Acute Myeloid LeukemiaPhase 2NCT00565058
Combination of GTI-2040 and Cytarabine in the Treatment of Refractory and Relapsed Acute Myeloid Leukemia (AML)
A Phase 2 study testing GTI-2040 in people with Acute Myeloid Leukemia, run by Aptose Biosciences Inc. at 7 sites in United States.
Trial summary
This is a Phase II trial conducted at multiple centers for evaluation of the pharmacodynamic activity and the overall response rate contributed by the combination agents of GTI-2040 and High Dose Cytarabine (HiDAC) in Refractory and Relapsed Acute Myeloid Leukemia (AML). Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- GTI-2040
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 7 sitesUnited States
- Study dates
- Aug 2007to Feb 2010
- 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 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Patients must have unequivocal histologic diagnosis of AML according to WHO classification.
- Patients must have (1) refractory AML, defined as a disease unresponsive to the initial treatment; or (2) relapsed AML, defined as disease that re-occurs after treatment with conventional or high dose chemotherapy, with or without autologous stem cell support.
- Patients previously treated with antisense oligonucleotides remain eligible in absence of significant or dose-limiting documented toxicities directly attributable to the antisense agents.
- Age 18-59 years old.
- Because no dosing or adverse event data are currently available on the use of GTI-2040 in combination with cytarabine in patients <18 years of age, children are excluded from this study but will be eligible for future pediatric Phase 2 combination trials.
- Eastern Cooperative Oncology Group (ECOG) performance status <= 2 (Karnofsky >60%).
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- Patients with central nervous system (CNS) involvement will be considered eligible for this study if no residual leukemic cells are detectable in the cerebral spinal fluid following intrathecal or radiation therapy.
- Central line catheter for administration of GTI-2040 infusion is required for all patients enrolled in the study.
- Ability to understand and the willingness to sign a written informed consent document. Written informed consent is required prior to any study procedures for screening or enrollment.
Who cannot join
- Patients who have had chemotherapy (with the exception of hydroxyurea) or radiotherapy within 4 weeks prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier. Patients who have received mitomycin C or nitrosurea require a 6 week recovery period before enrollment.
- Patients who have had prior allogeneic stem cell transplant.
- Patients may not be receiving any other investigational agents as part of ongoing treatment.
- Patients with the following abnormal clinical values (unless abnormalities in these parameters are directly attributable to malignancy):
- Resting cardiac ejection fraction < 50%
- Serum creatinine > 1.5 mg/dL
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- Total bilirubin > 2x upper limits of normal (ULN) (unless due to Gilbert's syndrome)
- aspartate aminotransferase (AST) and alanine aminotransferase (ALT) > 3x ULN
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to GTI-2040 or other agents used in the study.
- Patients who require chronic systemic anticoagulant therapy for medical conditions (e.g., previous history of deep venous thrombosis, atrial fibrillation etc.). Heparin administration to maintain central line patency (i.e. catheter flush) is not an exclusion.
- Patients with uncontrolled intercurrent illness including, but not limited to, ongoing or active infection requiring IV antibiotics, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia.
- Serious medical or psychiatric illness that would prevent informed consent or limit survival to < 4 weeks.
- Pregnancy or breastfeeding women. The potential for teratogenic effects and other risks for GTI-2040 in nursing infants are unknown. Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control) prior to study entry and for the duration of study participation.
- HIV-positive patients on combination antiretroviral therapy are ineligible because these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy. Appropriate studies will be undertaken in patients receiving combination antiretroviral therapy when indicated.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Northside Hospital
Atlanta, Georgia, 30342
Roswell Park Cancer Institute
Buffalo, New York, 14263
The Ohio State University
Columbus, Ohio, 43210-1240
Indiana Cancer Research Institute
Indianapolis, Indiana, 46202
The Mount Sinai Hospital
New York, New York, 10029
San Francisco Veterans Affairs Medical Center
San Francisco, California, 94121
UCSF Medical Center
San Francisco, California, 94121
About this study
Official title: A Phase II Simon Two-stage Multicenter Study and Pilot Pharmacodynamic Investigation of GTI 2040 in Combination With High Dose Cytarabine (HiDAC) in Refractory and Relapsed Acute Myeloid Leukemia (AML)
This is a Phase II trial conducted at multiple centers for evaluation of the pharmacodynamic activity and the overall response rate contributed by the combination agents of GTI-2040 and High Dose Cytarabine (HiDAC) in Refractory and Relapsed Acute Myeloid Leukemia (AML).
- Conditions
- Acute Myeloid Leukemia
- Being tested
- GTI-2040
- 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.
GTI-2040Biological
GTI-2040 will be administered one day after HiDAC in the pilot PD study and one day before HiDAC in the Phase II study for a cycle. Those who achieve a complete remission (CR) will be permitted to receive one cycle of consolidation of GTI-2040 and HiDAC
| Study group | Role | What they receive |
|---|---|---|
| Pilot | Experimental | Pilot PD Study (Delayed GTI-2040) Group: In the Pilot PD Study Group, addition of GTI-2040 is delayed until 24 hours after initiation of HiDAC. Receives: Biological: GTI-2040 |
| Phase II arm | Experimental | Phase II PD Study (Early GTI-2040) Group: In the Phase II PD Study Group, GTI-2040 is given 24 hours prior to addition of HiDAC. Receives: Biological: GTI-2040 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate of GTI-2040 Combined With HiDAC in Refractory or Relapsed AML
Overall Response was defined as whether or not the patient achieved complete remission (CR) and CR with incomplete blood count recovery (CRi) while on the study.
Measured: at 29-35 days
Secondary outcomes (1)
Summary of Treatment Emergent Adverse Events
An adverse event (AE) was defined as any unintended or undesirable experience that occurred during the course of the clinical investigation, regardless of whether or not it was considered to be study drug-related. This included any newly occurring event or a previous condition that had increased in severity or frequency since the administration of study drug.
Measured: 30 days after the last dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Nov 2007 (estimated)
- Study startAug 2007
- Primary completionSep 2009
- Study completionFeb 2010
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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aptose Biosciences Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Ohio State University
- Responsible party
- Sponsor
- Investigators
- Rebecca B Klisovic, MD, Principal investigator, Ohio State University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00565058
- Sponsor's protocol ID
- 2040AML201
- Status last verified
- Apr 2015
- First submitted
- 27 Nov 2007
- Last updated
- 27 Jul 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Simon Two-stage Multicenter Study and Pilot Pharmacodynamic Investigation of GTI 2040 in Combination With High Dose Cytarabine (HiDAC) in Refractory and Relapsed Acute Myeloid Leukemia (AML)
- ClinicalTrials.gov ID
- NCT00565058
- Acronym / sponsor ID
- 2040AML201
- Conditions
- Acute Myeloid Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Aptose Biosciences Inc.Industry (pharma, biotech, devices)
- Collaborators
- Ohio State University
- Study start
- Aug 2007
- Primary completion
- Sep 2009 (actual)
- Study completion
- Feb 2010 (actual)
- First posted
- 29 Nov 2007 (estimated)
- Results
- Posted 27 Jul 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase II trial conducted at multiple centers for evaluation of the pharmacodynamic activity and the overall response rate contributed by the combination agents of GTI-2040 and High Dose Cytarabine (HiDAC) in Refractory and Relapsed Acute Myeloid Leukemia (AML).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Pilot | Experimental | Biological: GTI-2040Pilot PD Study (Delayed GTI-2040) Group: In the Pilot PD Study Group, addition of GTI-2040 is delayed until 24 hours after initiation of HiDAC. |
| Phase II arm | Experimental | Biological: GTI-2040Phase II PD Study (Early GTI-2040) Group: In the Phase II PD Study Group, GTI-2040 is given 24 hours prior to addition of HiDAC. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (9 listed)
- Patients must have unequivocal histologic diagnosis of AML according to WHO classification.
- Patients must have (1) refractory AML, defined as a disease unresponsive to the initial treatment; or (2) relapsed AML, defined as disease that re-occurs after treatment with conventional or high dose chemotherapy, with or without autologous stem cell support.
- Patients previously treated with antisense oligonucleotides remain eligible in absence of significant or dose-limiting documented toxicities directly attributable to the antisense agents.
- Age 18-59 years old.
- Because no dosing or adverse event data are currently available on the use of GTI-2040 in combination with cytarabine in patients <18 years of age, children are excluded from this study but will be eligible for future pediatric Phase 2 combination trials.
Key exclusion criteria (14 listed)
- Patients who have had chemotherapy (with the exception of hydroxyurea) or radiotherapy within 4 weeks prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier. Patients who have received mitomycin C or nitrosurea require a 6 week recovery period before enrollment.
- Patients who have had prior allogeneic stem cell transplant.
- Patients may not be receiving any other investigational agents as part of ongoing treatment.
- Patients with the following abnormal clinical values (unless abnormalities in these parameters are directly attributable to malignancy):
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to GTI-2040 or other agents used in the 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 |
|---|---|---|
| GTI-2040GTI-2040 will be administered one day after HiDAC in the pilot PD study and one day before HiDAC in the Phase II study for a cycle. Those who achieve a complete remission (CR) will be permitted to receive one cycle of consolidation of GTI-2040 and HiDAC | Biological | Phase II arm, Pilot |
Primary outcome
Overall Response Rate of GTI-2040 Combined With HiDAC in Refractory or Relapsed AML
Time frame: at 29-35 days
Overall Response was defined as whether or not the patient achieved complete remission (CR) and CR with incomplete blood count recovery (CRi) while on the study.
1 secondary outcome
Summary of Treatment Emergent Adverse Events
Time frame: 30 days after the last dose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2015.
- 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.
Rebecca B Klisovic, MD principal investigator
Ohio State University
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GTI-2040 in people with Acute Myeloid Leukemia, run by Aptose Biosciences Inc. at 7 sites 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 9 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: finished, as of the sponsor's last update on 27 Jul 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites 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 Jul 2015). 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?
Overall Response Rate of GTI-2040 Combined With HiDAC in Refractory or Relapsed AML (time frame: at 29-35 days). 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 Jul 2015.