The Deferasirox-AmBisome Therapy for Mucormycosis (DEFEAT Mucor) Study
NCT ID: NCT00419770
Last Updated: 2023-10-16
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
20 participants
INTERVENTIONAL
2007-10-31
2010-12-31
Brief Summary
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Detailed Description
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Although the safety and efficacy of deferasirox have been extensively evaluated in iron-overloaded patients, there are minimal data in non-iron-overloaded patients or in infected patients. Therefore, the safety and efficacy of deferasirox in patients with mucormycosis is unclear, and confirming safety in the current study, at the currently planned dose, is required to lay the groundwork for a future phase III clinical trial.
This is a prospective, phase II, randomized, double-blinded, placebo-controlled study of liposomal amphotericin B (LAmB; AmBisome) plus deferasirox vs. LAmB plus placebo for mucormycosis infection. Twenty patients with proven or probable mucormycosis (except for isolated skin infection) by consensus EORTC/MSG criteria, who have received less than 14 days of antifungal therapy for mucormycosis, and who have had radiographic imaging by CT or MRI within the past 72 hours that shows evidence of infection, will be randomized to receive LAmB plus deferasirox or placebo (n = 10 per arm), with randomization stratified by study site.
The primary objective is to determine the safety and tolerability of adjunctive deferasirox therapy in patients being treated with LAmB for mucormycosis, and to obtain exploratory data on the efficacy of the iron chelation treatment. The exploratory efficacy endpoint will be the global response rate (composite of clinical and radiographic response) at end of study drug administration, as determined by a blinded adjudication committee.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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B
Deferasirox
deferasirox
20 mg/kg enterally per day
Liposomal amphotericin B
A
Placebo
Liposomal amphotericin B
Interventions
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deferasirox
20 mg/kg enterally per day
Placebo
Liposomal amphotericin B
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 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.
2 Years
ALL
No
Sponsors
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Gilead Sciences
INDUSTRY
Astellas Pharma Inc
INDUSTRY
Novartis
INDUSTRY
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
OTHER
Responsible Party
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Principal Investigators
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Brad Spellberg, MD
Role: PRINCIPAL_INVESTIGATOR
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Locations
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City of Hope National Medical Center
Duarte, California, United States
UCSF
San Francisco, California, United States
University of Miami
Miami, Florida, United States
Duke University
Durham, North Carolina, United States
Summa Health Systems
Akron, Ohio, United States
MD Anderson Cancer Center
Houston, Texas, United States
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States
Countries
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References
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Spellberg B, Edwards J Jr, Ibrahim A. Novel perspectives on mucormycosis: pathophysiology, presentation, and management. Clin Microbiol Rev. 2005 Jul;18(3):556-69. doi: 10.1128/CMR.18.3.556-569.2005.
Ibrahim AS, Edwards JE Jr, Fu Y, Spellberg B. Deferiprone iron chelation as a novel therapy for experimental mucormycosis. J Antimicrob Chemother. 2006 Nov;58(5):1070-3. doi: 10.1093/jac/dkl350. Epub 2006 Aug 23.
Reed C, Ibrahim A, Edwards JE Jr, Walot I, Spellberg B. Deferasirox, an iron-chelating agent, as salvage therapy for rhinocerebral mucormycosis. Antimicrob Agents Chemother. 2006 Nov;50(11):3968-9. doi: 10.1128/AAC.01065-06. Epub 2006 Sep 25. No abstract available.
Boelaert JR, Van Cutsem J, de Locht M, Schneider YJ, Crichton RR. Deferoxamine augments growth and pathogenicity of Rhizopus, while hydroxypyridinone chelators have no effect. Kidney Int. 1994 Mar;45(3):667-71. doi: 10.1038/ki.1994.89.
Spellberg B, Ibrahim AS, Chin-Hong PV, Kontoyiannis DP, Morris MI, Perfect JR, Fredricks D, Brass EP. The Deferasirox-AmBisome Therapy for Mucormycosis (DEFEAT Mucor) study: a randomized, double-blinded, placebo-controlled trial. J Antimicrob Chemother. 2012 Mar;67(3):715-22. doi: 10.1093/jac/dkr375. Epub 2011 Sep 20.
Other Identifiers
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12842
Identifier Type: -
Identifier Source: org_study_id
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