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Pilot Study to Assess Hematologic Response in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndromes Undergoing Monotherapy With Exjade (Deferasirox)

NCT02233504

Description:

The purpose of this trial is to examine the hematologic response rate of Exjade® in patients with AML and high risk MDS and chronic iron overload from blood transfusions. Deferasirox has been developed as an iron-chelating agent, and unlike deferoxamine, a previously developed iron chelator, deferasirox has the advantage of oral administration.

Related Conditions:
  • Acute Myeloid Leukemia
  • Myelodysplastic Syndromes
Recruiting Status:

Active, not recruiting

Phase:

Early Phase 1

Trial Eligibility

Document

Title

  • Brief Title: Pilot Study to Assess Hematologic Response in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndromes Undergoing Monotherapy With Exjade (Deferasirox)
  • Official Title: Pilot Study to Assess Hematologic Response in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndromes Undergoing Monotherapy With Exjade (Deferasirox)

Clinical Trial IDs

  • ORG STUDY ID: UPCC 40413
  • NCT ID: NCT02233504

Conditions

  • High Risk MDS or AML Patients

Interventions

DrugSynonymsArms
Exjade® (deferasirox, ICL670)

Purpose

The purpose of this trial is to examine the hematologic response rate of Exjade® in patients with AML and high risk MDS and chronic iron overload from blood transfusions. Deferasirox has been developed as an iron-chelating agent, and unlike deferoxamine, a previously developed iron chelator, deferasirox has the advantage of oral administration.

Trial Arms

NameTypeDescriptionInterventions

Eligibility Criteria

        Inclusion Criteria:

          -  Patients with high risk MDS or AML Exjade Pilot Assessing Hematologic Response in
             AML/MDS UPCC# 40413

          -  Newly diagnosed acute myeloid leukemia, not a candidate for intensive induction
             chemotherapy in the judgement of the investigator or unwilling to undergo intensive
             induction chemotherapy OR

          -  AML that is refractory or relapsed after treatment with a non-intensive regimen, and
             not a candidate for allogeneic transplant at this time OR

          -  MDS, IPSS 1.5 or greater, intolerant or with disease progression/lack of response to
             hypomethylating agents.

          -  Age ≥ 18 years and able to provide independent informed consent

          -  Serum Ferritin: For enrollment on the study: serum ferritin ≥ 500 ng/mL at screening.
             Samples must be obtained in the absence of concomitant infection. If transfusion is
             scheduled, draw serum ferritin PRIOR to transfusion.

          -  ECOG performance status 0-2.

          -  Sexually active women must use an effective method of contraception, or must have
             undergone clinically documented total hysterectomy and/or oophorectomy, or tubal
             ligation or be postmenopausal (defined as amenorrhea for at least 12 months)

        Exclusion Criteria:

          -  Prior therapy with intensive chemotherapy for AML

          -  Prior therapy with iron chelating agents within the last 6 months.

          -  Serum creatinine levels greater than 1.5x above the upper limit of normal

          -  AST or ALT levels greater than 5x the upper limit of normal

          -  Current therapy for AML or MDS (a ≥ 4 week washout period for any agent used to treat
             AML or MDS prior to first dose of study drug is required).

          -  Current therapy with hydrea to control leukocytosis.

          -  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) infection. In the absence of clinical suspicion for active infection,
             laboratory testing to assess Hepatitis B or C status will not be required at
             screening.

          -  Clinical history of HIV positive test result (ELISA or Western blot). Laboratory
             testing to assess HIV status will not be required at screening.

          -  Clinical or self reported history of drug or alcohol abuse within the 12 months prior
             to enrollment

          -  ECOG Performance Status > 2

          -  Uncontrolled systemic hypertension

          -  Severe cardiac insufficiency (NYHA III or IV), with uncontrolled and/or unstable
             cardiac or coronary artery disease not controlled by standard medical therapy

          -  Clinical diagnosis of or history of clinically relevant ocular toxicity related to
             iron chelation

          -  Systemic diseases (cardiovascular, renal, hepatic, etc.) which in the opinion of the
             investigator would prevent study treatment

          -  Pregnant or breast feeding women

          -  Treatment with systemic investigational drug within the past 4 weeks or topical
             investigational drug within the past 7 days or plan 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

          -  Hematopoietic stem cell transplant for MDS or AML.

          -  Active CNS leukemia Patients who are found to be ineligible after screening procedures
             will have the reason for ineligibility documented on the screening log. No further
             data will be collected in the CRF for these patients.
      
Maximum Eligible Age:N/A
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Number of Adverse Events
Time Frame:2 years
Safety Issue:
Description:

Details

Phase:Early Phase 1
Primary Purpose:Interventional
Overall Status:Active, not recruiting
Lead Sponsor:Abramson Cancer Center of the University of Pennsylvania

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