The Deferasirox-AmBisome Therapy for Mucormycosis (DEFEAT Mucor) Study - Study Results

Study Type:	Interventional
Study Design:	Allocation: Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Parallel Assignment; Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition:	Mucormycosis
Interventions:	Drug: deferasirox Drug: Placebo Drug: Liposomal amphotericin B

Participant Flow

Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
Deferasirox	Deferasirox plus liposomal amphotericin
Placebo	Placebo control plus background liposomal amphotericin

Participant Flow: Overall Study

	Deferasirox	Placebo
STARTED	11	9
COMPLETED	6	8
NOT COMPLETED	5	1
Withdrawal by Subject	5	1

Baseline Characteristics

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Reporting Groups

	Description
Deferasirox	Deferasirox plus liposomal amphotericin
Placebo	Placebo control plus background liposomal amphotericin

Baseline Measures

	Deferasirox	Placebo	Total
Number of Participants [units: participants]	11	9	20
Age [units: participants]
<=18 years	0	0	0
Between 18 and 65 years	8	7	15
>=65 years	3	2	5
Age [units: years] Median ( Full Range )	59 ( 30 to 71 )	47 ( 40 to 75 )	52 ( 30 to 75 )
Gender [units: participants]
Female	2	3	5
Male	9	6	15
Region of Enrollment [units: participants]
United States	11	9	20

Outcome Measures

1. Primary:

Total Adverse Events [ Time Frame: 30 Days After End of Therapy ]

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2. Primary:

Safety and Tolerability of Adjunctive Deferasirox Therapy in Patients Being Treated With LAmB for Mucormycosis [ Time Frame: 14 days ]

Results not yet posted. Anticipated Posting Date: No text entered. Safety Issue: Yes

3. Primary:

Global Response Rate (Composite of Clinical and Radiographic Response) at End of Study Drug Administration, as Determined by a Blinded Adjudication Committee [ Time Frame: 14 days ]

Results not yet posted. Anticipated Posting Date: No text entered. Safety Issue: No

4. Secondary:

Deferasirox Pharmacokinetic and Pharmacodynamic Parameters [ Time Frame: 7 days ]

Results not yet posted. Anticipated Posting Date: No text entered. Safety Issue: No

5. Secondary:

Survival, Radiographic Improvement, Clinical Response, Time to Survival, Deferasirox vs. Free Iron Level Correlation [ Time Frame: Up to 90 days ]

Results not yet posted. Anticipated Posting Date: No text entered. Safety Issue: No

Serious Adverse Events

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Other Adverse Events

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More Information

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Certain Agreements:

All Principal Investigators ARE employed by the organization sponsoring the study.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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Results Point of Contact:

Name/Title: Brad Spellberg
Organization: Los Angeles Biomedical Research Institute
phone: 310-781-3680
e-mail: bspellberg@labiomed.org

Publications:

Spellberg B, Edwards J Jr, Ibrahim A. Novel perspectives on mucormycosis: pathophysiology, presentation, and management. Clin Microbiol Rev. 2005 Jul;18(3):556-69. Review.

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. 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. 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.

Responsible Party:	Los Angeles Biomedical Research Institute
ClinicalTrials.gov Identifier:	NCT00419770 History of Changes
Other Study ID Numbers:	12842
Study First Received:	January 5, 2007
Results First Received:	June 29, 2011
Last Updated:	September 2, 2011
Health Authority:	United States: Institutional Review Board