Clinical Trial: The Deferasirox-AmBisome Therapy for Mucormycosis (DEFEAT Mucor) Study
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: The Deferasirox-AmBisome Therapy for Mucormycosis (DEFEAT Mucor) Study
Brief 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
Detailed Summary:
Because of its extremely high morbidity and mortality, it is imperative to look for new antifungal therapies to treat mucormycosis. The agents of mucormycosis are exquisitely sensitive to iron availability, and we and others have demonstrated that iron chelation therapy improves the survival of rodents with mucormycosis. Deferasirox (Exjade) is the first orally bioavailable iron chelator approved for use in the United States (US) by the Food and Drug Administration (FDA), with an indication for treatment of iron overload from chronic transfusions. In clinical studies, deferasirox has been well tolerated and effective in iron-overloaded patients.
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 deferasi
Sponsor: Los Angeles Biomedical Research Institute
Current Primary Outcome:
- Safety and Tolerability of Adjunctive Deferasirox Therapy in Patients Being Treated With LAmB for Mucormycosis [ Time Frame: 14 days ]
- 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 ]
- Total Adverse Events [ Time Frame: 30 Days After End of Therapy ]
Original Primary Outcome:
- Safety and Tolerability of Adjunctive Deferasirox Therapy in Patients Being Treated With LAmB for Mucormycosis
- Global Response Rate (Composite of Clinical and Radiographic Response) at End of Study Drug Administration, as Determined by a Blinded Adjudication Committee
Current Secondary Outcome:
- Deferasirox Pharmacokinetic and Pharmacodynamic Parameters [ Time Frame: 7 days ]
- Survival, Radiographic Improvement, Clinical Response, Time to Survival, Deferasirox vs. Free Iron Level Correlation [ Time Frame: Up to 90 days ]
Original Secondary Outcome: Deferasirox Pharmacokinetic and Pharmacodynamic Parameters
Information By: Los Angeles Biomedical Research Institute
Dates:
Date Received: January 5, 2007
Date Started: October 2007
Date Completion:
Last Updated: September 2, 2011
Last Verified: August 2011
