The Deferasirox-AmBisome Therapy for Mucormycosis (DEFEAT Mucor) Study
Condition: Mucormycosis
MeSH terms: Mucormycosis; Zygomycosis
Intervention: deferasirox; Placebo; Liposomal amphotericin B
Intervention description: 20 mg/kg enterally per day
Intervention MeSH terms: Amphotericin B; Liposomal amphotericin B; Deferasirox
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 20.0
Sex: All
Minimum age: 2 Years
Healthy volunteers: No
Keywords: zygomycosis; Rhizopus; iron; iron-chelation; adjunctive therapy

Summary:

      The purpose of this study is to determine if the addition of the medication, deferasirox, to
      standard antifungal therapy for the infection, mucormycosis, is safe and effective
    

Eligibility criteria:

        Inclusion Criteria:

          -  Age greater than 2 years.

          -  Proven or probable invasive mucormycosis, as defined by modification of consensus
             European Organization for Research and Treatment of Cancer (EORTC)/Mycosis Study Group
             (MSG) criteria. In brief, proven mucormycosis is defined as: 1) histopathologic or
             cytopathologic examination showing broad-based, aseptate, ribbon-like hyphae
             consistent with Mucorales from needle aspiration or biopsy specimen, with evidence of
             associated tissue damage (either microscopically or unequivocally by imaging); OR 2) a
             positive culture result for a sample obtained by sterile procedure from normally
             sterile and clinically or radiologically abnormal site consistent with infection,
             excluding urine and mucous membranes. Probable mucormycosis is defined as: 1) an
             at-risk host; AND 2) positive culture, cytology, or polymerase chain reaction (PCR)
             test (run at a CLIA-certified clinical microbiology laboratory) from sputum,
             bronchoalveolar lavage (BAL), endoscopy/colonoscopy, or sinus aspirate/biopsy; AND 3)
             1 major or 2 minor clinical criteria.

          -  Radiographic study by Computerized Tomography (CT) or Magnetic Resonance Imaging (MRI)
             has been obtained within 4 calendar days prior to enrollment and shows evidence of
             infection (i.e. focal nodule, mass, or abscess, or enhancement, or evidence of tissue
             edema or destruction that is not attributed to post-surgical reaction).

          -  Subject or authorized decision maker able to provide informed consent.

        Exclusion Criteria:

          -  High likelihood of death within the 48 h after enrollment (investigator's discretion).

          -  High likelihood of death due to factors unrelated to mucormycosis (e.g. due to
             uncontrolled and/or relapsed malignancy, severe graft versus host disease, other
             underlying diseases, etc.) within 30 days following enrollment (investigator's
             discretion).

          -  Patient unable to receive enteral medication (oral or via feeding tube).

          -  Infection limited to the supra-fascial skin (skin lesions in the presence of
             disseminated disease, deep invasive tissue infection spreading from a primary skin
             site, or subcutaneous infections extending to fascia are allowed).

          -  Patient has received > 14 days of polyene antifungal therapy (i.e. amphotericin B
             deoxycholate, liposomal amphotericin B, amphotericin B lipid complex, or amphotericin
             B colloidal dispersion) at the time of screening.

          -  Patient is already taking deferasirox therapy for any reason at the time of screening.

          -  Patient is allergic to or intolerant of deferasirox or LAmB.

          -  Patient has significant renal dysfunction at the time of screening, defined as serum
             creatinine of > 3 mg/dL or a calculated creatinine clearance of < 30 ml/min (by the
             Cockroft-Gault formula: (140 - age (yrs) * wt (kg)) * 0.85 (for females) / (72 * serum
             creatinine (mg/dL)).

          -  Patient has significant hepatic dysfunction at the time of screening, defined as BOTH
             an AST or ALT > 10 times the upper limit of normal, AND a direct (not total) bilirubin
             > 5 times the upper limit of normal.

          -  Women of child-bearing potential (those with menses within the last year) with a
             positive serum pregnancy test.

          -  Enrollment refused by the primary physician.