Deferasirox for Treating Patients Who Have Undergone Allogeneic Stem Cell Transplant and Have Iron Overload

June 7, 2019 updated by: City of Hope Medical Center

Deferasirox Treatment and Labile Plasma Iron in Iron Overloaded Patients Who Have Undergone Allogeneic Hematopoietic Stem Cell Transplantation

RATIONALE: Low dose deferasirox may be safe and effective in treating patients who have undergone hematopoietic stem cell transplant and have iron overload.

PURPOSE: This pilot clinical trial studies safety and tolerability of deferasirox in hematopoietic stem cell transplant recipients who have iron overload. Effect of low dose deferasirox on labile plasma iron is also examined.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

PRIMARY OBJECTIVES:

I. To determine labile plasma iron (LPI) levels in iron overloaded patients after allogeneic Hematopoietic Stem Cell Transplantation (HSCT).

II. To determine safety and tolerability of low dose deferasirox in the post allogeneic HSCT setting.

SECONDARY OBJECTIVES:

I. To determine ability of deferasirox to suppress LPI in allogeneic HSCT patients with serum ferritin over 1500 ng/ml.

II. To determine prevalence of elevated LPI in allogeneic HSCT recipients with serum ferritin over 1500 ng/ml.

III. To determine ability of low dose deferasirox to lower serum ferritin during the treatment period.

IV. To correlate LPI with serum ferritin in allogeneic HSCT recipients with serum ferritin over 1500 ng/ml.

OUTLINE: Patients receive deferasirox at 10 mg/kg once daily for 6 months in the absence of unacceptable toxicity. Labile plasma iron will be measured at baseline and at weeks 4, 12, and 24. Side effects of deferasirox will be recorded.

Study Type

Interventional

Enrollment (Actual)

1

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Duarte, California, United States, 91010
        • City of Hope

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion

  • Patients must have undergone a matched related donor, matched unrelated donor or cord blood Hematopoietic Stem Cell Transplant (HSCT) over 6 months ago
  • Patients currently on Desferal (desferrioxamine) therapy will require a one day wash out prior to the first dose of study drug
  • Serum ferritin >= 1500 ng/mL on two occasions two weeks apart at screening; samples must be obtained in the absence of concomitant infection
  • Normal C-reactive protein level at screening
  • Patients must be red cell transfusion independent for 2 months prior to enrollment
  • Sexually active women must use an effective method of contraception, or must have undergone clinical documented total hysterectomy and/or oophorectomy, or tubal ligation or be postmenopausal (defined as amenorrhea for at least 12 months)
  • Written informed consent by the patient

Exclusion

  • Chronic hepatic GVHD with serum total bilirubin over 2 mg/dL
  • Known hypersensitivity to deferasirox
  • Serum creatinine above the upper limit of normal
  • AST or ALT > 200 U/L during screening
  • Clinical or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA positive and ALT above the normal range)
  • History of HIV positive test result (ELISA or Western blot)
  • History of drug or alcohol abuse within the 12 months prior to enrollment
  • ECOG Performance Status > 2
  • Patients with a diagnosis of or history of clinically relevant ocular toxicity related to iron chelation
  • Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent study treatment
  • Pregnancy (as documented in required screening laboratory test) or breast feeding
  • Patients who received treatment with systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days or are planning to receive other investigational drugs while participating in the study
  • Other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of study drug
  • History of non-compliance to medical regimens or patients who are considered potentially unreliable and/or not cooperative

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: SUPPORTIVE_CARE
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Arm I
Patients receive oral deferasirox once daily for up to 6 months in the absence of unacceptable toxicity.
Drug: deferasirox
Given orally
Other Names:
  • Exjade
  • ICL670

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of Patients With Elevated Labile Plasma Iron (LPI) Above Threshold (0.5 Umol/L)
Time Frame: At baseline
At baseline

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Patients With LPI Below 0.5 Umol/L After Treatment
Time Frame: Assessed through 6 months from the start of treatment
In patients with LPI values above threshold 0.5umol/L at baseline, number of patients had LPI suppressed below this value after treatment. Measurement of LPI is done on plasma specimens.
Assessed through 6 months from the start of treatment
Number of Patients With Serum Ferritin Level Lower Than 1500 ng/mL After Treatment
Time Frame: Assessed through 6 months from the start of treatment
Number of patients, whose Serum Ferritin levels are lower than 1500 ng/mL at two consecutive study visits. Serum Ferritin levels are measured at screening (baseline), week 4, 8, 12, 16, 20, 24 and end of study.
Assessed through 6 months from the start of treatment
Correlation of LPI With Serum Ferritin
Time Frame: Assessed through 6 months from the start of treatment
Both LPI and Serum Ferritin levels are measured at screening (baseline), week 4, 12, 24 and end of study. The correlation between the levels of LPI and Serum Ferritin at screening, week 4, 12, 24 and end of study will be examined and plotted.
Assessed through 6 months from the start of treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

City of Hope Medical Center

Investigators

  • Principal Investigator: Vinod Pullarkat, MD, City of Hope Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

July 1, 2010

Primary Completion (ACTUAL)

August 9, 2011

Study Completion (ACTUAL)

August 9, 2011

Study Registration Dates

First Submitted

July 6, 2010

First Submitted That Met QC Criteria

July 8, 2010

First Posted (ESTIMATE)

July 9, 2010

Study Record Updates

Last Update Posted (ACTUAL)

June 18, 2019

Last Update Submitted That Met QC Criteria

June 7, 2019

Last Verified

November 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 09187
  • NCI-2010-01428

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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