamic lung volumes
Time frame: baseline and after three months of therapy
Static lung volumes
Time frame: baseline and after three months of therapy
airway resistances
Time frame: baseline and after three months of therapy
Lung clearance index
Time frame: baseline and after three months of therapy
6-minute walking test
Time frame: baseline and after three months of therapy
Sweat chloride test
Time frame: baseline and after three months of therapy
CFQ-R
Time frame: baseline and after three months of therapy
Digital lung auscultation
Time frame: baseline and after three months of therapy
Exacerbation frequency
Time frame: baseline and after three months of therapy
Results and safety
The study was stopped early (reason given: Patient inclusion has stopped after interim analysis.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2020.
- Individual participant dataSharing planned: No
Related trials for Cystic Fibrosis
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Stijn Verhulst, MD, PhD principal investigator
University Hospital, Antwerp
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Orkambi in people with Cystic Fibrosis, run by University Hospital, Antwerp at 1 site in Belgium.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 8 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Belgium. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Patient inclusion has stopped after interim analysis.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in specific image-based airway resistance (siRaw) (time frame: baseline and after three months of therapy). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Apr 2020.