Acute Myeloid LeukemiaPhase 1NCT04021368
RVU120 in Patients With Acute Myeloid Leukemia or High-risk Myelodysplastic Syndrome
A Phase 1 study testing RVU120(SEL120) in people with Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome, run by Ryvu Therapeutics SA at 10 sites in 2 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
- What is being tested?
- RVU120(SEL120)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 51 participants
- Where
- 10 sitesin 2 countries
- Study dates
- 4 Sep 2019to 21 May 2024
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- All the following criteria must be met for a patient to be eligible for the study:
- Written informed consent provided prior to any study-related procedure.
- Age ≥18 years.
- 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.
- Eastern Cooperative Oncology Group (ECOG) performance score of 0-2.
- Patients must have been off anti-cancer treatment prior to study.
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- Patients must have recovered from the toxic effects of previous treatments.
- 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);
- Adequate cardiac function
- Life expectancy of at least 12 weeks.
- 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.
- 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.
- Investigator considers the patient to be suitable for participation in the clinical study
Who cannot join
- Active central nervous system (CNS) leukemia.
- Previous treatment with CDK8-targeted therapy.
- Patients who have undergone major surgery within 28 days prior to first dose of study drug.
- Hematopoietic stem cell transplant within 120 days prior to first dose of study drug.
- Active acute graft versus host disease (GVHD)
- Infections and acute inflammatory conditions
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- Known seropositivity or history of HIV
- Known positive test of / or known active diagnosis of COVID-19 viral infection
- Ongoing significant liver disease
- Impairment of gastrointestinal function or gastrointestinal disease
- Ongoing drug-induced pneumonitis.
- Concurrent participation in another investigational clinical trial.
- 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.
- Significant cardiac dysfunction or poorly controlled angina.
- 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.
- Personal or family history of serious ventricular arrhythmia, or QT interval corrected for heart rate (QTc) ≥470 ms (Bazett's formula).
- 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.
- 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.
- Pregnant or breast-feeding females.
Trial locations and contacts
10 sites in 2 countries.
10 sites in 2 countries
United States5 sites
Northside Hospital
Atlanta, Georgia, 30342
MD Anderson Cancer Center
Houston, Texas, 77030
Sylvester Comprehensive Cancer Center, University of Miami Hospital and Clinics
Miami, Florida, 33136
Tennessee Oncology
Nashville, Tennessee, 37203
Washington University School of Medicine
St Louis, Missouri, 63110
Poland5 sites
Uniwersyteckie Centrum Kliniczne
Gdansk, 80-214
Świętokrzyskie Centrum Onkologii, Klinika Hematologii i Transplantacji Szpiku
Kielce, Świętokrzyskie Voivodeship, 25-734
MICS Centrum Medyczne Toruń
Torun, 87-100
Instytut Hematologii i Transfuzjologii
Warsaw, 02-776
Dolnośląskie Centrum Onkologii, Pulmonologii i Hematologii
Wroclaw, 53-413
About this study
Official title: A Phase Ib Study of RVU120 (SEL120) in Patients With Acute Myeloid Leukemia or High-risk Myelodysplastic Syndrome
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.
Read the detailed description
The study will determine the recommended phase II dose (RP2D) and safety of RVU120 (SEL120) given as monotherapy over a range of dose-levels, following a closely controlled dose escalation study design.
- Being tested
- RVU120(SEL120)
- Keywords
- Acute Myeloid Leukemia, Myelodysplastic Syndrome, CDK8/19 Inhibitor, RVU120 (SEL120), Clinical Trial, Phase Ib
- Medical subject headings
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RVU120(SEL120)Drug
RVU120(SEL120) will be administered as a single oral dose every other day (q.o.d.) for a total of 7 doses i.e. on Days 1, 3, 5, 7, 9, 11 and 13, in a 3-week treatment cycle.
| Study group | Role | What they receive |
|---|---|---|
| RVU120(SEL120) | Experimental | The first part of the study consists of dose-escalation cohorts where patients will receive ascending doses of RVU120(SEL120) to determine the recommended dose (RD) for further clinical development. The second part of the study is an enrichment cohort where additional 6 to 20 patients will be treated with RVU120(SEL120) to support the evaluation of the RD. Receives: Drug: RVU120(SEL120) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recommended dose (RD)
The RD will be determined using all available data, which will include dose limiting toxicities (DLTs) and other toxicities, signs of anti-leukemic activity, pharmacokinetic and pharmacodynamic characteristics.
Measured: Up to 2 years
Incidence of Adverse Events (Safety and Tolerability)
Safety and tolerability profile will be assessed by Common Terminology Criteria for Adverse Events v5.0 and summarized by type, frequency, and severity of adverse events.
Measured: Up to 2 years
Secondary outcomes (5)
The Maximum Observed Concentration (C[max])
Pharmacokinetic profile of RVU120(SEL120) will be characterized using the Maximum Observed Concentration (C[max]).
Measured: Up to 2 years
The Terminal Half-life (t[1/2])
Pharmacokinetic profile of RVU120(SEL120) will be characterized using the Terminal Half-life (t[1/2]).
Measured: Up to 2 years
The Area Under the Curve (AUC)
Pharmacokinetic profile of RVU120(SEL120) will be characterized using the Area Under the Curve (AUC).
Measured: Up to 2 years
The Volume of Distribution at Steady State (Vss)
Pharmacokinetic profile of RVU120(SEL120) will be characterized using the Volume of Distribution at Steady State (Vss).
Measured: Up to 2 years
Anti-leukemic activity
Anti-leukemic activity will be assessed by AML or MDS Response Criteria (Döhner et al., 2017, or Cheson et al., 2006, respectively).
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jul 2019
- Study start4 Sep 2019
- Primary completion21 May 2024
- Study completion21 May 2024
Why it stopped: RPD2 has been identified
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Dose escalating Cohorts with a safety expansion Cohort
- Masking
- None (open label)
- Enrolment
- 51 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ryvu Therapeutics SA
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04021368
- Sponsor's protocol ID
- CLI120-001
- Status last verified
- Sep 2026
- First submitted
- 12 Jul 2019
- Last updated
- 18 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No