Acute Myeloid LeukemiaPhase 2EXJADENCT02341495
Deferasirox, Cholecalciferol, and Azacitidine in the Treatment of Newly Diagnosed AML Patients Over 65
A Phase 2 study testing Deferasirox, Cholecalciferol and Azacitidine in people with Acute Myeloid Leukemia, run by Elizabeth Henry at 1 site in United States.
Trial summary
The purpose of this study is to find out if by giving a combination of 3 drugs the leukemia will go into complete remission (meaning the leukemia is completely gone), and to find out how long it stays away. The drugs used in this project are FDA approved and commercially available. Read more about this study
- Who is it for?
- Ages 65 to 89 years
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Deferasirox, Cholecalciferol and Azacitidine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2013to Nov 2016
- 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 65 to 89 years
- All sexes
- No healthy volunteers
- Older adult
Who may be able to join
- 65 or older (must have reached 65th birthday)
- Morphologically confirmed diagnosis of AML, excluding AML-M3
- Must have a Zubrod performance status of 0-3
Who cannot join
- Patients with known HIV infection are excluded. If HIV infection is suspected based on clinical condition, testing may be performed at the discretion of treatment team, but is not mandated prior to enrollment.
- Patients with central nervous system involvement by AML are excluded
- Patients with history of an active cancer (except basal cell and squamous cell cancers of the skin) within the previous 2 years are excluded
Trial locations and contacts
1 site in 1 country.
United States1 site
Loyola University Cardinal Bernardin Cancer Center
Maywood, Illinois, 60153
About this study
Official title: A Phase II Study Examining the Use of Deferasirox, Cholecalciferol, and Azacitidine in the Treatment of Newly Diagnosed Acute Myelogenous Leukemia (AML) in Elderly Patients
The purpose of this study is to find out if by giving a combination of 3 drugs the leukemia will go into complete remission (meaning the leukemia is completely gone), and to find out how long it stays away. The drugs used in this project are FDA approved and commercially available.
Read the detailed description
The majority of new cases of acute myeloid leukemia (AML) are diagnosed in patients older than 65 years of age. The median age of patients at time of diagnosis is 67 years. Standard therapies for AML often have significant toxicity and are poorly tolerated by this patient population. Treatment strategies for elderly patients with AML are limited. In those patients who do achieve complete remission, duration of remission tends to be short. Treatment at time of relapse is exceedingly difficult and outcomes are dismal. Thus, there is need for safer and more effective therapies for elderly patients with AML. This trial is a novel therapeutic combination of deferasirox (Exjade), cholecalciferol, and azacitidine in patients 65 years or older with newly diagnosed AML who are not fit for standard induction chemotherapy or hematopoietic stem-cell transplantation.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Deferasirox, Cholecalciferol, Azacitidine
- Keywords
- Azacitidine, Vidaza, Deferasirox, Exjade, Cholecalciferol, Vitamin D
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DeferasiroxDrug
Also called Exjade
Deferasirox (20mg/kg/day)on days 1-7 of protocol every 4 wks for 8 cycles PO
CholecalciferolDrug
Also called Vitamin D
Cholecalciferol(4,000 units/day) on days 1-7 of protocol every 4 wks for 8 cycles PO
AzacitidineDrug
Also called Vidaza
Azacitidine (75mg/m2 subcutaneous or IV administration) on days 1-7 of protocol 28 days for 32 wks
| Study group | Role | What they receive |
|---|---|---|
| Drug Treatment | Experimental | Deferasirox (20mg/kg/day)on days 1-7 of protocol, repeated every four weeks for 8 cycles given PO Cholecalciferol(4,000 units/day), on days 1-7 of protocol, repeated every four weeks for 8 cycles given PO Azacitidine (75mg/m2 subcutaneous or IV administration) on days 1-7 of protocol, repeated every four weeks for 8 cycles given either subcutaneously or IV Receives: Drug: Deferasirox; Drug: Cholecalciferol; Drug: Azacitidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Remission Rate
The Complete Remission Rate for all evaluable patients will be reported for the following treatment: azacitadine, vitamin D, and deferasirox (Exjade). The proportion of patients experiencing per-protocol CR will be calculated with a 90% exact confidence interval.
Measured: up to 5 years
Secondary outcomes (3)
Number of Patients With Adverse Events
Characterization of the safety of an induction regimen of azacitidine in combination with vitamin D and deferasirox (Exjade). The proportion of patients experiencing severe adverse events, and the number of patients experiencing severe adverse events probably or definitely related to treatment will be determined. Adverse events will be tabulated by category and graded by NCI CTCAE version 4.0
Measured: up to 5 years
Survival
The overall survival and relapse-free survival functions. Product-limit (Kaplan-Meier) estimate of the survival functions will be estimated, with 90% confidence intervals.
Measured: up to 5 years
Duration of Remission
The length of time for remission after achieving complete remission
Measured: up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jan 2015 (estimated)
- Study startFeb 2013
- Primary completionNov 2016
- Study completionNov 2016
Why it stopped: Study was terminated early due to low patient 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 Acute Myeloid LeukemiaStudies 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
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Elizabeth Henry
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Elizabeth Henry, Loyola University
- Investigators
- Elizabeth Henry, MD, Principal investigator, Faculty
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02341495
- Sponsor's protocol ID
- 204961
- Acronym
- EXJADE
- Status last verified
- Feb 2020
- First submitted
- 26 Sep 2013
- Last updated
- 27 Feb 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No: There is no plan to share individuals participant data.
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study Examining the Use of Deferasirox, Cholecalciferol, and Azacitidine in the Treatment of Newly Diagnosed Acute Myelogenous Leukemia (AML) in Elderly Patients
- ClinicalTrials.gov ID
- NCT02341495
- Acronym / sponsor ID
- EXJADE / 204961
- Conditions
- Acute Myeloid Leukemia
- Keywords
- Azacitidine, Vidaza, Deferasirox, Exjade, Cholecalciferol, Vitamin D
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study was terminated early due to low patient accrual.
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Elizabeth HenryUniversity, hospital or other
- Study start
- Feb 2013 (actual)
- Primary completion
- Nov 2016 (actual)
- Study completion
- Nov 2016 (actual)
- First posted
- 19 Jan 2015 (estimated)
- Results
- Posted 27 Feb 2020
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 study is to find out if by giving a combination of 3 drugs the leukemia will go into complete remission (meaning the leukemia is completely gone), and to find out how long it stays away. The drugs used in this project are FDA approved and commercially available.
Detailed description
The majority of new cases of acute myeloid leukemia (AML) are diagnosed in patients older than 65 years of age. The median age of patients at time of diagnosis is 67 years. Standard therapies for AML often have significant toxicity and are poorly tolerated by this patient population. Treatment strategies for elderly patients with AML are limited. In those patients who do achieve complete remission, duration of remission tends to be short. Treatment at time of relapse is exceedingly difficult and outcomes are dismal. Thus, there is need for safer and more effective therapies for elderly patients with AML. This trial is a novel therapeutic combination of deferasirox (Exjade), cholecalciferol, and azacitidine in patients 65 years or older with newly diagnosed AML who are not fit for standard induction chemotherapy or hematopoietic stem-cell transplantation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Drug Treatment | Experimental | Drug: Deferasirox, Drug: Cholecalciferol, Drug: AzacitidineDeferasirox (20mg/kg/day)on days 1-7 of protocol, repeated every four weeks for 8 cycles given PO Cholecalciferol(4,000 units/day), on days 1-7 of protocol, repeated every four weeks for 8 cycles given PO Azacitidine (75mg/m2 subcutaneous or IV administration) on days 1-7 of protocol, repeated every four weeks for 8 cycles given either subcutaneously or IV |
Who may qualify
- Ages
- 65 to 89 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Older adult
Key inclusion criteria (3 listed)
- 65 or older (must have reached 65th birthday)
- Morphologically confirmed diagnosis of AML, excluding AML-M3
- Must have a Zubrod performance status of 0-3
Key exclusion criteria (3 listed)
- Patients with known HIV infection are excluded. If HIV infection is suspected based on clinical condition, testing may be performed at the discretion of treatment team, but is not mandated prior to enrollment.
- Patients with central nervous system involvement by AML are excluded
- Patients with history of an active cancer (except basal cell and squamous cell cancers of the skin) within the previous 2 years are excluded
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: ExjadeDeferasirox (20mg/kg/day)on days 1-7 of protocol every 4 wks for 8 cycles PO | Drug | Drug Treatment |
| CholecalciferolAlso: Vitamin DCholecalciferol(4,000 units/day) on days 1-7 of protocol every 4 wks for 8 cycles PO | Drug | Drug Treatment |
| AzacitidineAlso: VidazaAzacitidine (75mg/m2 subcutaneous or IV administration) on days 1-7 of protocol 28 days for 32 wks | Drug | Drug Treatment |
Primary outcome
Complete Remission Rate
Time frame: up to 5 years
The Complete Remission Rate for all evaluable patients will be reported for the following treatment: azacitadine, vitamin D, and deferasirox (Exjade). The proportion of patients experiencing per-protocol CR will be calculated with a 90% exact confidence interval.
3 secondary outcomes
Number of Patients With Adverse Events
Time frame: up to 5 years
Survival
Time frame: up to 5 years
Duration of Remission
Time frame: up to 5 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No. There is no plan to share individuals participant data.
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Acute Biphenotypic Leukemia, Acute Myeloid Leukemia
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ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
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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.
Elizabeth Henry, MD principal investigator
Faculty
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, Cholecalciferol and Azacitidine in people with Acute Myeloid Leukemia, run by Elizabeth Henry 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 3 inclusion and 3 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 27 Feb 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 27 Feb 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?
Complete Remission Rate (time frame: up to 5 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 27 Feb 2020.