Actively Recruiting
Onvansertib for the Treatment of Recurrent or Refractory Chronic Myelomonocytic Leukemia and Myelodysplastic Syndrome/MPN Overlap Neoplasms
Led by Mayo Clinic · Updated on 2026-02-23
25
Participants Needed
1
Research Sites
243 weeks
Total Duration
On this page
Sponsors
M
Mayo Clinic
Lead Sponsor
N
National Cancer Institute (NCI)
Collaborating Sponsor
AI-Summary
What this Trial Is About
This phase I trial evaluates the safety, effectiveness, and best dose of onvansertib for the treatment of patients with chronic myelomonocytic leukemia and Myelodysplastic syndrome/myeloproliferative neoplasm (MDS/MPN) overlap neoplasms that has come back (recurrent) or that does not respond to treatment (refractory). Onvansertib is a drug that binds to and inhibits an enzyme called PLK1, preventing cancer cell proliferation and causing cell death.
CONDITIONS
Official Title
Onvansertib for the Treatment of Recurrent or Refractory Chronic Myelomonocytic Leukemia and Myelodysplastic Syndrome/MPN Overlap Neoplasms
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- History of WHO-defined proliferative CMML or MDS/MPN overlap neoplasm with white blood cell count ≥ 13,000/mm³ at diagnosis
- Relapsed or refractory after hydroxyurea treatment or at least 4 cycles of hypomethylating agents, or intolerant to these therapies
- Able and willing to provide written informed consent
- Willing to return for follow-up during the active monitoring phase
- Willing to provide mandatory bone marrow and blood specimens for research
- ECOG performance status 0, 1, or 2
- Recovered to grade 1 or baseline from previous therapy side effects except alopecia
- Platelet count ≥ 20,000/mm³ (or able to achieve with transfusion support)
- Total bilirubin ≤ 1.5 x upper limit normal (ULN) or ≤ 3 x ULN if Gilbert's syndrome
- ALT and AST ≤ 3 x ULN
- Estimated glomerular filtration rate (eGFR) ≥ 50 mL/min/m²
- Ability to complete questionnaires alone or with help
- For women of child-bearing potential: agreement to use contraception or abstain during the study and 180 days after
- Negative serum or urine pregnancy test ≤ 7 days prior to registration
You will not qualify if you...
- Previous exposure to investigational PLK1 inhibitors
- Transformation to acute leukemia on any prior bone marrow biopsy
- Prior allogeneic stem cell transplant with active moderate to severe graft-versus-host disease
- Active central nervous system disease
- Concurrent active cancers except treated nonmelanoma skin cancer or certain other cured cancers without disease for over 2 years
- NYHA class III/IV heart failure or active angina
- Chemotherapy or biologic therapy (except hydroxyurea) within 2 weeks before pre-registration
- Use of other investigational agents for primary disease
- Major surgery within 6 weeks before pre-registration
- Gastrointestinal disorders impairing oral drug absorption
- Unable or unwilling to swallow study drug
- Uncontrolled illness including infections, wounds, arrhythmias, pulmonary disease, or psychiatric/social issues
- Active HIV, hepatitis B or C with measurable viral load; exceptions apply
- Use of live vaccines ≤ 28 days before pre-registration
- Systemic immunosuppression with steroids ≥ 20 mg/day prednisone equivalent
- Any condition making study treatment unsafe or compliance unlikely
- Use of strong CYP3A4 inhibitors or inducers
- QTc interval > 470 ms (Fridericia's correction) unless corrected
- Pregnancy, nursing, or unwillingness to use contraception if of childbearing potential
- Increased risk of dangerous heart arrhythmias (Torsade des Pointes)
- Transformation to acute leukemia at registration biopsy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Mayo Clinic in Rochester
Rochester, Minnesota, United States, 55905
Actively Recruiting
Research Team
C
Clinical Trials Referral Office
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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