A Phase 2 interventional study of APX001 in Candidemia, Invasive Candidiases and Candida Infection, sponsored by Basilea Pharmaceutica. Completed at 2 sites in South Africa. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-09-16.
Sponsored by Basilea Pharmaceutica · Phase 2, Interventional, and Treatment
This is a multicenter, open-label, single arm study to evaluate the efficacy and safety of APX001 for the treatment of candidemia and/or invasive candidiasis caused by C. auris in patients aged 18 years and over with limited antifungal treatment options.
This is a multicenter, open-label, non-comparative, single arm study to evaluate the efficacy and safety of APX001 for the treatment of candidemia and/or invasive candidiasis caused by C. auris in patients aged 18 years and over with limited antifungal treatment options. The Study Drug Treatment Period will be up to a maximum of 42 days (inclusive of the loading dose [Study Day 1]). There will be a Follow up Period of 4 weeks (+4 days) after EOST. The total duration of participation in the study is up to approximately 10.5 weeks (inclusive of the Screening Period [168 hours prior to Baseline]).
73 studies on the registry are indexed under Candidemia; 5 are open to participants now.
This study's enrollment of 9 is below the median of 63 across 44 interventional studies indexed under Candidemia.
Browse Candidemia studies →Basilea Pharmaceutica is the lead sponsor of 60 studies on the registry; 7 are open to participants now.
Of its 22 completed or terminated interventional studies of FDA-regulated products, 15 (68%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CYP3A (CYP3A4) inhibitors and inducers
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
APX001 IV or oral for up to 42 days
Drug: APX001
Day 1: APX001 1000 mg IV BID over a 3-hour infusion Days 2-3: APX001 600 mg IV QD over a 3-hour infusion Days 4 - 42: APX001 600 mg IV QD over a 3-hour infusion or APX001 800 mg QD oral.
Also known as: fosmanogepix
Percentage of Participants With Treatment Success at End of Study Treatment (EOST) as Determined by Data Review Committee (DRC)
Treatment success was defined as meeting all of the following criteria: 1\) Two consecutive blood cultures negative for Candida species, and/or for participants with a deep-seated site of infection, at least 1 negative tissue culture or aspirate/fluid culture. For participants with a deep-seated site of infection involving visceral organs from which a tissue culture was not obtainable, resolution of the attributable clinical signs of infection recorded at Baseline, and as applicable, radiological improvement associated with the site of infection. 2) Alive at EOST and 3) No concomitant use of any other systemic antifungal therapies through EOST.
Time frame: EOST: any day from Day 1 up to maximum of Day 42
Time to First Negative Blood Culture
Time to first negative blood culture was defined as the number of days from date of first dose of study drug to the date of first negative blood culture plus 1. Participants without a negative blood culture at post-baseline visits were censored at the last assessment date. Kaplan-Meier method was used for analysis.
Time frame: Day 1 up to maximum of Day 42
Percentage of Participants With Mycological Outcomes at EOST and 2 and 4 Weeks After EOST
Mycological outcomes were determined based on eradication and presumed eradication. Eradication was defined as a negative blood (and/or other infection site) culture(s) for Candida species. Presumed eradication (applicable to invasive candidiasis) was defined as clinical resolution of invasive Candida species infection where tissue samples were unavailable. These would be applicable only if there were no concomitant or additional systemic antifungal usage.
Time frame: EOST: any day from Day 1 up to maximum of Day 42, 2 and 4 weeks after EOST
Percentage of Participants With Treatment Success at EOST Determined by Investigator
Treatment success was defined as meeting all of the following criteria: 1\) Two consecutive blood cultures negative for Candida species, and/or for participants with a deep-seated site of infection, at least 1 negative tissue culture or aspirate/fluid culture. For participants with a deep-seated site of infection involving visceral organs from which a tissue culture was not obtainable, resolution of the attributable clinical signs of infection recorded at Baseline, and as applicable, radiological improvement associated with the site of infection. 2) Alive at EOST and 3) No concomitant use of any other systemic antifungal therapies through EOST.
Time frame: EOST: any day from Day 1 up to maximum of Day 42
Percentage of Participants With Treatment Success at 2 and 4 Weeks After EOST Determined by Investigator and by the DRC
Treatment success was defined as meeting all of the following criteria: 1\) Two consecutive blood cultures negative for Candida species, and/or for participants with a deep-seated site of infection, at least 1 negative tissue culture or aspirate/fluid culture. For participants with a deep-seated site of infection involving visceral organs from which a tissue culture was not obtainable, resolution of the attributable clinical signs of infection recorded at Baseline, and as applicable, radiological improvement associated with the site of infection. 2) Alive at EOST and 3) No concomitant use of any other systemic antifungal therapies through EOST.
Time frame: 2 and 4 weeks after EOST (where EOST is any day from Day 1 up to maximum of Day 42)
All-Cause Mortality Through Study Day 30
Percentage of participants who died through study Day 30 is reported in this outcome measure.
Time frame: Day 1 through Day 30
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
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 Week 4 (+4 days) post last dose of study drug that were absent before treatment or that worsened relative to pretreatment state.
Time frame: Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days)
| Milestone | APX001 (Fosmanogepix) |
|---|---|
| Started | 9 |
| Completed | 7 |
| Not completed | 2 |
| Withdrew: Death | 2 |
Treatment success was defined as meeting all of the following criteria: 1\) Two consecutive blood cultures negative for Candida species, and/or for participants with a deep-seated site of infection, at least 1 negative tissue culture or aspirate/fluid culture. For participants with a deep-seated site of infection involving visceral organs from which a tissue culture was not obtainable, resolution of the attributable clinical signs of infection recorded at Baseline, and as applicable, radiological improvement associated with the site of infection. 2) Alive at EOST and 3) No concomitant use of any other systemic antifungal therapies through EOST.
| Percentage of participants | APX001 (Fosmanogepix) |
|---|---|
| Percentage of Participants With Treatment Success at End of Study Treatment (EOST) as Determined by Data Review Committee (DRC) | 88.9 (51.8 to 99.7) |
Time to first negative blood culture was defined as the number of days from date of first dose of study drug to the date of first negative blood culture plus 1. Participants without a negative blood culture at post-baseline visits were censored at the last assessment date. Kaplan-Meier method was used for analysis.
| Days | APX001 (Fosmanogepix) |
|---|---|
| Time to First Negative Blood Culture | 6 (5.0 to 15.0) |
Mycological outcomes were determined based on eradication and presumed eradication. Eradication was defined as a negative blood (and/or other infection site) culture(s) for Candida species. Presumed eradication (applicable to invasive candidiasis) was defined as clinical resolution of invasive Candida species infection where tissue samples were unavailable. These would be applicable only if there were no concomitant or additional systemic antifungal usage.
| Percentage of participants | APX001 (Fosmanogepix) |
|---|---|
| EOST | 66.7 (29.9 to 92.5) |
| 2 Weeks after EOST | 66.7 (29.9 to 92.5) |
| 4 Weeks after EOST | 66.7 (29.9 to 92.5) |
Treatment success was defined as meeting all of the following criteria: 1\) Two consecutive blood cultures negative for Candida species, and/or for participants with a deep-seated site of infection, at least 1 negative tissue culture or aspirate/fluid culture. For participants with a deep-seated site of infection involving visceral organs from which a tissue culture was not obtainable, resolution of the attributable clinical signs of infection recorded at Baseline, and as applicable, radiological improvement associated with the site of infection. 2) Alive at EOST and 3) No concomitant use of any other systemic antifungal therapies through EOST.
| Percentage of participants | APX001 (Fosmanogepix) |
|---|---|
| Percentage of Participants With Treatment Success at EOST Determined by Investigator | 88.9 (51.8 to 99.7) |
Treatment success was defined as meeting all of the following criteria: 1\) Two consecutive blood cultures negative for Candida species, and/or for participants with a deep-seated site of infection, at least 1 negative tissue culture or aspirate/fluid culture. For participants with a deep-seated site of infection involving visceral organs from which a tissue culture was not obtainable, resolution of the attributable clinical signs of infection recorded at Baseline, and as applicable, radiological improvement associated with the site of infection. 2) Alive at EOST and 3) No concomitant use of any other systemic antifungal therapies through EOST.
| Percentage of participants | APX001 (Fosmanogepix) |
|---|---|
| 2 Weeks after EOST: DRC | 66.7 (29.9 to 92.5) |
| 4 Weeks after EOST: DRC | 66.7 (29.9 to 92.5) |
| 2 Weeks after EOST: Investigator | 77.8 (40.0 to 97.2) |
| 4 Weeks after EOST: Investigator | 77.8 (40.0 to 97.2) |
Percentage of participants who died through study Day 30 is reported in this outcome measure.
| Percentage of participants | APX001 (Fosmanogepix) |
|---|---|
| All-Cause Mortality Through Study Day 30 | 11.1 |
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 Week 4 (+4 days) post last dose of study drug that were absent before treatment or that worsened relative to pretreatment state.
| Participants | APX001 (Fosmanogepix) |
|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | 9 |
Collected over Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days). Non-serious events are listed at a 2% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| APX001 (Fosmanogepix) | 2/9 (22.2%) | 2/9 (22.2%) | 9/9 (100%) |
| Event | APX001 (Fosmanogepix) |
|---|---|
| Multiple organ dysfunction syndromeGeneral disorders | 2/9 |
| Cardiac arrestCardiac disorders | 1/9 |
| PneumoniaInfections and infestations | 1/9 |
| HypotensionVascular disorders | 1/9 |
| Event | APX001 (Fosmanogepix) |
|---|---|
| PyrexiaGeneral disorders | 3/9 |
| ConstipationGastrointestinal disorders | 2/9 |
| Pneumonia bacterialInfections and infestations | 2/9 |
| PruritusSkin and subcutaneous tissue disorders | 2/9 |
| HypertensionVascular disorders | 2/9 |
| Angina pectorisCardiac disorders | 1/9 |
| Atrial fibrillationCardiac disorders | 1/9 |
| DiarrhoeaGastrointestinal disorders | 1/9 |
| NauseaGastrointestinal disorders | 1/9 |
| Generalised oedemaGeneral disorders | 1/9 |
Analysis was performed on all enrolled participants.
| Age, Continuous(Years) | APX001 (Fosmanogepix) |
|---|---|
| Mean | 49.8 ± 17.70 |
| Sex: Female, Male(Participants) | APX001 (Fosmanogepix) |
|---|---|
| Female | 3 |
| Male | 6 |
| Ethnicity (NIH/OMB)(Participants) | APX001 (Fosmanogepix) |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 9 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | APX001 (Fosmanogepix) |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 1 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 5 |
| White | 3 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Dec 2019 to primary completion in Nov 2020, 11 months.
Results were first posted in Jan 2023, 2 years 1 month after primary completion.
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This study is completed, as verified in Aug 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Fosmanogepix