Cystic FibrosisPhase 2NCT02443688

EMPIRE CF: A Phase 2 Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 in Adult Cystic Fibrosis (CF) Patients

A Phase 2 study testing CTX-4430 in people with Cystic Fibrosis, run by Celtaxsys, Inc. at 75 sites in 7 countries.

Completed

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

Official record

Search all trials

Trial summary

Who is it for?: Ages 18 to 30 years
Conditions the study is for: Cystic Fibrosis
What is being tested?: CTX-4430
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 200 participants
Where: 75 sitesin 7 countries
Study dates: 30 Oct 2015to 16 May 2018
Phase: Phase 2
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 to 30 years
All sexes
No healthy volunteers
Adult

Who may be able to join

Forced expiratory volume at one second (FEV1) ≥50 percent predicted at Screening
At least 1 pulmonary exacerbation in the 12 months before Screening

Who cannot join

Pregnant or nursing women
Medical condition that is unstable, could be adversely impacted by participation in the study, or could impact assessment of the study results
History of organ transplantation
History of alcoholism or drug abuse within 2 years before Screening
Regular use of a high-dose NSAID within 60 days before Screening

Trial locations and contacts

75 sites in 7 countries.

Country

City, postcode or hospital

75 sites in 7 countries

United States39 sites

Albany Medical College
Albany, New York, 12208
University of New Mexico
Albuquerque, New Mexico, 87131
Providence Health and Services
Anchorage, Alaska, 99508
University of Michigan
Ann Arbor, Michigan, 49286
Emory University
Atlanta, Georgia, 30322
Dell Children's Medical Center
Austin, Texas, 78723
University of Alabama
Birmingham, Alabama, 35233
Massachusetts General Hospital
Boston, Massachusetts, 02114
Boston Children's Hospital
Boston, Massachusetts, 02115
University of Chicago
Chicago, Illinois, 60637
UC Cincinnati Children's Hospital
Cincinnati, Ohio, 45267
National Jewish Health
Denver, Colorado, 80206
Harper University Hospital
Detroit, Michigan, 48201
Cook Children's Hospital
Fort Worth, Texas, 76104
University of Florida
Gainesville, Florida, 32610
Spectrum Health Butterworth Campus
Grand Rapids, Michigan, 49546
Hershey Medical Center
Hershey, Pennsylvania, 17033
University of Mississippi Medical Center
Jackson, Mississippi, 39216
University of Kansas Medical Center
Kansas City, Kansas, 66160
Universiy of Tennessee Medical Center UHS
Knoxville, Tennessee, 37920
Childrens Hospital Los Angeles
Los Angeles, California, 90027
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Yale University
New Haven, Connecticut, 06520
Columbia University Medical Center
New York, New York, 10032
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
Central Florida Pulmonary Group
Orlando, Florida, 32803
Pediatric Pulmonary and Cystic Fibrosis Clinic, Stanford University
Palo Alto, California, 94304
St. Francis Medical Center
Peoria, Illinois, 61637
Drexel University
Philadelphia, Pennsylvania, 19104
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Maine Medical Center
Portland, Maine, 04106
Oregon Health & Sciences University
Portland, Oregon, 97239
Virginia Commonwealth University
Richmond, Virginia, 23298
University of California Davis Medical Center
Sacramento, California, 95817
Sanford Clinical Research
Sioux Falls, South Dakota, 57104
SUNY Upstate Medical University
Syracuse, New York, 13210
Banner University of Arizona Medical Center
Tucson, Arizona, 85724
Wake Forest Hospital
Winston-Salem, North Carolina, 27157

Other trials in United States

United Kingdom11 sites

Belfast City Hospital
Belfast
Bristol Royal Infirmary
Bristol, BS2 8HW
Llandough Hospital
Cardiff, CF64 2XX
St James's University Hospital
Leeds, LS97TF
Liverpool Heart and Chest Hospital
Liverpool
King's College Hospital
London, SE5 9RS
Royal Brompton Hospital
London
University Hospital of South Manchester
Manchester
Royal Victoria Infirmary
Newcastle upon Tyne
University Hospital Southampton
Southampton
Royal Stoke University Hospital
Stoke-on-Trent

Other trials in United Kingdom

Germany10 sites

Charite' University
Berlin, 10117
Krankenhaus Donaustauf
Donaustauf
Carl-Gustav-Klinikum Dresden, Mukoviszidose Centrum "Christiane Herzog"
Dresden
Ruhrlandklinik Essen
Essen
Institut für klinische Forschung Pneumologie
Frankfurt
Klinikum der Johann Wolfgang Goethe-Universität
Frankfurt
Medizinische Hochschule Hannover
Hanover
Universitätsklinikum Jena CF Centre
Jena, 07740
Lungenärztliche Praxis München-Pasing
München-Pasing
Klinikum Stuttgart CF Ambulanz
Stuttgart

Other trials in Germany

France5 sites

Centre hospitalier de Dunkerque
Dunkirk
Hôpital Albert Michallon
Grenoble
Hopital Arnaud de Villeneuve
Montpellier, Cedex 5
Hôpital Cochin
Paris
CH Lyon Sud
Pierre-Bénite

Other trials in France

Italy5 sites

Ospedali Riuniti di Ancona
Ancona
Azienda Ospedaliero Universitaria
Catania
Azienda Ospedaliera A Meyer
Florence
IRCCS Ospedale Pediatrico Bambino
Rome
Ospedale Civile Maggiore
Verona

Other trials in Italy

Canada3 sites

University of Calgary
Calgary, Alberta, T2N 4N1
Ottawa Hospital
Ottawa, Ontario, K1H 8L6
St. Michael's Hospital
Toronto, Ontario, m5B 1W8

Other trials in Canada

Belgium2 sites

Hôpital Erasme
Brussels
UZ Leuven
Leuven

Other trials in Belgium

About this study

Official title: A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 Administered Orally Once-Daily for 48 Weeks in Adult Patients With Cystic Fibrosis

This study is a Phase 2, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of CTX-4430 administered once-daily for 48 weeks for treatment of CF.

Read the detailed description

This study is a Phase 2, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of CTX-4430 administered once-daily for 48 weeks for treatment of CF. A total of 195 pulmonary CF patients that meet all the inclusion and no exclusion criteria and provide written informed consent will be randomized to receive 50 mg CTX-4430, 100 mg CTX-4430, or placebo in a 1:1:1 ratio. Follow-up visits will be conducted approximately every 4 weeks from Week 4 to Week 52 (4 weeks after completion of treatment).

Conditions: Cystic Fibrosis
Being tested: CTX-4430, Placebo
Medical subject headings: Cystic Fibrosis

What participants receive

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

CTX-4430Drug
PlaceboDrug

Study groups in NCT02443688
Study group	Role	What they receive
50 mg CTX-4430	Experimental	Once daily oral capsule for 48 weeks Receives: Drug: CTX-4430
100 mg CTX-4430	Experimental	Once daily oral capsule for 48 weeks Receives: Drug: CTX-4430
Matching Placebo	Placebo comparator	Once daily oral capsule for 48 weeks Receives: Drug: Placebo

What is being measured

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

Difference From Placebo in Absolute Change From Baseline in Forced Expiratory Volume in 1 Second Percent Predicted (ppFEV1)
Difference from Placebo in absolute change from Baseline at Week 48 was assessed for FEV1 percent predicted.
Measured: Baseline, Week 48

Secondary outcomes (7)

Number of Pulmonary Exacerbations Through 48 Weeks
Rate of protocol-defined pulmonary exacerbations reported through the Week 48/Early Termination visit will be annualized where a year is defined by 52 weeks and will be analyzed using a negative binomial regression.
Measured: Week 48
Hazard Ratio Pulmonary Exacerbation While in the Study
Hazard Ratio of pulmonary exacerbation versus placebo for all subjects. Pulmonary exacerbations are defined as treatment with oral, inhaled, or intravenous antibiotic(s) for ≥4 of symptoms/signs per the modified Fuchs criteria.
Measured: Week 48
Subjects Without a Pulmonary Exacerbation While in the Study
Subjects who did not experience a protocol-defined pulmonary exacerbation during the study
Measured: Week 48
Relative Change (Percent Change) From Baseline in ppFEV1
Percent change from Baseline for ppFEV1 at 48 weeks was assessed.
Measured: Baseline, Week 48
Change From Baseline at 48 Weeks for Forced Vital Capacity Percent Predicted (FVC) and FEF25-75% (Forced Expiratory Flow During the Middle Half of the Forced Vital Capacity) Percent Predicted
Measured: Baseline, Week 48
Change From Baseline for Specified Biomarkers
Results were only calculated in subjects who had a verifiable result at the Baseline and Week 48 visits.
Measured: Baseline, Week 48
Change From Baseline for C-reactive Protein (Hs-CRP)
Results were only calculated in subjects who had a verifiable result at the Baseline and Week 48 visits.
Measured: Baseline, Week 48

Timeline

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

30 Oct 201516 May 2018

First posted14 May 2015 (estimated)
Study start30 Oct 2015
Primary completion20 Apr 2018
Study completion16 May 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.
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 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind
Who is masked: Participant, Care provider, Investigator
Enrolment: 200 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02443688
Sponsor's protocol ID: CTX-4430-CF-201
Status last verified: Aug 2019
First submitted: 8 May 2015
Last updated: 4 Sep 2019
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

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?

See locations

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All Cystic Fibrosis trials

Study at a glance

From the registry record, last updated by the sponsor on 4 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 Administered Orally Once-Daily for 48 Weeks in Adult Patients With Cystic Fibrosis
ClinicalTrials.gov ID: NCT02443688
Acronym / sponsor ID: CTX-4430-CF-201
Conditions: Cystic Fibrosis
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 200 participants (actual)
Lead sponsor: Celtaxsys, Inc.University, hospital or other
Study start: 30 Oct 2015 (actual)
Primary completion: 20 Apr 2018 (actual)
Study completion: 16 May 2018 (actual)
First posted: 14 May 2015 (estimated)
Results: Posted 25 Jul 2019

Clinical summary

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

This study is a Phase 2, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of CTX-4430 administered once-daily for 48 weeks for treatment of CF.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, care provider, investigator)
Number of arms: 3

Arm	Type	Receives
50 mg CTX-4430	Experimental	Drug: CTX-4430Once daily oral capsule for 48 weeks
100 mg CTX-4430	Experimental	Drug: CTX-4430Once daily oral capsule for 48 weeks
Matching Placebo	Placebo comparator	Drug: PlaceboOnce daily oral capsule for 48 weeks

Who may qualify

Ages: 18 to 30 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (2 listed)

Forced expiratory volume at one second (FEV1) ≥50 percent predicted at Screening
At least 1 pulmonary exacerbation in the 12 months before Screening

Key exclusion criteria (5 listed)

Pregnant or nursing women
Medical condition that is unstable, could be adversely impacted by participation in the study, or could impact assessment of the study results
History of organ transplantation
History of alcoholism or drug abuse within 2 years before Screening
Regular use of a high-dose NSAID within 60 days before Screening

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
CTX-4430	Drug	100 mg CTX-4430, 50 mg CTX-4430
Placebo	Drug	Matching Placebo

Primary outcome

Difference From Placebo in Absolute Change From Baseline in Forced Expiratory Volume in 1 Second Percent Predicted (ppFEV1)
Time frame: Baseline, Week 48
Difference from Placebo in absolute change from Baseline at Week 48 was assessed for FEV1 percent predicted.

7 secondary outcomes

Number of Pulmonary Exacerbations Through 48 Weeks
Time frame: Week 48
Hazard Ratio Pulmonary Exacerbation While in the Study
Time frame: Week 48
Subjects Without a Pulmonary Exacerbation While in the Study
Time frame: Week 48
Relative Change (Percent Change) From Baseline in ppFEV1
Time frame: Baseline, Week 48
Change From Baseline at 48 Weeks for Forced Vital Capacity Percent Predicted (FVC) and FEF25-75% (Forced Expiratory Flow During the Middle Half of the Forced Vital Capacity) Percent Predicted
Time frame: Baseline, Week 48
Change From Baseline for Specified Biomarkers
Time frame: Baseline, Week 48
Change From Baseline for C-reactive Protein (Hs-CRP)
Time frame: Baseline, Week 48

Results and safety

Results posted to the registry on 25 Jul 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
100 mg CTX-4430	66	54
50 mg CTX-4430	67	58
Matching Placebo	67	55

Primary outcome: Difference From Placebo in Absolute Change From Baseline in Forced Expiratory Volume in 1 Second Percent Predicted (ppFEV1)

Time frame: Baseline, Week 48. Mean (95% Confidence Interval). Unit: FEV1 percent predicted

Measure	100 mg CTX-4430 n = 66	50 mg CTX-4430 n = 67	Pooled CTX-4430 n = 133	Matching Placebo n = 66
Value	-1.30 (-3.22 to 0.62)	-3.76 (-5.65 to -1.86)	-2.53 (-3.88 to -1.18)	-2.69 (-4.63 to -0.75)

100 mg CTX-4430: Mean Difference (Final Values): 1.39 (95% CI -1.34 to 4.12), ANOVA
50 mg CTX-4430: Mean Difference (Final Values): -1.07 (95% CI -3.78 to 1.64), ANOVA
Pooled CTX-4430: Mean Difference (Final Values): 0.16 (95% CI -2.20 to 2.50), p = 0.45, ANOVA

Population analysed: Full Analysis Population - All subjects randomized to and receiving at least 1 dose of assigned treatment. The primary analysis was based upon the pooled results of the 100mg and 50mg doses.

7 secondary outcomes are reported in the full results.

Adverse events

Time frame: Baseline up to 28 days after the subject's last dose of study medication where the last dose is 48 weeks or early discontinuation of study drug.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
100 mg CTX-4430	27 of 66 (40.9%)	66 of 66 (100%)	0 of 66
50 mg CTX-4430	26 of 67 (38.8%)	67 of 67 (100%)	0 of 67
Matching Placebo	21 of 66 (31.8%)	65 of 66 (98.5%)	0 of 66

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 4 Sep 2019.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Cystic Fibrosis

RecruitingObservational study
Natural History of Noncirrhotic Portal Hypertension
Cystic Fibrosis, Immunologic Deficiency Syndrome
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 3
Compare Oral Itraconazole and Standard Care Versus Standard Care Alone in Patients With Non-cystic Fibrosis Related Bronchiectasis With Chronic Aspergillus Infection in Reducing Bronchiectasis Exacerbations
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Not yet recruitingObservational study
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Active, not recruitingObservational study
Feasibility and Acceptance of SonoHeal for Airway Clearance and Preliminary Comparison With the Standard of Care
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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.

Steven Rowe, MD principal investigator

University of Alabama at Birmingham, USA

Stuart Elborn, MD principal investigator

Royal Brompton Hospital, London UK

75 sites in 7 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing CTX-4430 in people with Cystic Fibrosis, run by Celtaxsys, Inc. at 75 sites in 7 countries.

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 2 inclusion and 5 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 4 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 75 sites in United States, United Kingdom, Germany, France, Italy and 2 more countries. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 25 Jul 2019). 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?

Difference From Placebo in Absolute Change From Baseline in Forced Expiratory Volume in 1 Second Percent Predicted (ppFEV1) (time frame: Baseline, Week 48). 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 NCT02443688.

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 4 Sep 2019.