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

NCT04148287 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: APX001-203C4791011
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 2

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.

Conditions

Candidemia; Invasive Candidiases; Candida Infection

Outcome Measures

Primary Outcomes (1)

• 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.

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

Secondary Outcomes (6)

• Time to First Negative Blood Culture

  Day 1 up to maximum of Day 42

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

  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

  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

  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

  Day 1 through Day 30

Study Arms (1)

APX001

EXPERIMENTAL

APX001 IV or oral for up to 42 days

Drug: APX001

Interventions

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.

Also known as: fosmanogepix

APX001

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Basilea Pharmaceutica: lead

Study Sites (2)

Netcare Union Hospital Trauma Surgeons

Alberton, Gauteng, 1449, South Africa

Location

Milpark Academic Trauma Centre

Johannesburg, Gauteng, 2193, South Africa

Location

Related Publications (1)

• 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.

  PMID: 37022196 RESULT

MeSH Terms

Conditions

Candidemia; Candidiasis, Invasive; Candidiasis

Condition Hierarchy (Ancestors)

Mycoses; Bacterial Infections and Mycoses; Infections; Invasive Fungal Infections; Fungemia; Sepsis; Systemic Inflammatory Response Syndrome; Inflammation; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Study Director
• Organization: Basilea Pharmaceutica International Ltd, Allschwil

marc.engelhardt@basilea.com

+41 79 701 0551

Study Officials

• Marc Engelhardt

  Basilea Pharmaceutica International Ltd, Allschwil

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open-label, single arm intervention. All patients will receive APX001. On Days 1-3, APX001 will be administered intravenously. Thereafter, if the patient is able to take oral medication, APX001 tablets will be taken orally.

Study Record Dates

• First Submitted: October 30, 2019
• First Posted: November 1, 2019
• Study Start: December 13, 2019
• Primary Completion: November 30, 2020
• Study Completion: December 31, 2020
• Last Updated: September 16, 2025
• Results First Posted: January 9, 2023

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

ZA (2)