Invasive Fungal InfectionsPhase 2AEGISNCT04240886
Open-label Study of APX001 for Treatment of Patients With Invasive Mold Infections Caused by Aspergillus or Rare Molds
A Phase 2 study testing fosmanogepix in people with Invasive Fungal Infections, run by Basilea Pharmaceutica at 15 sites in 4 countries.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Invasive Fungal Infections
- What is being tested?
- fosmanogepix
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 15 sitesin 4 countries
- Study dates
- 4 Jan 2020to 9 May 2022
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
Who cannot join
- 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.
Trial locations and contacts
15 sites in 4 countries.
15 sites in 4 countries
United States6 sites
City of Hope (City of Hope National Medical Center, City of Hope Medical Center)
Duarte, California, 91010
IP Address : City of Hope Investigational Drug Services (IDS)
Duarte, California, 91010
Duke Department of Pharmacy/Investigational Drug Service (IDS)
Durham, North Carolina, 27710
Duke Medicine Pavilion
Durham, North Carolina, 27710
Duke University Medical Center (Duke South Clinic)
Durham, North Carolina, 27710
Duke University Medical Center(Duke Hospital)
Durham, North Carolina, 27710
Israel4 sites
Pharmacy
Haifa, 3109601
Rambam Medical Center
Haifa, 3109601
Pharmacy
Ramat Gan, 5262160
Sheba Medical Center, Tel Hashomer
Ramat Gan, 5262160
Belgium3 sites
Cliniques Universitaires de Bruxelles Hôpital Erasme - Department of Infectious Diseases
Brussels, 1070
UZ Leuven - Department of Haematology
Leuven, 3000
CHU UCL Namur - Mont- Godinne University Hospital - Intensive Care Unit
Yvoir, 5530
Germany2 sites
Universitätsmedizin der Johannes Gutenberg-Universität Mainz III
Mainz, 55131
Klinikum Neuperlach, Klinik fur Hamatologie und Onkologie
München, Bavaria, 81737
About this study
Official title: 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).
- Conditions
- Invasive Fungal Infections
- Being tested
- fosmanogepix
- Keywords
- APX001, APX001A, fosmanogepix, manogepix, aspergillus, aspergillosis, covid, covid19, mold, mould, pulmonary aspergillosis, pulmonary aspergillus, galactomannan, Karius, SARS-CoV-2, fusarium, mucorales, mucor, rhizopus, scedosporium
- Medical subject headings
- Invasive Fungal Infections, Aspergillosis, COVID-19, Pulmonary Aspergillosis, Mycoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
fosmanogepixDrug
Also called APX001, E210
IV and oral fosmanogepix
| Study group | Role | What they receive |
|---|---|---|
| Cohort A: fosmanogepix (APX001) | Experimental | Not described Receives: Drug: fosmanogepix |
| Cohort B: fosmanogepix (APX001) | Experimental | Not described Receives: Drug: fosmanogepix |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Who Died After the First Dose of Study Drug 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.
Measured: After first dose on Day 1 through Day 42
Secondary outcomes (8)
Percentage of Participants With Global Response Based on Data Review Committee (DRC) Assessment at End of Study Drug Treatment (EOST)
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.
Measured: 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)
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.
Measured: 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)
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.
Measured: 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
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.
Measured: 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
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.
Measured: 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
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.
Measured: 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
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.
Measured: 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
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2020
- Study start4 Jan 2020
- Primary completion29 Mar 2022
- Study completion9 May 2022
Why it stopped: The Sponsor made a strategic decision to terminate the study, in order to prioritize a randomized comparative Phase 3 trial in the same indication. This decision was not based on any safety concerns
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Invasive Fungal InfectionsStudies 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Basilea Pharmaceutica
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Marc Engelhardt, Study director, Basilea Pharmaceutica International Ltd, Allschwil
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04240886
- Sponsor's protocol ID
- APX001-202
- Other IDs
- C4791010, 2019-001386-33, 2019
- Acronym
- AEGIS
- Status last verified
- Aug 2025
- First submitted
- 22 Jan 2020
- Last updated
- 16 Sep 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Hodges MR, Tawadrous M, Cornely OA, Thompson GR, Slavin MA, Maertens JA, Dadwal SS, Rahav G, Hazel S, Almas M, Jakate A, Pypstra R. Fosmanogepix for the Treatment of Invasive Mold Diseases Caused by Aspergillus Species and Rare Molds: A Phase 2, Open-Label Study (AEGIS). Clin Infect Dis. 2025 Dec 24;81(5):e302-e309. doi: 10.1093/cid/ciaf185.
PubMed 40203286
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT04240886
- Acronym / sponsor ID
- AEGIS / APX001-202
- Conditions
- Invasive Fungal Infections
- Keywords
- APX001, APX001A, fosmanogepix, manogepix, aspergillus, aspergillosis, covid, covid19
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The Sponsor made a strategic decision to terminate the study, in order to prioritize a randomized comparative Phase 3 trial in the same indication. This decision was not based on any safety concerns
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Basilea PharmaceuticaIndustry (pharma, biotech, devices)
- Study start
- 4 Jan 2020 (actual)
- Primary completion
- 29 Mar 2022 (actual)
- Study completion
- 9 May 2022 (actual)
- First posted
- 27 Jan 2020 (actual)
- Results
- Posted 19 May 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort A: fosmanogepix (APX001) | Experimental | Drug: fosmanogepix |
| Cohort B: fosmanogepix (APX001) | Experimental | Drug: fosmanogepix |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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).
Key exclusion criteria (4 listed)
- 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.
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 |
|---|---|---|
| fosmanogepixAlso: APX001, E210IV and oral fosmanogepix | Drug | Cohort A: fosmanogepix (APX001), Cohort B: fosmanogepix (APX001) |
Primary outcome
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.
8 secondary outcomes
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)
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)
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)
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)
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)
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)
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)
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 40203286Hodges MR, Tawadrous M, Cornely OA, Thompson GR, Slavin MA, Maertens JA, Dadwal SS, Rahav G, Hazel S, Almas M, Jakate A, Pypstra R. Fosmanogepix for the Treatment of Invasive Mold Diseases Caused by Aspergillus Species and Rare Molds: A Phase 2, Open-Label Study (AEGIS). Clin Infect Dis. 2025 Dec 24;81(5):e302-e309. doi: 10.1093/cid/ciaf185.
- Individual participant dataSharing planned: No
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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.
Marc Engelhardt study director
Basilea Pharmaceutica International Ltd, Allschwil
15 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing fosmanogepix in people with Invasive Fungal Infections, run by Basilea Pharmaceutica at 15 sites in 4 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 4 inclusion and 4 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: stopped early, as of the sponsor's last update on 16 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, Israel, Belgium, Germany. 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. 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 19 May 2023). 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?
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). 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 NCT04240886, which also lists 1 linked publication.
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 16 Sep 2025.