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

Phase 2

Completed

• Conditions: Candidemia
• Interventions: Drug: APX001

• Registration Number: NCT03604705
• Lead Sponsor: Basilea Pharmaceutica

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-07-19
• Study First Submitted QC: 2018-07-19
• Study First Posted: 2018-07-27
• Study Start: 2018-10-03
• Primary Completion: 2020-03-31
• Study Completion: 2020-07-02
• Results First Submitted: 2021-05-24
• Results First Submitted QC: 2021-07-14
• Results First Posted: 2021-08-05
• Status Verified: 2024-05
• Last Update Submitted: 2024-05-16
• Last Update Posted: 2024-06-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 21

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
APX001 Treatment	APX001	-

Primary Outcome Measures

Name	Time	Method
Treatment Success at End of Study Treatment (EOST) as Determined by the Data Review Committee (DRC)	One to forty-two days	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

Secondary Outcome Measures

Name	Time	Method
Time to First Negative Blood Culture	One to forty-nine days	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.
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

Trial Locations

Locations (29)

Augusta University (Georgia Regents University); 🇺🇸 Augusta, Georgia, United States

University of Chicago; 🇺🇸 Chicago, Illinois, United States

Universite Libre de Bruxelles (ULB) - Hopital Erasme; 🇧🇪 Brussels, Belgium

University Hospital Mont-Godinne; 🇧🇪 Yvoir, Belgium

Klinik I fur Innere Medizin- Uniklinik Koln; 🇩🇪 Cologne, Germany

Hopital Erasme; 🇧🇪 Brussels, Belgium

Hospital General Universitario de Alicante; 🇪🇸 Alicante, Spain

Infectious Diseases Unit; 🇮🇱 Tel Aviv, Israel

Hospital VLL D Hebron; 🇪🇸 Barcelona, Spain

Institut Jules Bordet,Service De Microbiologie; 🇧🇪 Brussels, Belgium

Hospital Universitario Mutua de Terrassa; 🇪🇸 Terrassa, Barcelona, Spain

Washington University; 🇺🇸 Saint Louis, Missouri, United States

Infectious Diseases Unit, Rambam Medical Center; 🇮🇱 Haifa, Israel

Sheba Medical Center; 🇮🇱 Tel HaShomer, Israel

Hospital Universitari Vall d'Hebron; 🇪🇸 Barcelona, Spain

Sourasky Medical Center; 🇮🇱 Tel Aviv, Israel

University of California, Davis; 🇺🇸 Davis, California, United States

University of Alabama at Birmingham School of Medicine; 🇺🇸 Birmingham, Alabama, United States

University of Alabama at Birmingham (UAB); 🇺🇸 Birmingham, Alabama, United States

University of Texas- Health Science Center and Medical School at Houston; 🇺🇸 Houston, Texas, United States

Duke University Hospital Medical Center; 🇺🇸 Durham, North Carolina, United States

Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz - III. Medizinische Klinik Haematologie/Onkologie; 🇩🇪 Mainz, Germany

Infectious Diseases Unit,Sheba Medical Center; 🇮🇱 Tel Hashomer, Israel

Institut Jules Bordet; 🇧🇪 Brussels, Belgium

CHU de Charleroi - Hopital Civil Marie Curie; 🇧🇪 Lodelinsart, Belgium

University Hospital Heidelberg; 🇩🇪 Heidelberg, Germany

Rambam Medical Center; 🇮🇱 Haifa, Israel

University of California-Davis Medical Center; 🇺🇸 Sacramento, California, United States

Mont-Godinne University Hospital; 🇧🇪 Yvoir, Belgium