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Acute Myeloid LeukemiaPhase 1NCT03063944

STAT Inhibitor OPB-111077, Decitabine and Venetoclax in Treating Patients With Acute Myeloid Leukemia That Is Refractory, Relapsed or Newly Diagnosed and Ineligible for Intensive Chemotherapy

A Phase 1 study testing STAT Inhibitor OPB-111077, Decitabine and Venetoclax in people with Acute Myeloid Leukemia and Recurrent Acute Myeloid Leukemia, run by Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This phase I trial studies the side effects and best dose of STAT inhibitor OPB-111077 when given together with decitabine and venetoclax in treating patients with acute myeloid leukemia that does not respond to treatment (refractory), has come back (relapsed), or is newly diagnosed and ineligible for intensive… Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
STAT Inhibitor OPB-111077, Decitabine and Venetoclax
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
37 participants
Where
1 siteUnited States
Study dates
17 Mar 2017to 4 Mar 2024
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 must have histologic evidence of high risk acute myeloid leukemia defined as one of the following:
  • Non-M3 AML refractory to standard primary induction therapy
  • Non-M3 AML relapsed after (i) any standard induction therapy (ii) any number of standard or experimental salvage therapies
  • Newly diagnosed non-M3 AML not eligible for intensive induction chemotherapy
  • Subjects must have an Eastern Cooperative Oncology Group (ECOG) performance status of 2 or less
  • Subjects must have a life expectancy of at least 4 weeks
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  • Subjects must be able to consume oral medication
  • Subjects must have recovered from the toxic effects of any prior chemotherapy to= < grade 1 (except alopecia)
  • Creatinine clearance (CrCL) >= 45
  • Total bilirubin =< 2 x upper limit of normal (ULN)
  • Serum glutamate pyruvate transaminase (SGPT) alanine aminotransferase (ALT) =< 2 x ULN
  • Negative pregnancy test for women with child-bearing potential
  • Patients must be able to sign consent and be willing and able to comply with scheduled visits, treatment plan and laboratory testing

Who cannot join

  • Subjects with FAB M3 (t(15;17)(q22;q21)[PML-RARalpha]) are not eligible
  • Subjects must not be receiving any chemotherapy agents (except hydroxyurea); intrathecal methotrexate and cytarabine are permissible
  • Subjects must not be receiving growth factors, except for erythropoietin
  • Subjects with a "currently active" second malignancy, other than non-melanoma skin cancer, carcinoma in situ of the cervix, resected incidental prostate cancer (staged pT2 with Gleason score =< 6 and postoperative prostate-specific antigen [PSA] < 0.5 ng/mL), or other adequately treated carcinoma-in-situ are ineligible; patients are not considered to have a "currently active" malignancy if they have completed therapy and are free of disease for >= 1 year
  • Subjects with uncontrolled high blood pressure, unstable angina, symptomatic congestive heart failure (New York Heart Association [NYHA] class 3), myocardial infarction within the past 6 months or serious uncontrolled cardiac arrhythmia are not eligible
  • Subjects with other severe concurrent disease which in the judgment of the investigator would make the patient inappropriate for entry into this study are ineligible
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  • Subjects must not have evidence of active leukemia in the central nervous system (CNS)
  • Subjects must not have received any investigational agents within 14 days or 5 half-lives (whichever is longer) of study entry
  • Subjects must not be pregnant or breastfeeding; pregnancy tests must be obtained for all females of child-bearing potential; pregnant or lactating patients are ineligible for this study; males or women of childbearing potential may not participate unless they have agreed to use an effective contraceptive method (defined as hormonal contraceptives, intrauterine devices, surgical contraceptives, or condoms)
  • Subjects who have uncontrolled infection are not eligible; patients must have any active infections under control; fungal disease must be stable for at least 2 weeks before study entry
  • Subjects with bacteremia must have documented negative blood cultures prior to study entry
  • Subjects who are suitable for and willing to receive standard intensive induction therapy
  • Subjects who are candidates for allogeneic transplantation, have a suitable donor, and are willing to undergo transplantation

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Sidney Kimmel Cancer Center at Thomas Jefferson University

    Philadelphia, Pennsylvania, 19171

Other trials in United States

About this study

Official title: Phase I Trial of OPB-111077 in Combination With Decitabine and Venetoclax for the Treatment of AML Refractory to or Ineligible for Intensive Chemotherapy

This phase I trial studies the side effects and best dose of STAT inhibitor OPB-111077 when given together with decitabine and venetoclax in treating patients with acute myeloid leukemia that does not respond to treatment (refractory), has come back (relapsed), or is newly diagnosed and ineligible for intensive chemotherapy. STAT inhibitor OPB-111077 and decitabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as venetoclax, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving STAT inhibitor OPB-111077, decitabine, and venetoclax may work better in treating patients with acute myeloid leukemia compared to decitabine alone.

Read the detailed description

PRIMARY OBJECTIVE:

  1. To determine the safety and tolerability of STAT inhibitor OPB-111077 (OPB-111077) in combination with decitabine and venetoclax.

SECONDARY OBJECTIVES:

  1. To describe any preliminary efficacy of OPB-111077 in combination with decitabine and venetoclax in patients with acute myeloid leukemia (AML).

II. To measure adenosine triphosphate (ATP) generation and perform metabolomics in patients with AML who are receiving OPB-111077, decitabine, and venetoclax.

III. To assess apoptosis and proliferation assays in patients with AML who are receiving OPB-111077, decitabine, and venetoclax.

OUTLINE: This is a dose escalation study of STAT inhibitor OPB-111077.

INDUCTION CYCLE 1: Patients receive STAT inhibitor OPB-111077 orally (PO) once daily (QD) on days 1-28, and venetoclax PO QD on days 4-28 of cycle 1. Patients also receive decitabine intravenously (IV) over approximately 1 hour on days 4-8 of cycle 1. Patients who achieve complete remission (CR) after 1 cycle move on to Maintenance, and patients who do not achieve a CR move on to Cycle 2.

INDUCTION CYCLE 2: Patients receive STAT inhibitor OPB-111077, venetoclax, and decitabine as in Induction Cycle 1. Patients who achieve CR, complete remission with incomplete hematologic recovery (CRi), partial remission (PR), or stable disease (or clinically significant hematologic improvement as determined by the investigator) move on to Maintenance.

MAINTENANCE: Patients receive STAT inhibitor OPB-111077, venetoclax, and decitabine as in Induction Cycle 1. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 3 months for 2 years.

Being tested
STAT Inhibitor OPB-111077, Decitabine, Venetoclax, Laboratory Biomarker Analysis
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.

  • STAT Inhibitor OPB-111077Drug

    Also called OPB-111077

    Given PO

  • DecitabineDrug

    Also called 2'-Deoxy-5-azacytidine, 4-Amino-1-(2-deoxy-beta-D-erythro-pentofuranosyl)-1,3,5-triazin-2(1H)-one, 2353-33-5, 5-Aza-2'-deoxycytidine

    Given IV

  • VenetoclaxDrug

    Also called 1257044-40-8, 4-(4-((2-(4-Chlorophenyl)-4, 4-dimethylcyclohex-1-en-1-yl)methyl)piperazin-1-yl)-N-((3-nitro-4-((tetrahydro-2H-pyran-4-ylmethyl)amino)phenyl)sulfonyl)-2-(1H-pyrrolo(2,3-b)pyridin-5-yloxy)benzamide, ABT-0199

    Given PO

  • Laboratory Biomarker AnalysisOther

    Correlative studies

Study groups in NCT03063944
Study groupRoleWhat they receive
Treatment (STAT inhibitor OPB-111077, venetoclax, decitabine)ExperimentalINDUCTION CYCLE 1: Patients receive STAT inhibitor OPB-111077 PO QD on days 1-28, and venetoclax PO QD on days 4-28 of cycle 1. Patients also receive decitabine IV over approximately 1 hour on days 4-8 of cycle 1. Patients who achieve complete remission (CR) after 1 cycle move on to Maintenance, and patients who do not achieve a CR move on to Cycle 2. INDUCTION CYCLE 2: Patients receive STAT inhibitor OPB-111077, venetoclax, and decitabine as in Induction Cycle 1. Patients who achieve CR, CRi, PR, or stable disease (or clinically significant hematologic improvement as determined by the investigator) move on to Maintenance. MAINTENANCE: Patients receive STAT inhibitor OPB-111077, venetoclax, and decitabine as in Induction Cycle 1. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Receives: Drug: STAT Inhibitor OPB-111077; Drug: Decitabine; Drug: Venetoclax; Other: Laboratory Biomarker Analysis

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of grade 4, non-hematologic dose limiting toxicities assessed by National Cancer Institute Common Toxicity Criteria for Adverse Events version 5.0

    Data analysis will be descriptive. All estimates of dose-specific rates (e.g., response and toxicity) will be presented with corresponding confidence intervals using the exact method. The method of Atkinson and Brown will be used for any rate related to definition of dose limiting toxicity, due to two-stage sampling. The method of Conover will be used otherwise.

    Measured: Up to 2 years

Secondary outcomes (6)

  1. Metabolomics as determined by bone marrow biopsy

    Data analysis will be descriptive.

    Measured: At the end of Cycle 1 (each cycle is 28 days)

  2. Adenosine triphosphate (ATP) generation as determined by bone marrow biopsy

    Data analysis will be descriptive.

    Measured: At the end of Cycle 1 (each cycle is 28 days)

  3. Apoptotic rate in blood and bone marrow assessed by apoptosis assays

    Data analysis will be descriptive.

    Measured: At the end of Cycle 1 (each cycle is 28 days)

  4. Cellular proliferation in blood and bone marrow assessed by proliferation assays

    Data analysis will be descriptive.

    Measured: At the end of Cycle 1 (each cycle is 28 days)

  5. Complete response as determined by bone marrow biopsy

    Data analysis will be descriptive

    Measured: At the end of Cycle 1 (each cycle is 28 days)

  6. Complete response in the absence of total platelet recovery determined by bone marrow biopsy

    Data analysis will be descriptive

    Measured: At the end of Cycle 1 (each cycle is 28 days)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Feb 2017
  2. Study start17 Mar 2017
  3. Primary completion18 Jan 2024
  4. Study completion4 Mar 2024

Resources

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
Single group
Masking
None (open label)
Enrolment
37 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03063944
Sponsor's protocol ID
16C.773
Other IDs
JT 10092
Status last verified
May 2025
First submitted
21 Feb 2017
Last updated
15 May 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing STAT Inhibitor OPB-111077, Decitabine and Venetoclax in people with Acute Myeloid Leukemia and Recurrent Acute Myeloid Leukemia, run by Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University 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 13 inclusion and 13 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 15 May 2025. 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 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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 grade 4, non-hematologic dose limiting toxicities assessed by National Cancer Institute Common Toxicity Criteria for Adverse Events version 5.0 (time frame: Up to 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03063944.

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 15 May 2025.

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