Skip to main content
OpenTrials
Completed

NCT Number: NCT04148287

An Open-label Study of APX001 for Treatment of Patients With Candidemia/Invasive Candidiasis Caused by Candida Auris

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Candidemia Bacterial Infections and Mycoses Candida Infection Candidiasis Candidiasis, Invasive Fungemia Infections Inflammation Invasive Candidiases Invasive Fungal Infections Mycoses Pathologic Processes Pathological Conditions, Signs and Symptoms Sepsis Systemic Inflammatory Response Syndrome

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Netcare Union Hospital Trauma Surgeons, Alberton, Gauteng, South Africa

Loading trial locations.

About this study

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]).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

APX001

Drug

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.

Other names: fosmanogepix

Primary outcomes

  1. Percentage of Participants With Treatment Success at End of Study Treatment (EOST) as Determined by Data Review Committee (DRC)

    Time frame: EOST: any day from Day 1 up to maximum of Day 42

    Treatment success was defined as meeting all of the following criteria:

    • 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.

Secondary outcomes

  1. Time to First Negative Blood Culture

    Time frame: Day 1 up to maximum of Day 42

    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.

  2. Percentage of Participants With Mycological Outcomes at EOST and 2 and 4 Weeks After EOST

    Time frame: EOST: any day from Day 1 up to maximum of Day 42, 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.

  3. Percentage of Participants With Treatment Success at EOST Determined by Investigator

    Time frame: EOST: any day from Day 1 up to maximum of Day 42

    Treatment success was defined as meeting all of the following criteria:

    • 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.

  4. Percentage of Participants With Treatment Success at 2 and 4 Weeks After EOST Determined by Investigator and by the DRC

    Time frame: 2 and 4 weeks after EOST (where EOST is any day from Day 1 up to maximum of Day 42)

    Treatment success was defined as meeting all of the following criteria:

    • 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.

  5. All-Cause Mortality Through Study Day 30

    Time frame: Day 1 through Day 30

    Percentage of participants who died through study Day 30 is reported in this outcome measure.

  6. Number of Participants With Treatment Emergent Adverse Events (TEAEs)

    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)

    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.

Sponsors and collaborators

Lead sponsor

Basilea Pharmaceutica

Industry

Registry information

Official study title

An Open-Label Study to Evaluate the Efficacy and Safety of APX001 in Patients With Candidemia and/or Invasive Candidiasis Caused by Candida Auris

Acronym: APEX

Important dates

Study start
2019
Primary completion
2020
Study completion
2020
First posted
Nov 1, 2019
Registry last updated
Sep 16, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.