AMLPhase 1/2NCT02795520
Pharmacological Study of Intravenous OTS167 in Patients With Refractory or Relapsed Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Advanced Myelodysplastic Syndromes, Advanced Myeloproliferative Neoplastic Disorders, or Advanced Chronic Myelogenous Leukemia
A Phase 1/2 study testing OTS167IV in people with AML and Advanced MDS, run by OncoTherapy Science, Inc. at 2 sites in United States.
Trial summary
The purpose of Phase I of this study is to test the safety and tolerability of the investigational drug, OTS167, and that of Phase II of this study is to confirm the potential response benefit of OTS167. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- AML, Advanced MDS, ALL
- What is being tested?
- OTS167IV
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 2 sitesUnited States
- Study dates
- Apr 2016to Sep 2021
- Phase
- Phase 1/2
- 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
- Diagnosis of:
- Relapsed or refractory AML (refractory to a standard anthracycline-based induction regimen or a hypomethylating agent for patients unfit for intensive chemotherapy or for whom no standard or curative therapy exists),
- ALL,
- Acute biphenotypic leukemia (assigned to the appropriate group by the treating physician by documented analysis of relevant laboratory values and pathology/cytogenetics),
- Advanced MDS defined as ≥5% bone marrow blasts or ≥2% blasts in the peripheral blood (including patients who have progressed following treatment with hypomethylating agents),
- Advanced MPN (excluding patients with ET, PV, or low risk MF), and MDS/MPN overlap syndrome with ≥5% bone marrow blasts or ≥2% blasts in the peripheral blood, or
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- Advanced CML after failure/progression of at least 3 prior TKIs
- Age ≥18 years
- No prior antineoplastic drug therapy for at least 14 days, with the exception of hydroxyurea, prior to starting OTS167. Patients with rapidly proliferative disease may continue to receive hydroxyurea
- Patients refractory to all approved therapies or for which no approved or conventional therapies are available
- Patients with a diagnosis of advanced CML must have been treated with 3 prior TKIs, and the last therapy must have been discontinued at least 14 days prior to starting OTS167
- Adequate organ function as defined below:
- Liver function (total bilirubin <2 mg/dL and aspartate aminotransferase and/or alanine aminotransferase <3 × upper limit of normal (ULN) or <5 × ULN if related to leukemic involvement)
- Renal function (creatinine <1.5 × ULN)
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2
- Negative urine pregnancy test within 1 week prior to Cycle 1 Day 1 for each woman of childbearing potential
- Able to understand the potential risks, benefits, and requirements of the study and are willing to provide informed consent; an informed consent form (ICF) for this study that is signed by the patient or his/her legally authorized representative is required prior to enrollment
Who cannot join
- Pregnant or breastfeeding patients (pregnant and breastfeeding women are excluded from this study because the agents used in this study may have unknown or unrecognized potential for teratogenic or abortifacient effects). Patients of childbearing potential must consent and agree to practice documented (type) adequate contraception during the course of on study treatment.
- Evidence of any form of active, uncontrolled, bacterial, viral (including hepatitis A, B, or C or known human immunodeficiency virus [HIV] seropositivity), or fungal infection. Patients who are positive for hepatitis B core antibody, hepatitis B surface antigen, or hepatitis C antibody must have a negative polymerase chain reaction (PCR) result before enrollment; those who are PCR-positive will be excluded. Evidence of congestive heart failure (New York Heart Association Class III or IV); myocardial infarction or stroke within 6 months; unstable angina; uncontrolled or unstable/medically important cardiac arrhythmia; prolonged QT interval corrected for heart rate (QTc) >450 msec (males) or >470 msec (females); uncontrolled epilepsy; uncontrolled bleeding; recent major surgical procedures within 30 days before Cycle 1 Day 1 without full recovery from the same; or any other serious comorbid medical condition that would preclude investigational study treatment
- Any psychiatric illness/social situations that would limit compliance with study requirements
- Documented hypersensitivity to any of the components of OTS167 or supportive care medicaments
- Central nervous system (CNS) leukemia
- MPN patients with ET, PV, or low risk MF
- Women of childbearing potential and men must agree prior to study entry to use appropriate contraception for the duration of study participation and until 30 days after receipt of the last dose of study drug
- Documented concurrent malignancy. Exceptions include cervical carcinoma in-situ, non-melanoma skin cancer (basal and squamous cell carcinoma), localized prostate cancer (Gleason score <6), and resected melanoma-in-situ. Other localized solid tumors in situ and other low risk cancers may also be exempt after discussion with the Sponsor Medical Monitor.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
University of Chicago
Chicago, Illinois, 60637
Weill Cornell Medicine
New York, New York, 10065
About this study
Official title: Phase I/II and Pharmacological Study of Intravenous OTS167 in Patients With Refractory or Relapsed Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Advanced Myelodysplastic Syndromes, Advanced Myeloproliferative Neoplastic Disorders, or Advanced Chronic Myelogenous Leukemia
The purpose of Phase I of this study is to test the safety and tolerability of the investigational drug, OTS167, and that of Phase II of this study is to confirm the potential response benefit of OTS167.
OTS167 is a maternal embryonic leucine zipper kinase (MELK) inhibitor which demonstrated antitumor properties in laboratory tests. It is being developed as an anti-cancer drug. In this study OTS167 will be administrated to patients with AML, ALL, advanced MDSs, advanced MPNs, or advanced CML.
- Conditions
- AML, Advanced MDS, ALL, Advanced CML, Advanced MPNs
- Being tested
- OTS167IV
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OTS167IVDrug
| Study group | Role | What they receive |
|---|---|---|
| OTS167IV | Experimental | Not described Receives: Drug: OTS167IV |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events assessed by CTCAE v4.03
Measured: Up to 30 days after last dose of study drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2016 (estimated)
- Study startApr 2016
- Primary completionMay 2021
- Study completionSep 2021
Why it stopped: slow patient accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AMLStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- OncoTherapy Science, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Olatoyosi Odenike, MBBS, Principal investigator, University of Chicago, Ilinois
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02795520
- Sponsor's protocol ID
- OTS167-SE02
- Status last verified
- Nov 2021
- First submitted
- 6 May 2016
- Last updated
- 24 Nov 2021
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II and Pharmacological Study of Intravenous OTS167 in Patients With Refractory or Relapsed Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Advanced Myelodysplastic Syndromes, Advanced Myeloproliferative Neoplastic Disorders, or Advanced Chronic Myelogenous Leukemia
- ClinicalTrials.gov ID
- NCT02795520
- Acronym / sponsor ID
- OTS167-SE02
- Conditions
- AML, Advanced MDS, ALL, Advanced CML, Advanced MPNs
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: slow patient accrual
- Enrolment
- 32 participants (actual)
- Lead sponsor
- OncoTherapy Science, Inc.Industry (pharma, biotech, devices)
- Study start
- Apr 2016 (actual)
- Primary completion
- May 2021 (actual)
- Study completion
- Sep 2021 (actual)
- First posted
- 10 Jun 2016 (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.
The purpose of Phase I of this study is to test the safety and tolerability of the investigational drug, OTS167, and that of Phase II of this study is to confirm the potential response benefit of OTS167.
OTS167 is a maternal embryonic leucine zipper kinase (MELK) inhibitor which demonstrated antitumor properties in laboratory tests. It is being developed as an anti-cancer drug. In this study OTS167 will be administrated to patients with AML, ALL, advanced MDSs, advanced MPNs, or advanced CML.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| OTS167IV | Experimental | Drug: OTS167IV |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Diagnosis of:
- Age ≥18 years
- No prior antineoplastic drug therapy for at least 14 days, with the exception of hydroxyurea, prior to starting OTS167. Patients with rapidly proliferative disease may continue to receive hydroxyurea
- Patients refractory to all approved therapies or for which no approved or conventional therapies are available
- Patients with a diagnosis of advanced CML must have been treated with 3 prior TKIs, and the last therapy must have been discontinued at least 14 days prior to starting OTS167
Key exclusion criteria (8 listed)
- Pregnant or breastfeeding patients (pregnant and breastfeeding women are excluded from this study because the agents used in this study may have unknown or unrecognized potential for teratogenic or abortifacient effects). Patients of childbearing potential must consent and agree to practice documented (type) adequate contraception during the course of on study treatment.
- Evidence of any form of active, uncontrolled, bacterial, viral (including hepatitis A, B, or C or known human immunodeficiency virus [HIV] seropositivity), or fungal infection. Patients who are positive for hepatitis B core antibody, hepatitis B surface antigen, or hepatitis C antibody must have a negative polymerase chain reaction (PCR) result before enrollment; those who are PCR-positive will be excluded. Evidence of congestive heart failure (New York Heart Association Class III or IV); myocardial infarction or stroke within 6 months; unstable angina; uncontrolled or unstable/medically important cardiac arrhythmia; prolonged QT interval corrected for heart rate (QTc) >450 msec (males) or >470 msec (females); uncontrolled epilepsy; uncontrolled bleeding; recent major surgical procedures within 30 days before Cycle 1 Day 1 without full recovery from the same; or any other serious comorbid medical condition that would preclude investigational study treatment
- Any psychiatric illness/social situations that would limit compliance with study requirements
- Documented hypersensitivity to any of the components of OTS167 or supportive care medicaments
- Central nervous system (CNS) leukemia
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 |
|---|---|---|
| OTS167IV | Drug | OTS167IV |
Primary outcome
Adverse events assessed by CTCAE v4.03
Time frame: Up to 30 days after last dose of study drug
Results and safety
The study was stopped early (reason given: slow patient accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Nov 2021.
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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.
Olatoyosi Odenike, MBBS principal investigator
University of Chicago, Ilinois
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing OTS167IV in people with AML and Advanced MDS, run by OncoTherapy Science, Inc. at 2 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 17 inclusion and 8 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 24 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 combined 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.
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?
The study was stopped early (reason given: slow patient accrual). 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?
Adverse events assessed by CTCAE v4.03 (time frame: Up to 30 days after last dose of study drug). 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 24 Nov 2021.