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Evaluating RVU120 for treating myelofibrosis

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)

PHASE2 · Ryvu Therapeutics SA · NCT06397313

This study is testing a new treatment called RVU120 for adults with myelofibrosis to see if it can help them feel better, either on its own or with another medication.

Quick facts

PhasePHASE2
Study typeInterventional
Enrollment230 (estimated)
Ages18 Years and up
SexAll
SponsorRyvu Therapeutics SA (industry)
Drugs / interventionsruxolitinib
Locations18 sites (Bologna and 17 other locations)
Trial IDNCT06397313 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of RVU120 in adult patients with intermediate or high-risk primary or secondary myelofibrosis. Participants will undergo a screening period followed by a 21-day treatment phase, with the option to receive RVU120 alone or in combination with ruxolitinib. The study includes a follow-up period of one year, during which participants will be monitored every three months. Eligible patients are those who have previously been treated or are ineligible for JAK inhibitor therapy.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with intermediate or high-risk myelofibrosis who have had an inadequate response to JAK inhibitors.

Not a fit: Patients who have not been diagnosed with myelofibrosis or those who have undergone hematopoietic stem cell transplant may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with difficult-to-treat myelofibrosis.

How similar studies have performed: While there have been studies on JAK inhibitors for myelofibrosis, the approach with RVU120 is novel and has not been extensively tested.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Age ≥18 years
2. Diagnosis of Primary or Secondary myelofibrosis (MF) according to the revised World Health Organization (WHO) criteria (Arber 2022).
3. Intermediate or high-risk disease.
4. 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.
5. 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).
6. 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.
7. Eastern Cooperative Oncology Group (ECOG) performance score 0-2.
8. Adequate hematologic function defined as:

   1. absolute neutrophil count (ANC) ≥1.0 × 109/L (without growth factor support)
   2. platelet count ≥50 × 109/L (Cohort 2 and Cohort 3 only)
9. 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).
10. 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.

Exclusion Criteria:

Each participant must not meet any of the following:

1. Peripheral blood blast count of ≥10% or bone marrow blast count of ≥10%.
2. Prior history of hematopoietic stem cell transplant.
3. Participation in any other study in which receipt of an investigational new drug occurred within 4 weeks prior to Cycle 1 Day 1.
4. Active known second malignancy with the exception of any of the following:

   1. Adequately treated basal cell carcinoma, squamous cell carcinoma of the skin, or in situ cervical cancer
   2. Adequately treated Stage I cancer from which the participant is currently in remission and has been in remission for ≥2 years
   3. Low-risk prostate cancer with a Gleason score \<7 and a prostate-specific antigen (PSA) level \<10 ng/mL
   4. Any other cancer from which the participant has been disease-free for ≥3 years.
5. Known or suspected allergy to RVU120 or RUX.
6. Impairment of gastrointestinal function or gastrointestinal disease
7. 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).
8. Ongoing systemic infection requiring antibiotic, antiviral, or antifungal treatment. Note: prophylactic treatment is allowed.
9. 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.
10. 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.
11. History of ventricular arrhythmia, or QTc ≥470 millisecond (Bazett's formula).
12. Known active human immunodeficiency virus (HIV) infection
13. Current active liver disease from any cause
14. Pregnant or lactating females.
15. 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.

Where this trial is running

Bologna and 17 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Myelofibrosis, intermediate, high-risk, primary, secondary

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.