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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.
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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 50 mg CTX-4430 | Experimental | Once daily oral capsule for 48 weeks |
|
| 100 mg CTX-4430 | Experimental | Once daily oral capsule for 48 weeks |
|
| Matching Placebo | Placebo Comparator | Once daily oral capsule for 48 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CTX-4430 | Drug |
| ||
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline, Week 48 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Week 48 |
| Hazard Ratio Pulmonary Exacerbation While in the Study |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Pulmonary Exacerbation Per Year for Participants With ppFEV1 >75 at Baseline | 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. | Week 48 |
| Hazard Ratio Pulmonary Exacerbation for Participants With ppFEV1 >75 at Baseline |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Steven Rowe, MD | University of Alabama at Birmingham, USA | Principal Investigator |
| Stuart Elborn, MD | Royal Brompton Hospital, London UK | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama | Birmingham | Alabama | 35233 | United States | ||
| Providence Health and Services |
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| ID | Title | Description |
|---|---|---|
| FG000 | 100 mg CTX-4430 | Once daily oral capsule for 48 weeks CTX-4430 |
| FG001 | 50 mg CTX-4430 | Once daily oral capsule for 48 weeks CTX-4430 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | May 4, 2016 | Apr 26, 2019 |
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|
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. |
| Week 48 |
| Subjects Without a Pulmonary Exacerbation While in the Study | Subjects who did not experience a protocol-defined pulmonary exacerbation during the study | Week 48 |
| Relative Change (Percent Change) From Baseline in ppFEV1 | Percent change from Baseline for ppFEV1 at 48 weeks was assessed. | 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 | 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. | 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. | Baseline, Week 48 |
Hazard ratio of pulmonary exacerbation versus placebo for all subjects |
| Week 48 |
| Subjects Without a Pulmonary Exacerbation by Participants With ppFEV1 >75 at Baseline | Subjects who did not experience a protocol-defined pulmonary exacerbation during the study. | Week 48 |
| Number of Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline | 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. | Week 48 |
| Hazard Ratio Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline | Hazard Ratio pulmonary exacerbation versus placebo for all subjects taking CFTR-modulating therapy at Baseline | Week 48 |
| Subjects Without a Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline | Subjects who did not experience a protocol-defined pulmonary exacerbation during the study. | Week 48 |
| Anchorage |
| Alaska |
| 99508 |
| United States |
| Banner University of Arizona Medical Center | Tucson | Arizona | 85724 | United States |
| Childrens Hospital Los Angeles | Los Angeles | California | 90027 | United States |
| Pediatric Pulmonary and Cystic Fibrosis Clinic, Stanford University | Palo Alto | California | 94304 | United States |
| University of California Davis Medical Center | Sacramento | California | 95817 | United States |
| National Jewish Health | Denver | Colorado | 80206 | United States |
| Yale University | New Haven | Connecticut | 06520 | United States |
| University of Florida | Gainesville | Florida | 32610 | United States |
| Central Florida Pulmonary Group | Orlando | Florida | 32803 | United States |
| Emory University | Atlanta | Georgia | 30322 | United States |
| University of Chicago | Chicago | Illinois | 60637 | United States |
| St. Francis Medical Center | Peoria | Illinois | 61637 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Maine Medical Center | Portland | Maine | 04106 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| University of Michigan | Ann Arbor | Michigan | 49286 | United States |
| Harper University Hospital | Detroit | Michigan | 48201 | United States |
| Spectrum Health Butterworth Campus | Grand Rapids | Michigan | 49546 | United States |
| University of Mississippi Medical Center | Jackson | Mississippi | 39216 | United States |
| University of New Mexico | Albuquerque | New Mexico | 87131 | United States |
| Albany Medical College | Albany | New York | 12208 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| SUNY Upstate Medical University | Syracuse | New York | 13210 | United States |
| Wake Forest Hospital | Winston-Salem | North Carolina | 27157 | United States |
| UC Cincinnati Children's Hospital | Cincinnati | Ohio | 45267 | United States |
| University of Oklahoma Health Sciences Center | Oklahoma City | Oklahoma | 73104 | United States |
| Oregon Health & Sciences University | Portland | Oregon | 97239 | United States |
| Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Drexel University | Philadelphia | Pennsylvania | 19104 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| Sanford Clinical Research | Sioux Falls | South Dakota | 57104 | United States |
| Universiy of Tennessee Medical Center UHS | Knoxville | Tennessee | 37920 | United States |
| Dell Children's Medical Center | Austin | Texas | 78723 | United States |
| Cook Children's Hospital | Fort Worth | Texas | 76104 | United States |
| Virginia Commonwealth University | Richmond | Virginia | 23298 | United States |
| Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| Hôpital Erasme | Brussels | Belgium |
| UZ Leuven | Leuven | Belgium |
| University of Calgary | Calgary | Alberta | T2N 4N1 | Canada |
| Ottawa Hospital | Ottawa | Ontario | K1H 8L6 | Canada |
| St. Michael's Hospital | Toronto | Ontario | m5B 1W8 | Canada |
| Centre hospitalier de Dunkerque | Dunkirk | France |
| Hôpital Albert Michallon | Grenoble | France |
| Hopital Arnaud de Villeneuve | Montpellier | Cedex 5 | France |
| Hôpital Cochin | Paris | France |
| CH Lyon Sud | Pierre-Bénite | France |
| Charite' University | Berlin | 10117 | Germany |
| Krankenhaus Donaustauf | Donaustauf | Germany |
| Carl-Gustav-Klinikum Dresden, Mukoviszidose Centrum "Christiane Herzog" | Dresden | Germany |
| Ruhrlandklinik Essen | Essen | Germany |
| Institut für klinische Forschung Pneumologie | Frankfurt | Germany |
| Klinikum der Johann Wolfgang Goethe-Universität | Frankfurt | Germany |
| Medizinische Hochschule Hannover | Hanover | Germany |
| Universitätsklinikum Jena CF Centre | Jena | 07740 | Germany |
| Lungenärztliche Praxis München-Pasing | München-Pasing | Germany |
| Klinikum Stuttgart CF Ambulanz | Stuttgart | Germany |
| Ospedali Riuniti di Ancona | Ancona | Italy |
| Azienda Ospedaliero Universitaria | Catania | Italy |
| Azienda Ospedaliera A Meyer | Florence | Italy |
| IRCCS Ospedale Pediatrico Bambino | Rome | Italy |
| Ospedale Civile Maggiore | Verona | Italy |
| Belfast City Hospital | Belfast | United Kingdom |
| Bristol Royal Infirmary | Bristol | BS2 8HW | United Kingdom |
| Llandough Hospital | Cardiff | CF64 2XX | United Kingdom |
| St James's University Hospital | Leeds | LS97TF | United Kingdom |
| Liverpool Heart and Chest Hospital | Liverpool | United Kingdom |
| King's College Hospital | London | SE5 9RS | United Kingdom |
| Royal Brompton Hospital | London | United Kingdom |
| University Hospital of South Manchester | Manchester | United Kingdom |
| Royal Victoria Infirmary | Newcastle upon Tyne | United Kingdom |
| University Hospital Southampton | Southampton | United Kingdom |
| Royal Stoke University Hospital | Stoke-on-Trent | United Kingdom |
| FG002 | Matching Placebo | Once daily oral capsule for 48 weeks Placebo |
| COMPLETED |
|
| NOT COMPLETED |
|
|
One subject in the matching placebo cohort was randomized but did not take any study drug. Therefore, the matching placebo group has 66 subjects listed instead of 67 subjects making it a total of 199 subjects who received study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | 100 mg CTX-4430 | Once daily oral capsule for 48 weeks CTX-4430 |
| BG001 | 50 mg CTX-4430 | Once daily oral capsule for 48 weeks CTX-4430 |
| BG002 | Matching Placebo | Once daily oral capsule for 48 weeks Placebo |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| On cystic fibrosis transmembrane conductance regulator (CFTR) Modulator Therapy: Yes | Count of Participants | Participants |
| ||||||||||||||||
| On CFTR Modulator Therapy: No | Count of Participants | Participants |
| ||||||||||||||||
| Number of Pulmonary Exacerbations in the year prior to Screening | Mean | Standard Deviation | pulmonary Exacerbations/year |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | 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. | Posted | Mean | 95% Confidence Interval | FEV1 percent predicted | Baseline, Week 48 |
|
|
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Full Analysis Population - All subjects randomized to and receiving at least 1 dose of assigned treatment. The statistical analysis plans states the two active doses will be compared to placebo individually as well as pooled. | Posted | Mean | 95% Confidence Interval | pulmonary exacerbations per year | Week 48 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | Full Analysis Population - All subjects randomized to and receiving at least 1 dose of assigned treatment. The statistical analysis plans states the two active doses will be compared to placebo individually as well as pooled. | Posted | Number | 95% Confidence Interval | hazard ratio | Week 48 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Subjects Without a Pulmonary Exacerbation While in the Study | Subjects who did not experience a protocol-defined pulmonary exacerbation during the study | Full Analysis Population - All subjects randomized to and receiving at least 1 dose of assigned treatment. | Posted | Count of Participants | Participants | Week 48 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Relative Change (Percent Change) From Baseline in ppFEV1 | Percent change from Baseline for ppFEV1 at 48 weeks was assessed. | Full Analysis Population - All subjects randomized to and receiving at least 1 dose of assigned treatment. (Observed Data). The statistical analysis plans states the two active doses will be compared to placebo individually as well as pooled. | Posted | Mean | 95% Confidence Interval | percent change from baseline | Baseline, Week 48 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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 | Full Analysis Population - All subjects randomized to and receiving at least 1 dose of assigned treatment. (Observed Data). The statistical analysis plans states the two active doses will be compared to placebo individually as well as pooled. | Posted | Mean | 95% Confidence Interval | absolute change of percent predicted | Baseline, Week 48 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline for Specified Biomarkers | Results were only calculated in subjects who had a verifiable result at the Baseline and Week 48 visits. | Full Analysis Population - All subjects randomized to and receiving at least 1 dose of assigned treatment and who had results at Baseline and Week 48. The statistical analysis plans states the two active doses will be compared to placebo individually as well as pooled. | Posted | Mean | 95% Confidence Interval | log10(mcg/mL) | Baseline, Week 48 |
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| Secondary | 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. | Full Analysis Population - All subjects randomized to and receiving at least 1 dose of assigned treatment and who had results at Baseline and Week 48. The statistical analysis plans states the two active doses will be compared to placebo individually as well as pooled. | Posted | Mean | 95% Confidence Interval | mg/dL | Baseline, Week 48 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Number of Pulmonary Exacerbation Per Year for Participants With ppFEV1 >75 at Baseline | 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. | Population of subjects with Baseline ppFEV1 >75. The statistical analysis plans states the two active doses will be compared to placebo individually as well as combined. The statistical analysis plans states the two active doses will be compared to placebo individually as well as pooled. | Posted | Mean | 95% Confidence Interval | pulmonary exacerbations per year | Week 48 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Hazard Ratio Pulmonary Exacerbation for Participants With ppFEV1 >75 at Baseline | Hazard ratio of pulmonary exacerbation versus placebo for all subjects | Population of subjects with Baseline ppFEV1 >75. The statistical analysis plan states the two active doses will be compared to placebo individually as well as pooled. | Posted | Number | 95% Confidence Interval | hazard ratio | Week 48 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Subjects Without a Pulmonary Exacerbation by Participants With ppFEV1 >75 at Baseline | Subjects who did not experience a protocol-defined pulmonary exacerbation during the study. | Population of subjects with Baseline ppFEV1 >75. | Posted | Count of Participants | Participants | Week 48 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Number of Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline | 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. | Population of subjects taking CFTR modulating therapy at Baseline. The statistical analysis plans states the two active doses will be compared to placebo individually as well as pooled. | Posted | Mean | 95% Confidence Interval | pulmonary exacerbations per year | Week 48 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Hazard Ratio Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline | Hazard Ratio pulmonary exacerbation versus placebo for all subjects taking CFTR-modulating therapy at Baseline | Population of subjects taking CFTR modulating therapy at Baseline. The statistical analysis plans states the two active doses will be compared to placebo individually as well as pooled. | Posted | Number | 95% Confidence Interval | hazard ratio | Week 48 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Subjects Without a Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline | Subjects who did not experience a protocol-defined pulmonary exacerbation during the study. | Population of subjects taking CFTR modulating therapy at Baseline. | Posted | Count of Participants | Participants | Week 48 |
|
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| Post-Hoc | Number of Pulmonary Exacerbation by Baseline FEV1 Percent Predicted at Week 48 | 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. | Subject's FEV1 percent predicted at screening are presented below. | Posted | Mean | 95% Confidence Interval | pulmonary exacerbations per year | Week 48 |
|
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.
An adverse event (AE) is any untoward medical occurrence in a subject administered a pharmaceutical product and does not necessarily have a causal relationship with this product. For purposes of this trial, AEs will be reported from the signing of the study informed consent (ICF) through study completion or early termination. A Treatment-Emergent AE is an AE that occurs after the first dose of study medication.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 100 mg CTX-4430 | Once daily oral capsule for 48 weeks CTX-4430 | 0 | 66 | 27 | 66 | 66 | 66 |
| EG001 | 50 mg CTX-4430 | Once daily oral capsule for 48 weeks CTX-4430 | 0 | 67 | 26 | 67 | 67 | 67 |
| EG002 | Matching Placebo | Once daily oral capsule for 48 weeks Placebo | 0 | 66 | 21 | 66 | 65 | 66 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Haemolytic Anaemia | Blood and lymphatic system disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Chronic Sinusitis | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Lower respiratory tract infection fungal | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Nocardiosis | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Pneumonia mycoplasmal | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Scedosporium infection | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Lower limb fracture | Injury, poisoning and procedural complications | MedDRA (18.0) | Non-systematic Assessment |
| |
| Borderline serous tumour of ovary | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (18.0) | Non-systematic Assessment |
| |
| Calculus ureteric | Renal and urinary disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Acute Respiratory Failure | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Nasal septum deviation | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Antibiotic therapy | Surgical and medical procedures | MedDRA (18.0) | Non-systematic Assessment |
| |
| Therapeutic embolisation | Surgical and medical procedures | MedDRA (18.0) | Non-systematic Assessment |
| |
| Therapy Change | Surgical and medical procedures | MedDRA (18.0) | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Forced expiratory volume decreased | Investigations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Sputum Abnormal | Investigations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA (18.0) | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Paranasal sinus hypersecretion | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Rales | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (18.0) | Non-systematic Assessment |
|
PI is free to publish results of the study after (1) the first multi-center publication or (2) 24 months after close of the study, whichever occurs first.
Proposed publications are to be submitted to the sponsor for review and comment for a period of at least 90 days (which may be extended under certain circumstances related to protection of intellectual property as well as for deletion of any Confidential Information).
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Director, Clincal Operations | Celtaxsys, Inc. | 4702060153 | 123 | sbirnbaum@celtaxsys.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 15, 2018 | Apr 26, 2019 | SAP_001.pdf |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000625351 | acebilustat |
Not provided
Not provided
Not provided
| Between 18 and 65 years |
|
| >=65 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Mean Difference (Final Values) |
| -1.07 |
| Standard Error of the Mean |
| 1.37 |
| 2-Sided |
| 95 |
| -3.78 |
| 1.64 |
| Other |
Single Group Difference from Placebo Mean Change from Baseline with 95% CI |
| ANOVA | 0.45 | 0.1 alpha level (2-sided) prespecified | Mean Difference (Final Values) | 0.16 | Standard Error of the Mean | 1.2 | 2-Sided | 95 | -2.20 | 2.50 | Superiority | Difference from placebo of pooled arms (100mg CTX-4430 and 50mg CTX-4430) in change from baseline in ppFEV1 at Week 48. |
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Once daily oral capsule for 48 weeks
Placebo
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