Open-label Study of APX001 for Treatment of Patients With Invasive Mold Infections Caused by Aspergillus or Rare Molds (AEGIS)

September 3, 2025 updated by: Basilea Pharmaceutica

A Phase 2, Open-Label Study to Evaluate the Safety and Efficacy of APX001 in the Treatment of Patients With Invasive Mold Infections Caused by Aspergillus Species or Rare Molds

This is a Phase 2, multicenter study to evaluate APX001 for the treatment of invasive fungal infections caused by Aspergillus spp. or rare molds (eg, Scedosporium spp., Fusarium spp., and Mucorales fungi).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
21

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Brussels, Belgium, 1070
        • Cliniques Universitaires de Bruxelles Hôpital Erasme - Department of Infectious Diseases
      • Leuven, Belgium, 3000
        • UZ Leuven - Department of Haematology
      • Yvoir, Belgium, 5530
        • CHU UCL Namur - Mont- Godinne University Hospital - Intensive Care Unit
  • Germany
      • Mainz, Germany, 55131
        • Universitatsmedizin Der Johannes Gutenberg-Universitat Mainz Iii
    • Bavaria
      • München, Bavaria, Germany, 81737
        • Klinikum Neuperlach, Klinik fur Hamatologie und Onkologie
  • Israel
      • Haifa, Israel, 3109601
        • Rambam Medical Center
      • Haifa, Israel, 3109601
        • Pharmacy
      • Ramat Gan, Israel, 5262160
        • Pharmacy
      • Ramat Gan, Israel, 5262160
        • Sheba Medical Center, Tel Hashomer
  • United States
    • California
      • Duarte, California, United States, 91010
        • City of Hope (City of Hope National Medical Center, City of Hope Medical Center)
      • Duarte, California, United States, 91010
        • IP Address : City of Hope Investigational Drug Services (IDS)
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke Department of Pharmacy/Investigational Drug Service (IDS)
      • Durham, North Carolina, United States, 27710
        • Duke Medicine Pavilion
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center (Duke South Clinic)
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center(Duke Hospital)

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Males or females, 18 years or older.
  • Patients with proven or probable IMI caused by Aspergillus spp. Patients who present with IMI due to other filamentous fungi (eg, Scedosporium spp., Fusarium spp., and Mucorales fungi such as Mucor spp. or Rhizopus spp.) may also be enrolled.
  • Have limited or no treatment options due to documented or anticipated resistance, contraindication, intolerance, or lack of clinical response to SOC antifungal therapy, as advocated by the relevant regional/country treatment guidelines.
  • Patients where the Investigator considers that there is a potential advantage of using APX001 over current SOC (eg, broad spectrum of activity, emergence of IMI during antifungal prophylaxis, activity against resistant mold pathogens, IV and PO formulations, favorable DDI profile, favorable hepatic and renal safety profile, wide tissue distribution including brain), and/or where the SOC antifungal therapy carries significant risk of toxicity or treatment failure (eg, DDI risk, safety/toxicity risk, site of infection not accessible by SOC).

Exclusion Criteria:

  • Refractory hematologic malignancy.
  • Chronic aspergillosis, aspergilloma, or allergic bronchopulmonary aspergillosis.
  • Treatment with systemic (PO, IV, or inhaled) mold active antifungal therapy for 120 hours immediately before initial dosing. Note: patients with invasive fungal infection caused by a mold with documented resistance to or lack of coverage by the prior SOC in question, may have received >120 hours prior treatment and remain eligible for the study.
  • Evidence of significant hepatic dysfunction.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Cohort A: fosmanogepix (APX001)
Drug: fosmanogepix
IV and oral fosmanogepix
Other Names:
  • APX001
  • E210
Experimental: Cohort B: fosmanogepix (APX001)
Drug: fosmanogepix
IV and oral fosmanogepix
Other Names:
  • APX001
  • E210

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Percentage of Participants Who Died After the First Dose of Study Drug Through Day 42
Time Frame: After first dose on Day 1 through Day 42
Percentage of participants who died after first dose in the study through Day 42 were reported in this outcome measure. This outcome measure included all deaths from Day 1 through Day 42.
After first dose on Day 1 through Day 42

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Percentage of Participants With Global Response Based on Data Review Committee (DRC) Assessment at End of Study Drug Treatment (EOST)
Time Frame: Any day from Day 1 until end of study treatment (any day up to Day 42)
Global response was classified as treatment success (complete or partial response [CR or PR]) or treatment failure (stable response [SR], progression of fungal disease [PD], or death) as determined by DRC. CR: survival within prespecified period of observation (PPOB), resolution of all attributable (att) symptoms and signs of disease and radiological (rad) abnormalities, and mycological (myco) evidence of eradication of disease. PR: survival within POB, improvement in att symptoms and signs of disease and rad abnormalities, and evidence of clearance of cultures or reduction of fungal burden. SR: survival within PPOB, minor or no improvement in fungal disease but no evidence of progression based on composite of clinical, rad and myco criteria, PD or death. PD: evidence of progressive fungal disease based on composite of clinical, rad and myco criteria. Death: death during PPOB, regardless of attribution. Data is presented for EOST which could have preceded Day 42 for some participants.
Any day from Day 1 until end of study treatment (any day up to Day 42)
Percentage of Participants With Treatment Success or Treatment Failure for Global Response Based on Data Review Committee (DRC) Assessment at End of Study Drug Treatment (EOST)
Time Frame: Any day from Day 1 until end of study treatment (any day up to Day 42)
Global response was classified as treatment success (CR or PR) or treatment failure (SR, PD or death) as determined by DRC. CR: survival within prespecified POB, resolution of all attributable symptoms and signs of disease and rad abnormalities and myco evidence of eradication of disease. PR: survival within prespecified POB, improvement in attributable symptoms and signs of disease and rad abnormalities and evidence of clearance of cultures or reduction of fungal burden. SR: survival within prespecified POB, minor or no improvement in fungal disease, but no evidence of progression, based on composite of clinical, rad, and myco criteria, PD or death. PD: evidence of PD based on a composite of clinical, rad, and myco criteria. Death: death during prespecified period of evaluation, regardless of attribution. Percentage of participants with treatment success (CR, PR) and treatment failure (SR, PD, death) along with two-sided exact binomial 80% confidence interval is presented.
Any day from Day 1 until end of study treatment (any day up to Day 42)
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame: Day 1 up to maximum of 31 days of follow up post last dose of study treatment, where maximum treatment duration was 42 days (maximum up to 73 days)
An adverse event was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs were events between first dose of study drug and up to 4 weeks post last dose of study treatment that were absent before treatment or that worsened relative to pretreatment state. An SAE was any untoward medical occurrence that occurred, at any dose: resulted in death; required inpatient hospitalization or prolongation of existing hospitalization; was life-threatening; resulted in persistent or significant disability/ incapacity; was a congenital anomaly/birth defect and other important medical events. AEs included SAEs and all non-SAEs.
Day 1 up to maximum of 31 days of follow up post last dose of study treatment, where maximum treatment duration was 42 days (maximum up to 73 days)
Number of Participants With Clinically Significant Abnormality in Vital Signs
Time Frame: Day 1 up to maximum of 31 days of follow up post last dose of study treatment, where maximum treatment duration was 42 days (maximum up to 73 days)
Vital signs included body temperature, blood pressure, heart rate, respiratory rate, and oxygen saturation. Number of participants with clinically significant abnormality in vital signs as judged by investigator were reported in this outcome measure.
Day 1 up to maximum of 31 days of follow up post last dose of study treatment, where maximum treatment duration was 42 days (maximum up to 73 days)
Number of Participants With Clinically Significant Abnormality in Laboratory Test Evaluations
Time Frame: Day 1 up to maximum of 31 days of follow up post last dose of study treatment, where maximum treatment duration was 42 days (maximum up to 73 days)
Clinical laboratory assessments included serum chemistry (bilirubin, aspartate aminotransferase, alanine aminotransferase, alkaline phosphatase, creatinine, creatine kinase), hematology (including hemoglobin, hematocrit, platelets, leukocytes, lymphocytes, neutrophils, basophils, eosinophils and monocytes), coagulation (including Prothrombin time [PT]/ International normalized ratio [INR]), and urinalysis. Number of participants with clinically significant abnormality in any laboratory parameter as judged by investigator and reported as adverse events were presented.
Day 1 up to maximum of 31 days of follow up post last dose of study treatment, where maximum treatment duration was 42 days (maximum up to 73 days)
Number of Participants With Clinically Significant Abnormal Electrocardiogram (ECG) Findings
Time Frame: Day 1 up to maximum of 31 days of follow up post last dose of study treatment, where maximum treatment duration was 42 days (maximum up to 73 days)
ECG parameters included PR interval, QRS interval, QT interval, QT interval corrected using the Fridericia's formula (QTcF), QT interval corrected using the Bazett's formula (QTcB), RR interval and heart rate. Number of participants with clinically significant abnormal ECG findings as judged by investigator were presented.
Day 1 up to maximum of 31 days of follow up post last dose of study treatment, where maximum treatment duration was 42 days (maximum up to 73 days)
Number of Participants With Clinically Significant Change From Baseline in Physical and Neurological Examinations
Time Frame: Day 1 up to maximum of 31 days of follow up post last dose of study treatment, where maximum treatment duration was 42 days (maximum up to 73 days)
Physical examination included an assessment of general appearance, skin, eyes, heart, chest, abdomen, and a neurological examination. Components of the neurological examination included cranial nerve, sensory, and motor examination; reflex and gait testing; and coordination assessment. Clinically significant changes were judged by investigator.
Day 1 up to maximum of 31 days of follow up post last dose of study treatment, where maximum treatment duration was 42 days (maximum up to 73 days)
Plasma Concentrations of Fosmanogepix
Time Frame: Days 1, 2, 3, 4, 7: Pre-dose and 3 hours post-dose; Days 6, 13, 14: 3 hours post-dose, Day 15: pre-dose
Days 1, 2, 3, 4, 7: Pre-dose and 3 hours post-dose; Days 6, 13, 14: 3 hours post-dose, Day 15: pre-dose

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Time Frame
All-Cause Mortality
Time Frame: Day 84
Day 84

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Basilea Pharmaceutica

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Marc Engelhardt, Basilea Pharmaceutica International Ltd, Allschwil

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 4, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 29, 2022

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 9, 2022

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 22, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 23, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 27, 2020

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 16, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 3, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • APX001-202
  • C4791010 (Other Identifier: Alias Study Number)
  • 2019-001386-33 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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