An Efficacy and Safety Study of APX001 in Non-Neutropenic Patients With Candidemia

An Open-Label Study to Evaluate the Efficacy and Safety of APX001 in Non Neutropenic Patients With Candidemia, With or Without Invasive Candidiasis, Inclusive of Patients With Suspected Resistance to Standard of Care Antifungal Treatment

This is a multicenter, open-label, non-comparative, single-arm study to evaluate the efficacy and safety of APX001 for the first-line treatment for candidemia including suspected or confirmed antifungal-resistant candidemia in non-neutropenic patients 18 yeas of age and older.

Suspicion of antifungal-resistant candidemia is sufficient (documented resistance is not required for enrollment). The Study Drug Treatment Period of APX001 will be a maximum of 14 days. After completion of 14 days study drug therapy, if further antifungal treatment is indicated to complete treatment of candidemia in accordance with standard practice guidelines, fluconazole (unless susceptibility results warrant alternative antifungal therapy) may commence for up to a further 7 days. There will be a Follow up Period of 4 weeks (+4 days) after EOT. The total duration of participation in the study is up to approximately 7.5 weeks.

This study will be conducted at approximately 20 sites in the United States and globally.

Study Overview

• Status: Completed
• Conditions: Candidemia
• Intervention / Treatment: Drug: APX001

• Study Type: Interventional
• Enrollment (Actual): 21
• Phase: Phase 2

Contacts and Locations

Study Locations

• Belgium
  • Brussels, Belgium
    • Institut Jules Bordet
  • Brussels, Belgium, 1070
    • Hôpital Erasme
  • Brussels, Belgium
    • Universite Libre de Bruxelles (ULB) - Hopital Erasme
  • Brussels, Belgium, 1000
    • Institut Jules Bordet,Service De Microbiologie
  • Lodelinsart, Belgium
    • CHU de Charleroi - Hopital Civil Marie Curie
  • Yvoir, Belgium
    • University Hospital Mont-Godinne
  • Yvoir, Belgium, 5530
    • Mont-Godinne University Hospital
• Germany
  • Cologne, Germany
    • Klinik I fur Innere Medizin- Uniklinik Koln
  • Heidelberg, Germany
    • University Hospital Heidelberg
  • Mainz, Germany
    • Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz - III. Medizinische Klinik Haematologie/Onkologie
• Israel
  • Haifa, Israel
    • Rambam Medical Center
  • Haifa, Israel, 3109601
    • Infectious Diseases Unit, Rambam Medical Center
  • Tel Aviv, Israel
    • Sourasky Medical Center
  • Tel Aviv, Israel, 6423906
    • Infectious Diseases Unit
  • Tel Litwinsky, Israel
    • Sheba Medical Center
  • Tel Litwinsky, Israel, 52621
    • Infectious Diseases Unit,Sheba Medical Center
• Spain
  • Alicante, Spain
    • Hospital General Universitario de Alicante
  • Barcelona, Spain, 08035
    • Hospital Universitari Vall d'Hebron
  • Barcelona, Spain
    • Hospital VLL D Hebron
  • Barcelona
    • Terrassa, Barcelona, Spain
      • Hospital Universitario Mutua de Terrassa
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35249
      • University of Alabama at Birmingham (UAB)
    • Birmingham, Alabama, United States, 35233
      • University of Alabama at Birmingham School of Medicine
  • California
    • Davis, California, United States, 95616
      • University of California, Davis
    • Sacramento, California, United States, 95817
      • University Of California-Davis Medical Center
  • Georgia
    • Augusta, Georgia, United States, 30901
      • Augusta University (Georgia Regents University)
  • Illinois
    • Chicago, Illinois, United States, 60637
      • University of Chicago
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Washington University
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Hospital Medical Center
  • Texas
    • Houston, Texas, United States, 77030
      • University of Texas- Health Science Center and Medical School at Houston

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Key Inclusion Criteria:

• Provision of written consent
• Adults ages 18 and above male or female
• New diagnosis of candidemia
• Able to have pre-existing intravascular catheters removed and replaced (as necessary)

Key Exclusion Criteria:

• neutropenia
• deep-seated Candida-related infections
• hepatosplenic candidiasis
• received more than 2 days of prior systemic antifungal treatment for current candidemia episode
• severe hepatic impairment

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: APX001 Treatment	Drug: APX001; APX001

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment Success at End of Study Treatment (EOST) as Determined by the Data Review Committee (DRC)	Treatment Success is defined as meeting all of the following criteria: Two consecutive blood cultures negative for Candida spp. Alive at EOST No concomitant use of any other systemic antifungal therapies through end of study treatment	One to forty-two days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to First Negative Blood Culture	Time to first negative blood culture was defined as the number of days from first dose date of study drug to the date of first post-Baseline negative blood culture + 1. Patients without a negative blood culture at post-Baseline visits were censored at the last assessment date.	One to forty-nine days
Percentage of Patients With Mycological Outcomes at End of Study Treatment (EOST), End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT)		End of study treatment (EOST), end of treatment (EOT), and 2 and 4 weeks after end of treatment (EOT)
Percentage of Patients With Treatment Success at End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT)		2 and 4 weeks after end of treatment (EOT)
Overall Survival at Study Day 30		Day 30
Number of Patients With Treatment Emergent Adverse Events (TEAEs)		One to forty-nine days

Collaborators and Investigators

• Sponsor: Basilea Pharmaceutica
• Investigators: Study Director: Marc Engelhardt, Basilea Pharmaceutica International Ltd, Allschwil

Publications and helpful links

General Publications

• Pappas PG, Vazquez JA, Oren I, Rahav G, Aoun M, Bulpa P, Ben-Ami R, Ferrer R, Mccarty T, Thompson GR, Schlamm H, Bien PA, Barbat SH, Wedel P, Oborska I, Tawadrous M, Hodges MR. Clinical safety and efficacy of novel antifungal, fosmanogepix, for the treatment of candidaemia: results from a Phase 2 trial. J Antimicrob Chemother. 2023 Oct 3;78(10):2471-2480. doi: 10.1093/jac/dkad256.

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): October 3, 2018
• Primary Completion (Actual): March 31, 2020
• Study Completion (Actual): July 2, 2020

Study Registration Dates

• First Submitted: July 19, 2018
• First Submitted That Met QC Criteria: July 19, 2018
• First Posted (Actual): July 27, 2018

Study Record Updates

• Last Update Posted (Estimated): September 16, 2025
• Last Update Submitted That Met QC Criteria: September 3, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Invasive Fungal Infections; Pathologic Processes; Infections; Sepsis; Systemic Inflammatory Response Syndrome; Inflammation; Bacterial Infections and Mycoses; Mycoses; Candidiasis, Invasive; Candidiasis; Fungemia; Pathological Conditions, Signs and Symptoms; Candidemia
• Other Study ID Numbers: APX001-201; C4791009 (Other Identifier: Alias Study Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No