Trial · NCT04240886
Open-label Study of APX001 for Treatment of Patients With Invasive Mold Infections Caused by Aspergillus or Rare Molds
NCT04240886, Phase 2, Terminated, Basilea Pharmaceutica; results first posted 2023-05-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Open-label Study of APX001 for Treatment of Patients With Invasive Mold Infections Caused by Aspergillus or Rare Molds. Trials, record NCT04240886. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04240886
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Open-label Study of APX001 for Treatment of Patients With Invasive Mold Infections Caused by Aspergillus or Rare Molds
- NCT number
- NCT04240886
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 21 participants, actual
- Lead sponsor
- Basilea Pharmaceutica
- Conditions
- Invasive Fungal Infections
- Interventions
- fosmanogepix
- First posted
- 2020-01-27
- Last updated
- 2025-09-16
- Other registries' IDs
- eudract:2019-001386-33
- Results first posted
- 2023-05-19
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Who Died After the First Dose of Study Drug Through Day 42 | 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. |
| Secondary | Percentage of Participants With Global Response Based on Data Review Committee (DRC) Assessment at End of Study Drug Treatment (EOST) | 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. |
| Secondary | 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) | 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. |
| Secondary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (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) | 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. |
| Secondary | Number of Participants With Clinically Significant Abnormality in Vital Signs | 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. |
| Secondary | Number of Participants With Clinically Significant Abnormality in Laboratory Test Evaluations | 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. |
| Secondary | Number of Participants With Clinically Significant Abnormal Electrocardiogram (ECG) Findings | 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. |
| Secondary | Number of Participants With Clinically Significant Change From Baseline in Physical and Neurological Examinations | 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. |
| Secondary | Plasma Concentrations of Fosmanogepix | 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 |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants Who Died After the First Dose of Study Drug Through Day 42 | After first dose on Day 1 through Day 42 | The modified Intent-to-Treat (mITT) population included all participants who entered in the study, received at least 1 dose of study drug, and had a diagnosis of proven or probable IMI confirmed by the DRC. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Global Response Based on Data Review Committee (DRC) Assessment at End of Study Drug Treatment (EOST) | Any day from Day 1 until end of study treatment (any day up to Day 42) | mITT population included all participants who entered in the study, received at least 1 dose of study drug, and had a diagnosis of proven or probable IMI confirmed by the DRC. | Percentage of participants | NUMBER |
| SECONDARY | 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) | Any day from Day 1 until end of study treatment (any day up to Day 42) | mITT population included all participants who entered in the study, received at least 1 dose of study drug, and had a diagnosis of proven or probable IMI confirmed by the DRC. | Percentage of participants | NUMBER |
| SECONDARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (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) | Safety population included all participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Clinically Significant Abnormality in Vital Signs | 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) | Safety population included all participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Clinically Significant Abnormality in Laboratory Test Evaluations | 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) | Safety population included all participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Clinically Significant Abnormal Electrocardiogram (ECG) Findings | 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) | Safety population included all participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Clinically Significant Change From Baseline in Physical and Neurological Examinations | 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) | Safety population included all participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Plasma Concentrations of Fosmanogepix | 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 | The Pharmacokinetic (PK) Population included all participants who received any amount of study drug and had evaluable PK data. Here, Number Analyzed signifies participants evaluable at specified time points. | nanogram per milliliter | GEOMETRIC_MEAN |
Source ClinicalTrials.gov
Results by group28
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants Who Died After the First Dose of Study Drug Through Day 42 | APX001 (Fosmanogepix): Cohort A | 25.0 | 80% Confidence Interval 12.7 to 41.5 | Percentage of participants | |
| Percentage of Participants With Global Response Based on Data Review Committee (DRC) Assessment at End of Study Drug Treatment (EOST) | APX001 (Fosmanogepix): Cohort A | CR | 20.0 | Percentage of participants | |
| Percentage of Participants With Global Response Based on Data Review Committee (DRC) Assessment at End of Study Drug Treatment (EOST) | APX001 (Fosmanogepix): Cohort A | PR | 20.0 | Percentage of participants | |
| Percentage of Participants With Global Response Based on Data Review Committee (DRC) Assessment at End of Study Drug Treatment (EOST) | APX001 (Fosmanogepix): Cohort A | SR | 10.0 | Percentage of participants | |
| Percentage of Participants With Global Response Based on Data Review Committee (DRC) Assessment at End of Study Drug Treatment (EOST) | APX001 (Fosmanogepix): Cohort A | PD | 30.0 | Percentage of participants | |
| Percentage of Participants With Global Response Based on Data Review Committee (DRC) Assessment at End of Study Drug Treatment (EOST) | APX001 (Fosmanogepix): Cohort A | Death | 20.0 | Percentage of participants | |
| 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) | APX001 (Fosmanogepix): Cohort A | Treatment Success | 40.0 | 80% Confidence Interval 24.9 to 56.7 | Percentage of participants |
| 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) | APX001 (Fosmanogepix): Cohort A | Treatment Failure | 60.0 | 80% Confidence Interval 43.3 to 75.1 | Percentage of participants |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | APX001 (Fosmanogepix): Cohort A | TEAEs | 21 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | APX001 (Fosmanogepix): Cohort A | SAEs | 13 | Participants |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Phase | STARTED | APX001 (Fosmanogepix): Cohort A | 21 | |
| Treatment Phase | COMPLETED | APX001 (Fosmanogepix): Cohort A | 11 | |
| Treatment Phase | NOT COMPLETED | APX001 (Fosmanogepix): Cohort A | 10 | |
| Follow-up Phase | STARTED | APX001 (Fosmanogepix): Cohort A | 17 | |
| Follow-up Phase | COMPLETED | APX001 (Fosmanogepix): Cohort A | 13 | |
| Follow-up Phase | NOT COMPLETED | APX001 (Fosmanogepix): Cohort A | 4 |
Source ClinicalTrials.gov
Adverse events161
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | APX001 (Fosmanogepix): Cohort A | 2 | 21 | |
| other | Disseminated intravascular coagulation | Blood and lymphatic system disorders | APX001 (Fosmanogepix): Cohort A | 1 | 21 | |
| other | Eosinophilia | Blood and lymphatic system disorders | APX001 (Fosmanogepix): Cohort A | 3 | 21 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | APX001 (Fosmanogepix): Cohort A | 2 | 21 | |
| other | Pancytopenia | Blood and lymphatic system disorders | APX001 (Fosmanogepix): Cohort A | 1 | 21 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | APX001 (Fosmanogepix): Cohort A | 1 | 21 | |
| other | Palpitations | Cardiac disorders | APX001 (Fosmanogepix): Cohort A | 1 | 21 | |
| other | Pericarditis | Cardiac disorders | APX001 (Fosmanogepix): Cohort A | 1 | 21 | |
| other | Sinus tachycardia | Cardiac disorders | APX001 (Fosmanogepix): Cohort A | 1 | 21 | |
| other | Tachycardia | Cardiac disorders | APX001 (Fosmanogepix): Cohort A | 3 | 21 |
1 to 10 of 161Source ClinicalTrials.gov
See all 161 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | APX001 (Fosmanogepix): Cohort A | 9 | 21 |
| Total, other adverse events | APX001 (Fosmanogepix): Cohort A | 21 | 21 |
| Total, serious adverse events | APX001 (Fosmanogepix): Cohort A | 13 | 21 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 40203286 | RESULT |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
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