Omacetaxine in Patients With Intermediate-1 and Higher Risk Myelodysplastic Syndrome (MDS) Post Hypomethylating Agent (HMA) Failure

NCT02159872 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: 2013-0870NCI-2014-01483
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical research study is learn if omacetaxine can help to control myelodysplastic syndrome (MDS). The safety of this drug will also be studied. This is an investigational study. Omacetaxine is FDA approved and commercially available for the treatment of chronic myelogenous leukemia (CML). It is investigational to use omacetaxine in patients with MDS. The study doctor can explain how the study drug is designed to work. Up to 80 participants will be enrolled in this study. All will take part at MD Anderson.

Conditions

Leukemia

Keywords

Leukemia; Myelodysplastic Syndrome; MDS; Omacetaxine; Synribo; Homoharringtonine

Outcome Measures

Primary Outcomes (2)

• Overall Survival (OS)

  Overall survival defined as the time from treatment start to the time of death. Overall survival continuously monitored using the Bayesian method.

  Up to 2.5 Years

• Number of Participants With a Response

  Response is Complete Response (CR) + Partial Response (PR) + Hematologic Improvement (HI). CR is the normalization of the peripheral blood and bone marrow with \</= 5% bone marrow blasts, a peripheral blood granulocyte count \>/= (1.0x10\^9/L, and a platelet count \>/= 100x10\^9/L). PR is the same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if \<15% at start of treatment. HI meets all of the criteria for CR except for platelet recovery to \>/=100x10\^9L.

  Up to 2 years

Study Arms (1)

Omacetaxine

EXPERIMENTAL

Omacetaxine 1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle. Participant may continue taking the study drug for up to 24 cycles of treatment.

Drug: Omacetaxine

Interventions

Omacetaxine DRUG

1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle.

Also known as: Synribo, Homoharringtonine

Omacetaxine

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age \>/= 18 years
• Diagnosis of MDS confirmed within 10 weeks prior to study entry according to WHO criteria. Patients are either not eligible for or choose not to proceed with a stem cell transplant.
• MDS classified as follows: RAEB-1 (5%-9% BM blasts); RAEB-2 (10%-19% BM Blasts); CMML (5%-19% BM blasts); RAEB-t (20%-29% BM blasts) AND/OR by IPSS: intermediate-1 and high risk patients.
• No response, progression, or relapse (according to 2006 IWG criteria) following at least 4 cycles of either azacitidine or decitabine, which were completed within the last 2 years - AND/OR - intolerance to azacitidine or decitabine defined as drug-related \>/= grade 3 hepatic or renal toxicity leading to treatment discontinuation during the preceding 2 years.
• Eastern Cooperative Oncology Group (ECOG) performance status of \</= 2.
• Willing to adhere to and comply with all prohibitions and restrictions specified in the protocol.
• Patient (or patient's legally authorized representative) must have signed an informed consent document indicating that the patient understands the purpose of and procedures required for the study and is willing to participate in the study.)

You may not qualify if:

• Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia.
• Active infection not adequately responding to appropriate antibiotics (i.e. ongoing temperatures of \>/= 38 degree Celsius).
• Total bilirubin \>/= 1.5 mg/dL and not related to hemolysis or Gilbert's disease. Patients with total bilirubin \>/= 1.5 mg/dL to 3 mg/dL are eligible if at least 75% of the bilirubin is indirect.
• Alanine transaminase (ALT/SGPT) or aspartate transaminase (AST/SGOT) \>/= 2.5 x the upper limit of normal.
• Serum creatinine \> 1.5 mg/dL.
• Female patients who are pregnant or lactating.
• Patients with reproductive potential who are unwilling to following contraception requirements (including condom use for males with sexual partners, and for females: prescription oral contraceptives \[birth control pills\], contraceptive injections, intrauterine devices \[IUD\], double-barrier method \[spermicidal jelly or foam with condoms or diaphragm\], contraceptive patch, or surgical sterilization) throughout the study.
• Female patients with reproductive potential who do not have a negative urine or blood beta-human chorionic gonadotropin (beta HCG) pregnancy test at screening.
• Patients receiving any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy.
• Prior hydroxyurea for control of leukocytosis or use of hematopoietic growth factors (eg, G-CSF, GM-CSF, procrit, aranesp, thrombopoietins) is allowed at any time prior to or during study if considered to be in the best interest of the patient.
• Psychiatric illness or social situation that would limit the patient's ability to comply with study requirements.

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• Teva Pharmaceuticals USA: collaborator

Study Sites (1)

University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• University of Texas MD Anderson Cancer Center Website

MeSH Terms

Conditions

Leukemia; Myelodysplastic Syndromes

Interventions

Homoharringtonine

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Harringtonines; Alkaloids; Heterocyclic Compounds; Benzazepines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds, 4 or More Rings

Results Point of Contact

• Title: Elias Jabbour MD/Professor
• Organization: The University of Texas MD Anderson Cancer Center

ejabbour@mdaderson.org

713-792-4764

Study Officials

• Elias Jabbour, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 6, 2014
• First Posted: June 10, 2014
• Study Start: May 18, 2015
• Primary Completion: April 14, 2020
• Study Completion: April 14, 2020
• Last Updated: June 9, 2021
• Results First Posted: June 9, 2021

Record last verified: 2021-05

Locations

US (1)