Trial Outcomes & Findings for Deferoxamine for Iron Overload Before Allogeneic Stem Cell Transplantation (NCT NCT00658411)

Results Overview

All patients meeting the criteria for Severe iron overload as defined by BOTH: ferritin ≥ 1000 ng/ml and liver iron content(LIC) ≥ 5 mg/gdw were enrolled and received chelation therapy with Deferoxamine. All patients who received chelation therapy were monitored for grade 3 or above toxicity Attributable to Deferoxamine(grades defined by the CTCAE Version 3). The number of participants with grade 3 or higher toxicities were measured and used to determine the safety of chelation therapy.

Recruitment status

TERMINATED

Study phase

NA

Target enrollment

5 participants

Primary outcome timeframe

Baseline , 6 month, 1 year

Results posted on

2013-04-09

Participant Flow

Adult patients with AML, ALL, MDS scheduled for HSCT with myeloablative conditioning, who in addition were found to have both a serum ferritin ≥ 1000 ng/ml and a liver iron content (LIC) \> 5 mg/g dry weight (mg/gdw) based on hepatic T2\* measurement, were offered enrollment on the chelation study.

Participant milestones

Participant milestones
Measure	All Patients Deferoxamine for \>= 2 weeks prior to stem cells
Overall Study STARTED	5
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Deferoxamine for Iron Overload Before Allogeneic Stem Cell Transplantation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Patients n=5 Participants Deferoxamine prior to stem cells
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age Continuous	48 years STANDARD_DEVIATION 9.8 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	5 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline , 6 month, 1 year

Population: Patients who met criteria for iron overload pre-transplant, as defined by the protocol, were enrolled on study for chelation therapy. Those patients who received therapy were monitored for toxicities using the CTCAE version 3.0.

All patients meeting the criteria for Severe iron overload as defined by BOTH: ferritin ≥ 1000 ng/ml and liver iron content(LIC) ≥ 5 mg/gdw were enrolled and received chelation therapy with Deferoxamine. All patients who received chelation therapy were monitored for grade 3 or above toxicity Attributable to Deferoxamine(grades defined by the CTCAE Version 3). The number of participants with grade 3 or higher toxicities were measured and used to determine the safety of chelation therapy.

Outcome measures

Outcome measures
Measure	Deferoxamine n=5 Participants All patients received a maximum dose of 50mg/kg/d of deferoxamine as chelation therapy for at least 2 weeks prior to receiving myeloablative transplant.	Relapse (Deferoxamine) Patients who received Deferoxamine and relapsed post transplant at 1 year.	Disease-Free Survival (Deferoxamine) Patients who received Deferoxamine and are currently alive without incidence of relapse at 1 year.	Overall Survival (Deferoxamine) Patients who received Deferoxamine and have relapsed and is alive at 1 year.
Safety of Deferoxamine Therapy Determined by the Number of Participants With Grade 3 or Higher Toxicities. Baseline	5 Participants 9.8 • Interval 20.0 to 52.0	—	—	—
Safety of Deferoxamine Therapy Determined by the Number of Participants With Grade 3 or Higher Toxicities. 6 month	0 Participants	—	—	—
Safety of Deferoxamine Therapy Determined by the Number of Participants With Grade 3 or Higher Toxicities. 1 year	0 Participants	—	—	—

SECONDARY outcome

Timeframe: 1 year

Population: Stopped early for poor accrual

Survival information for the 5 patients who were treated with deferoxamine was collected. This information was used to determine transplant-related mortality, relapse, disease-free and overall survival.

Outcome measures

Outcome measures
Measure	Deferoxamine n=5 Participants All patients received a maximum dose of 50mg/kg/d of deferoxamine as chelation therapy for at least 2 weeks prior to receiving myeloablative transplant.	Relapse (Deferoxamine) n=5 Participants Patients who received Deferoxamine and relapsed post transplant at 1 year.	Disease-Free Survival (Deferoxamine) n=5 Participants Patients who received Deferoxamine and are currently alive without incidence of relapse at 1 year.	Overall Survival (Deferoxamine) n=5 Participants Patients who received Deferoxamine and have relapsed and is alive at 1 year.
1-year Post-Transplant Survival	0 participants	0 participants	5 participants	0 participants

Adverse Events

Deferoxamine

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Deferoxamine n=5 participants at risk All patients received a maximum dose of 50mg/kg/d of deferoxamine as chelation therapy for at least 2 weeks prior to receiving myeloablative transplant.
Vascular disorders Hypotension	20.0% 1/5 • Number of events 5 • All patients were monitored for toxicities from the time of chelation to study completion (1 year post transplant).

Other adverse events

Adverse event data not reported

Additional Information

Philippe Armand, MD, PhD

Dana-Farber Cancer Institute

Phone: 617-632-2305

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place