Acute Myelogenous Leukemia in RelapsePhase 1NCT02267863
A Study of APTO-253 in Patients With Relapsed or Refractory AML or MDS
A Phase 1 study testing APTO-253 in people with Acute Myelogenous Leukemia in Relapse and Acute Myelogenous Leukemia, Relapsed, Adult, run by Aptose Biosciences Inc. at 13 sites in United States.
Trial summary
This study is being done to evaluate the safety and effectiveness of APTO-253 for the treatment of patients with the condition of acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS) for which either the standard treatment has failed, is no longer effective, or can no longer be administered safely or… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myelogenous Leukemia in Relapse, Acute Myelogenous Leukemia, Relapsed, Adult, Acute Myelogenous Leukemia, Adult
- What is being tested?
- APTO-253
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 13 sitesUnited States
- Study dates
- Oct 2014to Sep 2021
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 ≥18 years old
- Life expectancy of at least 2 months
- Off previous cancer therapy for at least 14 days, or 5 half-lives for noncytotoxic agents prior to first study treatment administration
- Patients must have a calculated creatinine clearance >60 mL/min
- Acceptable hematologic, renal and liver functions and coagulation status parameters
Who cannot join
- Patients with GVHD requiring systemic immunosuppressive therapy
- Uncontrolled leptomeningeal disease, auto-immune hemolytic anemia and uncontrolled and clinical significant disease related metabolic disorder
- Clinically significant intravascular coagulation
- Treatment with other investigational drugs within 14 days prior to first study treatment administration
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
United States13 sites
University of Michigan
Ann Arbor, Michigan, 48109
Emory University; Winship Cancer Institute
Atlanta, Georgia, 30322
St. Vincent Frontier Cancer Center
Billings, Montana, 59102
University Hospital
Cleveland, Ohio, 44106
Baylor Research Institute
Dallas, Texas, 75246
Prisma Health, Institute for Translational Oncology Research
Greenville, South Carolina, 29605
MD Anderson Cancer Center
Houston, Texas, 77030
UC San Diego Moores Cancer Center
La Jolla, California, 92093-0698
Ochsner Cancer Institute
New Orleans, Louisiana, 70121
University of California, Irvine
Orange, California, 92868
Oregon Health & Science University
Portland, Oregon, 97239
University of Rochester; Wilmot Cancer Institute Clinical Trials Office
Rochester, New York, 14643
University of Arizona Cancer Center
Tucson, Arizona, 85724
About this study
Official title: A Phase Ia/b Dose Escalation and Expansion, Multicenter, Open-label, Safety, Pharmacokinetic and Pharmacodynamic Study of APTO-253 in Patients With Relapsed or Refractory Acute Myelogenous Leukemia or High-Risk Myelodysplasia
This study is being done to evaluate the safety and effectiveness of APTO-253 for the treatment of patients with the condition of acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS) for which either the standard treatment has failed, is no longer effective, or can no longer be administered safely or poses a risk for your general well being.
Read the detailed description
This is a multicenter, open-label, Phase Ia/b dose escalation study of safety, pharmacodynamics, and pharmacokinetics of APTO-253 in ascending cohorts (3+3 design) to determine the MTD or recommended dose in patients with relapsed or refractory acute myelogenous leukemia (AML) or high-risk MDS patients. This is to be followed by a cohort expansion phase at the MTD or recommended dose.
- Conditions
- Acute Myelogenous Leukemia in Relapse, Acute Myelogenous Leukemia, Relapsed, Adult, Acute Myelogenous Leukemia, Adult, Acute Myelogenous Leukemia, High Risk Myelodysplasia
- Being tested
- APTO-253
- Keywords
- AML, MDS, Leukemia, Acute Myeloid Leukemia, Aptose, APTO-253, Kinase Inhibitor
- Medical subject headings
- Leukemia, Myeloid, Acute, Recurrence, Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
APTO-253Drug
APTO-253 will be given in ascending doses starting at 20 mg/m2 until the maximum tolerated dose or recommended dose is reached.
| Study group | Role | What they receive |
|---|---|---|
| Dose Escalation and Expansion | Experimental | APTO-253 will be given in ascending doses in patients with relapsed or refractory AML or high risk MDS (escalation cohort), until the maximum tolerated dose or recommended dose is reached. Followed by up to 30 patients enrolled in the expansion cohort at the recommended dose. Receives: Drug: APTO-253 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events of APTO-253
To determine the safety and tolerability of APTO-253 by assessing treatment-related adverse events as assessed by CTCAE v4.0.
Measured: Cycle 1 (28 days)
Maximum tolerated dose and dose limiting toxicities
To determine the maximum tolerated dose (MTD) and the dose limiting toxicities (DLT) of APTO-253 when given on days 1, 8, 15, and 22 of each 28-day cycle.
Measured: Cycle 1 (28 days)
Establish recommended dose for future development of APTO-253
To establish the dose of APTO-253 recommended for future development of APTO-253 for patients with specific types of hematologic malignancies.
Measured: Up to 7 months
Secondary outcomes (8)
Pharmacokinetic variables including maximum plasma concentration (Cmax)
Pharmacokinetic variables including maximum plasma concentration (Cmax)
Measured: Cycle 1 (28 days)
Pharmacokinetic variables including minimum plasma concentration (Cmin)
Pharmacokinetic variables including minimum plasma concentration (Cmin)
Measured: Cycle 1 (28 days)
Pharmacokinetic variables including Area Under the Curve (AUC)
Pharmacokinetic variables including Area Under the Curve (AUC)
Measured: Cycle 1 (28 days)
Pharmacokinetic variables including volume of distribution
Pharmacokinetic variables including volume of distribution
Measured: Cycle 1 (28 days)
Pharmacokinetic variables including clearance
Pharmacokinetic variables including clearance
Measured: Cycle 1 (28 days)
Pharmacokinetic variables including serum half-life
Pharmacokinetic variables including serum half-life
Measured: Cycle 1 (28 days)
Assess for any evidence of antitumor activity of APTO-253 by hematologic and bone marrow evaluations in acute leukemia and MDS.
To observe patients for any evidence of antitumor activity of APTO-253 by hematologic and bone marrow evaluations in acute leukemia and MDS.
Measured: Average 2 Cycles (8 weeks)
Determine the ability of APTO-253 to alter the expression of pharmacodynamic biomarkers of drug effect.
To determine the ability of APTO-253 to alter the expression of pharmacodynamic biomarkers of drug effect.
Measured: Average 2 Cycles (8 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Oct 2014 (estimated)
- Study startOct 2014
- Primary completionSep 2021
- Study completionSep 2021
Why it stopped: Drug manufacturing process and procedure review
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myelogenous Leukemia in RelapseStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aptose Biosciences Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Rafael Bejar, MD., PhD., Study director, Aptose Biosciences Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02267863
- Sponsor's protocol ID
- 253-HEM1-01
- Status last verified
- Aug 2022
- First submitted
- 3 Oct 2014
- Last updated
- 22 Aug 2022
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase Ia/b Dose Escalation and Expansion, Multicenter, Open-label, Safety, Pharmacokinetic and Pharmacodynamic Study of APTO-253 in Patients With Relapsed or Refractory Acute Myelogenous Leukemia or High-Risk Myelodysplasia
- ClinicalTrials.gov ID
- NCT02267863
- Acronym / sponsor ID
- 253-HEM1-01
- Conditions
- Acute Myelogenous Leukemia in Relapse, Acute Myelogenous Leukemia, Relapsed, Adult, Acute Myelogenous Leukemia, Adult, Acute Myelogenous Leukemia, High Risk Myelodysplasia
- Keywords
- AML, MDS, Leukemia, Acute Myeloid Leukemia, Aptose, APTO-253, Kinase Inhibitor
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Drug manufacturing process and procedure review
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Aptose Biosciences Inc.Industry (pharma, biotech, devices)
- Study start
- Oct 2014
- Primary completion
- Sep 2021 (actual)
- Study completion
- Sep 2021 (actual)
- First posted
- 20 Oct 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is being done to evaluate the safety and effectiveness of APTO-253 for the treatment of patients with the condition of acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS) for which either the standard treatment has failed, is no longer effective, or can no longer be administered safely or poses a risk for your general well being.
Detailed description
This is a multicenter, open-label, Phase Ia/b dose escalation study of safety, pharmacodynamics, and pharmacokinetics of APTO-253 in ascending cohorts (3+3 design) to determine the MTD or recommended dose in patients with relapsed or refractory acute myelogenous leukemia (AML) or high-risk MDS patients. This is to be followed by a cohort expansion phase at the MTD or recommended dose.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dose Escalation and Expansion | Experimental | Drug: APTO-253APTO-253 will be given in ascending doses in patients with relapsed or refractory AML or high risk MDS (escalation cohort), until the maximum tolerated dose or recommended dose is reached. Followed by up to 30 patients enrolled in the expansion cohort at the recommended dose. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Patients ≥18 years old
- Life expectancy of at least 2 months
- Off previous cancer therapy for at least 14 days, or 5 half-lives for noncytotoxic agents prior to first study treatment administration
- Patients must have a calculated creatinine clearance >60 mL/min
- Acceptable hematologic, renal and liver functions and coagulation status parameters
Key exclusion criteria (4 listed)
- Patients with GVHD requiring systemic immunosuppressive therapy
- Uncontrolled leptomeningeal disease, auto-immune hemolytic anemia and uncontrolled and clinical significant disease related metabolic disorder
- Clinically significant intravascular coagulation
- Treatment with other investigational drugs within 14 days prior to first study treatment administration
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 |
|---|---|---|
| APTO-253APTO-253 will be given in ascending doses starting at 20 mg/m2 until the maximum tolerated dose or recommended dose is reached. | Drug | Dose Escalation and Expansion |
Primary outcome
Incidence of treatment-emergent adverse events of APTO-253
Time frame: Cycle 1 (28 days)
To determine the safety and tolerability of APTO-253 by assessing treatment-related adverse events as assessed by CTCAE v4.0.
Maximum tolerated dose and dose limiting toxicities
Time frame: Cycle 1 (28 days)
To determine the maximum tolerated dose (MTD) and the dose limiting toxicities (DLT) of APTO-253 when given on days 1, 8, 15, and 22 of each 28-day cycle.
Establish recommended dose for future development of APTO-253
Time frame: Up to 7 months
To establish the dose of APTO-253 recommended for future development of APTO-253 for patients with specific types of hematologic malignancies.
8 secondary outcomes
Pharmacokinetic variables including maximum plasma concentration (Cmax)
Time frame: Cycle 1 (28 days)
Pharmacokinetic variables including minimum plasma concentration (Cmin)
Time frame: Cycle 1 (28 days)
Pharmacokinetic variables including Area Under the Curve (AUC)
Time frame: Cycle 1 (28 days)
Pharmacokinetic variables including volume of distribution
Time frame: Cycle 1 (28 days)
Pharmacokinetic variables including clearance
Time frame: Cycle 1 (28 days)
Pharmacokinetic variables including serum half-life
Time frame: Cycle 1 (28 days)
Assess for any evidence of antitumor activity of APTO-253 by hematologic and bone marrow evaluations in acute leukemia and MDS.
Time frame: Average 2 Cycles (8 weeks)
Determine the ability of APTO-253 to alter the expression of pharmacodynamic biomarkers of drug effect.
Time frame: Average 2 Cycles (8 weeks)
Results and safety
The study was stopped early (reason given: Drug manufacturing process and procedure review). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Aug 2022.
- Aptose BiosciencesLink provided by the sponsor.
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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.
Rafael Bejar, MD., PhD. study director
Aptose Biosciences Inc.
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing APTO-253 in people with Acute Myelogenous Leukemia in Relapse and Acute Myelogenous Leukemia, Relapsed, Adult, run by Aptose Biosciences Inc. at 13 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 5 inclusion and 4 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 22 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Drug manufacturing process and procedure review). Results are not available in the source record.
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?
Incidence of treatment-emergent adverse events of APTO-253 (time frame: Cycle 1 (28 days)). The study lists 3 primary outcomes in all. 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 22 Aug 2022.