A Phase Ib Study of RVU120 (SEL120) in Patients With Acute Myeloid Leukemia or High-risk Myelodysplastic Syndrome RVU120

What's the purpose of this trial?

This first-in-human study will evaluate RVU120 (SEL120), a novel small molecule CDK8/19 inhibitor, in patients with Acute Myeloid Leukemia (AML) or High-risk Myelodysplastic Syndrome (HR-MDS), in terms of selection of the recommended dose for further clinical development and assessment of safety, tolerability, preliminary anti-leukemic activity, as well as pharmacokinetic and pharmacodynamic profiles.

This trial is currently open and accepting patients.

What will happen during the trial?

You may be able to join this trial if you:

The following criteria is a partial list of reasons why patients may be eligible to participate in this clinical trial. Further evaluation with a medical professional is required.

Inclusion Criteria:

All the following criteria must be met for a patient to be eligible for the study:

1. Written informed consent provided prior to any study-related procedure.
2. Age ≥18 years.
3. AML diagnosis according to the 2016 World Health Organisation (WHO) classification (Arber et al. 2016) with relapsed or refractory disease with no available therapy who have exhausted the applicable standard of care; or Myelodysplastic Syndrome (MDS) diagnosis according to the 2016 WHO classification (Arber et al. 2016) with high-risk disease per the Revised International Prognostic Scoring System (IPSS-R \>4.5) and with relapsed or refractory disease with no available therapy who have exhausted the applicable standard of care.
4. Eastern Cooperative Oncology Group (ECOG) performance score of 0-2.
5. Patients must have been off anti-cancer treatment prior to study.
6. Patients must have recovered from the toxic effects of previous treatments.
7. Peripheral white blood cell (WBC) count \<10x10\^9/L; Platelet count \>10,000/μL; Serum albumin ≥ 30g/L (3.0g/dL); Normal coagulation (elevated INR, prothrombin time or APTT \<1.3 x ULN acceptable); AST and ALT ≤3x ULN; Total bilirubin ≤1.5 x ULN; Creatinine clearance ≥60 mL/min (Cockcroft-Gault formula);
8. Adequate cardiac function
9. Life expectancy of at least 12 weeks.
10. For females of childbearing potential (FCBP), a negative pregnancy test must be confirmed before enrolment. FCBP must commit to use a highly effective method of contraception during study participation and until 6 months after the last dose of study drug. Females must also refrain from donating blood or egg (ovum) during the same time period.
11. For males, an effective barrier method of contraception must be used during study participation until 6 months after the last dose of study drug, if the patient is sexually active with a FCBP. Males must also refrain from donating blood or sperm during the same time-period.
12. Investigator considers the patient to be suitable for participation in the clinical study

Exclusion Criteria:

1. Active central nervous system (CNS) leukemia.
2. Previous treatment with CDK8-targeted therapy.
3. Patients who have undergone major surgery within 28 days prior to first dose of study drug.
4. Hematopoietic stem cell transplant within 120 days prior to first dose of study drug.
5. Active acute graft versus host disease (GVHD)
6. Infections and acute inflammatory conditions
7. Known seropositivity or history of HIV
8. Known positive test of / or known active diagnosis of COVID-19 viral infection
9. Ongoing significant liver disease
10. Impairment of gastrointestinal function or gastrointestinal disease
11. Ongoing drug-induced pneumonitis.
12. Concurrent participation in another investigational clinical trial.
13. Taking any medications, herbal supplements or other substances (including smoking) that are known to be strong inhibitors or inducers of CYP1A2 or that can prolong Q wave to T wave (QT) interval and/or cause torsade de pointes within less than 5 half-lives, prior to first dose of study drug.
14. Significant cardiac dysfunction or poorly controlled angina.
15. Currently taking drugs that are documented, in the drug package insert, to have a risk of causing prolonged QTc or torsades de pointes (TdP) within 5 half-lives prior to first dose of study drug.
16. Personal or family history of serious ventricular arrhythmia, or QT interval corrected for heart rate (QTc) ≥470 ms (Bazett's formula).
17. Any other prior or current medical condition or extenuating circumstance that, in the Investigator's opinion, could jeopardize patient safety or interfere with the objectives of the study.
18. Prior history of malignancies other than AML or MDS, unless the patient has been free of the disease for 5 years or more prior to Screening.
19. Pregnant or breast-feeding females.

Additional Trial Information

Phase 1

Enrollment: 112 patients (estimated)

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Published Results

CDK 8/19 Kinase Inhibitor RVU120 in Patients with AML or Higher-Risk MDS: Safety and Efficacy Results from New Dose Escalation Cohorts

November 15, 2022

Up to July 25, 2022, 17 patients have been treated with RVU120 at doses from 10 mg to 110 mg per day on day 1, 3, 5, 7, 9, 11 and 13 in a 21-day cycle. Three patients had HR-MDS and 14 had AML, relapsed/refractory to a median of 3 prior lines of therapy, median age was 71 years and ECOG PS was 0-1 in 10 patients and 2 in 7 patients. Most frequent all grade Adverse Events (AE) were nausea (in 55% of patients), thrombocytopenia (33%) and febrile neutropenia (33%), hypokalemia (27%) and vomiting (27%). Drug discontinuation due to AE occurred in 5 patients, Clinical benefit was observed in 11 evaluable patients. Best response was 1 CR and 10 disease stabilizations (SD), 2 of which associated with Hematological Improvement due to increased Erythroid differentiation and/or Platelet recovery, and 4 with BM blast reduction. At the dose level of 100 mg, one patient with AML, refractory to 4 prior lines of therapy and with GATA2 rearrangement, achieved SD and is ongoing at Cycle 4 with platelet recovery and RBC transfusion independence; 1 patient with HR-MDS, progressing after 5 lines of therapy, showed a SD with BM blasts clearance at flow cytometry in an unscheduled efficacy evaluation. The third patient dosed at 100 mg died due to SAE of pneumonia with no post treatment BM evaluation. 2 additional patients (1 treated at 75 mg and 1 at 85 mg) are still ongoing after nearly 6 months of treatment. Both achieved SD. All patients' CD34+ blasts were tested for relative STAT5 phosphorylation changes from baseline. Results showed a clear correlation between pSTAT5 inhibition and RVU120 exposure.

Trial Locations

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Northside Hospital (Atlanta)

Atlanta, GA

Open and Accepting


Washington University School of Medicine

Saint Louis, MO

Open and Accepting


Sarah Cannon Tennessee Oncology- Nashville (25th Ave) Centennial Clinic

Nashville, TN

Open and Accepting


MD Anderson Cancer Center The University of Texas

Houston, TX

Open and Accepting
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