Iron OverloadPhase 2NCT01159067
Deferasirox for Treating Patients Who Have Undergone Allogeneic Stem Cell Transplant and Have Iron Overload
A Phase 2 study testing deferasirox in people with Iron Overload and Accelerated Phase Chronic Myelogenous Leukemia, run by City of Hope Medical Center at 1 site in United States.
Trial summary
RATIONALE: Low dose deferasirox may be safe and effective in treating patients who have undergone hematopoietic stem cell transplant and have iron overload. PURPOSE: This pilot clinical trial studies safety and tolerability of deferasirox in hematopoietic stem cell transplant recipients who have iron overload. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Iron Overload, Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
- What is being tested?
- deferasirox
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2010to 9 Aug 2011
- 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 undergone a matched related donor, matched unrelated donor or cord blood Hematopoietic Stem Cell Transplant (HSCT) over 6 months ago
- Patients currently on Desferal (desferrioxamine) therapy will require a one day wash out prior to the first dose of study drug
- Serum ferritin >= 1500 ng/mL on two occasions two weeks apart at screening; samples must be obtained in the absence of concomitant infection
- Normal C-reactive protein level at screening
- Patients must be red cell transfusion independent for 2 months prior to enrollment
- Sexually active women must use an effective method of contraception, or must have undergone clinical documented total hysterectomy and/or oophorectomy, or tubal ligation or be postmenopausal (defined as amenorrhea for at least 12 months)
- Written informed consent by the patient
Who cannot join
- Chronic hepatic GVHD with serum total bilirubin over 2 mg/dL
- Known hypersensitivity to deferasirox
- Serum creatinine above the upper limit of normal
- AST or ALT > 200 U/L during screening
- 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)
- History of HIV positive test result (ELISA or Western blot)
Show all 14Show fewer
- History of drug or alcohol abuse within the 12 months prior to enrollment
- ECOG Performance Status > 2
- Patients with a diagnosis of or history of clinically relevant ocular toxicity related to iron chelation
- Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent study treatment
- Pregnancy (as documented in required screening laboratory test) or breast feeding
- Patients who received treatment with systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days or are planning 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Deferasirox Treatment and Labile Plasma Iron in Iron Overloaded Patients Who Have Undergone Allogeneic Hematopoietic Stem Cell Transplantation
RATIONALE: Low dose deferasirox may be safe and effective in treating patients who have undergone hematopoietic stem cell transplant and have iron overload.
PURPOSE: This pilot clinical trial studies safety and tolerability of deferasirox in hematopoietic stem cell transplant recipients who have iron overload. Effect of low dose deferasirox on labile plasma iron is also examined.
Read the detailed description
PRIMARY OBJECTIVES:
- To determine labile plasma iron (LPI) levels in iron overloaded patients after allogeneic Hematopoietic Stem Cell Transplantation (HSCT).
II. To determine safety and tolerability of low dose deferasirox in the post allogeneic HSCT setting.
SECONDARY OBJECTIVES:
- To determine ability of deferasirox to suppress LPI in allogeneic HSCT patients with serum ferritin over 1500 ng/ml.
II. To determine prevalence of elevated LPI in allogeneic HSCT recipients with serum ferritin over 1500 ng/ml.
III. To determine ability of low dose deferasirox to lower serum ferritin during the treatment period.
IV. To correlate LPI with serum ferritin in allogeneic HSCT recipients with serum ferritin over 1500 ng/ml.
OUTLINE: Patients receive deferasirox at 10 mg/kg once daily for 6 months in the absence of unacceptable toxicity. Labile plasma iron will be measured at baseline and at weeks 4, 12, and 24. Side effects of deferasirox will be recorded.
- Conditions
- Iron Overload, Accelerated Phase Chronic Myelogenous 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)
- Being tested
- deferasirox
- Keywords
- HSCT, iron overload, deferasirox, labile plasma iron
- Medical subject headings
- Iron Overload, Leukemia, Myeloid, Accelerated Phase, Congenital Abnormalities, Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative, Blast Crisis, Pdgfra-Associated Chronic Eosinophilic Leukemia, Leukemia, Myelomonocytic, Chronic, Leukemia, Neutrophilic, Chronic
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
| Study group | Role | What they receive |
|---|---|---|
| Arm I | Experimental | Patients receive oral deferasirox once daily for up to 6 months in the absence of unacceptable toxicity. Receives: Drug: deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients With Elevated Labile Plasma Iron (LPI) Above Threshold (0.5 Umol/L)
Measured: At baseline
Secondary outcomes (3)
Number of Patients With LPI Below 0.5 Umol/L After Treatment
In patients with LPI values above threshold 0.5umol/L at baseline, number of patients had LPI suppressed below this value after treatment. Measurement of LPI is done on plasma specimens.
Measured: Assessed through 6 months from the start of treatment
Number of Patients With Serum Ferritin Level Lower Than 1500 ng/mL After Treatment
Number of patients, whose Serum Ferritin levels are lower than 1500 ng/mL at two consecutive study visits. Serum Ferritin levels are measured at screening (baseline), week 4, 8, 12, 16, 20, 24 and end of study.
Measured: Assessed through 6 months from the start of treatment
Correlation of LPI With Serum Ferritin
Both LPI and Serum Ferritin levels are measured at screening (baseline), week 4, 12, 24 and end of study. The correlation between the levels of LPI and Serum Ferritin at screening, week 4, 12, 24 and end of study will be examined and plotted.
Measured: Assessed through 6 months from the start of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2010 (estimated)
- Study startJul 2010
- Primary completion9 Aug 2011
- Study completion9 Aug 2011
Why it stopped: Low enrollment
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 Iron OverloadStudies of the same condition, open and under way.
- More trials run by City of Hope Medical 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
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- City of Hope Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Vinod Pullarkat, MD, Principal investigator, City of Hope Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01159067
- Sponsor's protocol ID
- 09187
- Other IDs
- NCI-2010-01428
- Status last verified
- Nov 2017
- First submitted
- 6 Jul 2010
- Last updated
- 18 Jun 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Deferasirox Treatment and Labile Plasma Iron in Iron Overloaded Patients Who Have Undergone Allogeneic Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT01159067
- Acronym / sponsor ID
- 09187
- Conditions
- Iron Overload, Accelerated Phase Chronic Myelogenous 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)
- Keywords
- HSCT, iron overload, deferasirox, labile plasma iron
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Low enrollment
- Enrolment
- 1 participants (actual)
- Lead sponsor
- City of Hope Medical CenterUniversity, hospital or other
- Study start
- Jul 2010
- Primary completion
- 9 Aug 2011 (actual)
- Study completion
- 9 Aug 2011 (actual)
- First posted
- 9 Jul 2010 (estimated)
- Results
- Posted 29 May 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
RATIONALE: Low dose deferasirox may be safe and effective in treating patients who have undergone hematopoietic stem cell transplant and have iron overload.
PURPOSE: This pilot clinical trial studies safety and tolerability of deferasirox in hematopoietic stem cell transplant recipients who have iron overload. Effect of low dose deferasirox on labile plasma iron is also examined.
Detailed description
PRIMARY OBJECTIVES:
- To determine labile plasma iron (LPI) levels in iron overloaded patients after allogeneic Hematopoietic Stem Cell Transplantation (HSCT).
II. To determine safety and tolerability of low dose deferasirox in the post allogeneic HSCT setting.
SECONDARY OBJECTIVES:
- To determine ability of deferasirox to suppress LPI in allogeneic HSCT patients with serum ferritin over 1500 ng/ml.
II. To determine prevalence of elevated LPI in allogeneic HSCT recipients with serum ferritin over 1500 ng/ml.
III. To determine ability of low dose deferasirox to lower serum ferritin during the treatment period.
IV. To correlate LPI with serum ferritin in allogeneic HSCT recipients with serum ferritin over 1500 ng/ml.
OUTLINE: Patients receive deferasirox at 10 mg/kg once daily for 6 months in the absence of unacceptable toxicity. Labile plasma iron will be measured at baseline and at weeks 4, 12, and 24. Side effects of deferasirox will be recorded.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm I | Experimental | Drug: deferasiroxPatients receive oral deferasirox once daily for up to 6 months in the absence of unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Patients must have undergone a matched related donor, matched unrelated donor or cord blood Hematopoietic Stem Cell Transplant (HSCT) over 6 months ago
- Patients currently on Desferal (desferrioxamine) therapy will require a one day wash out prior to the first dose of study drug
- Serum ferritin >= 1500 ng/mL on two occasions two weeks apart at screening; samples must be obtained in the absence of concomitant infection
- Normal C-reactive protein level at screening
- Patients must be red cell transfusion independent for 2 months prior to enrollment
Key exclusion criteria (14 listed)
- Chronic hepatic GVHD with serum total bilirubin over 2 mg/dL
- Known hypersensitivity to deferasirox
- Serum creatinine above the upper limit of normal
- AST or ALT > 200 U/L during screening
- 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)
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 |
Primary outcome
Number of Patients With Elevated Labile Plasma Iron (LPI) Above Threshold (0.5 Umol/L)
Time frame: At baseline
3 secondary outcomes
Number of Patients With LPI Below 0.5 Umol/L After Treatment
Time frame: Assessed through 6 months from the start of treatment
Number of Patients With Serum Ferritin Level Lower Than 1500 ng/mL After Treatment
Time frame: Assessed through 6 months from the start of treatment
Correlation of LPI With Serum Ferritin
Time frame: Assessed through 6 months from the start of treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2019.
- 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.
Vinod Pullarkat, MD principal investigator
City of Hope Medical Center
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 in people with Iron Overload and Accelerated Phase Chronic Myelogenous Leukemia, run by City of Hope Medical 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 7 inclusion and 14 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 18 Jun 2019. 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 29 May 2019). 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?
Number of Patients With Elevated Labile Plasma Iron (LPI) Above Threshold (0.5 Umol/L) (time frame: At baseline). 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 18 Jun 2019.