Study to Evaluate the Efficacy and Safety of Ibrexafungerp in Patients With Fungal Diseases That Are Refractory to or Intolerant of Standard Antifungal Treatment

Study Overview
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Brief Summary
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This is a multicenter, open label, non-comparator, single arm study to evaluate the efficacy and safety of ibrexafungerp (SCY-078) in patients ≥ 18 years of age with a documented fungal disease that has been intolerant or refractory (rIFI) to Standard of Care (SoC) antifungal treatment.

Detailed Description
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This is a multicenter, open label, non-comparator, single arm study to evaluate the efficacy and safety of ibrexafungerp in patients ≥ 18 years of age with a documented invasive and/or severe fungal disease that has been intolerant or refractory (rIFI) to Standard of Care (SoC) antifungal treatment. Patients will be treated with ibrexafungerp for up to 180 days. Treatment beyond 180 days and combination therapy with other antifungal agents may be allowed under special circumstances to be agreed upon by the Investigator and the Sponsor. Subjects must have a proven or probable fungal disease and meet all study criteria to be considered for enrollment. Eligible subjects must also have documented evidence of failure of, intolerance to, or toxicity related to a currently approved SoC antifungal treatment. Subjects will also be considered for enrollment if they have an eligible fungal disease and, in the judgement of the investigator, the subject cannot receive approved oral antifungal options (e.g. susceptibility of the organism or risk for drug-drug interactions) and a continued IV antifungal therapy is not desirable or feasible due to clinical or logistical circumstances. Following a screening visit, there will be up to 15 treatment visits, a follow-up visit and 2 follow-up contacts.

Official Title
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Open-Label Study to Evaluate the Efficacy and Safety of SCY-078 (Ibrexafungerp) in Patients With Fungal Diseases That Are Refractory to or Intolerant of Standard Antifungal Treatment (FURI)

Conditions
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Invasive Candidiasis, Mucocutaneous Candidiasis, Coccidioidomycosis, Histoplasmosis, Blastomycosis, Chronic Pulmonary Aspergillosis, Allergic Bronchopulmonary Aspergillosis, Invasive Pulmonary Aspergillosis, Recurrent Vulvovaginal Candidiasis, Other Emerging Fungi

Intervention / Treatment
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* Drug: Ibrexafungerp


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Must have a documented eligible invasive and/or severe fungal disease that is refractory or intolerant to Standard-of-Care treatment Be able to tolerate medication orally or through a nasogastric (NG) tube or percutaneous endoscopic gastrostomy (PEG) tube Be able to understand and sign a written informed consent form (ICF), which must be obtained prior to treatment and any study-related procedures. Be able to understand and sign a consent or authorization form which shall permit the use, disclosure and transfer of the subject's personal health information. (e.g., in the U.S. HIPAA Authorization form). Be able to understand and follow all study-related procedures including study drug administration. Agree to use a medically acceptable method of contraception while receiving protocol-assigned product. Key Exclusion Criteria: An invasive fungal disease with CNS involvement. Subject has an inappropriately controlled fungal disease source (e.g., persistent catheters that cannot be removed and are likely the source of infection). Subject is hemodynamically unstable, requiring vasopressor medication for blood pressure support. A life expectancy < 30 days. Subject with abnormal liver test parameters: aspartate aminotransferase (AST), alanine aminotransferase (ALT) >10 x the upper limit of normal (ULN), and/or total bilirubin > 5 x ULN. Subject is pregnant or lactating. Subject has used an investigational drug within 30 days prior to the baseline visit.

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
Interventional Model Description: Open label, non-comparator, single arm
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Ibrexafungerp (SCY-078)<br>Ibrexafungerp (SCY-078), orally administered QD for up to 180 days. | Drug: Ibrexafungerp<br>* Experimental Study Drug<br>* Other names: SCY-078;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Assessment of Global Response | The proportion of subjects with a complete or partial Global Response (GR) at the End of Treatment Visit. | Up to 180 days of study treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Assessment of Recurrence of Baseline Fungal Infection | The proportion of subjects with a recurrence of the baseline fungal infectoin | up to 42 days after end of study treatment | 
| Assessment of survival | Proportion of surviving subjects | Day 42, Day 84 |