MyelofibrosisPhase 2POTAMI-61NCT06397313
RVU120 in Patients With Intermediate or High-risk, Primary or Secondary Myelofibrosis
A Phase 2 study testing RVU120 and Ruxolitinib in people with Myelofibrosis, run by Ryvu Therapeutics SA at 18 sites in 2 countries.
Trial summary
The objective of this clinical trial is to evaluate the efficacy (how well the drug works), safety, pharmacokinetics (PK), and pharmacodynamics (PD) of the study drug, RVU120, in treating adult patients with intermediate or high-risk, primary or secondary myelofibrosis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelofibrosis
- What is being tested?
- RVU120 and Ruxolitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 18 sitesin 2 countries
- Study dates
- 19 Sep 2024to 30 Jun 2026
- 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
- Age ≥18 years
- Diagnosis of Primary or Secondary myelofibrosis (MF) according to the revised World Health Organization (WHO) criteria (Arber 2022).
- Intermediate or high-risk disease.
- Resistant or refractory to prior Janus kinase (JAK) inhibitor treatment or ineligible for JAK inhibitor treatment in the opinion of the investigator; or Suboptimal response to JAK inhibitor treatment. Note: a suboptimal response to JAK inhibitor treatment is defined as spleen size increase by palpation >25% after the first 3 months of treatment with a JAK inhibitor or persistent splenomegaly (spleen volume of >450 cm3) after at least 6 months of JAK inhibitor treatment and presence of 1 symptom score ≥5 or 2 symptom scores ≥3, new or persistent red blood cell (RBC) transfusion dependence; or may include participants naïve to previous treatment with JAK inhibitor.
- Measurable splenomegaly as demonstrated by palpable spleen measuring ≥5 cm below the left costal margin. The edge of the spleen should be measured from the mid-clavicular line on the left side of the abdomen to the point of greatest splenic protrusion; or spleen volume of ≥450 cm3 measured by magnetic resonance imaging(MRI) or computed tomography (CT).
- Active symptoms of MF as demonstrated by a symptom score of at least 5 points (on a 0 to 10 scale) on at least one of the symptoms or a score of 3 or greater on at least 2 of the following symptoms: night sweats, itchiness, abdominal discomfort, pain under ribs on left side, early satiety, bone or muscle pain, and inactivity.
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- Eastern Cooperative Oncology Group (ECOG) performance score 0-2.
- Adequate hematologic function defined as:
- absolute neutrophil count (ANC) ≥1.0 × 109/L (without growth factor support)
- platelet count ≥50 × 109/L (Cohort 2 and Cohort 3 only)
- Adequate renal function defined as calculated or measured creatinine clearance (CrCl) of ≥30 mL/minute using the formula of Cockcroft and Gault (see Section 15).
- Adequate liver function defined as (a) aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤3 × upper limit of normal (ULN); (b) alkaline phosphatase (ALP) ≤2 × ULN (ALP ≤5 × ULN for participants with isozymes specific to bone); (c) bilirubin <2 × ULN or bilirubin ≤3 × ULN if due to Gilbert's disease.
Who cannot join
- Each participant must not meet any of the following:
- Peripheral blood blast count of ≥10% or bone marrow blast count of ≥10%.
- Prior history of hematopoietic stem cell transplant.
- Participation in any other study in which receipt of an investigational new drug occurred within 4 weeks prior to Cycle 1 Day 1.
- Active known second malignancy with the exception of any of the following:
- Adequately treated basal cell carcinoma, squamous cell carcinoma of the skin, or in situ cervical cancer
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- Adequately treated Stage I cancer from which the participant is currently in remission and has been in remission for ≥2 years
- Low-risk prostate cancer with a Gleason score <7 and a prostate-specific antigen (PSA) level <10 ng/mL
- Any other cancer from which the participant has been disease-free for ≥3 years.
- Known or suspected allergy to RVU120 or RUX.
- Impairment of gastrointestinal function or gastrointestinal disease
- Major surgical procedure or significant traumatic injury within 28 days Placement of a vascular access device or minor surgery is permitted within 14 days before Cycle 1 Day 1 (provided that the wound has healed).
- Ongoing systemic infection requiring antibiotic, antiviral, or antifungal treatment. Note: prophylactic treatment is allowed.
- Significant cardiac dysfunction defined as myocardial infarction within 12 months of Cycle 1 Day 1, New York Heart Association (NYHA) Class III or IV heart failure, uncontrolled dysrhythmias, poorly controlled angina (Section 17), or left ventricular ejection fraction (LVEF) <40% as per echocardiography or multiple gated acquisition (MUGA) scan.
- Taking any medications, herbal supplements, or other substances (including smoking) that are known to be strong inhibitors or moderate/strong inducers or sensitive substrates of CYP1A2, within less than 5 half-lives prior to Cycle 1 Day 1.
- History of ventricular arrhythmia, or QTc ≥470 millisecond (Bazett's formula).
- Known active human immunodeficiency virus (HIV) infection
- Current active liver disease from any cause
- Pregnant or lactating females.
- Any other prior or current medical or psychiatric condition, intercurrent illness, surgical history, physical or electrocardiogram (ECG) findings, laboratory abnormalities, or extenuating circumstance (e.g., alcohol or drug addiction) that, in the Investigator's opinion, could jeopardize participant safety or interfere with the objectives of the study.
Trial locations and contacts
18 sites in 2 countries.
18 sites in 2 countries
Poland13 sites
Wojewódzki Szpital Specjalistyczny w Białej Podlaskiej
Biała Podlaska
Szpital Uniwersytecki nr 2 im. dr Jana Biziela
Bydgoszcz
M2M Med. Sp. z o.o. Sp. j.
Chorzów
Centrum Wsparcia Badań Klinicznych UCK Ośrodek Badań Klinicznych Wczesnych Faz
Gdansk
Pratia Hematologia Sp. z o.o.
Katowice
Świętokrzyskie Centrum Onkologii Samodzielny Publiczny Zakład Opieki Zdrowotnej
Kielce
Samodzielny Publiczny Zakład Opieki Zdrowotnej Szpital Uniwersytecki w Krakowie
Krakow
Samodzielny Publiczny Szpital Kliniczny Nr 1 w Lublinie
Lublin
Wojewódzki Szpital Zespolony im. L. Rydygiera w Toruniu
Torun
Lux Med Onkologia Sp. z o.o.
Warsaw
Wojskowy Instytut Medyczny Państwowy Instytut Badawczy
Warsaw
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu
Wroclaw
Szpital Uniwersytecki Imienia Karola Marcinkowskiego w Zielonej Gorze Sp. z o. o.
Zielona Góra
Italy5 sites
Policlinico Sant'Orsola-Malpighi
Bologna
Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
Brescia
Azienda Ospedaliero Universitaria Policlinico "G. Rodolico - San Marco"
Catania
Istituto Romagnolo per lo Studio dei Tumori Dino Amadori IRST S.r.l.
Meldola
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
Milan
About this study
Official title: An Open-Label Clinical Trial of RVU120 as Monotherapy and in Combination With Ruxolitinib in Patients With Intermediate or High-Risk, Primary or Secondary Myelofibrosis (POTAMI-61)
The objective of this clinical trial is to evaluate the efficacy (how well the drug works), safety, pharmacokinetics (PK), and pharmacodynamics (PD) of the study drug, RVU120, in treating adult patients with intermediate or high-risk, primary or secondary myelofibrosis. RVU120 will be given as a single agent or in combination with ruxolitinib.
Read the detailed description
The study schedule consists of a screening period up to 28 days, a 21-day treatment period, an end of treatment visit (30 days) and a 1-year follow-up where participants will be contacted every 3 months for assessment. Study duration for each participant will vary depending on the number of 21-day treatment cycles received. The study is open to participants aged ≥18 years with intermediate or high-risk, primary or secondary MF who have been previously treated, are ineligible for, or had a suboptimal response to JAK inhibitor therapy. Participants must have adequate organ function (kidney, liver) and no history of hematopoietic stem cell transplant. Participants 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.
- Conditions
- Myelofibrosis
- Being tested
- RVU120, Ruxolitinib
- Keywords
- intermediate, high-risk, primary, secondary
- Medical subject headings
- Primary Myelofibrosis, Neoplasm Metastasis
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
RuxolitinibDrug
Also called RUX
Ruxolitinib is a kinase inhibitor which inhibits Janus Associated Kinases (JAKs) JAK1 and JAK2
| Study group | Role | What they receive |
|---|---|---|
| RVU120 | Experimental | Cohort 1 RVU120 is administered at 250 mg as a single agent every other day on days 1, 3, 5, 7, 9, 11, and 13 of 21-day treatment cycles, or at an adjusted dose, to participants with intermediate or high-risk, primary or secondary MF who have been previously treated with or are ineligible for treatment with a JAK inhibitor. Receives: Drug: RVU120 |
| RVU120 + ruxolitinib | Experimental | Cohort 2 RVU120 is administered at 250 mg every other day on days 1, 3, 5, 7, 9, 11, and 13 of 21-day treatment cycles or at an adjusted dose, in combined with ruxolitinib administered orally twice daily following the dosing instructions in current prescribing information, to participants with intermediate or high risk, primary or secondary MF experiencing suboptimal response to JAK inhibitor. Receives: Drug: RVU120; Drug: Ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the number of participants with a response (anti-cancer activity) to RVU120 when administered as a single agent or in combination with ruxolitinib
The proportion of participants with spleen volume reduction (SVR) of ≥35% after 24 weeks of study treatment evaluated by magnetic resonance imaging (MRI) or computed tomography (CT)
Measured: 12 months
Secondary outcomes (9)
To further evaluate the number of participants having a response (anti-cancer activity and/or clinical benefit) to RVU120 when administered as a single agent or in combination with ruxolitinib
The proportion of participants with ≥1 Grade bone marrow fibrosis improvement after 24 weeks of study treatment
Measured: 12 months
Duration of spleen response
Assessed as the time from initial SVR of ≥35% by MRI/CT until disease progression or death, whichever occurs first
Measured: 12 months
Number of participants with leukemic transformation
Assessed as the proportion of participants with bone marrow blast counts of at least 20%, or peripheral blast counts of at least 20% lasting 2 weeks
Measured: 12 months
Number of participants with hematological (clinical) improvement
Assessed as the proportion of participants with hematological (clinical) improvement as defined by International Working Group (IWG) Consensus Criteria (see Tefferi et al., 2006 for further details)
Measured: 12 months
Progression-Free Survival (PFS)
Assessed as time from the time from first treatment to the first occurrence of disease progression or death
Measured: 12 months
Overall Survival (OS)
Assessed as time from the time from first treatment to death
Measured: 12 months
Incidence and severity of adverse events (AEs)
Assessed as the number and grade of adverse events assessed by CTCAE v5.0
Measured: 12 months
Change in symptom burden
Assessed as the proportion of participants achieving at least a 50% reduction in symptom burden after 24 weeks of commencing study treatment as assessed by the Total Symptom Score (TSS) using the Myelofibrosis Symptom Assessment Form (MFSAF). Each item is rated on a scale from 0 (Absent) to 10 (Worst Imaginable).
Measured: Baseline and week 24
Absolute change in TSS from baseline
Assessed as the number of participants with absolute change in Total Symptom Score (TSS) from baseline assessed using the Myelofibrosis Symptom Assessment Form (MFSAF). Each item is rated on a scale from 0 (Absent) to 10 (Worst Imaginable).
Measured: Baseline and week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 May 2024
- Study start19 Sep 2024
- Primary completion30 Jun 2026
- Study completion30 Jun 2026
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 MyelofibrosisStudies 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 36 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
- NCT06397313
- Sponsor's protocol ID
- POTAMI-61
- Acronym
- POTAMI-61
- Status last verified
- Sep 2026
- First submitted
- 29 Apr 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
- An Open-Label Clinical Trial of RVU120 as Monotherapy and in Combination With Ruxolitinib in Patients With Intermediate or High-Risk, Primary or Secondary Myelofibrosis (POTAMI-61)
- ClinicalTrials.gov ID
- NCT06397313
- Acronym / sponsor ID
- POTAMI-61 / POTAMI-61
- Conditions
- Myelofibrosis
- Keywords
- intermediate, high-risk, primary, secondary
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Ryvu Therapeutics SAIndustry (pharma, biotech, devices)
- Study start
- 19 Sep 2024 (actual)
- Primary completion
- 30 Jun 2026 (actual)
- Study completion
- 30 Jun 2026 (actual)
- First posted
- 2 May 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 objective of this clinical trial is to evaluate the efficacy (how well the drug works), safety, pharmacokinetics (PK), and pharmacodynamics (PD) of the study drug, RVU120, in treating adult patients with intermediate or high-risk, primary or secondary myelofibrosis. RVU120 will be given as a single agent or in combination with ruxolitinib.
Detailed description
The study schedule consists of a screening period up to 28 days, a 21-day treatment period, an end of treatment visit (30 days) and a 1-year follow-up where participants will be contacted every 3 months for assessment. Study duration for each participant will vary depending on the number of 21-day treatment cycles received. The study is open to participants aged ≥18 years with intermediate or high-risk, primary or secondary MF who have been previously treated, are ineligible for, or had a suboptimal response to JAK inhibitor therapy. Participants must have adequate organ function (kidney, liver) and no history of hematopoietic stem cell transplant. Participants 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| RVU120 | Experimental | Drug: RVU120Cohort 1 RVU120 is administered at 250 mg as a single agent every other day on days 1, 3, 5, 7, 9, 11, and 13 of 21-day treatment cycles, or at an adjusted dose, to participants with intermediate or high-risk, primary or secondary MF who have been previously treated with or are ineligible for treatment with a JAK inhibitor. |
| RVU120 + ruxolitinib | Experimental | Drug: RVU120, Drug: RuxolitinibCohort 2 RVU120 is administered at 250 mg every other day on days 1, 3, 5, 7, 9, 11, and 13 of 21-day treatment cycles or at an adjusted dose, in combined with ruxolitinib administered orally twice daily following the dosing instructions in current prescribing information, to participants with intermediate or high risk, primary or secondary MF experiencing suboptimal response to JAK inhibitor. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Age ≥18 years
- Diagnosis of Primary or Secondary myelofibrosis (MF) according to the revised World Health Organization (WHO) criteria (Arber 2022).
- Intermediate or high-risk disease.
- Resistant or refractory to prior Janus kinase (JAK) inhibitor treatment or ineligible for JAK inhibitor treatment in the opinion of the investigator; or Suboptimal response to JAK inhibitor treatment. Note: a suboptimal response to JAK inhibitor treatment is defined as spleen size increase by palpation >25% after the first 3 months of treatment with a JAK inhibitor or persistent splenomegaly (spleen volume of >450 cm3) after at least 6 months of JAK inhibitor treatment and presence of 1 symptom score ≥5 or 2 symptom scores ≥3, new or persistent red blood cell (RBC) transfusion dependence; or may include participants naïve to previous treatment with JAK inhibitor.
- Measurable splenomegaly as demonstrated by palpable spleen measuring ≥5 cm below the left costal margin. The edge of the spleen should be measured from the mid-clavicular line on the left side of the abdomen to the point of greatest splenic protrusion; or spleen volume of ≥450 cm3 measured by magnetic resonance imaging(MRI) or computed tomography (CT).
Key exclusion criteria (20 listed)
- Peripheral blood blast count of ≥10% or bone marrow blast count of ≥10%.
- Prior history of hematopoietic stem cell transplant.
- Participation in any other study in which receipt of an investigational new drug occurred within 4 weeks prior to Cycle 1 Day 1.
- Active known second malignancy with the exception of any of the following:
- Known or suspected allergy to RVU120 or RUX.
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, RVU120 + ruxolitinib |
| RuxolitinibAlso: RUXRuxolitinib is a kinase inhibitor which inhibits Janus Associated Kinases (JAKs) JAK1 and JAK2 | Drug | RVU120 + ruxolitinib |
Primary outcome
To evaluate the number of participants with a response (anti-cancer activity) to RVU120 when administered as a single agent or in combination with ruxolitinib
Time frame: 12 months
The proportion of participants with spleen volume reduction (SVR) of ≥35% after 24 weeks of study treatment evaluated by magnetic resonance imaging (MRI) or computed tomography (CT)
9 secondary outcomes
To further evaluate the number of participants having a response (anti-cancer activity and/or clinical benefit) to RVU120 when administered as a single agent or in combination with ruxolitinib
Time frame: 12 months
Duration of spleen response
Time frame: 12 months
Number of participants with leukemic transformation
Time frame: 12 months
Number of participants with hematological (clinical) improvement
Time frame: 12 months
Progression-Free Survival (PFS)
Time frame: 12 months
Overall Survival (OS)
Time frame: 12 months
Incidence and severity of adverse events (AEs)
Time frame: 12 months
Change in symptom burden
Time frame: Baseline and week 24
Absolute change in TSS from baseline
Time frame: Baseline and week 24
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.
18 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RVU120 and Ruxolitinib in people with Myelofibrosis, run by Ryvu Therapeutics SA at 18 sites in 2 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 12 inclusion and 20 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 18 sites in Poland, Italy. 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?
To evaluate the number of participants with a response (anti-cancer activity) to RVU120 when administered as a single agent or in combination with ruxolitinib (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.