Phase 1 Study to Determine the Safety and Efficacy of Onvansertib, A Novel, Oral, PLK1 Inhibitor in Patients With Proliferative Chronic Myelomonocytic Leukemia (CMML) and Myelodysplastic Syndrome/MPN Overlap Neoplasms Relapsed/Refractory or Intolerant to Available Therapies
Mayo Clinic
Summary
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.
Description
PRIMARY OBJECTIVE: I. Characterization of adverse events (AEs) by type, incidence, severity \[graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0\], seriousness, and relationship to treatment; effects on vital signs and laboratory parameters; changes from baseline in electrocardiograms (ECGs), physical examinations, weight, and Eastern Cooperative Oncology Group (ECOG) performance status. SECONDARY OBJECTIVES: I. Efficacy: complete response (CR) rate, according to the 2015 myelodysplastic syndrome (MDS)/myeloproliferative neoplasm (MP…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: PRE-REGISTRATION - INCLUSION CRITERIA: * Age \>= 18 years * History of World Health Organization (WHO)-defined diagnosis of proliferative CMML (WBC count \>= 13,000/mm\^3 at time of diagnosis), or MDS/MPN overlap neoplasm with WBC count \>= 13,000/mm3 at time of diagnosis (atypical CML and MDS/MPN-NOS). * NOTE: Hydroxyurea or hypomethylating induced leukopenia does not preclude inclusion. Discussion of WBC \< 13,000/mm\^3 due to treatment at the time of preregistration must be discussed with the Sponsor/Principal Investigator * Relapsed/refractory following treatment w…
Interventions
- ProcedureBiospecimen Collection
Undergo collection of blood samples
- ProcedureBone Marrow Aspiration and Biopsy
Undergo bone marrow aspiration and biopsy
- DrugOnvansertib
Given PO
- ProcedureUltrasound Imaging
Undergo ultrasound imaging
Location
- Mayo Clinic in RochesterRochester, Minnesota