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

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

Sponsors

Lead Sponsor: City of Hope Medical Center

Source City of Hope Medical Center
Brief Summary

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.

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.

Overall Status Terminated
Start Date 2010-07-01
Completion Date 2011-08-09
Primary Completion Date 2011-08-09
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Number of Patients With Elevated Labile Plasma Iron (LPI) Above Threshold (0.5 Umol/L) At baseline
Secondary Outcome
Measure Time Frame
Number of Patients With LPI Below 0.5 Umol/L After Treatment Assessed through 6 months from the start of treatment
Number of Patients With Serum Ferritin Level Lower Than 1500 ng/mL After Treatment Assessed through 6 months from the start of treatment
Correlation of LPI With Serum Ferritin Assessed through 6 months from the start of treatment
Enrollment 1
Condition
Intervention

Intervention Type: Drug

Intervention Name: deferasirox

Description: Given orally

Arm Group Label: Arm I

Eligibility

Criteria:

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

Gender:

All

Minimum Age:

18 Years

Maximum Age:

N/A

Healthy Volunteers:

No

Overall Official
Last Name Role Affiliation
Vinod Pullarkat, MD Principal Investigator City of Hope Medical Center
Location
Facility: City of Hope
Location Countries

United States

Verification Date

2017-11-01

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: Arm I

Type: Experimental

Description: Patients receive oral deferasirox once daily for up to 6 months in the absence of unacceptable toxicity.

Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Primary Purpose: Supportive Care

Masking: None (Open Label)

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 [email protected]. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Research News