High Risk MDS or AML PatientsEarly Phase 1NCT02233504
Pilot Study to Assess Hematologic Response in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndromes Undergoing Monotherapy With Exjade (Deferasirox)
A Early Phase 1 study testing Exjade® (deferasirox, ICL670) in people with High Risk MDS or AML Patients, run by Abramson Cancer Center at Penn Medicine at 1 site in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- High Risk MDS or AML Patients
- What is being tested?
- Exjade® (deferasirox, ICL670)
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2014to 2 Oct 2017
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Abramson Cancer Center of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
About this study
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.
- Conditions
- High Risk MDS or AML Patients
- Being tested
- Exjade® (deferasirox, ICL670)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Exjade® (deferasirox, ICL670)Drug
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Adverse Events
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2014 (estimated)
- Study startAug 2014
- Primary completionOct 2017
- Study completion2 Oct 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for High Risk MDS or AML PatientsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Abramson Cancer Center at Penn Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- James Mangan, MD, PhD, Principal investigator, Abramson Cancer Center at Penn Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02233504
- Sponsor's protocol ID
- UPCC 40413
- Status last verified
- Apr 2020
- First submitted
- 4 Sep 2014
- Last updated
- 8 Apr 2020
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study to Assess Hematologic Response in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndromes Undergoing Monotherapy With Exjade (Deferasirox)
- ClinicalTrials.gov ID
- NCT02233504
- Acronym / sponsor ID
- UPCC 40413
- Conditions
- High Risk MDS or AML Patients
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Abramson Cancer Center at Penn MedicineUniversity, hospital or other
- Study start
- Aug 2014 (actual)
- Primary completion
- Oct 2017 (actual)
- Study completion
- 2 Oct 2017 (actual)
- First posted
- 8 Sep 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (20 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Exjade® (deferasirox, ICL670) | Drug | – |
Primary outcome
Number of Adverse Events
Time frame: 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
James Mangan, MD, PhD principal investigator
Abramson Cancer Center at Penn Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Exjade® (deferasirox, ICL670) in people with High Risk MDS or AML Patients, run by Abramson Cancer Center at Penn Medicine at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Adverse Events (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Apr 2020.