- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02233504
Pilot Study to Assess Hematologic Response in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndromes Undergoing Monotherapy With Exjade (Deferasirox)
April 7, 2020 updated by: Abramson Cancer Center of the University of Pennsylvania
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.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Anticipated)
25
Phase
- Early Phase 1
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
-
-
Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- Abramson Cancer Center of the University of Pennsylvania
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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 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.
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: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Number of Adverse Events
Time Frame: 2 years
|
2 years
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Investigators
- Principal Investigator: James Mangan, MD, PhD, Abramson Cancer Center of the University of Pennsylvania
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 (Actual)
August 1, 2014
Primary Completion (Actual)
October 1, 2017
Study Completion (Actual)
October 2, 2017
Study Registration Dates
First Submitted
September 4, 2014
First Submitted That Met QC Criteria
September 4, 2014
First Posted (Estimate)
September 8, 2014
Study Record Updates
Last Update Posted (Actual)
April 8, 2020
Last Update Submitted That Met QC Criteria
April 7, 2020
Last Verified
April 1, 2020
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Neoplasms by Histologic Type
- Neoplasms
- Bone Marrow Diseases
- Hematologic Diseases
- Precancerous Conditions
- Leukemia
- Leukemia, Myeloid
- Myelodysplastic Syndromes
- Leukemia, Myeloid, Acute
- Preleukemia
- Molecular Mechanisms of Pharmacological Action
- Chelating Agents
- Sequestering Agents
- Iron Chelating Agents
- Deferasirox
Other Study ID Numbers
- UPCC 40413
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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