Trial Outcomes & Findings for The Deferasirox-AmBisome Therapy for Mucormycosis (DEFEAT Mucor) Study (NCT NCT00419770)
NCT ID: NCT00419770
Last Updated: 2023-10-16
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
20 participants
Primary outcome timeframe
14 days
Results posted on
2023-10-16
Participant Flow
Participant milestones
| Measure |
Deferasirox
Deferasirox (20 mg/kg/d for 14 days) plus liposomal amphotericin (minimum dose of 5 mg/kg thrice weekly)
|
Placebo
Placebo control plus background liposomal amphotericin
|
|---|---|---|
|
Overall Study
STARTED
|
11
|
9
|
|
Overall Study
COMPLETED
|
6
|
8
|
|
Overall Study
NOT COMPLETED
|
5
|
1
|
Reasons for withdrawal
| Measure |
Deferasirox
Deferasirox (20 mg/kg/d for 14 days) plus liposomal amphotericin (minimum dose of 5 mg/kg thrice weekly)
|
Placebo
Placebo control plus background liposomal amphotericin
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
5
|
1
|
Baseline Characteristics
The Deferasirox-AmBisome Therapy for Mucormycosis (DEFEAT Mucor) Study
Baseline characteristics by cohort
| Measure |
Deferasirox
n=11 Participants
Deferasirox plus liposomal amphotericin
|
Placebo
n=9 Participants
Placebo control plus background liposomal amphotericin
|
Total
n=20 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Age, Continuous
|
59 years
n=5 Participants
|
47 years
n=7 Participants
|
52 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
11 participants
n=5 Participants
|
9 participants
n=7 Participants
|
20 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 14 daysOutcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: 14 daysOutcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: 30 Days After End of TherapyOutcome measures
| Measure |
Deferasirox
n=11 Participants
Deferasirox plus liposomal amphotericin
|
Placebo
n=9 Participants
Placebo control plus background liposomal amphotericin
|
|---|---|---|
|
Total Adverse Events
|
16 Events
|
16 Events
|
SECONDARY outcome
Timeframe: 7 daysOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 90 daysOutcome measures
Outcome data not reported
Adverse Events
Deferasirox
Serious events: 8 serious events
Other events: 8 other events
Deaths: 0 deaths
Placebo
Serious events: 4 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Deferasirox
n=11 participants at risk
Deferasirox plus liposomal amphotericin
|
Placebo
n=9 participants at risk
Placebo control plus background liposomal amphotericin
|
|---|---|---|
|
Infections and infestations
Progressive Infection
|
9.1%
1/11 • Number of events 1
|
0.00%
0/9
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Progression of underlying disease
|
27.3%
3/11 • Number of events 3
|
0.00%
0/9
|
|
General disorders
Respiratory Failure
|
18.2%
2/11 • Number of events 2
|
22.2%
2/9 • Number of events 2
|
|
General disorders
Renal Failure
|
9.1%
1/11 • Number of events 1
|
0.00%
0/9
|
|
Gastrointestinal disorders
Liver failure
|
9.1%
1/11 • Number of events 1
|
0.00%
0/9
|
|
Cardiac disorders
Myocardial infarction
|
9.1%
1/11 • Number of events 1
|
0.00%
0/9
|
|
Gastrointestinal disorders
Vomiting/interolerance of study drug
|
9.1%
1/11 • Number of events 1
|
11.1%
1/9 • Number of events 1
|
|
Nervous system disorders
Intracranial bleeding
|
9.1%
1/11 • Number of events 1
|
0.00%
0/9
|
|
Cardiac disorders
arrhythmia
|
0.00%
0/11
|
11.1%
1/9 • Number of events 1
|
|
Hepatobiliary disorders
alveolar hemorrhage
|
0.00%
0/11
|
11.1%
1/9 • Number of events 1
|
|
Infections and infestations
bacterial pneumonia
|
9.1%
1/11 • Number of events 1
|
11.1%
1/9 • Number of events 1
|
|
General disorders
Fever
|
0.00%
0/11
|
11.1%
1/9 • Number of events 1
|
Other adverse events
| Measure |
Deferasirox
n=11 participants at risk
Deferasirox plus liposomal amphotericin
|
Placebo
n=9 participants at risk
Placebo control plus background liposomal amphotericin
|
|---|---|---|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/11
|
11.1%
1/9 • Number of events 1
|
|
Blood and lymphatic system disorders
Exophthalmos
|
0.00%
0/11
|
11.1%
1/9 • Number of events 1
|
|
Eye disorders
Blurry vision
|
0.00%
0/11
|
11.1%
1/9 • Number of events 1
|
|
Gastrointestinal disorders
Diarrhea
|
9.1%
1/11 • Number of events 1
|
11.1%
1/9 • Number of events 1
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/11
|
11.1%
1/9 • Number of events 1
|
|
Metabolism and nutrition disorders
Elevated creatinine
|
0.00%
0/11
|
11.1%
1/9 • Number of events 1
|
|
Metabolism and nutrition disorders
Electrolyte abnormalities
|
45.5%
5/11 • Number of events 5
|
11.1%
1/9 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Myoclonus
|
0.00%
0/11
|
11.1%
1/9 • Number of events 1
|
|
Nervous system disorders
Altered mental status
|
0.00%
0/11
|
22.2%
2/9 • Number of events 3
|
|
General disorders
Respiratory distress
|
9.1%
1/11 • Number of events 1
|
0.00%
0/9
|
|
Musculoskeletal and connective tissue disorders
Rash
|
9.1%
1/11 • Number of events 1
|
0.00%
0/9
|
Additional Information
Brad Spellberg
Los Angeles Biomedical Research Institute
Phone: 310-781-3680
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place