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

University of Minnesota (UMN)

Status and phase

Terminated

Phase 2

Conditions

Breast Cancer

Multiple Myeloma and Plasma Cell Neoplasm

Neuroblastoma

Iron Overload

Lymphoma

Ovarian Cancer

Leukemia

Myelodysplastic Syndromes

Treatments

Drug: deferasirox

Study type

Interventional

Funder types

Other

Identifiers

NCT00602446

CDR0000584690

UMN-MT2007-11R (Other Identifier)

UMN-2007LS065 (Other Identifier)

NOVARTIS-CICL670AUS12 (Other Identifier)

Details and patient eligibility

About

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.

Full 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.

Enrollment

4 patients

Sex

All

Ages

18 to 70 years old

Volunteers

No Healthy Volunteers

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