An Open-label Study of APX001 for Treatment of Patients With Candidemia/Invasive Candidiasis Caused by Candida Auris
Condition: Candidemia; Invasive Candidiases; Candida Infection
MeSH terms: Candidiasis; Candidemia; Candidiasis, Invasive
Intervention: APX001
Intervention description: 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.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Intervention model description: 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.
Masking: None (Open Label)
Enrollment: 9.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

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.
    

Eligibility criteria:

        Inclusion Criteria:

          -  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

        Exclusion Criteria:

          -  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