Omacetaxine in Treating Patients With Chronic Myeloid Leukemia (CML) With the T315I BCR-ABL Gene Mutation

A multi-center phase II open-label study to evaluate the efficacy and safety of subcutaneous administration of omacetaxine in the treatment of patients with chronic myeloid leukemia (CML) in chronic, accelerated and blast phase who have tested positive for the T315I bcr-abl point mutation.

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ANZCTR

Registry ID

ACTRN12609000373279

Enrollment

100

Registered

2009-05-27

Start date

2009-03-11

Completion date

Unknown

Last updated

2026-02-04

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

Brief summary

This study looks at the effectiveness of chemotherapy with the drug Omacetaxine in people with chronic myeloid leukemia (CML) who have the Philadelphia chromosome (T315I bcr-abl gene) mutation. Who is it for? You can join this study if you have: - chronic myeloid leukemia (CML) (cancer of blood cells) which is in chronic, accelerated, or blast phase - tested positive for the T315I bcr-abl point mutation - had no success with tyrosine kinase inhibitor (iminitab - Glivec) treatment. Trial details All participants will be treated with induction course cycles consisting of subcutaneous Omacetaxine administered twice daily for 14 consecutive days every 28 days. Participants who demonstrate a response may receive maintenance therapy for up to 24 months, consisting of subcutaneous Omacetaxine twice daily for 7 days every 28 days. Participants will be evaluated every 7 days with complete blood and platelet counts while undergoing therapy; the number of consecutive doses of Omacetaxine or intervals between subsequent cycles may be adjusted as necessary, according to guidelines provided in the treatment plan. Omacetaxine causes programmed cell death in myeloid cancer cells. Patients will be monitored from the beginning of treatment until any return of the disease or until they die. The study aims to measure the effectiveness of treatment, and investigates the tolerance to and toxicity of Omacetaxine.

Interventions

Omacetaxine mepesuccinate Patient will receive omacetaxine in treatment cycles. Each treatment cycle will have a duration of 28 days and treatment cycles will continue for upto 24 months - without a break between cycles. Dose = 1.25 mg/m2 twice daily for the first 14 days during induction treatment cycles and 1.25 mg/m2 twice a day for the first 7 days during maintenance treatment cycles. Duration: upto 6 months for induction and ~18 months for maintenance (total of 24 months on study). Mode o

Study design

Allocation

Non-randomised trial

Intervention model

Single group

Primary purpose

Treatment

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

1. Male or female patients, age 18 years or older. 2. Philadelphia chromosome (Ph) positive chronic myelogenous leukemia in either chronic, accelerated, or blast phase. 3. The patient will have the T315I BCR-ABL gene mutation. 4. Patients will have failed prior imatinib therapy. 5. Acceptable Renal and Liver Function. 6. Eastern Cooperative Oncology Group (ECOG) performance status 0-2. 7. Sexually active patients and their partners must use an effective double barrier method of contraception.

Exclusion criteria

1. New York Heart Association (NYHA) class III or IV heart disease, active ischemia or any other uncontrolled cardiac condition such as angina pectoris, clinically significant cardiac arrhythmia and requiring therapy, uncontrolled hypertension or congestive heart failure Myocardial infarction in the previous 12 weeks. 2. Lymphoid Ph+ blast crisis. 3. Pregnant or lactating. 4. Any medical or psychiatric condition, which may compromise the ability to give written informed consent or to comply with the study protocol. 5. Patient is candidate (and has a donor identified) for bone marrow or blood stem cell transplantation. 6. Patient is enrolled in another clinical investigation within 30 days of enrollment or is receiving another investigational agent.

Outcome results