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

• Sponsor: Masonic Cancer Center, University of Minnesota (Other)
• Overall Status: Terminated
• CT.gov ID: NCT00602446
• Collaborator: (none)
• Enrollment: 4
• Location: 1
• Arm: 1
• Duration (Months): 28
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

RATIONALE: Deferasirox may be effective in treating iron overload caused by blood transfusions in patients who have undergone donor stem cell transplant.

PURPOSE: This phase II trial is studying the side effects and how well deferasirox works in treating patients with iron overload after donor stem cell transplant.

Condition or Disease	Intervention/Treatment	Phase
Breast Cancer; Iron Overload; Leukemia; Lymphoma; Multiple Myeloma and Plasma Cell Neoplasm; Myelodysplastic Syndromes; Neuroblastoma; Ovarian Cancer	Drug: deferasirox	Phase 2

Detailed Description

OBJECTIVES:

Primary

• To evaluate the safety of deferasirox given over 6 months in reducing liver iron concentration in patients with transfusional iron overload after undergoing allogeneic hematopoietic stem cell transplantation.

Secondary

• To evaluate the efficacy of deferasirox in reducing liver iron overload in these patients.

OUTLINE: This is a multicenter study.

Patients receive oral deferasirox once daily for 6 months in the absence of disease progression or unacceptable toxicity.

After completion of study therapy, patients are followed at 4 weeks.

Study Design

• Study Type: Interventional
• Actual Enrollment: 4 participants
• Allocation: Non-Randomized
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Supportive Care
• Official Title: Open-Label Single-Arm Pilot Study of Deferasirox (Exjade®) in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron Overload
• Study Start Date: Aug 1, 2007
• Actual Primary Completion Date: Sep 1, 2009
• Actual Study Completion Date: Dec 1, 2009

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Deferasirox Treated Includes patients that were treated with deferasirox for 6 months.	Drug: deferasirox; 20 mg/kg once daily orally for 6 months; Other Names: Exjade

Outcome Measures

Primary Outcome Measures

1. Number of Patients Not Completing Treatment [6 Months]

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

Secondary Outcome Measures

1. Reduction in Liver Iron Concentration After Study Drug [6 Months]

   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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 70 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Confirmed diagnosis of iron overload, defined as serum ferritin > 1,000 ng/mL and liver iron concentration ≥ 5 mg iron/g on tissue proton transverse relaxation rates Magnetic Resonance Imaging (MRI)

• Underwent prior allogeneic hematopoietic stem cell transplantation (HSCT) using either myeloablative or reduced-intensity conditioning at least 12 months ago

• No evidence of relapse or progression of the primary disease for which allogeneic HSCT was performed

• Patients who have become red-cell transfusion independent (i.e., no red cell transfusions within the past 3 months) as well as patients who require red cell transfusions are eligible

• Meets one of the following criteria:

• Ineligible for phlebotomy (hemoglobin < 11 g/dL, poor intravenous access, or unable to undergo phlebotomy every 4 weeks)

• Have failed treatment with phlebotomy (serum ferritin > 50% of baseline after 3 months of phlebotomy)

• Refused phlebotomy

• ECOG performance status of 0-2

• Life expectancy ≥ 6 months

• Adequate renal function defined as serum creatinine < or = 1.6 mg/dL and creatinine clearance of > or = 60 ml/min calculated using the Crockcroft-Gault formula on 2 occasions within 30 days of enrollment

• Sexually active men and women must use an effective method of contraception. Alternatively, women must have undergone clinically documented total hysterectomy and/or oophorectomy, or tubal ligation or be postmenopausal.

• Must be able to give written informed consent.

• Prior therapy with deferoxamine allowed provided it was completed ≥ 12 months ago

Exclusion Criteria:

• Contraindication for performing MRI or inability to undergo MRI because of claustrophobia or weight (>350 pounds).

• Inability to take medications orally.

• Uncontrolled bacterial, viral, or fungal infection

• ANC ≥ 1,000/mm³

• Hemoglobin ≥ 8.0 g/dL

• Platelet count ≥ 50,000/mm³

• Aspartate aminotransferance (AST) and alanine aminotransferase (ALT) ≤ 5 times the upper limit of normal

• Less than 4 weeks since prior and no concurrent systemic investigational drug

• Less than 7 days since prior and no concurrent topical investigational drug. Concurrent non-investigational medications needed to treat concomitant medical conditions are allowed, with the exception of other chelating agents. Concurrent growth factors such as epoetin alfa, darbepoetin alfa, filgrastim (G-CSF), and sargramostim (GM-CSF) allowed. Concurrent irradiated packed red-cell and platelet transfusions allowed as clinically indicated. Concurrent low-doses of vitamin C supplements (≤ 200 mg/day) allowed.

• Concurrent iron supplements or multivitamins with iron.

• Aluminum-containing antacid therapies may not be taken simultaneously with deferasirox, but may be taken 2 hours before or after administration of deferasirox

• On dialysis or status post-renal transplantation

• Pregnant or nursing

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Masonic Cancer Center at University of Minnesota	Minneapolis	Minnesota	United States	55455

Sponsors and Collaborators

• Masonic Cancer Center, University of Minnesota

Investigators

• Principal Investigator: Linda J. Burns, MD, Masonic Cancer Center, University of Minnesota

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Masonic Cancer Center, University of Minnesota
• ClinicalTrials.gov Identifier: NCT00602446
• Other Study ID Numbers: CDR0000584690; UMN-2007LS065; UMN-MT2007-11R; NOVARTIS-CICL670AUS12
• First Posted: Jan 28, 2008
• Last Update Posted: Dec 28, 2017
• Last Verified: Dec 1, 2017

• Keywords provided by Masonic Cancer Center, University of Minnesota: iron overload
• Additional relevant MeSH terms: Multiple Myeloma; Neuroblastoma; Neoplasms, Plasma Cell; Plasmacytoma; Myelodysplastic Syndromes; Iron Overload; Deferasirox

Study Results

Participant Flow

Recruitment Details	Only one site (Masonic Cancer Center) enrolled patients in this study.
Pre-assignment Detail	4 patients were consented, however, one withdrew consent before receiving treatment.

Arm/Group Title	Deferasirox Treated
Arm/Group Description	Includes patients that were treated with deferasirox for 6 months.
Period Title: Overall Study
STARTED	3
COMPLETED	3
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Deferasirox Treated
Arm/Group Description	Includes patients that were treated with deferasirox for 6 months.
Overall Participants	3
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	3 100%
>=65 years	0 0%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	45.7 (5.0)
Sex: Female, Male (Count of Participants)
Female	0 0%
Male	3 100%
Region of Enrollment (participants) [Number]
United States	3 100%

Outcome Measures

1. Primary Outcome

Title	Number of Patients Not Completing Treatment
Description	Number of patients who discontinued deferasirox during 6 month daily treatment due to drug related toxicity
Time Frame	6 Months

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Deferasirox Treated
Arm/Group Description	Includes patients that were treated with deferasirox for 6 months.
Measure Participants	3
Number [Participants]	0 0%

2. Secondary Outcome

Title	Reduction in Liver Iron Concentration After Study Drug
Description	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.
Time Frame	6 Months

Outcome Measure Data

Analysis Population Description

All patients included in count.

Arm/Group Title	Deferasirox Treated
Arm/Group Description	Includes patients that were treated with deferasirox for 6 months.
Measure Participants	3
Mean (Standard Deviation) [milligrams/gram]	5.6 (4.8)

Adverse Events

Time Frame	6 Months
Arm/Group Title	Deferasirox Treated
Arm/Group Description	Includes patients that were treated with deferasirox for 6 months.
All Cause Mortality
	Deferasirox Treated
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Deferasirox Treated
	Affected / at Risk (%)	# Events
Total	0/3 (0%)
Other (Not Including Serious) Adverse Events
	Deferasirox Treated
	Affected / at Risk (%)	# Events
Total	3/3 (100%)
Blood and lymphatic system disorders
Increased serum creatinine	2/3 (66.7%)	2
Gastrointestinal disorders
Diarrhea	3/3 (100%)	3
Skin and subcutaneous tissue disorders
Rash	2/3 (66.7%)	2

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

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

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.