Can a targeted drug outperform standard care for rare blood cancers?

NCT ID NCT02577926

Disease control Sponsor: RWTH Aachen University Source: ClinicalTrials.gov ↗

First seen Aug 13, 2026 · Last updated Aug 14, 2026 · Updated 1 time

Summary

This trial compares the drug ruxolitinib against the best available standard treatments for people with high-risk polycythemia vera (PV) or essential thrombocythemia (ET), two rare blood cancers that cause overproduction of blood cells and can lead to clots, bleeding, and severe symptoms. Participants receive either ruxolitinib or a standard therapy chosen by their doctor, such as hydroxyurea or interferon. The main goal is to see if ruxolitinib leads to a higher rate of complete clinicohematologic response, meaning better control of blood counts and symptoms, while also monitoring side effects.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: Ruxolitinib, a JAK1/2 inhibitor, compared to best available therapy (e.g., hydroxyurea, anagrelide, interferon).
What this could lead to: If successful, ruxolitinib could become a more effective treatment option for high-risk PV and ET, potentially improving symptom control and reducing complications.
What could go wrong: This is a phase 2 trial, so results are preliminary. Ruxolitinib may not prove superior to existing therapies, and it carries risks like infection and blood count changes.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

207 people

The number who actually took part.

Started

Oct 2015

Expected to finish

Dec 2028

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. 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 2. Patient must be 18 years of age or older 3. Patient´s ECOG performance status must be 0-2 4. 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 * 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) 5. 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. 6. 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 7. 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 8. Patients must be able to swallow and retain oral medication Exclusion Criteria: 1. Patients who meet criteria for post PV-MF or post ET-MF (IWG-MRT) 2. Patients who have received previous ruxolitinib treatment 3. Patients who have a history of anaphylaxis following exposure to the BAT drug of choice 4. Patients who have an inadequate bone marrow reserve as demonstrated by ANC ≤ 1 x 109/l OR platelet count \<50 x 109/l 5. Patients who have known hepatitis B or C or HIV infection 6. 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 7. Patients who have history of active substance or alcohol abuse within the last year 8. Female patients who are pregnant or nursing 9. 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 10. 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 11. 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 12. Patients who have uncontrolled bacterial, viral, or fungal infection 13. Patients who have any medical condition requiring prolonged use of oral corticosteroids with a dose of more than 20 mg per day (\> 1 month) 14. Patients who have severe cerebral dysfunction and/or legal incapacity 15. Patients who have had active splanchnic vein thrombosis within the last 3 months (includes Budd-Chiari, portal vein, splenic and mesenteric thrombosis) 16. Patients who have thyroid dysfunction which is not adequately controlled 17. 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 18. 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) 19. Patients with a diagnosis of galactose or lactose intolerance or a glucose-galactose- malabsortion

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Conditions

The condition(s) this trial relates to.

acquired polycythemia vera essential thrombocythemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Essential thrombocythemia (ET) Polycythemia vera (PV)

Contacts and locations

Locations

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

Berlin, 13353, Germany
III. Medizinischen Klinik des Klinikums rechts der Isar der TU München

Müchen, Bavaria, 81675, Germany
Johanniter-Krankenhaus Rheinhausen GmbH Hämatologie / Internistische Onkologie / Tagesklinik

Duisburg, North Rhine-Westphalia, 47228, Germany
Klinikum Chemnitz gGmbH Klinik für Innere Medizin III

Chemnitz, Saxony, 09113, Germany
Klinikum Nürnberg Nord Medizinische Klinik 5

Nuremberg, Bavaria, 90419, Germany
Marienhospital

Düsseldorf, North Rhine-Westphalia, 40479, Germany
Mühlenkreiskliniken Johannes Wesling Klinikum Minden Klinik für Hämatologie, Onkologie und Palliativmedizin

Minden, North Rhine-Westphalia, 32429, Germany
Rems-Murr Klinikum Winnenden

Winnenden, Baden-Wurttemberg, 71364, Germany
Studienzentrum Aschaffenburg

Aschaffenburg, Bavaria, 63739, Germany
UNIVERSITÄTSKLINIKUM Schleswig-Holstein - Klinik für Hämatologie und Onkologie, Campus Lübeck

Lübeck, Germany
Uniklinik RWTH Aachen

Aachen, North Rhine-Westphalia, 52074, Germany
Universitätsklinik Jena - Klinik für Innere Medizin II

Jena, 07705, Germany
Universitätsklinikum Bonn Medizinische Klinik und Poliklinik III

Bonn, North Rhine-Westphalia, 53105, Germany
Universitätsklinikum Dresden Medizinische Klinik und Poliklinik I

Dresden, Saxony, 01307, Germany
Universitätsklinikum Düsseldorf Klinik für Hämatologie, Onkologie und Klinische Immunologie

Düsseldorf, North Rhine-Westphalia, 40225, Germany
Universitätsklinikum Essen Klinik für Hämatologie

Essen, North Rhine-Westphalia, 45122, Germany
Universitätsklinikum Freiburg - Klinik für Innere Medizin I

Freiburg im Breisgau, 79106, Germany
Universitätsklinikum Halle (Saale)

Halle, Saxony-Anhalt, 06120, Germany
Universitätsklinikum Hamburg Eppendorf Klinik und Poliklinik für Onkologie, Hämatologie und KMT mit Sektion Pneumologie

Hamburg, Free and Hanseatic City of Hamburg, 20246, Germany
Universitätsklinikum Magdeburg

Magdeburg, Sachesen-Anhalt, 39120, Germany
Universitätsklinikum Ulm Klinik für Innere Medizin III

Ulm, Baden-Wurttemberg, 89081, Germany
Universitätsmedizin Mainz III. Medizinische Klinik und Poliklinik

Mainz, Hesse, 55131, Germany
Universitätsmedizin Mannheim III. Medizinische Klinik Hämatologie und Internistische Onkologie

Mannheim, Baden-Wurttemberg, 68167, Germany

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