Phase 2
N=9
An Open-label Study of APX001 for Treatment of Patients With Candidemia/Invasive Candidiasis Caused by Candida Auris
Candidemia · Invasive Candidiases · Candida Infection
Bottom Line
View on ClinicalTrials.gov: NCT04148287 ↗Enrolled (actual)
9
Serious AEs
22.2%
Results posted
Jan 2023
Primary outcome: Primary: Percentage of Participants With Treatment Success at End of Study Treatment (EOST) as Determined by Data Review Committee (DRC) — 88.9 Percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- APX001 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Basilea Pharmaceutica
- Primary completion
- Nov 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Treatment Success at End of Study Treatment (EOST) as Determined by Data Review Committee (DRC) |
88.9 | — |
| SECONDARY Time to First Negative Blood Culture |
6 | — |
| SECONDARY Percentage of Participants With Mycological Outcomes at EOST and 2 and 4 Weeks After EOST |
66.7; 66.7; 66.7 | — |
| SECONDARY Percentage of Participants With Treatment Success at EOST Determined by Investigator |
88.9 | — |
| SECONDARY Percentage of Participants With Treatment Success at 2 and 4 Weeks After EOST Determined by Investigator and by the DRC |
66.7; 66.7; 77.8; 77.8 | — |
| SECONDARY All-Cause Mortality Through Study Day 30 |
11.1 | — |
| SECONDARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) |
9 | — |
Summary
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.
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT04148287). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.