Phase 2
N=48
Omacetaxine in Patients With Intermediate-1 and Higher Risk Myelodysplastic Syndrome (MDS) Post Hypomethylating Agent (HMA) Failure
Leukemia
Bottom Line
View on ClinicalTrials.gov: NCT02159872 ↗Enrolled (actual)
48
Serious AEs
62.5%
Results posted
Jun 2021
Primary outcome: Primary: Overall Survival (OS) — 7.5 Months
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Omacetaxine (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- M.D. Anderson Cancer Center
- Primary completion
- Apr 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overall Survival (OS) |
7.5 | — |
| PRIMARY Number of Participants With a Response |
16 | — |
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.
Eligibility Criteria
Inclusion Criteria
- 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 /= 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.
Data sourced from ClinicalTrials.gov (NCT02159872). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.