Clinical trial NCT00602446: Deferasirox in Treating Patients With Iron Overload After Undergoing a Donor Stem Cell Transplant
Official title: Open-Label Single-Arm Pilot Study of Deferasirox (Exjade®) in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron Overload
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Breast Cancer
Iron Overload
Leukemia
Lymphoma
Multiple Myeloma and Plasma Cell Neoplasm
Myelodysplastic Syndromes
Neuroblastoma
Ovarian Cancer
Keywords: iron overload
Lead sponsor: Masonic Cancer Center, University of Minnesota
Sponsor class: OTHER
First posted: 2008-01-28
Enrollment: 4
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Number of Patients Not Completing Treatment
timeFrame: 6 Months
Secondary outcomes:
measure: Reduction in Liver Iron Concentration After Study Drug
timeFrame: 6 Months
Other outcomes:
Locations:
city: Minneapolis
country: United States
facility: Masonic Cancer Center at University of Minnesota
state: Minnesota
Study officials:
affiliation: Masonic Cancer Center, University of Minnesota
name: Linda J. Burns, MD
role: PRINCIPAL_INVESTIGATOR
References: