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

NCT ID: NCT04148287

Last Updated: 2025-09-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-12-13
• Study Completion Date: 2020-12-31

Brief 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.

Detailed Description

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]).

Conditions

Candidemia; Invasive Candidiases; Candida Infection

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

APX001

APX001 IV or oral for up to 42 days

Group Type: EXPERIMENTAL

APX001

Intervention Type: 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.

Interventions

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.

Intervention Type: DRUG

Other Intervention Names

fosmanogepix

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Basilea Pharmaceutica

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Marc Engelhardt

Role: STUDY_DIRECTOR

Basilea Pharmaceutica International Ltd, Allschwil

Locations

Netcare Union Hospital Trauma Surgeons

Alberton, Gauteng, South Africa

Milpark Academic Trauma Centre

Johannesburg, Gauteng, South Africa

Countries

South Africa

References

Vazquez JA, Pappas PG, Boffard K, Paruk F, Bien PA, Tawadrous M, Ople E, Wedel P, Oborska I, Hodges MR. Clinical Efficacy and Safety of a Novel Antifungal, Fosmanogepix, in Patients with Candidemia Caused by Candida auris: Results from a Phase 2 Trial. Antimicrob Agents Chemother. 2023 May 17;67(5):e0141922. doi: 10.1128/aac.01419-22. Epub 2023 Apr 6.

Reference Type: RESULT

PMID: 37022196 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

C4791011

Identifier Type: OTHER

Identifier Source: secondary_id

APX001-203

Identifier Type: -

Identifier Source: org_study_id