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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Candidemia

Intervention / Treatment
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* Drug: APX001


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: APX001 Treatment<br> | Drug: APX001<br>* APX001<br>|

What is the study measuring?
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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 |