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A Phase 3 Efficacy and Safety Study of Fosmanogepix for the Treatment of Adult Participants With Candidemia and/or Invasive Candidiasis.

Phase 3
Recruiting
Conditions
Candidemia
Candidiasis, Invasive
Interventions
Registration Number
NCT05421858
Lead Sponsor
Basilea Pharmaceutica
Brief Summary

The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called Fosmanogepix) for the potential treatment of candidemia and/or invasive candidiasis, a life-threatening fungal infection caused by several species of yeast called Candida.

The study is seeking patients who have a diagnosis of candidemia and/or invasive candidiasis.

Two-thirds of all patients will receive the study medication fosmanogepix Intravenous (IV) infusion followed by optional fosmanogepix tablets. One-third of all patients will receive a standard of care regimen of caspofungin Intravenous (IV) infusion followed by optional fluconazole capsules.

Fosmanogepix or caspofungin will first be given as an Intravenous (IV) infusion directly into a vein in the arm each day at the study clinic. Fosmanogepix tablets or fluconazole capsules will be taken orally by mouth daily either at the study clinic, or at home if patients are well enough to be discharged from the hospital.

The treatment effect in patients receiving fosmanogepix to those receiving caspofungin/ fluconazole will be compared. The primary aim is to show that fosmanogepix is not inferior (not worse) to caspofungin/ fluconazole with a noninferiority margin of 15%.

The duration of study treatment and number of study visits will vary depending on how long the patient will be treated for the infection. Treatment will continue for a maximum of 6 weeks depending on when the infection is cleared and whether other symptoms related to the infection have improved. There will also be a follow-up visit 6 weeks after the study treatment was stopped.

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
450
Inclusion Criteria
  1. Patients ≥ 18 years (or the minimum country-specific age of consent if > 18) at Screening who have provided signed informed consent indicating that they understand the purpose of, and procedures required for, the study, and are willing to participate in the study. If the patient is unable to consent for himself/herself, a legally authorized representative must provide informed consent on his/her behalf.
  2. Diagnosis of candidemia and/or invasive candidiasis based on a blood or non-blood specimen obtained within ≤ 96 hours (4 days) before randomization, and on clinical criteria judged attributable to candidemia/invasive candidiasis occurring at any time from ≤ 12 hours prior to the qualifying positive index culture being taken through to randomization.
  3. Patient's condition allows for appropriate infection source control measures, including removal of pre-existing intravascular catheters and devices, if necessary.
Exclusion Criteria
  1. Existing infection

    1. Infection known to be due to Candida krusei, in blood or any other normally sterile site.
    2. Inappropriate fungal infection source control.
    3. Diagnosis of certain deep-seated Candida infections.
  2. Life expectancy of < 72 hours in the opinion of the investigator.

  3. Requirement, or expected requirement, for hemodialysis, peritoneal dialysis, or hemofiltration.

  4. Ongoing neurological disorders, including specified conditions presenting with a CTCAE Grade ≥ 2 (neurological symptoms that are considered to be a consequence of the current episode of candidemia / invasive candidiasis are not exclusionary).

  5. Patients with known human immunodeficiency virus infection, who have CD4+ count < 200/mm3 or viral load > 400 copies/mL), or who have had an active opportunistic infection within 6 months prior to Screening.

  6. Other medical or psychiatric condition or laboratory abnormality that may increase the risk of study participation or make the patient inappropriate for the study.

  7. Current use of any prohibited concomitant medications or those unwilling/unable to use a permitted concomitant medication.

  8. Received > 2 days (> 48 hours) equivalent of prior systemic antifungal treatment at approved doses and frequency to treat the current episode of candidemia and/or invasive candidiasis (e.g., > 2 doses of a once daily antifungal agent or > 4 doses of a twice daily antifungal agent), within the 96 hours prior to randomization (except for non-susceptible Candida spp. and for patients who develop candidemia or invasive candidiasis while on prophylaxis with an azole or amphotericin B).

  9. Previous administration with an investigational drug or investigational vaccine within 30 days or 5 half-lives preceding the first dose of study drug used in this study (whichever is longer).

  10. Prior participation in this or any previous study of fosmanogepix.

  11. Moderate or severe hepatic impairment, known active viral hepatitis B or C, ALT or AST ≥ 5 × ULN or total bilirubin > 3 × ULN unless this is due to isolated hyperbilirubinemia or documented Gilbert's syndrome.

  12. Female patient is pregnant or lactating.

  13. Known hypersensitivity to fosmanogepix, manogepix, caspofungin, any echinocandin, fluconazole or to any of their excipients.

  14. Investigator site staff directly involved in the conduct of the study and their family members, site staff otherwise supervised by the investigator, and Sponsor and Sponsor delegate employees directly involved in the conduct of the study and their family members.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Fosmanogepix IV/oralPlaceboFosmanogepix will be administered as an Intravenous (IV) infusion given directly into a vein. There is an option to switch from the IV infusion to the oral form of fosmanogepix which is taken by mouth. Matching placebos for caspofungin and fluconazole will also be administered (a placebo does not have any medicine in it but looks just like the caspofungin and fluconazole).
Caspofungin IV/ Fluconazole oralPlaceboCaspofungin will be administered as an intravenous (IV) infusion given directly into a vein. There is an option to switch from the IV infusion to oral fluconazole which is taken by mouth. Matching placebos for fosmanogepix will also be administered (a placebo does not have any medicine in it but looks just like the medicine fosmanogepix being studied).
Fosmanogepix IV/oralFosmanogepixFosmanogepix will be administered as an Intravenous (IV) infusion given directly into a vein. There is an option to switch from the IV infusion to the oral form of fosmanogepix which is taken by mouth. Matching placebos for caspofungin and fluconazole will also be administered (a placebo does not have any medicine in it but looks just like the caspofungin and fluconazole).
Caspofungin IV/ Fluconazole oralCaspofunginCaspofungin will be administered as an intravenous (IV) infusion given directly into a vein. There is an option to switch from the IV infusion to oral fluconazole which is taken by mouth. Matching placebos for fosmanogepix will also be administered (a placebo does not have any medicine in it but looks just like the medicine fosmanogepix being studied).
Caspofungin IV/ Fluconazole oralFluconazoleCaspofungin will be administered as an intravenous (IV) infusion given directly into a vein. There is an option to switch from the IV infusion to oral fluconazole which is taken by mouth. Matching placebos for fosmanogepix will also be administered (a placebo does not have any medicine in it but looks just like the medicine fosmanogepix being studied).
Primary Outcome Measures
NameTimeMethod
Proportion of patients alive at Day 30Day 30
Proportion of patients with an overall response of treatment success at end of study treatment (EOST)EOST (up to Day 42)
Secondary Outcome Measures
NameTimeMethod
Proportion of patients with clinical response of success at Day 7, 14, EOIV, EOST, Follow-up 6- weeks after EOSTDay 14, EOIV (up to Day 42), EOST (up to Day 42), Follow-up 6-weeks after EOST]
Time to first negative blood culture in patients on fosmanogepix compared to caspofungin/fluconazoleBaseline up to follow-up 6-weeks after EOST (approximately up to 12,5 weeks)
Proportion of patients with an overall response of treatment success at Day 7Day 7
Proportion of patients with an overall response of treatment success at end of IV treatment (EOIV)up to Day 42
Proportion of patients with an overall response of treatment success (sustained) at follow-up 6 weeks after EOSTapproximately up to 12,5 weeks
Proportion of patients with mycological response of eradication or presumed eradication at Day 7, 14, EOIV, EOST, Follow-up 6-weeks after EOSTDay 14, EOIV (up to Day 42), EOST (up to Day 42), Follow-up 6-weeks after EOST
Incidence of treatment-emergent adverse event (TEAEs), serious adverse events (SAEs), treatment-related adverse events (AEs), adverse events of special interest (AESI)and AEs leading to discontinuationScreening up to follow-up 6-weeks after EOST (approximately up to 12,5 weeks)
Number of patients with clinically significant laboratory abnormalitiesBaseline up to follow-up 6-weeks after EOST (approximately up to 12,5 weeks)
Number of patients with abnormal neurological examination findingsBaseline up to follow-up 6-weeks after EOST (approximately up to 12,5 weeks)
Assessment of 12-lead electrocardiogram (ECGs)Baseline up to follow-up 6-weeks after EOST (approximately up to 12,5 weeks)
Plasma concentrations versus time of fosmanogepix (prodrug) and manogepix (active moiety)Day 3: 0, 3, 6, 9 and 24 hours post-dose; Day 7, 14, 21, 28, 35; EOST: 72 and 192 hours post-dose
Proportion of patients with an overall response of treatment success at Day 14Day 14

Trial Locations

Locations (86)

University of Alabama at Birmingham School of Medicine, Department of Medicine

🇺🇸

Birmingham, Alabama, United States

UC Davis Medical Center

🇺🇸

Sacramento, California, United States

Emory University Hospital - Clifton Road

🇺🇸

Atlanta, Georgia, United States

Indiana University Methodist Hospital

🇺🇸

Indianapolis, Indiana, United States

University of Kentucky College of Medicine

🇺🇸

Lexington, Kentucky, United States

University of Michigan Health System (UMHS) - A. Alfred Taubman Health Care Center

🇺🇸

Ann Arbor, Michigan, United States

Henry Ford Hospital, Department of Medicine, Division of Infectious Diseases

🇺🇸

Detroit, Michigan, United States

University of Minnesota

🇺🇸

Minneapolis, Minnesota, United States

Washington University School of Medicine, Infectious Diseases Clinical Research Unit

🇺🇸

Saint Louis, Missouri, United States

University of North Carolina at Chapel Hill

🇺🇸

Chapel Hill, North Carolina, United States

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

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