AnemiaPhase 2NCT02943668
Deferasirox in Treating Patients With Very Low, Low, or Intermediate-Risk Red Blood Cell Transfusion Dependent Anemia or Myelodysplastic Syndrome
A Phase 2 study testing Deferasirox and Laboratory Biomarker Analysis in people with Anemia and Myelodysplastic Syndrome, run by Fred Hutchinson Cancer Center at 1 site in United States.
Trial summary
This phase II trial studies how well deferasirox works in treating patients with very low, low, or intermediate-risk anemia or myelodysplastic syndrome that depends on red blood cell transfusions. Deferasirox may treat too much iron in the blood caused by blood transfusions. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Anemia, Myelodysplastic Syndrome
- What is being tested?
- Deferasirox and Laboratory Biomarker Analysis
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Mar 2017to 17 Dec 2018
- 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
- Capable of giving written informed consent prior to any study-specific procedures
- Diagnosis of MDS as defined by the World Health Organization (WHO) diagnostic criteria
- Have very low, low or intermediate-risk disease by the Revised International Prognostic Scoring System (IPSS-R)
- Baseline serum ferritin level >= 100 ng/mL
- Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
- Anemia defined as: hemoglobin =< 10.0 g/dL
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- Bilirubin =< 1.5 times upper limit of normal (ULN)
- Alanine aminotransferase (ALT), and aspartate aminotransferase (AST) =< 3.5 times ULN
- Serum creatinine =< 1.5 x ULN
- Estimated glomerular filtration rate (GFR) > 40 mL/min
- Males and females with reproductive potential must agree to use medically approved contraceptive precautions during the study and for 3 months following the last dose of deferasirox
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of study treatment; effective contraception methods include:
- Placement of an intrauterine device (IUD) or intrauterine system (IUS)
- Barrier methods of contraception: condom or occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/vaginal suppository
- Total abstinence or (when this is in line with the preferred and usual lifestyle of the subject); periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception
- Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy) or tubal ligation at least six weeks before taking study treatment; in case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment
- Male sterilization (at least 6 months prior to screening); for female subjects on the study, the vasectomized male partner should be the sole partner for that subject
- Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks ago; in the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of child bearing potential; sexually active males must use a condom during intercourse while taking drug and for 28 days after stopping study medication and should not father a child in this period; a condom is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid
- Females with childbearing potential* must have had a negative urine or serum pregnancy test =< 7 days before the first dose of deferasirox and must also not be breastfeeding
- Reliable and willing to make themselves available for the duration of the study and are willing to follow study procedures
Who cannot join
- If the patient is currently receiving erythroid stimulating agents (ESA) with plans to continue during study, less than 2 months duration of ESA prior to starting study drug and no dose escalation within 2 months of start of study drug
- If the patient is being treated with granulocyte-colony stimulating factor (GCSF) and/or a TPO-mimetic (for example, eltrombopag or romiplostim) with plans to continue during the study: Less than 2 months duration of GCSF or the TPO-mimetic treatment prior to starting study drug; or GCSF and/or TPO-mimetic has been added to ESA therapy within 2 months of start of study drug
- If patient is being treated with lenalidomide with plans to continue during the study: Stable dose for less than 3 months prior to start of study drug
- If patient is being treated with hypomethylating agents (HMA) (for example, azacitidine or decitabine) with plans to continue during the study: Stable dose for less than 6 months prior to start of study drug
- Currently enrolled in, or discontinued within the last 14 days from a clinical trial involving an investigational product or non-approved use of a drug, or concurrently enrolled in any other type of medical research judged not to be scientifically or medically compatible with this study
- Presence of >= 10% blast by morphologic examination of bone marrow aspirate or biopsy
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- Platelets =< 50,000
- Microcytosis on screening blood cell count (CBC) (mean corpuscular volume [MCV] < 81 fL)
- Active gastrointestinal (GI) ulceration or hemorrhage
- Have a serious preexisting medical condition that, in the opinion of the investigator would preclude participation in the study (for example a GI disorder causing clinically significant symptoms such as nausea, vomiting, and diarrhea, or malabsorption syndrome) or that would result in a life expectancy of less than 1 year
- Known hypersensitivity to deferasirox
- History of non-transfusional hemosiderosis
- Prior hematopoietic stem cell transplant for the diagnosis of MDS
- A second primary malignancy that in the judgment of the principal investigator (PI) or designee may affect the interpretation of results
- Have an active fungal, bacterial, and/or known viral infection including human immunodeficiency virus (HIV) or viral (A, B, or C) hepatitis
- Currently using aluminum-containing antacid products
- History of clinically significant auditory or ocular toxicity with ICT
Trial locations and contacts
1 site in 1 country.
United States1 site
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, 98109
About this study
Official title: A Phase II Study of Deferasirox in Patients With Myelodysplastic Syndromes Who Are Anemic With Iron Overload
This phase II trial studies how well deferasirox works in treating patients with very low, low, or intermediate-risk anemia or myelodysplastic syndrome that depends on red blood cell transfusions. Deferasirox may treat too much iron in the blood caused by blood transfusions.
Read the detailed description
PRIMARY OBJECTIVES:
- To assess the activity of iron chelation therapy (ICT) with deferasirox, in patients with anemia due to myelodysplastic syndrome (MDS).
SECONDARY OBJECTIVES:
- Reduction in red blood cell (RBC) transfusion requirements. II. Hematologic improvement. III. Change in serum ferritin levels from baseline to the end of the study as measured on a monthly basis.
IV. Safety and tolerability of deferasirox.
EXPLORATORY OBJECTIVES:
- Blood and marrow samples will be taken to study erythropoiesis and the impact of iron overload on erythropoiesis.
OUTLINE: Patients receive deferasirox orally (PO) once daily (QD). Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up for 30 days.
- Conditions
- Anemia, Myelodysplastic Syndrome
- Being tested
- Deferasirox, Laboratory Biomarker Analysis
- Medical subject headings
- Anemia, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DeferasiroxDrug
Also called Exjade
Given PO
Laboratory Biomarker AnalysisOther
Correlative studies
| Study group | Role | What they receive |
|---|---|---|
| Treatment (deferasirox) | Experimental | Patients receive deferasirox PO QD. Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity. Receives: Drug: Deferasirox; Other: Laboratory Biomarker Analysis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Patients That Achieve Erythroid Hematologic Improvement.
As defined by the modified International Working Group (IWG) response criteria:
Erythroid response (pretreatment, <11 g/dL):
- Hgb increase by ≥ 1.5 g/dL
- Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 wk compared with the pretreatment transfusion number in the previous 8 wk. Only RBC transfusions given for a Hgb of ≤ 0.9 g/dL pretreatment will count in the RBC transfusion response evaluation.
Platelet response (pretreatment, < 100 x 10^9/L)
- Absolute increase of ≥ 30 x 10^9/L for patients starting with > 20 x 10^9/L platelets
- Increase from < 20 x 10^9/L to > 20 x 10^9/L and by at least 100%
Neutrophil response (pretreatment, < 1.0 x 10^9/L)
- At least 100% increase and an absolute increase > 0.5 x 10^9/L
Measured: At 6 months
Secondary outcomes (3)
Change in Red Blood Cell (RBC) Transfusion Requirements
Assessed monthly for up to twelve months.
Measured: Baseline up to 12 months
Change in Serum Ferritin Levels
Assessed monthly for up to twelve months.
Measured: Baseline up to 12 months
Proportion of Patients Who Achieve Granulocyte or Platelet Hematologic Improvement
As defined by the modified International Working Group (IWG) response criteria:
Erythroid response (pretreatment, <11 g/dL):
- Hgb increase by ≥ 1.5 g/dL
- Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 wk compared with the pretreatment transfusion number in the previous 8 wk. Only RBC transfusions given for a Hgb of ≤ 0.9 g/dL pretreatment will count in the RBC transfusion response evaluation.
Platelet response (pretreatment, < 100 x 10^9/L)
- Absolute increase of ≥ 30 x 10^9/L for patients starting with > 20 x 10^9/L platelets
- Increase from < 20 x 10^9/L to > 20 x 10^9/L and by at least 100%
Neutrophil response (pretreatment, < 1.0 x 10^9/L)
- At least 100% increase and an absolute increase > 0.5 x 10^9/L
Measured: At 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Oct 2016 (estimated)
- Study start2 Mar 2017
- Primary completion17 Dec 2018
- Study completion17 Dec 2018
Why it stopped: Terminated due to low accrual
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 AnemiaStudies of the same condition, open and under way.
- More trials run by Fred Hutchinson Cancer CenterThe 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fred Hutchinson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI), Novartis Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Bart Scott, Principal investigator, Fred Hutch/University of Washington Cancer Consortium
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02943668
- Sponsor's protocol ID
- 9422
- Other IDs
- NCI-2016-01457, 9422, P30CA015704
- Status last verified
- Apr 2020
- First submitted
- 21 Oct 2016
- Last updated
- 1 Jul 2020
- 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 1 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Deferasirox in Patients With Myelodysplastic Syndromes Who Are Anemic With Iron Overload
- ClinicalTrials.gov ID
- NCT02943668
- Acronym / sponsor ID
- 9422
- Conditions
- Anemia, Myelodysplastic Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated due to low accrual
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Fred Hutchinson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI), Novartis Pharmaceuticals
- Study start
- 2 Mar 2017 (actual)
- Primary completion
- 17 Dec 2018 (actual)
- Study completion
- 17 Dec 2018 (actual)
- First posted
- 25 Oct 2016 (estimated)
- Results
- Posted 1 Jul 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II trial studies how well deferasirox works in treating patients with very low, low, or intermediate-risk anemia or myelodysplastic syndrome that depends on red blood cell transfusions. Deferasirox may treat too much iron in the blood caused by blood transfusions.
Detailed description
PRIMARY OBJECTIVES:
- To assess the activity of iron chelation therapy (ICT) with deferasirox, in patients with anemia due to myelodysplastic syndrome (MDS).
SECONDARY OBJECTIVES:
- Reduction in red blood cell (RBC) transfusion requirements. II. Hematologic improvement. III. Change in serum ferritin levels from baseline to the end of the study as measured on a monthly basis.
IV. Safety and tolerability of deferasirox.
EXPLORATORY OBJECTIVES:
- Blood and marrow samples will be taken to study erythropoiesis and the impact of iron overload on erythropoiesis.
OUTLINE: Patients receive deferasirox orally (PO) once daily (QD). Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up for 30 days.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (deferasirox) | Experimental | Drug: Deferasirox, Other: Laboratory Biomarker AnalysisPatients receive deferasirox PO QD. Treatment continues for up to 52 weeks in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- Capable of giving written informed consent prior to any study-specific procedures
- Diagnosis of MDS as defined by the World Health Organization (WHO) diagnostic criteria
- Have very low, low or intermediate-risk disease by the Revised International Prognostic Scoring System (IPSS-R)
- Baseline serum ferritin level >= 100 ng/mL
- Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
Key exclusion criteria (17 listed)
- If the patient is currently receiving erythroid stimulating agents (ESA) with plans to continue during study, less than 2 months duration of ESA prior to starting study drug and no dose escalation within 2 months of start of study drug
- If the patient is being treated with granulocyte-colony stimulating factor (GCSF) and/or a TPO-mimetic (for example, eltrombopag or romiplostim) with plans to continue during the study: Less than 2 months duration of GCSF or the TPO-mimetic treatment prior to starting study drug; or GCSF and/or TPO-mimetic has been added to ESA therapy within 2 months of start of study drug
- If patient is being treated with lenalidomide with plans to continue during the study: Stable dose for less than 3 months prior to start of study drug
- If patient is being treated with hypomethylating agents (HMA) (for example, azacitidine or decitabine) with plans to continue during the study: Stable dose for less than 6 months prior to start of study drug
- Currently enrolled in, or discontinued within the last 14 days from a clinical trial involving an investigational product or non-approved use of a drug, or concurrently enrolled in any other type of medical research judged not to be scientifically or medically compatible with this study
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: ExjadeGiven PO | Drug | Treatment (deferasirox) |
| Laboratory Biomarker AnalysisCorrelative studies | Other | Treatment (deferasirox) |
Primary outcome
Proportion of Patients That Achieve Erythroid Hematologic Improvement.
Time frame: At 6 months
As defined by the modified International Working Group (IWG) response criteria: Erythroid response (pretreatment, <11 g/dL): 1. Hgb increase by ≥ 1.5 g/dL 2. Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 wk compared with the pretreatment transfusion number in the previous 8 wk. Only RBC transfusions given for a Hgb of ≤ 0.9 g/dL pretreatment will count in the RBC transfusion response evaluation. Platelet response (pretreatment, < 100 x 10^9/L) 1. Absolute increase of ≥ 30 x 10^9/L for patients starting with > 20 x 10^9/L platelets 2. Increase from < 20 x 10^9/L to > 20 x 10^9/L and by at least 100% Neutrophil response (pretreatment, < 1.0 x 10^9/L) 1) At least 100% increase and an absolute increase > 0.5 x 10^9/L
3 secondary outcomes
Change in Red Blood Cell (RBC) Transfusion Requirements
Time frame: Baseline up to 12 months
Change in Serum Ferritin Levels
Time frame: Baseline up to 12 months
Proportion of Patients Who Achieve Granulocyte or Platelet Hematologic Improvement
Time frame: At 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jul 2020.
- 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.
Bart Scott principal investigator
Fred Hutch/University of Washington Cancer Consortium
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 and Laboratory Biomarker Analysis in people with Anemia and Myelodysplastic Syndrome, run by Fred Hutchinson Cancer Center 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 20 inclusion and 17 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: stopped early, as of the sponsor's last update on 1 Jul 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?
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 1 Jul 2020). 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?
Proportion of Patients That Achieve Erythroid Hematologic Improvement. (time frame: At 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 1 Jul 2020.