Deferasirox in Treating Iron Overload Caused By Blood Transfusions in Patients With Hematologic Malignancies

Phase 2

Completed

• Conditions: Adult Langerhans Cell Histiocytosis; Adult Acute Lymphoblastic Leukemia in Remission; Adult Acute Myeloid Leukemia With t(15;17)(q22;q12); Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); Adult Grade III Lymphomatoid Granulomatosis; Angioimmunoblastic T-cell Lymphoma; Contiguous Stage II Adult Burkitt Lymphoma; Contiguous Stage II Adult Diffuse Large Cell Lymphoma; Contiguous Stage II Adult Diffuse Mixed Cell Lymphoma; Contiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma
• Interventions: Drug: deferasirox; Other: laboratory biomarker analysis; Other: enzyme-linked immunosorbent assay

• Registration Number: NCT01273766
• Lead Sponsor: Wake Forest University Health Sciences

Brief Summary

RATIONALE: Deferasirox may remove excess iron from the body caused by blood transfusions.

PURPOSE: This clinical trial studies deferasirox in treating iron overload caused by blood transfusions in patients with hematologic malignancies.

Detailed Description

PRIMARY OBJECTIVES: I. To determine the effects of the iron-chelating agent deferasirox on changes in: neutrophil function; macrophage function; lymphocyte function.

SECONDARY OBJECTIVES: I. To determine the effect of chelation on the incidence of bacterial, viral and fungal infections documented by clinical, microbiologically-proven versus radiologically-proven criteria. II. To determine the effect of iron chelation on mortality and morbidity with incidence of the following parameters: Need for hospitalization; Duration of hospitalization; Need for ventilatory support; Need for exchange transfusion/apheresis; Need for treatment with antifungals or antibiotics for documented infections.

OUTLINE: Patients receive oral deferasirox once daily for up to 6 months or until blood counts recover in the absence of disease progression or unacceptable toxicity.

Study Timeline

• Study Start: 2011-01
• Study First Submitted: 2011-01-07
• Study First Submitted QC: 2011-01-07
• Study First Posted: 2011-01-10
• Primary Completion: 2012-03
• Results First Submitted: 2013-06-20
• Results First Submitted QC: 2013-11-05
• Results First Posted: 2013-11-27
• Study Completion: 2014-12
• Status Verified: 2018-08
• Last Update Submitted: 2018-08-07
• Last Update Posted: 2018-09-07

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 16

Inclusion Criteria

• Patients must have a pathology confirmed diagnosis of one of the following: myelodysplastic syndrome (MDS); acute leukemia; multiple myeloma; myelofibrosis; lymphoma; chronic anemia; sickle cell anemia
• Iron score >= 2
• Absolute Neutrophil Count (ANC) >= 1,000
• Platelets >= 50,000
• Albumin >= 2 g/dL
• Alkaline phosphatase =< 5X Upper Limit of Normal (ULN)
• Total bilirubin =< 1.5
• Creatinine =< 2X age-appropriate Upper Limit of Normal (ULN) OR creatinine clearance >= 40 ml/min
• Serum Glutamic Oxaloacetic Transaminase (SGOT) [AST] and Serum Glutamic Pyruvic Transaminase (SGPT) [ALT] =< 5X Upper Limit of Normal (ULN)
• Eastern Cooperative Oncology Group(ECOG) performance status of 0 or 1
• Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation; should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately
• Ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria

• Patients with active disease undergoing chemotherapy treatment
• Patient who have been treated with rituximab or immunomodulating drugs =< 1 month prior to enrollment
• HIV-positive patients
• Hepatitis-C positive patients
• Women who are pregnant or breastfeeding
• Patients on hemodialysis/patients with renal failure
• Patients with sepsis or acute illness
• Known hypersensitivity to deferasirox
• Patients with moderate or severe hearing loss as defined by audiogram

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Arm I	laboratory biomarker analysis	Patients receive oral deferasirox once daily for up to 6 months or until blood counts recover in the absence of disease progression or unacceptable toxicity.
Arm I	enzyme-linked immunosorbent assay	Patients receive oral deferasirox once daily for up to 6 months or until blood counts recover in the absence of disease progression or unacceptable toxicity.
Arm I	deferasirox	Patients receive oral deferasirox once daily for up to 6 months or until blood counts recover in the absence of disease progression or unacceptable toxicity.

Primary Outcome Measures

Name	Time	Method
Changes in Mean Neutrophil Values (as Measured by Lab) for Arm 1 (Other Arms Were Used for Calibration Only)	Baseline, up to 6 months	Changes in Neutrophils between baseline and mean neutrophils values during treatment (measured after each dose)

Secondary Outcome Measures

Name	Time	Method
Need for Hospitalization, Ventilator Support, Exchange Transfusion/Apheresis or Treatment With Antifungals or Antibiotics	Baseline, up to 6 months	Records will be assessed at baseline and prospectively while on study.
Cumulative Incidence of Documented Bacterial, Fungal, and Viral Infections	Baseline, up to 6 months	Records will be assessed at baseline and prospectively while on study.

Trial Locations

Locations (1)

Comprehensive Cancer Center of Wake Forest University; 🇺🇸 Winston-Salem, North Carolina, United States