Acute Undifferentiated LeukemiaPhase 2NCT01273766
Deferasirox in Treating Iron Overload Caused By Blood Transfusions in Patients With Hematologic Malignancies
A Phase 2 study testing deferasirox, laboratory biomarker analysis and enzyme-linked immunosorbent assay in people with Acute Undifferentiated Leukemia and Adult Acute Lymphoblastic Leukemia in Remission, run by Wake Forest University Health Sciences at 1 site in United States.
Trial summary
RATIONALE: Deferasirox may remove excess iron from the body caused by blood transfusions. PURPOSE: This clinical trial studies deferasirox in treating iron overload caused by blood transfusions in patients with hematologic malignancies. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- What is being tested?
- deferasirox, laboratory biomarker analysis and enzyme-linked immunosorbent assay
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2011to Dec 2014
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 must have a pathology confirmed diagnosis of one of the following: myelodysplastic syndrome (MDS); acute leukemia; multiple myeloma; myelofibrosis; lymphoma; chronic anemia; sickle cell anemia
- Iron score >= 2
- Absolute Neutrophil Count (ANC) >= 1,000
- Platelets >= 50,000
- Albumin >= 2 g/dL
- Alkaline phosphatase =< 5X Upper Limit of Normal (ULN)
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- Total bilirubin =< 1.5
- Creatinine =< 2X age-appropriate Upper Limit of Normal (ULN) OR creatinine clearance >= 40 ml/min
- Serum Glutamic Oxaloacetic Transaminase (SGOT) [AST] and Serum Glutamic Pyruvic Transaminase (SGPT) [ALT] =< 5X Upper Limit of Normal (ULN)
- Eastern Cooperative Oncology Group(ECOG) performance status of 0 or 1
- Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation; should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately
- Ability to understand and the willingness to sign a written informed consent document
Who cannot join
- Patients with active disease undergoing chemotherapy treatment
- Patient who have been treated with rituximab or immunomodulating drugs =< 1 month prior to enrollment
- HIV-positive patients
- Hepatitis-C positive patients
- Women who are pregnant or breastfeeding
- Patients on hemodialysis/patients with renal failure
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- Patients with sepsis or acute illness
- Known hypersensitivity to deferasirox
- Patients with moderate or severe hearing loss as defined by audiogram
Trial locations and contacts
1 site in 1 country.
United States1 site
Comprehensive Cancer Center of Wake Forest University
Winston-Salem, North Carolina, 27157
About this study
Official title: Impact of Intervention With Deferasirox on the Immune Function of Patients With Hematologic Diseases and Transfusion-Related Iron Overload
RATIONALE: Deferasirox may remove excess iron from the body caused by blood transfusions.
PURPOSE: This clinical trial studies deferasirox in treating iron overload caused by blood transfusions in patients with hematologic malignancies.
Read the detailed description
PRIMARY OBJECTIVES: I. To determine the effects of the iron-chelating agent deferasirox on changes in: neutrophil function; macrophage function; lymphocyte function.
SECONDARY OBJECTIVES: I. To determine the effect of chelation on the incidence of bacterial, viral and fungal infections documented by clinical, microbiologically-proven versus radiologically-proven criteria. II. To determine the effect of iron chelation on mortality and morbidity with incidence of the following parameters: Need for hospitalization; Duration of hospitalization; Need for ventilatory support; Need for exchange transfusion/apheresis; Need for treatment with antifungals or antibiotics for documented infections.
OUTLINE: Patients receive oral deferasirox once daily for up to 6 months or until blood counts recover in the absence of disease progression or unacceptable toxicity.
- Conditions
- Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
- Being tested
- deferasirox, laboratory biomarker analysis, enzyme-linked immunosorbent assay
- Medical subject headings
- Leukemia, Biphenotypic, Acute, Congenital Abnormalities, Histiocytosis, Langerhans-Cell, Lymphoma, Extranodal NK-T-Cell, Lymphoma, Large-Cell, Anaplastic, Immunoblastic Lymphadenopathy, Intraocular Lymphoma, Leukemia, Mast-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
deferasiroxDrug
Also called Exjade, ICL670
Given orally
laboratory biomarker analysisOther
Correlative studies
enzyme-linked immunosorbent assayOther
Also called ELISA
Correlative studies
| Study group | Role | What they receive |
|---|---|---|
| Arm I | Experimental | Patients receive oral deferasirox once daily for up to 6 months or until blood counts recover in the absence of disease progression or unacceptable toxicity. Receives: Drug: deferasirox; Other: laboratory biomarker analysis; Other: enzyme-linked immunosorbent assay |
| control arm | No intervention | blood tested on healthy patients |
| correlative | No intervention | treated off study with or without oral deferasirox (patient choice) but lab draws to gather lab analysis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in Mean Neutrophil Values (as Measured by Lab) for Arm 1 (Other Arms Were Used for Calibration Only)
Changes in Neutrophils between baseline and mean neutrophils values during treatment (measured after each dose)
Measured: Baseline, up to 6 months
Secondary outcomes (2)
Need for Hospitalization, Ventilator Support, Exchange Transfusion/Apheresis or Treatment With Antifungals or Antibiotics
Records will be assessed at baseline and prospectively while on study.
Measured: Baseline, up to 6 months
Cumulative Incidence of Documented Bacterial, Fungal, and Viral Infections
Records will be assessed at baseline and prospectively while on study.
Measured: Baseline, up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2011 (estimated)
- Study startJan 2011
- Primary completionMar 2012
- Study completionDec 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Acute Undifferentiated LeukemiaStudies of the same condition, open and under way.
- More trials run by Wake Forest University Health SciencesThe sponsor's other registered studies.
- 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
- Phase 2
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Wake Forest University Health Sciences
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Mary Ann Knovich, MD, Principal investigator, Wake Forest University Health Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01273766
- Sponsor's protocol ID
- IRB00015287
- Other IDs
- NCI-2010-02228, CCCWFU 97710
- Status last verified
- Aug 2018
- First submitted
- 7 Jan 2011
- Last updated
- 7 Sep 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Impact of Intervention With Deferasirox on the Immune Function of Patients With Hematologic Diseases and Transfusion-Related Iron Overload
- ClinicalTrials.gov ID
- NCT01273766
- Acronym / sponsor ID
- IRB00015287
- Conditions
- Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Wake Forest University Health SciencesUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Jan 2011
- Primary completion
- Mar 2012 (actual)
- Study completion
- Dec 2014 (actual)
- First posted
- 10 Jan 2011 (estimated)
- Results
- Posted 27 Nov 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
RATIONALE: Deferasirox may remove excess iron from the body caused by blood transfusions.
PURPOSE: This clinical trial studies deferasirox in treating iron overload caused by blood transfusions in patients with hematologic malignancies.
Detailed description
PRIMARY OBJECTIVES: I. To determine the effects of the iron-chelating agent deferasirox on changes in: neutrophil function; macrophage function; lymphocyte function.
SECONDARY OBJECTIVES: I. To determine the effect of chelation on the incidence of bacterial, viral and fungal infections documented by clinical, microbiologically-proven versus radiologically-proven criteria. II. To determine the effect of iron chelation on mortality and morbidity with incidence of the following parameters: Need for hospitalization; Duration of hospitalization; Need for ventilatory support; Need for exchange transfusion/apheresis; Need for treatment with antifungals or antibiotics for documented infections.
OUTLINE: Patients receive oral deferasirox once daily for up to 6 months or until blood counts recover in the absence of disease progression or unacceptable toxicity.
Study design
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Arm I | Experimental | Drug: deferasirox, Other: laboratory biomarker analysis, Other: enzyme-linked immunosorbent assayPatients receive oral deferasirox once daily for up to 6 months or until blood counts recover in the absence of disease progression or unacceptable toxicity. |
| control arm | No intervention | –blood tested on healthy patients |
| correlative | No intervention | –treated off study with or without oral deferasirox (patient choice) but lab draws to gather lab analysis |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Patients must have a pathology confirmed diagnosis of one of the following: myelodysplastic syndrome (MDS); acute leukemia; multiple myeloma; myelofibrosis; lymphoma; chronic anemia; sickle cell anemia
- Iron score >= 2
- Absolute Neutrophil Count (ANC) >= 1,000
- Platelets >= 50,000
- Albumin >= 2 g/dL
Key exclusion criteria (9 listed)
- Patients with active disease undergoing chemotherapy treatment
- Patient who have been treated with rituximab or immunomodulating drugs =< 1 month prior to enrollment
- HIV-positive patients
- Hepatitis-C positive patients
- Women who are pregnant or breastfeeding
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 |
|---|---|---|
| deferasiroxAlso: Exjade, ICL670Given orally | Drug | Arm I |
| laboratory biomarker analysisCorrelative studies | Other | Arm I |
| enzyme-linked immunosorbent assayAlso: ELISACorrelative studies | Other | Arm I |
Primary outcome
Changes in Mean Neutrophil Values (as Measured by Lab) for Arm 1 (Other Arms Were Used for Calibration Only)
Time frame: Baseline, up to 6 months
Changes in Neutrophils between baseline and mean neutrophils values during treatment (measured after each dose)
2 secondary outcomes
Need for Hospitalization, Ventilator Support, Exchange Transfusion/Apheresis or Treatment With Antifungals or Antibiotics
Time frame: Baseline, up to 6 months
Cumulative Incidence of Documented Bacterial, Fungal, and Viral Infections
Time frame: Baseline, up to 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Sep 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Mary Ann Knovich, MD principal investigator
Wake Forest University Health Sciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing deferasirox, laboratory biomarker analysis and enzyme-linked immunosorbent assay in people with Acute Undifferentiated Leukemia and Adult Acute Lymphoblastic Leukemia in Remission, run by Wake Forest University Health Sciences 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 12 inclusion and 9 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 7 Sep 2018. 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?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 Nov 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Changes in Mean Neutrophil Values (as Measured by Lab) for Arm 1 (Other Arms Were Used for Calibration Only) (time frame: Baseline, up to 6 months). 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 7 Sep 2018.