Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| C4791009 | Other Identifier | Alias Study Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This is a multicenter, open-label, non-comparative, single-arm study to evaluate the efficacy and safety of APX001 for the first-line treatment for candidemia including suspected or confirmed antifungal-resistant candidemia in non-neutropenic patients 18 yeas of age and older.
Suspicion of antifungal-resistant candidemia is sufficient (documented resistance is not required for enrollment). The Study Drug Treatment Period of APX001 will be a maximum of 14 days. After completion of 14 days study drug therapy, if further antifungal treatment is indicated to complete treatment of candidemia in accordance with standard practice guidelines, fluconazole (unless susceptibility results warrant alternative antifungal therapy) may commence for up to a further 7 days. There will be a Follow up Period of 4 weeks (+4 days) after EOT. The total duration of participation in the study is up to approximately 7.5 weeks.
This study will be conducted at approximately 20 sites in the United States and globally.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| APX001 Treatment | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| APX001 | Drug | APX001 |
|
| Measure | Description | Time Frame |
|---|---|---|
| Treatment Success at End of Study Treatment (EOST) as Determined by the Data Review Committee (DRC) | Treatment Success is defined as meeting all of the following criteria: Two consecutive blood cultures negative for Candida spp. Alive at EOST No concomitant use of any other systemic antifungal therapies through end of study treatment | One to forty-two days |
| Measure | Description | Time Frame |
|---|---|---|
| Time to First Negative Blood Culture | Time to first negative blood culture was defined as the number of days from first dose date of study drug to the date of first post-Baseline negative blood culture + 1. Patients without a negative blood culture at post-Baseline visits were censored at the last assessment date. | One to forty-nine days |
Not provided
Key Inclusion Criteria:
Key Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Marc Engelhardt | Basilea Pharmaceutica International Ltd, Allschwil | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham School of Medicine | Birmingham | Alabama | 35233 | United States | ||
| University of Alabama at Birmingham (UAB) |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37596890 | Result | Pappas PG, Vazquez JA, Oren I, Rahav G, Aoun M, Bulpa P, Ben-Ami R, Ferrer R, Mccarty T, Thompson GR, Schlamm H, Bien PA, Barbat SH, Wedel P, Oborska I, Tawadrous M, Hodges MR. Clinical safety and efficacy of novel antifungal, fosmanogepix, for the treatment of candidaemia: results from a Phase 2 trial. J Antimicrob Chemother. 2023 Oct 3;78(10):2471-2480. doi: 10.1093/jac/dkad256. |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | APX001 Treatment | APX001: APX001 IV administration followed by APX001 oral tablet administration |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 3, 2019 | Apr 21, 2021 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Percentage of Patients With Mycological Outcomes at End of Study Treatment (EOST), End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT) |
| End of study treatment (EOST), end of treatment (EOT), and 2 and 4 weeks after end of treatment (EOT) |
| Percentage of Patients With Treatment Success at End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT) | 2 and 4 weeks after end of treatment (EOT) |
| Overall Survival at Study Day 30 | Day 30 |
| Number of Patients With Treatment Emergent Adverse Events (TEAEs) | One to forty-nine days |
| Birmingham |
| Alabama |
| 35249 |
| United States |
| University of California, Davis | Davis | California | 95616 | United States |
| University of California-Davis Medical Center | Sacramento | California | 95817 | United States |
| Augusta University (Georgia Regents University) | Augusta | Georgia | 30901 | United States |
| University of Chicago | Chicago | Illinois | 60637 | United States |
| Washington University | St Louis | Missouri | 63110 | United States |
| Duke University Hospital Medical Center | Durham | North Carolina | 27710 | United States |
| University of Texas- Health Science Center and Medical School at Houston | Houston | Texas | 77030 | United States |
| Institut Jules Bordet,Service De Microbiologie | Brussels | 1000 | Belgium |
| Hopital Erasme | Brussels | 1070 | Belgium |
| Institut Jules Bordet | Brussels | Belgium |
| Universite Libre de Bruxelles (ULB) - Hopital Erasme | Brussels | Belgium |
| CHU de Charleroi - Hopital Civil Marie Curie | Lodelinsart | Belgium |
| Mont-Godinne University Hospital | Yvoir | 5530 | Belgium |
| University Hospital Mont-Godinne | Yvoir | Belgium |
| Klinik I fur Innere Medizin- Uniklinik Koln | Cologne | Germany |
| University Hospital Heidelberg | Heidelberg | Germany |
| Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz - III. Medizinische Klinik Haematologie/Onkologie | Mainz | Germany |
| Infectious Diseases Unit, Rambam Medical Center | Haifa | 3109601 | Israel |
| Rambam Medical Center | Haifa | Israel |
| Infectious Diseases Unit | Tel Aviv | 6423906 | Israel |
| Sourasky Medical Center | Tel Aviv | Israel |
| Infectious Diseases Unit,Sheba Medical Center | Tel Litwinsky | 52621 | Israel |
| Sheba Medical Center | Tel Litwinsky | Israel |
| Hospital Universitario Mutua de Terrassa | Terrassa | Barcelona | Spain |
| Hospital General Universitario de Alicante | Alicante | Spain |
| Hospital Universitari Vall d'Hebron | Barcelona | 08035 | Spain |
| Hospital VLL D Hebron | Barcelona | Spain |
| COMPLETED |
|
| NOT COMPLETED |
|
Treatment Period - MITT
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Treatment Period - MITT | Evaluation of APX001 for the first-line treatment for candidemia, including suspected or confirmed antifungal-resistant candidemia, in non-neutropenic patients ≥ 18 years of age who had at least 1 positive blood culture within the 96 hours prior to starting study drug. Modified Intent-to-Treat (MITT) Population. The MITT Population contained 20 (95.2%) patients. |
| 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 |
| |||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Region of Enrollment | Number | participants |
|
| 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 | Treatment Success at End of Study Treatment (EOST) as Determined by the Data Review Committee (DRC) | Treatment Success is defined as meeting all of the following criteria: Two consecutive blood cultures negative for Candida spp. Alive at EOST No concomitant use of any other systemic antifungal therapies through end of study treatment | Posted | Count of Participants | Participants | One to forty-two days |
|
|
| |||||||||||||||||||||||||||
| Secondary | Time to First Negative Blood Culture | Time to first negative blood culture was defined as the number of days from first dose date of study drug to the date of first post-Baseline negative blood culture + 1. Patients without a negative blood culture at post-Baseline visits were censored at the last assessment date. | Posted | Mean | Standard Deviation | Days | One to forty-nine days |
|
| |||||||||||||||||||||||||||
| Secondary | Percentage of Patients With Mycological Outcomes at End of Study Treatment (EOST), End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT) | Posted | Count of Participants | Participants | End of study treatment (EOST), end of treatment (EOT), and 2 and 4 weeks after end of treatment (EOT) |
|
| |||||||||||||||||||||||||||||
| Secondary | Percentage of Patients With Treatment Success at End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT) | Posted | Number | percentage of participants | 2 and 4 weeks after end of treatment (EOT) |
|
| |||||||||||||||||||||||||||||
| Secondary | Overall Survival at Study Day 30 | Posted | Count of Participants | Participants | Day 30 |
|
|
| ||||||||||||||||||||||||||||
| Secondary | Number of Patients With Treatment Emergent Adverse Events (TEAEs) | Posted | Count of Participants | Participants | One to forty-nine days |
|
|
|
One to forty-nine days
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Safety Population | Twenty-one patients were dosed with APX001 and composed the ITT/Safety Population. | 5 | 21 | 9 | 21 | 20 | 21 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Septic shock | Infections and infestations | Systematic Assessment |
| ||
| Bacteraemia | Infections and infestations | Systematic Assessment |
| ||
| Bacterial sepsis | Infections and infestations | Systematic Assessment |
| ||
| Enterobacter sepsis | Infections and infestations | Systematic Assessment |
| ||
| Necrotising fasciitis | Infections and infestations | Systematic Assessment |
| ||
| Sepsis | Infections and infestations | Systematic Assessment |
| ||
| Stenotrophomonas sepsis | Infections and infestations | Systematic Assessment |
| ||
| Systemic candida | Infections and infestations | Systematic Assessment |
| ||
| Urinary tract infection bacterial | Infections and infestations | Systematic Assessment |
| ||
| Cardiac failure congestive | Cardiac disorders | Systematic Assessment |
| ||
| Cardio-respiratory arrest | Cardiac disorders | Systematic Assessment |
| ||
| Euthanasia | General disorders | Systematic Assessment |
| ||
| General physical health deterioration | General disorders | Systematic Assessment |
| ||
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Interstitial lung disease | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Leukopenia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Gastrointestinal fistula | Gastrointestinal disorders | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bacteraemia | Infections and infestations | Systematic Assessment |
| ||
| Septic shock | Infections and infestations | Systematic Assessment |
| ||
| Diarrhoea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
| ||
| Nausea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Oedema peripheral | General disorders | Systematic Assessment |
| ||
| Pyrexia | General disorders | Systematic Assessment |
| ||
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Acute kidney injury | Renal and urinary disorders | Systematic Assessment |
| ||
| Hydronephrosis | Renal and urinary disorders | Systematic Assessment |
| ||
| Renal failure | Renal and urinary disorders | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Basilea Pharmaceutica International Ltd, Allschwil | +41 79 701 0551 | marc.engelhardt@basilea.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 27, 2020 | Apr 21, 2021 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D058387 | Candidemia |
| ID | Term |
|---|---|
| D058365 | Candidiasis, Invasive |
| D002177 | Candidiasis |
| D009181 | Mycoses |
| D001423 | Bacterial Infections and Mycoses |
| D007239 | Infections |
| D000072742 | Invasive Fungal Infections |
| D016469 | Fungemia |
| D018805 | Sepsis |
| D018746 | Systemic Inflammatory Response Syndrome |
| D007249 | Inflammation |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Israel |
|
| Spain |
|
| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
|
|
|
| Title | Measurements |
|---|---|
|
|