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

NCT03604705 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: APX001-201C4791009
• Study Type: interventional
• Target: 21
• Locations: 5 countries
• Sites: 29

Brief Summary

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.

Conditions

Candidemia

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (5)

• Time to First Negative Blood Culture

  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

Study Arms (1)

APX001 Treatment

EXPERIMENTAL

Drug: APX001

Interventions

APX001 DRUG

APX001

APX001 Treatment

Eligibility Criteria

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

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Basilea Pharmaceutica: lead

Study Sites (29)

University of Alabama at Birmingham School of Medicine

Birmingham, Alabama, 35233, United States

Location

University of Alabama at Birmingham (UAB)

Birmingham, Alabama, 35249, United States

Location

University of California, Davis

Davis, California, 95616, United States

Location

University of California-Davis Medical Center

Sacramento, California, 95817, United States

Location

Augusta University (Georgia Regents University)

Augusta, Georgia, 30901, United States

Location

University of Chicago

Chicago, Illinois, 60637, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

Duke University Hospital Medical Center

Durham, North Carolina, 27710, United States

Location

University of Texas- Health Science Center and Medical School at Houston

Houston, Texas, 77030, United States

Location

Institut Jules Bordet,Service De Microbiologie

Brussels, 1000, Belgium

Location

Hopital Erasme

Brussels, 1070, Belgium

Location

Institut Jules Bordet

Brussels, Belgium

Location

Universite Libre de Bruxelles (ULB) - Hopital Erasme

Brussels, Belgium

Location

CHU de Charleroi - Hopital Civil Marie Curie

Lodelinsart, Belgium

Location

Mont-Godinne University Hospital

Yvoir, 5530, Belgium

Location

University Hospital Mont-Godinne

Yvoir, Belgium

Location

Klinik I fur Innere Medizin- Uniklinik Koln

Cologne, Germany

Location

University Hospital Heidelberg

Heidelberg, Germany

Location

Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz - III. Medizinische Klinik Haematologie/Onkologie

Mainz, Germany

Location

Infectious Diseases Unit, Rambam Medical Center

Haifa, 3109601, Israel

Location

Rambam Medical Center

Haifa, Israel

Location

Infectious Diseases Unit

Tel Aviv, 6423906, Israel

Location

Sourasky Medical Center

Tel Aviv, Israel

Location

Infectious Diseases Unit,Sheba Medical Center

Tel Litwinsky, 52621, Israel

Location

Sheba Medical Center

Tel Litwinsky, Israel

Location

Hospital Universitario Mutua de Terrassa

Terrassa, Barcelona, Spain

Location

Hospital General Universitario de Alicante

Alicante, Spain

Location

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital VLL D Hebron

Barcelona, Spain

Location

Related Publications (1)

• 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.

  PMID: 37596890 RESULT

MeSH Terms

Conditions

Candidemia

Condition Hierarchy (Ancestors)

Candidiasis, Invasive; Candidiasis; 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
• 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

Study Record Dates

• First Submitted: July 19, 2018
• First Posted: July 27, 2018
• Study Start: October 3, 2018
• Primary Completion: March 31, 2020
• Study Completion: July 2, 2020
• Last Updated: September 16, 2025
• Results First Posted: August 5, 2021

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

IL (6); ES (4); BE (7); US (9); DE (3)