Hematopoietic and Lymphoid Cell NeoplasmPhase 1/2NCT04874194

Omacetaxine and Venetoclax for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome Harboring Mutant RUNX1

A Phase 1/2 study testing Omacetaxine Mepesuccinate and Venetoclax in people with Hematopoietic and Lymphoid Cell Neoplasm and Recurrent Acute Biphenotypic Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Hematopoietic and Lymphoid Cell Neoplasm, Recurrent Acute Biphenotypic Leukemia, Recurrent Acute Myeloid Leukemia
What is being tested?: Omacetaxine Mepesuccinate and Venetoclax
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 24 participants
Where: 1 siteUnited States
Study dates: 17 Dec 2021to 16 Sep 2024
Phase: Phase 1/2
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 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Patients with a diagnosis of relapsed or refractory acute myeloid leukemia (AML) (or biphenotypic or bilineage leukemia including a myeloid component) or myelodysplastic syndrome
For myelodysplastic syndrome (MDS) patients, patients must have no response, progression, or relapse following at least 4 cycles of azacytidine or decitabine; and/or intolerance defined as grade >= 3 drug-related toxicity precluding continued therapy
Age >= 18 years
Subjects must have documented RUNX1 gene mutation
Eastern Cooperative Oncology Group (ECOG) performance status =< 2
Creatinine < 2 unless related to the disease

Show all 11Show fewer

Direct bilirubin < 2x upper limit of normal (ULN) unless increase is due to Gilbert's disease or leukemic involvement
Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) < 3x ULN unless considered due to leukemic involvement
In the absence of rapidly proliferative disease, the interval from prior treatment to time of initiation will be at least 7 days for cytotoxic or non-cytotoxic (i.e. immunotherapy) agents. Oral hydroxyurea and/or cytarabine (up to 2 g/m^2) for patients with rapidly proliferative disease is allowed before the start of study therapy, as needed, for clinical benefit and after discussion with the principal investigator (PI)
Male subjects must agree to refrain from unprotected sex and sperm donation from initial study drug administration until 90 days after the last dose of study drug
Willing and able to provide informed consent

Who cannot join

Patients with t(15;17) karyotypic abnormality or acute promyelocytic leukemia (French-American-British [FAB] class M3-AML)
Patients with any concurrent uncontrolled clinically significant medical condition including active infection or psychiatric illness, which could place the patient at unacceptable risk of study treatment
Patients with active graft-versus-host-disease (GVHD) status post stem cell transplant (patients without active GVHD on chronic suppressive immunosuppression and/or phototherapy for chronic skin GVHD are permitted after discussion with the PI)
Patients with any severe gastrointestinal or metabolic condition which could interfere with the absorption of oral study medications
Known active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection or known human immunodeficiency virus (HIV) infection
Subject has a white blood cell count > 25 x 10^9/L. (Note: Hydroxyurea is permitted to meet this criterion.)
Nursing women, women of childbearing potential (WOCBP) with positive urine pregnancy test, or women of childbearing potential who are not willing to maintain adequate contraception
Appropriate highly effective method(s) of contraception include oral or injectable hormonal birth control, intrauterine device (IUD), and double barrier methods (for example a condom in combination with a spermicide)

Trial locations and contacts

1 site in 1 country.

United States1 site

M D Anderson Cancer Center
Houston, Texas, 77030

Other trials in United States

About this study

Official title: Phase Ib/II Study of Omacetaxine and Venetoclax for Patients With Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome Harboring Mutant RUNX1

This phase Ib/II trial best dose, possible benefits and/or side effects of omacetaxine and venetoclax in treating patients with acute myeloid leukemia or myelodysplastic syndrome that has come back (recurrent) or does not respond to treatment (refractory) and have a genetic change RUNX1. Drugs used in chemotherapy, such as omacetaxine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Venetoclax may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Giving omacetaxine and venetoclax may help to control the disease.

Read the detailed description

PRIMARY OBJECTIVES:

To determine the safety and tolerability and recommended phase 2 dose (RP2D) of omacetaxine in combination with venetoclax for patients with relapsed/refractory acute myeloid leukemia or myelodysplastic syndrome harboring a RUNX1 mutation. (Phase 1b) II. To determine the efficacy of omacetaxine in combination with venetoclax for patients with relapsed/refractory acute myeloid leukemia or myelodysplastic syndrome harboring a RUNX1 mutation. (Phase II)

SECONDARY OBJECTIVES:

To determine duration of response (DOR), event-free survival (EFS), and overall survival (OS).

II. To evaluate occurrence of minimal residual disease (MRD) negative status by multiparameter flow cytometry and molecular evaluation.

OUTLINE: This is a phase I, dose de-escalation study followed by a phase II study.

Patients receive omacetaxine subcutaneously (SC) twice daily (BID) on days 2-3 or 2-4, and venetoclax orally (PO) on days 1-7, 1-10 or 1-14. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up within 30 days, then every 3 months for 3 years.

Conditions: Hematopoietic and Lymphoid Cell Neoplasm, Recurrent Acute Biphenotypic Leukemia, Recurrent Acute Myeloid Leukemia, Recurrent Myelodysplastic Syndrome, Refractory Acute Biphenotypic Leukemia, Refractory Acute Myeloid Leukemia
Being tested: Omacetaxine Mepesuccinate, Venetoclax
Medical subject headings: Hematologic Neoplasms, Leukemia, Biphenotypic, Acute, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Omacetaxine MepesuccinateDrug
Also called Ceflatonin, Cephalotaxine, 4-methyl 2-hydroxy-2-(4-hydroxy-4-methylpentyl)butanedioate (ester), [3(R)]- (9CI), CGX-635, HHT
Given SC
VenetoclaxDrug
Also called ABT-0199, ABT-199, ABT199, GDC-0199
Given PO

Study groups in NCT04874194
Study group	Role	What they receive
Ph 1 Arm A (AML) Dose 0	Experimental	Participants receive omacetaxine SC BID on days 2-4, and venetoclax PO on days 1-10 . Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Receives: Drug: Omacetaxine Mepesuccinate; Drug: Venetoclax
Ph 1 Arm B (MDS) Dose 0	Experimental	Participants receive omacetaxine SC BID on days 2-4, and venetoclax PO on days 1-10 . Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Receives: Drug: Omacetaxine Mepesuccinate; Drug: Venetoclax
Ph 1 Arm A (AML) Dose +1	Experimental	Participants receive omacetaxine SC BID on days 2-4, and venetoclax PO on days 1-14. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Receives: Drug: Omacetaxine Mepesuccinate; Drug: Venetoclax
Ph 1 Arm B (MDS) Dose + 1	Experimental	Participants receive omacetaxine SC BID on days 2-4, and venetoclax PO on days 1-14. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Receives: Drug: Omacetaxine Mepesuccinate; Drug: Venetoclax
Ph 2 Arm A (AML) Dose +1	Experimental	Participants receive omacetaxine SC BID on days 2-4, and venetoclax PO on days 1-14. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Receives: Drug: Omacetaxine Mepesuccinate; Drug: Venetoclax
Ph 2 Arm B (MDS) Dose + 1	Experimental	Participants receive omacetaxine SC BID on days 2-4, and venetoclax PO on days 1-14. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Receives: Drug: Omacetaxine Mepesuccinate; Drug: Venetoclax

What is being measured

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

Recommended Phase 2 Dose (RP2D) of Omacetaxine in Combination With Venetoclax
The RP2D will be selected at the end of the Phase 1b portion based on safety data , in Arms A and B independently. Preliminary efficacy and PK data for each dose level may also be considered as appropriate.
Measured: Up to 30 days
Number of Participant to Achieve Complete Remission
Complete Remission for AML is defined as: Absolute neutrophil count > 10^3/UL, platelets . 10^5/UL, red cell transfusion independence, absence of extramedullary disease, and bone marrow with < 5% blasts. Complete Remission for MDS is defined as: Absolute neutrophil count > 10^3/UL, platelets . 10^5/UL, hemoglobin >11 g/dl, and bone marrow with < 5% blasts. Peripheral dysplasia will be noted.
Measured: At day 28, and 3 cycles.

Secondary outcomes (3)

Event-free Survival (EFS)
Time from date of treatment start until the date of failure or death from any cause.
Measured: Up to Two years, 8 months, 30 days
Overall Survival (OS)
The Kaplan-Meier method will be used to estimate the probabilities. Log-rank tests will be used to compare among subgroups of patients in terms of OS.
Measured: Up to Two years, 8 months, 30 days
Duration of Response
Response date to loss of response or last follow up.
Measured: Up to Two years, 8 months, 30 days

Timeline

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

17 Dec 202116 Sep 2024

First posted5 May 2021
Study start17 Dec 2021
Primary completion16 Sep 2024
Study completion16 Sep 2024

Why it stopped: The study terminated early because the company was longer providing the investigational product.

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.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Hematopoietic and Lymphoid Cell NeoplasmStudies of the same condition, open and under way.
More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
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: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 24 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04874194
Sponsor's protocol ID: 2020-0890
Other IDs: NCI-2021-03341, 2020-0890
Status last verified: Oct 2025
First submitted: 1 May 2021
Last updated: 3 Nov 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

DiNardo CD, Jen WY, Montalban-Bravo G, Wang X, Loghavi S, Lavu S, Short NJ, Chien K, Issa GC, Pemmaraju N, Yilmaz M, Andreeff M, Borthakur G, Kadia TM, Daver NG, Garcia-Manero G, Mill CP, Su X, Fiskus W, Bhalla KN. Omacetaxine and venetoclax in relapsed/refractory acute myeloid leukemia or myelodysplastic syndrome with mutant RUNX1. Blood Neoplasia. 2025 Jul 25;2(4):100145. doi: 10.1016/j.bneo.2025.100145. eCollection 2025 Nov.
PubMed 40979071

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 3 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Phase Ib/II Study of Omacetaxine and Venetoclax for Patients With Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome Harboring Mutant RUNX1
ClinicalTrials.gov ID: NCT04874194
Acronym / sponsor ID: 2020-0890
Conditions: Hematopoietic and Lymphoid Cell Neoplasm, Recurrent Acute Biphenotypic Leukemia, Recurrent Acute Myeloid Leukemia, Recurrent Myelodysplastic Syndrome, Refractory Acute Biphenotypic Leukemia, Refractory Acute Myeloid Leukemia
Phase: Phase 1/2
Study type: Interventional
Recruitment status: TerminatedReason stopped: The study terminated early because the company was longer providing the investigational product.
Enrolment: 24 participants (actual)
Lead sponsor: M.D. Anderson Cancer CenterUniversity, hospital or other
Study start: 17 Dec 2021 (actual)
Primary completion: 16 Sep 2024 (actual)
Study completion: 16 Sep 2024 (actual)
First posted: 5 May 2021 (actual)
Results: Posted 3 Nov 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

PRIMARY OBJECTIVES:

To determine the safety and tolerability and recommended phase 2 dose (RP2D) of omacetaxine in combination with venetoclax for patients with relapsed/refractory acute myeloid leukemia or myelodysplastic syndrome harboring a RUNX1 mutation. (Phase 1b) II. To determine the efficacy of omacetaxine in combination with venetoclax for patients with relapsed/refractory acute myeloid leukemia or myelodysplastic syndrome harboring a RUNX1 mutation. (Phase II)

SECONDARY OBJECTIVES:

To determine duration of response (DOR), event-free survival (EFS), and overall survival (OS).

II. To evaluate occurrence of minimal residual disease (MRD) negative status by multiparameter flow cytometry and molecular evaluation.

OUTLINE: This is a phase I, dose de-escalation study followed by a phase II study.

After completion of study treatment, patients are followed up within 30 days, then every 3 months for 3 years.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 6

Arm	Type	Receives
Ph 1 Arm A (AML) Dose 0	Experimental	Drug: Omacetaxine Mepesuccinate, Drug: VenetoclaxParticipants receive omacetaxine SC BID on days 2-4, and venetoclax PO on days 1-10 . Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity.
Ph 1 Arm B (MDS) Dose 0	Experimental	Drug: Omacetaxine Mepesuccinate, Drug: VenetoclaxParticipants receive omacetaxine SC BID on days 2-4, and venetoclax PO on days 1-10 . Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity.
Ph 1 Arm A (AML) Dose +1	Experimental	Drug: Omacetaxine Mepesuccinate, Drug: VenetoclaxParticipants receive omacetaxine SC BID on days 2-4, and venetoclax PO on days 1-14. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity.
Ph 1 Arm B (MDS) Dose + 1	Experimental	Drug: Omacetaxine Mepesuccinate, Drug: VenetoclaxParticipants receive omacetaxine SC BID on days 2-4, and venetoclax PO on days 1-14. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity.
Ph 2 Arm A (AML) Dose +1	Experimental	Drug: Omacetaxine Mepesuccinate, Drug: VenetoclaxParticipants receive omacetaxine SC BID on days 2-4, and venetoclax PO on days 1-14. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity.
Ph 2 Arm B (MDS) Dose + 1	Experimental	Drug: Omacetaxine Mepesuccinate, Drug: VenetoclaxParticipants receive omacetaxine SC BID on days 2-4, and venetoclax PO on days 1-14. Treatment repeats every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (11 listed)

Patients with a diagnosis of relapsed or refractory acute myeloid leukemia (AML) (or biphenotypic or bilineage leukemia including a myeloid component) or myelodysplastic syndrome
For myelodysplastic syndrome (MDS) patients, patients must have no response, progression, or relapse following at least 4 cycles of azacytidine or decitabine; and/or intolerance defined as grade >= 3 drug-related toxicity precluding continued therapy
Age >= 18 years
Subjects must have documented RUNX1 gene mutation
Eastern Cooperative Oncology Group (ECOG) performance status =< 2

Key exclusion criteria (8 listed)

Patients with t(15;17) karyotypic abnormality or acute promyelocytic leukemia (French-American-British [FAB] class M3-AML)
Patients with any concurrent uncontrolled clinically significant medical condition including active infection or psychiatric illness, which could place the patient at unacceptable risk of study treatment
Patients with active graft-versus-host-disease (GVHD) status post stem cell transplant (patients without active GVHD on chronic suppressive immunosuppression and/or phototherapy for chronic skin GVHD are permitted after discussion with the PI)
Patients with any severe gastrointestinal or metabolic condition which could interfere with the absorption of oral study medications
Known active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection or known human immunodeficiency virus (HIV) infection

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
Omacetaxine MepesuccinateAlso: Ceflatonin, Cephalotaxine, 4-methyl 2-hydroxy-2-(4-hydroxy-4-methylpentyl)butanedioate (ester), [3(R)]- (9CI), CGX-635, HHTGiven SC	Drug	Ph 1 Arm A (AML) Dose +1, Ph 1 Arm A (AML) Dose 0, Ph 1 Arm B (MDS) Dose + 1, Ph 1 Arm B (MDS) Dose 0, Ph 2 Arm A (AML) Dose +1, Ph 2 Arm B (MDS) Dose + 1
VenetoclaxAlso: ABT-0199, ABT-199, ABT199, GDC-0199Given PO	Drug	Ph 1 Arm A (AML) Dose +1, Ph 1 Arm A (AML) Dose 0, Ph 1 Arm B (MDS) Dose + 1, Ph 1 Arm B (MDS) Dose 0, Ph 2 Arm A (AML) Dose +1, Ph 2 Arm B (MDS) Dose + 1

Primary outcome

Recommended Phase 2 Dose (RP2D) of Omacetaxine in Combination With Venetoclax
Time frame: Up to 30 days
The RP2D will be selected at the end of the Phase 1b portion based on safety data , in Arms A and B independently. Preliminary efficacy and PK data for each dose level may also be considered as appropriate.
Number of Participant to Achieve Complete Remission
Time frame: At day 28, and 3 cycles.
Complete Remission for AML is defined as: Absolute neutrophil count > 10^3/UL, platelets . 10^5/UL, red cell transfusion independence, absence of extramedullary disease, and bone marrow with < 5% blasts. Complete Remission for MDS is defined as: Absolute neutrophil count > 10^3/UL, platelets . 10^5/UL, hemoglobin >11 g/dl, and bone marrow with < 5% blasts. Peripheral dysplasia will be noted.

3 secondary outcomes

Event-free Survival (EFS)
Time frame: Up to Two years, 8 months, 30 days
Overall Survival (OS)
Time frame: Up to Two years, 8 months, 30 days
Duration of Response
Time frame: Up to Two years, 8 months, 30 days

Results and safety

Results posted to the registry on 3 Nov 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Ph 1 Arm A (AML) Dose 0	3	3
Ph 1 Arm B (MDS) Dose 0	2	2
Ph 1 Arm A (AML) Dose +1	9	9
Ph 1 Arm B (MDS) Dose + 1	0	0
Ph 2 Arm A (AML) Dose +1	10	10
Ph 2 Arm B (MDS) Dose + 1	0	0

Primary outcome: Recommended Phase 2 Dose (RP2D) of Omacetaxine in Combination With Venetoclax

Time frame: Up to 30 days. Number. Unit: mg/m2

Measure	Ph 1 Arm A (AML) Dose 0, Arm B (MDS) Dose 0, Arm A (AML) Dose +1 n = 14	Ph 1 Arm B (MDS) Dose + 1 n = 0
Omacetaxine (days 2-4)	1.25	–
Venetoclax (days 1-14)	400	–

Population analysed: RP2D was only assessed in the phase 1 portion of the study. In the phase 1b portion of the MDS arm, 2 patients were treated at dose level 0 before the study was closed by the sponsor.

Primary outcome: Number of Participant to Achieve Complete Remission

Time frame: At day 28, and 3 cycles.. Count of participants. Unit: Participants

Measure	Ph 1 Arm A (AML) Dose 0 n = 2	Ph 1 Arm B (MDS) Dose 0 n = 2	Ph 1 Arm A (AML) Dose +1 n = 9	Ph 1 Arm B (MDS) Dose + 1 n = 0	Ph 2 Arm A (AML) Dose +1 n = 8	Ph 2 Arm B (MDS) Dose + 1 n = 0
Value	0	2	0	–	0	–

Population analysed: Of the 24 patients enrolled in the study, three were not evaluable for response. One participant in Ph 1 Arm A (AML) Dose 0 was not evaluable for response. Two participants in Ph 2 Arm A (AML) Dose +1 were not available for response.

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: Up to Two years, 8 months, 30 days. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Ph 1 Arm A (AML) Dose 0	3 of 3 (100%)	1 of 3 (33.3%)	1 of 3
Ph 1 Arm B (MDS) Dose 0	2 of 2 (100%)	1 of 2 (50%)	0 of 2
Ph 1 Arm A (AML) Dose +1	5 of 9 (55.6%)	5 of 9 (55.6%)	1 of 9
Ph 1 Arm B (MDS) Dose + 1	0 of 0	0 of 0	0
Ph 2 Arm A (AML) Dose +1	7 of 10 (70%)	4 of 10 (40%)	2 of 10
Ph 2 Arm B (MDS) Dose + 1	0 of 0	0 of 0	0

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 40979071DiNardo CD, Jen WY, Montalban-Bravo G, Wang X, Loghavi S, Lavu S, Short NJ, Chien K, Issa GC, Pemmaraju N, Yilmaz M, Andreeff M, Borthakur G, Kadia TM, Daver NG, Garcia-Manero G, Mill CP, Su X, Fiskus W, Bhalla KN. Omacetaxine and venetoclax in relapsed/refractory acute myeloid leukemia or myelodysplastic syndrome with mutant RUNX1. Blood Neoplasia. 2025 Jul 25;2(4):100145. doi: 10.1016/j.bneo.2025.100145. eCollection 2025 Nov.
M D Anderson Cancer CenterLink 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.

Courtney DiNardo, MD principal investigator

M.D. Anderson Cancer Center

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 11 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 3 Nov 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 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?

Yes. Results are posted in the registry (first posted 3 Nov 2025). 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?

Recommended Phase 2 Dose (RP2D) of Omacetaxine in Combination With Venetoclax (time frame: Up to 30 days). The study lists 2 primary outcomes in all. 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 NCT04874194, which also lists 1 linked publication.

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 3 Nov 2025.