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Polycythemia Vera (PV)Phase 2Ruxo-BEATNCT02577926

The Ruxo-BEAT Trial in Patients With High-risk Polycythemia Vera or High-risk Essential Thrombocythemia

A Phase 2 study testing Ruxolitinib and BAT in people with Polycythemia Vera (PV) and Essential Thrombocythemia (ET), run by RWTH Aachen University at 23 sites in Germany.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

The Philadelphia chromosome negative myeloproliferative neoplasms (MPN) comprise a group of clonal hematological malignancies that are characterized by chronic myeloproliferation, splenomegaly, different degrees of bone marrow fibrosis, and disease-related symptoms including pruritus, night sweats, fever, weight loss… Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Ruxolitinib and BAT
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
207 participants
Where
23 sitesGermany
Study dates
Oct 2015to Dec 2028
Phase
Phase 2
Are there results?
Not yet: the study is under way

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 provide written informed consent prior to studyspecific procedures or assessments which are not routinely performed for diagnosis or monitoring of PV or ET, and the subjects must be willing to comply with treatment and to follow up assessments and procedures
  • Patient must be 18 years of age or older
  • Patient´s ECOG performance status must be 0-2
  • Patient must fulfill WHO 2008 diagnostic criteria for either polycythemia vera (PV) or essential thrombocythemia (ET). Moreover, PV- and ET-patients have to be classified as high risk according to defined criteria.
  • For patients with high risk PV OR PV with indication for cytoreductive therapy due to progressive myeloproliferation, AT LEAST ONE of the following must be fulfilled (according to DGHO onkopedia) (Barbui, et al., 2011). (Passamonti, 2009):
  • Age > 60 years
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  • Previous documented thrombosis or thromboembolism
  • Platelet count > 1500 x 109/L
  • Poor tolerance of phlebotomy or frequent phlebotomy requirement
  • Symptomatic or progressive splenomegaly
  • Severe disease-related symptoms (according to the investigators definition)
  • Progressive leukocytosis with leukocyte count > 20 x 109/L
  • For patients with high risk ET, AT LEAST ONE of the following must be fulfilled (according to DGHO guidelines):
  • Age > 60 years
  • Platelet count> 1500 x 109/L
  • Previous thrombosis or thromboembolism
  • Previous severe hemorrhage related to ET (defined as decrease of Hgb of at least 2 g/dl)
  • Patients must fulfill the following criteria regarding prior therapy:
  • PV patients:
  • Never treated with cytoreductive drugs except hydroyurea, anagrelide, or interferon for up to 6 weeks maximum (phlebotomy and/or aspirin are allowed)
  • ET patients:
  • Naïve and pretreated patients may be entered in this trial.
  • Patient must have adequate liver function as indicated by a total bilirubin, AST, and ALT ≤ 2 of the institutional upper limit of normal (ULN) value, unless directly attributable to the patient's MPN
  • Patient must have a creatinine clearance >40ml/min calculated according to the modified formula of Cockcroft and Gault, eGFR, or directly measured after 24h-urine collection
  • Patients must be able to swallow and retain oral medication

Who cannot join

  • Patients who meet criteria for post PV-MF or post ET-MF (IWG-MRT)
  • Patients who have received previous ruxolitinib treatment
  • Patients who have a history of anaphylaxis following exposure to the BAT drug of choice
  • Patients who have an inadequate bone marrow reserve as demonstrated by ANC ≤ 1 x 109/l OR platelet count <50 x 109/l
  • Patients who have known hepatitis B or C or HIV infection
  • Patients who suffer from other severe, concurrent diseases, including tuberculosis, serious cardiac functional dysfunction (class III or IV as defined by the New York Heart Association Classification), uncontrolled diabetes, uncontrolled hypertension, severe pulmonary disease (i.e. COPD with hypoxemia), or major organ malfunction that could interfere with the patient's ability to participate in the study
Show all 24Show fewer
  • Patients who have history of active substance or alcohol abuse within the last year
  • Female patients who are pregnant or nursing
  • Patients who have participated in another interventional trial and/or used investigational agents or concurrent anticancer treatment for concomitant disease within the last 4 weeks of registration
  • Any circumstance at the time of study entry that would preclude completion of the study or the required follow-up prohibits inclusion into this study
  • Subjects who have had an active malignancy during the previous 3 years except for treated cervical intraepithelial neoplasia, basal cell carcinoma of the skin, or squamous cell carcinoma of the skin, each with no evidence for recurrence in the past 3 years
  • Patients who have uncontrolled bacterial, viral, or fungal infection
  • Patients who have any medical condition requiring prolonged use of oral corticosteroids with a dose of more than 20 mg per day (> 1 month)
  • Patients who have severe cerebral dysfunction and/or legal incapacity
  • Patients who have had active splanchnic vein thrombosis within the last 3 months (includes Budd-Chiari, portal vein, splenic and mesenteric thrombosis)
  • Patients who have thyroid dysfunction which is not adequately controlled
  • Fertile men or women of childbearing potential cannot be included unless they are:
  • surgically sterile or > 2 years after the onset of menopause and/or
  • willing to use a highly effective contraceptive method (Pearl Index <1) such as oral contraceptives, intrauterine device, sexual abstinence, or barrier method of contraception (i.e. condoms) in conjunction with spermicidal jelly during study treatment
  • Patients who are taking any of the following prohibited medication:
  • clarithromycin, telithromycin, troleandomycin (antibiotics)
  • ritonavir, indinavir, saquinavir, nelfinavir, amprenavir, lopinavir (HIV protease inhibitors)
  • itraconazole, ketoconazole, voriconazole, fluconazole (antifungals)
  • Patients with a diagnosis of galactose or lactose intolerance or a glucose-galactose- malabsortion

Trial locations and contacts

23 sites in 1 country.

23 sites in 1 country

Germany23 sites

  • Uniklinik RWTH Aachen

    Aachen, North Rhine-Westphalia, 52074

  • Studienzentrum Aschaffenburg

    Aschaffenburg, Bavaria, 63739

  • Charite Universitätsmedizin Berlin; Medizinische Klinik m.S. Hämatologie, Onkologie und Tumorimmunologie

    Berlin, 13353

  • Universitätsklinikum Bonn Medizinische Klinik und Poliklinik III

    Bonn, North Rhine-Westphalia, 53105

  • Klinikum Chemnitz gGmbH Klinik für Innere Medizin III

    Chemnitz, Saxony, 09113

  • Universitätsklinikum Dresden Medizinische Klinik und Poliklinik I

    Dresden, Saxony, 01307

  • Johanniter-Krankenhaus Rheinhausen GmbH Hämatologie / Internistische Onkologie / Tagesklinik

    Duisburg, North Rhine-Westphalia, 47228

  • Universitätsklinikum Düsseldorf Klinik für Hämatologie, Onkologie und Klinische Immunologie

    Düsseldorf, North Rhine-Westphalia, 40225

  • Marienhospital

    Düsseldorf, North Rhine-Westphalia, 40479

  • Universitätsklinikum Essen Klinik für Hämatologie

    Essen, North Rhine-Westphalia, 45122

  • Universitätsklinikum Freiburg - Klinik für Innere Medizin I

    Freiburg im Breisgau, 79106

  • Universitätsklinikum Halle (Saale)

    Halle, Saxony-Anhalt, 06120

  • Universitätsklinikum Hamburg Eppendorf Klinik und Poliklinik für Onkologie, Hämatologie und KMT mit Sektion Pneumologie

    Hamburg, Free and Hanseatic City of Hamburg, 20246

  • Universitätsklinik Jena - Klinik für Innere Medizin II

    Jena, 07705

  • UNIVERSITÄTSKLINIKUM Schleswig-Holstein - Klinik für Hämatologie und Onkologie, Campus Lübeck

    Lübeck

  • Universitätsklinikum Magdeburg

    Magdeburg, Sachesen-Anhalt, 39120

  • Universitätsmedizin Mainz III. Medizinische Klinik und Poliklinik

    Mainz, Hesse, 55131

  • Universitätsmedizin Mannheim III. Medizinische Klinik Hämatologie und Internistische Onkologie

    Mannheim, Baden-Wurttemberg, 68167

  • Mühlenkreiskliniken Johannes Wesling Klinikum Minden Klinik für Hämatologie, Onkologie und Palliativmedizin

    Minden, North Rhine-Westphalia, 32429

  • III. Medizinischen Klinik des Klinikums rechts der Isar der TU München

    Müchen, Bavaria, 81675

  • Klinikum Nürnberg Nord Medizinische Klinik 5

    Nuremberg, Bavaria, 90419

  • Universitätsklinikum Ulm Klinik für Innere Medizin III

    Ulm, Baden-Wurttemberg, 89081

  • Rems-Murr Klinikum Winnenden

    Winnenden, Baden-Wurttemberg, 71364

Other trials in Germany

About this study

Official title: Ruxolitinib Versus Best Available Therapy in Patients With High-risk Polycythemia Vera or High-risk Essential Thrombocythemia - The Ruxo-BEAT Trial

The Philadelphia chromosome negative myeloproliferative neoplasms (MPN) comprise a group of clonal hematological malignancies that are characterized by chronic myeloproliferation, splenomegaly, different degrees of bone marrow fibrosis, and disease-related symptoms including pruritus, night sweats, fever, weight loss, cachexia, and diarrhea. In addition, due to elevated numbers of leucocytes, erythrocytes and/or platelets, the disease course can be complicated by thromboembolic disease, hemorrhage, and leukemic transformation as well as myelofibrosis.

Patients with polycythemia vera (PV) typically harbor an increased number of blood cells from all three hematopoietic cell lineages due to clonal amplification of hematopoetic stem cells, while patients with essential thrombocythemia (ET) typically show a predominant expansion of the megakaryocytic lineage. Most patients with PV below the age of 60 years are currently being treated with acetylsalicylic acid +/- phlebotomy only, and patients with low-risk ET have an almost normal life expectancy and often do not require specific treatment. However, PV- as well as ET-patients with a higher risk for complications require cytoreductive treatment. In addition, constitutional symptoms can be unbearable to patients even in the absence of bona fide high risk factors, and these patients may similarly benefit from antineoplastic therapy.

Read the detailed description

Polycythemia vera (PV) and essential thrombocythemia (ET) are classical Philadelphia-negative myeloproliferative neoplasms (MPN) that are characterized by an excess of cells in the peripheral blood, clonal bone marrow hyperplasia, and extramedullary hematopoiesis. The symptoms of these patients may range from asymptomatic disease to symptomatic disease that may significantly affect their activities of daily living, such as severe generalized pruritus, night sweats and fevers, erythromelalgia, bone and muscle pain, weight loss, and fatigue. Moreover, the patients may develop thromboembolic and hemorrhagic complications, transition to myelofibrosis (MF), and transformation to acute leukemia. In principle, the only potentially curative therapy for MPNs is allogenic stem cell transplantation (allo-SCT). However, due to significant transplant-associated morbidity and mortality, this therapeutic option is only applied in exceptional cases of ET or PV. The majority of patients do not qualify for allo-SCT since the risks of this treatment clearly outweigh the potential benefits. Moreover, even with a non-transplantation approach, patients with ET and PV have a life expectancy comparable to or close to healthy age-matched control persons. For patients with standard risk PV, phlebotomy and acetylsalicylic acid are standard of care (target hematocrit below 45 %), while patients with standard risk ET should receive either no specific treatment or acetylsalicylic acid (provided that no microvascular symptoms or secondary acquired von Willebrand syndrome are present).

However, in patients who are at high risk to develop thromboembolic or hemorrhagic complications (high-risk patients), cytoreductive treatment is generally indicated to prevent these potentially life-threatening complications. In PV and ET, high risk patients are characterized by advanced age (> 60 years) and / or a history of thromboembolic or hemorrhagic events {1,2,3}. In ET, a platelet count > 1500 x 109/l is associated with an increased risk of bleeding, and thus should result in a platelet lowering treatment {2}. In PV, in addition to the risk-score based therapy, cytoreduction is also required in patients with progressive or marked myeloproliferation (leukocytosis, thrombocytosis, symptomatic splenomegaly, increase of frequency of phlebotomy requirement), or devastating constitutional symptoms {1,2,4}. In Germany, best available therapy (BAT) includes approved drugs such as hydroxyurea (HU; approved for both PV and ET) and anagrelide (approved for second-line treatment of ET) and non-approved options such as alpha-interferon, pipobroman, busulfan (in elderly patients), and radioactive phosphorus (32P). In rare cases, patients may also benefit from splenic irradiation or splenectomy.

Ruxolitinib is a JAK1/2-specific tyrosine kinase inhibitor (TKI) which has been approved for the treatment of symptomatic myelofibrosis. The compound was shown to be superior to hydroxyurea in reducing splenomegaly and constitutional symptoms. Ruxolitinib is currently studied in phase 2 and phase 3 clinical trials for HU-resistant or HU-intolerant PV and ET. The aim of the present study is to assess the feasibility, efficacy, and safety of ruxolitinib treatment vs. BAT in patients with high-risk PV or -ET.

Being tested
Ruxolitinib, BAT
Medical subject headings
Polycythemia Vera, Thrombocythemia, Essential

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RuxolitinibDrug

    Also called Study drug, Jakavi

    Ruxolitinib is a JAK1/2-specific tyrosine kinase inhibitor (TKI) which has been approved for the treatment of symptomatic myelofibrosis. The compound was shown to be superior to hydroxyurea in reducing splenomegaly and constitutional symptoms.

  • BATDrug

    Also called Control Treatment

    BAT is at the choice of the investigator (monotherapy with i.e. hydroxyurea, anagrelide, interferon, busulfan, immunomodulators etc).

Study groups in NCT02577926
Study groupRoleWhat they receive
RuxolitinibExperimentalRuxolitinib will be administered orally at a dose of 10 mg twice daily (both PV and ET) for two consecutive years.
Receives: Drug: Ruxolitinib
Best available therapy (BAT)Active comparatorBAT may include all currently used treatment options. BAT is at the choice of the investigator (monotherapy with i.e. hydroxyurea, anagrelide, interferon, busulfan, immunomodulators etc). BAT will be administrated for two consecutive years.
Receives: Drug: BAT

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The rate of complete clinicohematologic response rate (CHR) as defined by Barosi et al 2009

    Measured: at month 6

Secondary outcomes (8)

  1. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

    Measured: at month 6 and 12

  2. The complete response rate (CR) at month 6 as defined by Barosi et al Blood 2013 (revised ELN response criteria)

    Measured: month 6

  3. The rate of complete responses (CHR) at month 12 as defined by Barosi et al Blood 2009

    Measured: month 12

  4. The efficacy as assessed by the absence of phlebotomy (Hct <45%)

    Measured: through study completion, an average of 2 years

  5. The efficacy as assessed by the reduction in spleen size (palpable spleen that is reduced by > 50% from baseline measured by palpation and ultrasound) OR platelet count < 600 x 10^9/l (ET)

    Measured: through study completion, an average of 2 years

  6. Proportion of subjects achieving both durable absence of phlebotomy eligibility AND durable spleen volume reduction measured by palpation and ultrasound (PV) OR durable platelet count <600 x 10^9/l (ET) (durable defined as >3 months) {Barosi et al 2013)

    Measured: through study completion, an average of 2 years

  7. The rate of overall clinicohematologic remissions (CR + PR) according to both guidelines (Barosi et al 2009 and 2013)

    Measured: through study completion, an average of 2 years

  8. Safety of both regimen

    Adverse events will be assessed according to CTCAE 4.0 throughout the study until 30 days after EoT for patients in both regimens

    Measured: through study completion, an average of 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Oct 2015 (estimated)
  2. Study startOct 2015
  3. Primary completionDec 2027 (estimated)
  4. Study completionDec 2028 (estimated)

Resources

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
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
207 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02577926
Sponsor's protocol ID
12-181
Acronym
Ruxo-BEAT
Status last verified
Aug 2026
First submitted
1 Oct 2015
Last updated
13 Aug 2026
Results posted
No
Data monitoring committee
Yes
Individual data sharing
No

Publications

  • Isfort S, Manz K, Teichmann LL, Crysandt M, Burchert A, Hochhaus A, Saussele S, Kiani A, Gothert JR, Illmer T, Schafhausen P, Al-Ali HK, Stegelmann F, Hanel M, Pfeiffer T, Giagounidis A, Franke GN, Koschmieder S, Fabarius A, Ernst T, Warnken-Uhlich M, Wolber U, Kohn D, Pfirrmann M, Wolf D, Brummendorf TH; German CML study group. Step-in dosing of bosutinib in pts with chronic phase chronic myeloid leukemia (CML) after second-generation tyrosine kinase inhibitor (TKI) therapy: results of the Bosutinib Dose Optimization (BODO) Study. Ann Hematol. 2023 Oct;102(10):2741-2752. doi: 10.1007/s00277-023-05394-0. Epub 2023 Aug 18.

    PubMed 37592092

  • Koschmieder S, Isfort S, Wolf D, Heidel FH, Hochhaus A, Schafhausen P, Griesshammer M, Wolleschak D, Platzbecker U, Dohner K, Jost PJ, Parmentier S, Schaich M, von Bubnoff N, Stegelmann F, Maurer A, Crysandt M, Gezer D, Kortmann M, Franklin J, Frank J, Hellmich M, Brummendorf TH; German Study Group for Myeloproliferative Neoplasms (GSG-MPN). Efficacy and safety of ruxolitinib in patients with newly-diagnosed polycythemia vera: futility analysis of the RuxoBEAT clinical trial of the GSG-MPN study group. Ann Hematol. 2023 Feb;102(2):349-358. doi: 10.1007/s00277-022-05080-7. Epub 2022 Dec 23.

    PubMed 36564535

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ruxolitinib and BAT in people with Polycythemia Vera (PV) and Essential Thrombocythemia (ET), run by RWTH Aachen University at 23 sites in Germany.

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 25 inclusion and 24 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: under way, not looking for participants, as of the sponsor's last update on 13 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 23 sites in Germany. 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?

This study is still under way. Results are not available in the source record yet.

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?

The rate of complete clinicohematologic response rate (CHR) as defined by Barosi et al 2009 (time frame: at month 6). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02577926, which also lists 2 linked publications.

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 13 Aug 2026.

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