An Efficacy and Safety Study of Ivacaftor in Patients With Cystic Fibrosis and Two Splicing Mutations

A Phase 3 study testing Ivacaftor in people with Cystic Fibrosis, run by Medical University of South Carolina.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

This postmarketing N of 2 study is designed to evaluate the efficacy and safety of open-label ivacaftor treatment in two sisters with cystic fibrosis and pancreatic sufficiency. Read more about this study

Who is it for?: Ages 18 years and older, women only
Conditions the study is for: Cystic Fibrosis
What is being tested?: Ivacaftor
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 2 participants
Where: No sites listed yet
Study dates: 1 Nov 2016to May 2018
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 18 years and older
Women only
No healthy volunteers
Adult, Older adult

Who may be able to join

Subjects are >18 years of age and able to provide informed consent.
Subjects reside in the US and are willing to be treated with ivacaftor.
Subjects have the splicing mutation of interest.
Subjects are willing and able to perform requirements of the study.

Who cannot join

There are no relevant exclusion criteria for this n-of-2 study.

About this study

This postmarketing N of 2 study is designed to evaluate the efficacy and safety of open-label ivacaftor treatment in two sisters with cystic fibrosis and pancreatic sufficiency.

Read the detailed description

The two sisters have a splicing mutation that is predicted to respond favorably to ivacaftor therapy. In addition to measurement of usual clinical outcomes (i.e. lung function, nutritional status), there is great interest on the impact on nontuberculous mycobacteria (NTM) airways infection.

Subjects will undergo sputum cultures at baseline and monthly during treatment, initially in the absence of anti-NTM therapy but with the intent to treat with antibiotics if there is persistence of the infection in cultures. Other clinical outcomes will include changes in sweat chloride, lung function and weight. Safety measures will include periodic assessment of liver enzymes. All serious and non-serious adverse events will be collected

Conditions: Cystic Fibrosis
Being tested: Ivacaftor
Medical subject headings: Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

IvacaftorDrug
Subjects will be treated with ivacaftor for 6 months and followed for 7 months and will undergo assessments along the way to measure sweat chloride and sputum amounts.

Study groups in NCT02934698
Study group	Role	What they receive
Ivacaftor	Experimental	There is only one arm to this study. The two sisters with Cystic Fibrosis will both receive Ivacaftor for 6 months for their treatment. Receives: Drug: Ivacaftor

What is being measured

The primary outcome is the main question the study is designed to answer.

Forced Expiratory Volume
Absolute change in percent predicted in 1 second FEV1 from baseline through week 24
Measured: 24 weeks

Secondary outcomes (2)

Sputum Results
Achievement of mycobacterial culture conversion (negative culture)
Measured: 24 weeks
Sweat Chloride
Testing efficacy through gathering absolute change in sweat chloride from baseline through week 24
Measured: 24 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1 Nov 2016May 2018

First posted17 Oct 2016 (estimated)
Study start1 Nov 2016
Primary completionMay 2018
Study completionMay 2018

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Cystic FibrosisStudies of the same condition, open and under way.
More trials run by Medical University of South CarolinaThe sponsor's other registered studies.
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 3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 2 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02934698
Sponsor's protocol ID: Pro00052526
Status last verified: Jul 2018
First submitted: 13 Oct 2016
Last updated: 6 Aug 2018
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
Non-cystic Fibrosis Bronchiectasis
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete

All Cystic Fibrosis trials

Study at a glance

From the registry record, last updated by the sponsor on 6 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Efficacy and Safety Study of Ivacaftor in Patients With Cystic Fibrosis and Two Splicing Mutations
ClinicalTrials.gov ID: NCT02934698
Acronym / sponsor ID: Pro00052526
Conditions: Cystic Fibrosis
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 2 participants (actual)
Lead sponsor: Medical University of South CarolinaUniversity, hospital or other
Collaborators: Vertex Pharmaceuticals Incorporated
Study start: 1 Nov 2016 (actual)
Primary completion: May 2018 (actual)
Study completion: May 2018 (actual)
First posted: 17 Oct 2016 (estimated)
Results: Posted 6 Aug 2018

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

This postmarketing N of 2 study is designed to evaluate the efficacy and safety of open-label ivacaftor treatment in two sisters with cystic fibrosis and pancreatic sufficiency.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Ivacaftor	Experimental	Drug: IvacaftorThere is only one arm to this study. The two sisters with Cystic Fibrosis will both receive Ivacaftor for 6 months for their treatment.

Who may qualify

Ages: 18 years and older
Sex: Female only
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Subjects are >18 years of age and able to provide informed consent.
Subjects reside in the US and are willing to be treated with ivacaftor.
Subjects have the splicing mutation of interest.
Subjects are willing and able to perform requirements of the study.

Key exclusion criteria (1 listed)

There are no relevant exclusion criteria for this n-of-2 study.

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
IvacaftorSubjects will be treated with ivacaftor for 6 months and followed for 7 months and will undergo assessments along the way to measure sweat chloride and sputum amounts.	Drug	Ivacaftor

Primary outcome

Forced Expiratory Volume
Time frame: 24 weeks
Absolute change in percent predicted in 1 second FEV1 from baseline through week 24

2 secondary outcomes

Sputum Results
Time frame: 24 weeks
Sweat Chloride
Time frame: 24 Weeks

Results and safety

Results posted to the registry on 6 Aug 2018, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Ivacaftor	2	2

Primary outcome: Forced Expiratory Volume

Time frame: 24 weeks. Mean (Full Range). Unit: percentage of predicted

Measure	Ivacaftor n = 2
Value	4 (3 to 5)

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: 24 weeks. Other adverse events reported at 1% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Ivacaftor	0 of 2 (0%)	2 of 2 (100%)	0 of 2

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 6 Aug 2018.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

Related trials for Cystic Fibrosis

RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
Non-cystic Fibrosis Bronchiectasis
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete

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.

Patrick A Flume, MD principal investigator

Medical University of South Carolina

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Ivacaftor in people with Cystic Fibrosis, run by Medical University of South Carolina.

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 4 inclusion and 1 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: finished, as of the sponsor's last update on 6 Aug 2018. Status can change between updates, so confirm with the study contact before making plans.

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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 6 Aug 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Forced Expiratory Volume (time frame: 24 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02934698.

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 6 Aug 2018.