Acute Myeloid Leukemia (AML)Phase 2RIVER-52NCT06268574
Safety and Efficacy of RVU120 for Treatment of Relapsed/Refractory AML
A Phase 2 study testing RVU120 in people with Acute Myeloid Leukemia (AML) and High-risk Myelodysplastic Syndrome, run by Ryvu Therapeutics SA at 41 sites in 4 countries.
Trial summary
The goal of this study is to assess the safety, tolerability, anti-tumor activity (efficacy), pharmacokinetics (PK), and pharmacodynamics (PD) of the agent RVU120 when administered to adult patients with relapsed or refractory acute myeloid leukemia (AML) or relapsed or progressing high-risk myelodysplastic syndrome… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia (AML), High-risk Myelodysplastic Syndrome
- What is being tested?
- RVU120
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 62 participants
- Where
- 41 sitesin 4 countries
- Study dates
- 23 Jan 2024to 5 Sep 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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
- Subjects must sign a written informed consent document and complete study related procedures
- Patients must have a diagnosis of AML or HR-MDS (per 2022 WHO classification) with MDS confirmed as high risk with IPSS-R
- Patients must have relapsed or refractory AML (per ELN 2022 criteria)
- Patients must have relapsed or progressing HR-MDS (per IWG response criteria)
- Patients must have failed first-line treatment and have no alternative therapeutic options likely to produce clinical benefit
- Patients must have ECOG performance status of 0 to 2
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- Patients must have adequate end organ function defined as:
- WBC < 30 x 10(9)/L on Day 1 prior to first dose of study drug
- Platelet count > 10,000/mcL on Day 1 prior to first dose of study drug
- Serum albumin ≥ 25 g/L (2.5 g/dL)
- Normal coagulation (elevated international normalized ratio [INR], prothrombin time or activated partial thromboplastin time [APTT] <1.3 x the upper limit of normal [ULN] acceptable)
- AST (aspartate transaminase) and ALT (alanine transaminase) ≤ 3 x ULN (upper limit of normal)
- Total bilirubin ≤ 3 x ULN
- Creatinine clearance (Cockcroft \& Gault formula) ≥ 30 mL/min
Who cannot join
- Active central nervous system (CNS) leukemia.
- Diagnosis of acute promyelocytic leukemia (APL), the M3 subtype of AML.
- Previous treatment with CDK8 and/or CDK19-targeted therapy.
- 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, ≥Grade 2 acute graft versus host disease (GVHD), active moderate-to-severe chronic GVHD, or requirement for systemic immunosuppressive medications for GVHD
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- Evidence of ongoing and uncontrolled systemic bacterial, fungal, or viral infection and acute inflammatory conditions (including pancreatitis).
- Known seropositivity or history of active viral infection with human immunodeficiency virus (HIV).
- 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 may interfere with the metabolism of the study drug
- Significant cardiac dysfunction defined as myocardial infarction within 12 months of first dose of study drug, New York Heart Association (NYHA) Class III or IV heart failure, uncontrolled dysrhythmias, poorly controlled angina or left ventricular ejection fraction (LVEF) <40% as per echocardiography or multiple gated acquisition (MUGA) scan.
- History of ventricular arrhythmia, or QTc ≥470 ms (Bazett's formula).
- Prior history of malignancies other than AML, unless the participant has been free of the disease for 5 years or more prior to Screening
- Pregnant or breast-feeding.
Trial locations and contacts
41 sites in 4 countries.
41 sites in 4 countries
Poland12 sites
Wojewodzki Szpital Specjalistyczny w Bialej Podlaskiej
Biała Podlaska, 21-500
Uniwersyteckie Centrum Kliniczne
Gdansk, 80-214
Narodowy Instytut Onkologii Im. Marii Sklodowskiej-Curie Panstwowy Instytut Badawczy
Gliwice, 44-102
Pratia Hematologia Sp. z o.o.
Katowice, 40-519
Szpital Wojewodzki Im. Dr. Ludwika Rydygiera w Suwalkach
Suwałki, 16-400
MICS Centrum Medyczne Toruń
Torun, 87-100
Specjalistyczny Szpital Im. Dra Alfreda Sokolowskiego
Wałbrzych, 58-309
MTZ Clinical Research
Warsaw, Mazowieckie Województwo, 02-172
Instytut Hematologii I Transfuzjologii
Warsaw, 02-776
Wojskowy Instytut Medyczny Panstwowy Instytut Badawczy
Warsaw, 04-141
Dolnoslaskie Centrum Onkologii Pulmonologii i Hematologii
Wroclaw, 53-439
Szpital Uniwersytecki Imienia Karola Marcinkowskiego w Zielonej Gorze Sp. z o. o.
Zielona Góra, 65-046
Italy11 sites
Azienda Ospedaliero Universitaria Delle Marche
Ancona, 60126
Univerisity of Bologna Policlinico Sant'Orsola
Bologna, 40138
Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
Brescia, 25123
Careggi University Hospital
Florence, 50134
Ospedale Vito Fazzi Lecce
Lecce, 73100
Istituto Romagnolo Per Lo Studio Dei Tumori Dino Amadori IRST S.r.l.
Meldola, 47014
ASST Grande Ospedale Metropolitano Niguarda
Milan, 20162
Azienda Ospedaliero Universitaria Pisana
Pisa, 56126
Azienda Ospedaliera Policlinico Universitario Tor Vergata
Roma, 00133
Humanitas Mirasole S.p.A.
Rozzano, 20089
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
Turin, 10126
Spain10 sites
Hospital de la Santa Creu i de Sant Pau
Barcelona
Hospital del Mar
Barcelona
Institut Catala d'Oncologia Hospitalet
Barcelona
Complejo Hospitalario De Caceres - Hospital General San Pedro De Alcantara
Cáceres
Hospital Universitario La Paz (HULP)
Madrid
MD Anderson Cancer Center Madrid
Madrid
Hospital Regional Universitario de Málaga
Málaga
Clinica Universidad de Navarra
Pamplona
Virgen del Rocío University Hospital
Seville
Hospital Universitario La Fe
Valencia
France8 sites
Centre Hospitalier Universitaire Grenoble Alpes
La Tronche
Centre Hospitalier Le Mans
Le Mans
Centre Hospitalier Universitaire de Lille (CHU Lille)
Lille
Institut Paoli Calmettes (IPC)
Marseille
Centre Hospitalier Universitaire (CHU) de Nice - Hopital L'Archet I
Nice
Centre Hospitalier Universitaire de Nimes (CHU) - Institut de Cancerologie du Gard
Nîmes
Publique-Hopitaux de Paris (AP-HP) - Hopital Saint-Louis
Paris
UNICANCER - Centre Henri-Becquerel
Rouen
About this study
Official title: A Multicenter, Open-Label Clinical Trial of RVU120 in Patients With Relapsed or Refractory High-Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia With or Without NPM1 Mutation (RIVER-52)
The goal of this study is to assess the safety, tolerability, anti-tumor activity (efficacy), pharmacokinetics (PK), and pharmacodynamics (PD) of the agent RVU120 when administered to adult patients with relapsed or refractory acute myeloid leukemia (AML) or relapsed or progressing high-risk myelodysplastic syndrome (HR-MDS) and who have no alternative therapies available. The study consists of two parts. Part 1 will assess the safety and tolerability of the dosages given and the level of anti-tumor activity or clinical response. Based on the results from part 1 the study will continue to enrol patient into Part 2 which will continue to evaluate safety and tolerability and anti-tumor activity in a larger number of patients.
Read the detailed description
Patients entering the study will undergo a Screening Period of up to 21 days, a Treatment Period where they will take the drug every other day (7 times in 13 days) in cycles of 21 days, an End of Treatment period (lasting approximately 30 days after last dose), and a 1-year Follow-up Period where participants will be contacted every 3 months for progression and survival status. In Part 1, patients with AML or HR-MDS will be enrolled. All patients will receive RVU120 until the patient meets eligibility for transplant, until there is disease progression or if there are signs of intolerance. A patient may withdraw from the study at any time at their own request or may be withdrawn at any time at the discretion of the Investigator. Depending on the outcome of part 1, part 2 may include patients with HR-MDS and AML irrespective of NPM1 mutation status.
- Being tested
- RVU120
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RVU120Drug
Also called SEL120
RVU120 is a potent, selective inhibitor of CDK8 and its paralog CDK19
| Study group | Role | What they receive |
|---|---|---|
| RVU120 single agent | Experimental | RVU120 oral capsules administered at dose of 250 mg every other day on Days 1-13 of each 21-day cycle of treatment. Receives: Drug: RVU120 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Remission (CR), with and without measurable residual disease (MRD)
Rate of CR, CRh, or CRi
Measured: 12 months
Secondary outcomes (11)
Overall response rate
Overall response rate including CR, CRh, CRi, MLFS, or PR in AML patients or CR, PR, or marrow CR HR-MDS patients
Measured: 12 months
Duration of response
Time from first response to hematologial replase or death
Measured: 12 months
Progression-free survival
Time from first treatment to the first occurrence of disease progression or death
Measured: 12 months
Overall survival
Time from first treatment to death
Measured: 12 months
Incidence of Adverse Events (Safety and Tolerability)
Number and grade of adverse events assessed by CTCAE v5.0
Measured: Up to 24 months
Percentage of participants bridged to hematopoietic stem cell transplantation
Number of hematopoietic stem cell transplantations following response
Measured: 12 months
Maximum Plasma Concentration (Cmax)
Assessment of the peak plasma concentration (Cmax)
Measured: 12 months
Maximum Plasma Concentration (Tmax)
Assessment of the time to peak plasma concentration (Tmax)
Measured: 12 months
Area Under the Concentration Time-Curve (AUC)
Assessed of the area under the plasma concentration versus time curve (AUC)
Measured: 12 months
Impact of treatment on hematological malignancy patient-reported outcomes (HM-PRO)
Assess changes in summary scores of the HM-PRO using a three point impact scale (Not at all, A little, A lot) and a three-point severity scale (Not at all, Mild, Severe)
Measured: Up to 12 months
Impact of treatment on health-related quality of life (QOL-E)
Assess changes in summary scores of the QOL-E questionnaires using a four point scale in order of impact or severity from better to worse outcome (eg, Never, Rarely, Sometimes, Often)
Measured: Up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2024
- Study start23 Jan 2024
- Primary completion30 Jun 2025
- Study completion5 Sep 2025
Why it stopped: Sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia (AML)Studies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single Group Assignment
- Masking
- None (open label)
- Enrolment
- 62 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
- NCT06268574
- Sponsor's protocol ID
- RIVER-52
- Acronym
- RIVER-52
- Status last verified
- Sep 2026
- First submitted
- 5 Jan 2024
- Last updated
- 16 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-Label Clinical Trial of RVU120 in Patients With Relapsed or Refractory High-Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia With or Without NPM1 Mutation (RIVER-52)
- ClinicalTrials.gov ID
- NCT06268574
- Acronym / sponsor ID
- RIVER-52 / RIVER-52
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision
- Enrolment
- 62 participants (actual)
- Lead sponsor
- Ryvu Therapeutics SAIndustry (pharma, biotech, devices)
- Study start
- 23 Jan 2024 (actual)
- Primary completion
- 30 Jun 2025 (actual)
- Study completion
- 5 Sep 2025 (actual)
- First posted
- 20 Feb 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this study is to assess the safety, tolerability, anti-tumor activity (efficacy), pharmacokinetics (PK), and pharmacodynamics (PD) of the agent RVU120 when administered to adult patients with relapsed or refractory acute myeloid leukemia (AML) or relapsed or progressing high-risk myelodysplastic syndrome (HR-MDS) and who have no alternative therapies available. The study consists of two parts. Part 1 will assess the safety and tolerability of the dosages given and the level of anti-tumor activity or clinical response. Based on the results from part 1 the study will continue to enrol patient into Part 2 which will continue to evaluate safety and tolerability and anti-tumor activity in a larger number of patients.
Detailed description
Patients entering the study will undergo a Screening Period of up to 21 days, a Treatment Period where they will take the drug every other day (7 times in 13 days) in cycles of 21 days, an End of Treatment period (lasting approximately 30 days after last dose), and a 1-year Follow-up Period where participants will be contacted every 3 months for progression and survival status. In Part 1, patients with AML or HR-MDS will be enrolled. All patients will receive RVU120 until the patient meets eligibility for transplant, until there is disease progression or if there are signs of intolerance. A patient may withdraw from the study at any time at their own request or may be withdrawn at any time at the discretion of the Investigator. Depending on the outcome of part 1, part 2 may include patients with HR-MDS and AML irrespective of NPM1 mutation status.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single Group Assignment
| Arm | Type | Receives |
|---|---|---|
| RVU120 single agent | Experimental | Drug: RVU120RVU120 oral capsules administered at dose of 250 mg every other day on Days 1-13 of each 21-day cycle of treatment. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Subjects must sign a written informed consent document and complete study related procedures
- Patients must have a diagnosis of AML or HR-MDS (per 2022 WHO classification) with MDS confirmed as high risk with IPSS-R
- Patients must have relapsed or refractory AML (per ELN 2022 criteria)
- Patients must have relapsed or progressing HR-MDS (per IWG response criteria)
- Patients must have failed first-line treatment and have no alternative therapeutic options likely to produce clinical benefit
Key exclusion criteria (17 listed)
- Active central nervous system (CNS) leukemia.
- Diagnosis of acute promyelocytic leukemia (APL), the M3 subtype of AML.
- Previous treatment with CDK8 and/or CDK19-targeted therapy.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RVU120Also: SEL120RVU120 is a potent, selective inhibitor of CDK8 and its paralog CDK19 | Drug | RVU120 single agent |
Primary outcome
Complete Remission (CR), with and without measurable residual disease (MRD)
Time frame: 12 months
Rate of CR, CRh, or CRi
11 secondary outcomes
Overall response rate
Time frame: 12 months
Duration of response
Time frame: 12 months
Progression-free survival
Time frame: 12 months
Overall survival
Time frame: 12 months
Incidence of Adverse Events (Safety and Tolerability)
Time frame: Up to 24 months
Percentage of participants bridged to hematopoietic stem cell transplantation
Time frame: 12 months
Maximum Plasma Concentration (Cmax)
Time frame: 12 months
Maximum Plasma Concentration (Tmax)
Time frame: 12 months
Area Under the Concentration Time-Curve (AUC)
Time frame: 12 months
Impact of treatment on hematological malignancy patient-reported outcomes (HM-PRO)
Time frame: Up to 12 months
Impact of treatment on health-related quality of life (QOL-E)
Time frame: Up to 12 months
Results and safety
The study was stopped early (reason given: Sponsor decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
41 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RVU120 in people with Acute Myeloid Leukemia (AML) and High-risk Myelodysplastic Syndrome, run by Ryvu Therapeutics SA at 41 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 16 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 41 sites in Poland, Italy, Spain, France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Sponsor decision). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Complete Remission (CR), with and without measurable residual disease (MRD) (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2026.