Trial Outcomes & Findings for Deferasirox in Treating Patients With Iron Overload After Undergoing a Donor Stem Cell Transplant (NCT NCT00602446)

Results Overview

Number of patients who discontinued deferasirox during 6 month daily treatment due to drug related toxicity

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

6 Months

Results posted on

2017-12-28

Participant Flow

Only one site (Masonic Cancer Center) enrolled patients in this study.

4 patients were consented, however, one withdrew consent before receiving treatment.

Participant milestones

Participant milestones
Measure	Deferasirox Treated Includes patients that were treated with deferasirox for 6 months.
Overall Study STARTED	3
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Deferasirox in Treating Patients With Iron Overload After Undergoing a Donor Stem Cell Transplant

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Deferasirox Treated n=3 Participants Includes patients that were treated with deferasirox for 6 months.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	45.7 years STANDARD_DEVIATION 5.0 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 Months

Population: Note: 1 patient had a transient decrease in hemoglobin that required discontinuation of treatment for 2 weeks; subsequently restarted at same dose and completed therapy.

Number of patients who discontinued deferasirox during 6 month daily treatment due to drug related toxicity

Outcome measures

Outcome measures
Measure	Deferasirox Treated n=3 Participants Includes patients that were treated with deferasirox for 6 months.
Number of Patients Not Completing Treatment	0 Participants

SECONDARY outcome

Timeframe: 6 Months

Population: All patients included in count.

Efficacy as measured by reduction in liver iron concentration (LIC) after 6 months of the study drug compared to baseline (LIC at baseline minus LIC at 6 months). This shows the mean reduction for the 3 subjects treated in this study.

Outcome measures

Outcome measures
Measure	Deferasirox Treated n=3 Participants Includes patients that were treated with deferasirox for 6 months.
Reduction in Liver Iron Concentration After Study Drug	5.6 milligrams/gram Standard Deviation 4.8

Adverse Events

Deferasirox Treated

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Deferasirox Treated n=3 participants at risk Includes patients that were treated with deferasirox for 6 months.
Skin and subcutaneous tissue disorders Rash	66.7% 2/3 • Number of events 2 • 6 Months
Gastrointestinal disorders Diarrhea	100.0% 3/3 • Number of events 3 • 6 Months
Blood and lymphatic system disorders Increased serum creatinine	66.7% 2/3 • Number of events 2 • 6 Months

Additional Information

Linda J. Burns, MD

Masonic Cancer Center, University of Minnesota

Phone: 612-624-8144

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place