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New drug shows promise against superbug fungus in small trial

NCT ID NCT04148287

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 26, 2026 · Updated 1 time

Summary

This Phase 2 trial tested an experimental antifungal drug called APX001 (fosmanogepix) in 9 adults with blood or deep-organ infections caused by Candida auris, a fungus that often resists standard treatments. The drug was given by IV or mouth for up to 42 days. The goal was to see if the infection cleared and patients survived without needing other antifungals. Because the study was very small and had no placebo group, the results are encouraging but not conclusive.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
APX001 (fosmanogepix), an antifungal drug given by IV or mouth
What this could lead to
If it works, this could offer a new treatment option for people with dangerous Candida auris infections that resist standard antifungals.
What could go wrong
This was a very small study (9 people) with no comparison group, so results are preliminary. The drug may not work as well in larger, more diverse groups, and side effects are still being studied.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

9 people

The number who actually took part.

Started

Dec 2019

Finished

Dec 2020

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Limited or no treatment options due to resistance, contraindication, intolerance or lack of clinical response to standard of care antifungal therapy, as advocated by the relevant regional/country treatment guidelines * Established mycological and clinical diagnosis of candidemia and/or invasive candidiasis caused by Candida auris * Able to have pre-existing intravascular catheters removed and replaced (if necessary) * Females of childbearing potential with male partners, and males with female partner(s) of childbearing potential, must agree to use 2 forms of highly effective contraception throughout the duration of the study and for 90 days following the last study drug administration. Females of childbearing potential must have a negative urine pregnancy test within 96 hours prior study entry. * Wiling to participate in the study, willing to give written informed consent, and willing to comply with the study restrictions; where permitted by local regulations, written informed consent from a legal authorized representative (LAR) will be obtained for patients who are unable to give consent Exclusion Criteria: * Life expectancy of less than 7 days in the opinion of the Investigator * Human immunodeficiency virus-infected patients who are receiving antiretroviral therapy that are moderate to strong inducers of CYP3A4, or who have detectable viremia, or who have had an active opportunistic infection within 6 months prior * Alanine aminotransferase or aspartate aminotransferase greater than or equal to 5 times the upper limit of normal * Total bilirubin greater than 3 time the upper limit of normal, unless isolated hyperbilirubinemia or due to documented Gilbert's disease * Pregnant or lactating female patient * Inappropriate fungal infection source control * Investigational drug administered within 30 days prior to dosing or five half-lives whichever is longer * Diagnosis of deep-seated Candida-related infections causing hardware associated septic arthritis, osteomyelitis, endocarditis, myocarditis, hepatosplenic candidiasis, or a central nervous system infection or site of infection that would require antifungal therapy to exceed the maximal duration of study drug treatment

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Milpark Academic Trauma Centre

    Johannesburg, Gauteng, 2193, South Africa

  • Netcare Union Hospital Trauma Surgeons

    Alberton, Gauteng, 1449, South Africa

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