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Ruxolitinib

Phase 3

Polycythemia Vera | Small molecule | Hematology |Novartis AG|Last Updated: Apr 28, 2026

Success Probability
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Market & Valuation
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Trial Design
RandomizedACTIVE_CONTROLLEDDMCBiomarker
Total Trials3
Total Enrollment896
FDA Designations
No designations recorded
Clinical trial landscape

Ruxolitinib · 11 trials · 12 indications

Phase 3 7Phase 2 3Phase 1 1
NCT04362137Study to Assess the Efficacy and Safety of Ruxolitinib in Patients With COVID-19 Associated Cytokine StormCytokine Storm (Covid-19)
COMPLETED432 Analytics
NCT04116502MITHRIDATE: Ruxolitinib Versus Hydroxycarbamide or Interferon as First Line Therapy in High Risk Polycythemia VeraPolycythemia Vera
RECRUITING586 Analytics
NCT02913261Safety and Efficacy of Ruxolitinib Versus Best Available Therapy in Patients With Corticosteroid-refractory Acute Graft vs. Host Disease After Allogeneic Stem Cell TransplantationCorticosteroid Refractory Acute Graft vs Host Disease
COMPLETED310 Analytics
NCT02292446Expanded Treatment Protocol (ETP) of Ruxolitinib in Patients With Polycythemia Vera Who Were Hydroxyurea Resistant or Intolerant and for Whom no Treatment Alternatives Was Available.Polycythemia Vera
COMPLETED161 Analytics
NCT02087059A Clinical Study of Ruxolitinib in Patients With Primary Myelofibrosis (PM), Post-polycythemia Vera (PV) Myelofibrosis, or Post-essential Thrombocythemia (ET) MyelofibrosisPrimary Myelofibrosis (MF)
COMPLETED51 Analytics
NCT02038036Ruxolitinib Efficacy and Safety in Patients With HU Resistant or Intolerant Polycythemia Vera vs Best Available Therapy.Polycythemia Vera
COMPLETED149 Analytics
NCT00934544Controlled Myelofibrosis Study With Oral Janus-associated Kinase (JAK) Inhibitor Treatment-II: The COMFORT-II TrialMyelofibrosis
COMPLETED219 Analytics
PHASE3COMPLETED
Study to Assess the Efficacy and Safety of Ruxolitinib in Patients With COVID-19 Associated Cytokine Storm
Cytokine Storm (Covid-19)Unlock trial analytics
PHASE3RECRUITING
MITHRIDATE: Ruxolitinib Versus Hydroxycarbamide or Interferon as First Line Therapy in High Risk Polycythemia Vera
Polycythemia VeraUnlock trial analytics
PHASE3COMPLETED
Safety and Efficacy of Ruxolitinib Versus Best Available Therapy in Patients With Corticosteroid-refractory Acute Graft vs. Host Disease After Allogeneic Stem Cell Transplantation
Corticosteroid Refractory Acute Graft vs Host DiseaseUnlock trial analytics
PHASE3COMPLETED
Expanded Treatment Protocol (ETP) of Ruxolitinib in Patients With Polycythemia Vera Who Were Hydroxyurea Resistant or Intolerant and for Whom no Treatment Alternatives Was Available.
Polycythemia VeraUnlock trial analytics
PHASE3COMPLETED
A Clinical Study of Ruxolitinib in Patients With Primary Myelofibrosis (PM), Post-polycythemia Vera (PV) Myelofibrosis, or Post-essential Thrombocythemia (ET) Myelofibrosis
Primary Myelofibrosis (MF)Unlock trial analytics
PHASE3COMPLETED
Ruxolitinib Efficacy and Safety in Patients With HU Resistant or Intolerant Polycythemia Vera vs Best Available Therapy.
Polycythemia VeraUnlock trial analytics
PHASE3COMPLETED
Controlled Myelofibrosis Study With Oral Janus-associated Kinase (JAK) Inhibitor Treatment-II: The COMFORT-II Trial
MyelofibrosisUnlock trial analytics
Study Endpoints
Primary Endpoints
Proportion of Patients Who Die, Develop Respiratory Failure [Require Mechanical Ventilation] or Require Intensive Care Unit (ICU) Care
Day 1 - Day 29

Efficacy is measured by a composite endpoint of proportion of patients who die, develop respiratory failure \[require mechanical ventilation\], or require intensive care unit \[ICU\] care for the treatment of COVID-19. Analyses are cumulative, thus analysis on Day 29 includes all events till that day. Patients who developed respiratory failure and/or required ICU at randomization are excluded from the analysis.

Event Free Survival (EFS)
the time from randomisation to the date of the first major thrombosis/haemorrhage, death,transformation to Myelodysplastic Syndromes, Acute Myeloid Leukaemia or Post-polycythemia Vera Myelofibrosis, if within the ~3 year trial period

Event Free Survival

Overall Response Rate (ORR) at Day 28
Day 28

Overall response rate at Day 28 after randomization was defined as the percentage participants in each arm demonstrating a complete response (CR) or partial response (PR), based on investigator assessment \& according to standard criteria, without requirement for additional systemic therapies for an earlier progression, mixed response or non-response. Scoring of response was relative to the organ stage at the time of randomization. CR was defined as a score of 0 for the aGvHD grading in all evaluable organs that indicates complete resolution of all signs \& symptoms of aGvHD in all evaluable organs without administration of additional systemic therapies for any earlier progression, mixed response or non-response of aGvHD. PR was defined as improvement of 1 stage in 1 or more organs involved with aGvHD signs or symptoms without progression in other organs or sites without administration of additional systemic therapies for an earlier progression, mixed response or non-response of aGvHD.

Number of Participants With Adverse Events - All Grades
Baseline up to approximately 26 months

Summary of adverse events (all grades).

Number of Participants With Adverse Events as a Measure of Safety and Tolerability
24 weeks
Number of Participants Achieving Hematocrit (Hct) Control at Week 28
Week 28

Proportion of patients achieving Hct control at Week 28 was defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 28, with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8. Phlebotomy eligibility was defined by: * Confirmed Hct \> 45% that is at least 3 percentage points higher than the Hct obtained at Baseline Or * Confirmed Hct \> 48% The confirmation occurred 2 to 14 days subsequent to the initial observation.

Percentage of Participants With at Least 35% Reduction in Spleen Volume From Baseline at Week 48
Baseline, Week 48

The change in spleen volume from baseline to week 48 was measured by magnetic resonance imaging (MRI) (or by computer tomography (CT) for participants unable to undergo MRI) and was calculated only for participants who had an evaluable spleen volume at baseline. The percentage of participants achieving a greater than or equal to 35% reduction in spleen volume from baseline to week 48 was then calculated by treatment group.

Percentage of Participants With at Least 50% Reduction in Spleen Length From Baseline at Week 24
Baseline up to week 24

Percentage of participants achieving a 50% reduction in spleen length at week 24. For subjects with palpable spleen at baseline and non-palpable at post-baseline, the post-baseline spleen are imputed as 0. Subjects who had palpable, but missing spleen length at baseline is excluded from the analysis. Subjects with missing spleen length at Week 24 or who withdraw earlier from the study are considered as a non-responder. The 95% CI is computed using exact Clopper-Pearson method.

Change of Hematocrit Adjusted Volume of Red Blood Cells (RBC)
week 6 to week 30 interval

Change of RBC transfusion requirement measured as percent change of the hematocrit-adjusted volume of transfused RBC and observed during within on-treatment interval (any time-points of RBC transfusion between week 6 and week 30 driven by the individual patient's need) compared to baseline (defined by pre-treatment interval between Week - 24 to start of treatment).

Percentage of Participants With at Least 35% Reduction in Spleen Volume From Baseline at Week 24
24 weeks

The primary measure of spleen size was by MRI. MRIs were performed with a body coil because the objective was to measure organ volume only, not to assess for lesions. MRIs were performed by local radiologists who were instructed not to provide a quantitative measure of spleen volume, but could provide a qualitative assessment such as enlarged, smaller, larger, etc. The scans from an individual patient were to be read by a central reader upon transfer from the site radiologist.

Phase I: Measurement of Pharmacokinetic (PK) Parameter, AUClast, in aGvHD and SR-aGvHD Patients
Day 1: at predose, 0.5,1,1.5, 2, 4, 6, 9 hours post dose

Measurement in acute GvHD and SR-acute GvHD subjects used extensive PK sampling in Groups 1-3 sparse sampling in Group 4. AUClast: The AUC from time zero to the last measurable concentration sampling time (Tlast).

Phase I: Measurement of PK Parameter, Cmax, in aGvHD and SR-aGvHD Patients
Day 1: at predose, 0.5,1,1.5, 2, 4, 6, 9 hours post dose

Measurement in acute GvHD and SR-acute GvHD subjects used extensive PK sampling in Groups 1-3 and sparse sampling in Group 4. Cmax: The maximum (peak) observed plasma drug concentration

Phase I: Measurement of PK Parameter, T1/2, in aGvHD and SR-aGvHD Patients
Day 1: at predose, 0.5,1,1.5, 2, 4, 6, 9 hours post dose

Measurement in acute GvHD and SR-acute GvHD subjects used extensive PK sampling in Groups 1-3 and sparse sampling in Group 4. T1/2: The elimination half-life associated with the terminal slope (Lambda\_z ) of a semi logarithmic concentration-time curve

Phase I: Measurement of PK Parameter, Ctrough, in aGvHD and SR-aGvHD Patients
Day 7 at pre-dose

Measurement in acute GvHD and SR-acute GvHD subjects used extensive PK sampling in Groups 1-3 and sparse sampling in Group 4. Ctrough: The minimum observed plasma concentration at the end of an administration interval (corresponding to the pre-dose concentration prior to the following administration).

Phase I: Age-based Determination of Recommended Phase 2 Dose (RP2D) Using AUClast
Day 1: at predose, 0.5,1,1.5, 2, 4, 6, 9 hours post dose

Phase I: Age-based determination of RP2D in Groups 2 and 3 and was based on observed PK parameters in Groups 1-3 * Group 2: age ≥ 6 to \< 12 years * Group 3: age ≥ 2 to \< 6 years AUClast: The AUC from time zero to the last measurable concentration sampling time (Tlast).

Phase I: Age-based Determination of Recommended Phase 2 Dose (RP2D) Using Cmax
Day 1: at predose, 0.5,1,1.5, 2, 4, 6, 9 hours post dose

Phase I: Age-based determination of RP2D in Groups 2 and 3 and was based on observed PK parameters in Groups 1-3 * Group 2: age ≥ 6 to \< 12 years * Group 3: age ≥ 2 to \< 6 years Cmax: The maximum (peak) observed plasma drug concentration.

Phase I: Age-based Determination of Recommended Phase 2 Dose (RP2D) Using Ctrough
Day 7 at pre-dose

Phase I: Age-based determination of RP2D in Groups 2 and 3 and was based on observed PK parameters in Groups 1-3 * Group 2: age ≥ 6 to \< 12 years * Group 3: age ≥ 2 to \< 6 years Ctrough: The minimum observed plasma concentration at the end of an administration interval (corresponding to the pre-dose concentration prior to the following administration).

Phase II: Overall Response Rate (ORR)
Day 28

Phase II: ORR is defined as the percentage of patients demonstrating a complete response (CR) or partial response (PR) without requirement for additional systemic therapies for an earlier progression, mixed response or non-response. Scoring of response was relative to the organ stage at the start of the study treatment. Complete response (CR) is defined as a score of 0 for the aGvHD grading in all evaluable organs that indicates complete resolution of all signs and symptoms of aGvHD in all evaluable organs without administration of additional systemic therapy for any earlier progression, mixed response or non-response of aGvHD. Partial response (PR) is defined as improvement of 1 stage in 1 or more organs involved with aGvHD signs or symptoms without progression in other organs or sites without administration of additional systemic therapy for an earlier progression, mixed response or non-response of aGvHD.

Secondary Endpoints
Clinical Status
Baseline, Day 15, Day 29
Percentage of Patients With at Least Two-point Improvement From Baseline in Clinical Status
Baseline, Day 15, Day 29
Percentage of Patients With at Least One-point Improvement From Baseline in Clinical Status
Baseline, Day 15, Day 29
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Ruxolitinib 5 mgEXPERIMENTALRuxolitinib 5 mg tablets twice daily (b.i.d.) for 14 days with possible extension of treatment to 28 days
PlaceboPLACEBO_COMPARATORMatching-image placebo for 14 days with possible extension of treatment to 28 days
A- RuxolitinibEXPERIMENTALTreatment with Ruxolitinib
B- Hydroxycarbamide OR Interferon AACTIVE_COMPARATORBest Available Therapy (BAT), Treatment with hydroxycarbamide OR Interferon A
RuxolitinibEXPERIMENTALThese patients were administered Ruxolitinib orally twice per day (b.i.d) at a dose of 10 mg bid, as two 5-mg tablets. Ruxolitinib was taken without regards to food.
Best Available Therapy (BAT)ACTIVE_COMPARATORThese patients were administered BAT per the Investigator's best judgement based on a specific list of BAT.
All patientsEXPERIMENTALAll patients will receive ruxolitinib at a starting dose of 10 mg twice daily which could be titrated to most appropriate dose. Dose was not to exceed 25 mg bid nor be less than 5 mg once a day
All SubjectsEXPERIMENTAL10 mg BID (2 tablets of 5mg) was self-administered as starting dose for all patients. This dose was maintained for the first 12 weeks and titrated up thereafter unless they had met criteria for dose hold or dose reduction. Dose was to have been increased or decreased per standardized dosing paradigm and not to have exceeded 25 mg bid.
INC424 (ruxolitinib) - Study TreatmentEXPERIMENTALRegularly transfused adult patients with thalassemia and spleen enlargement.
Interventions
NameTypeDescription
RuxolitinibDRUGRuxolitinib 5 mg tablets
PlaceboDRUGMatching-image placebo
HydroxycarbamideDRUGVia standard hospital mechanisms
Interferon-AlphaDRUGAny formulation, via standard hospital mechanisms
Ruxolitinib (RUX)DRUGRuxolitinib was provided as 5 mg tablets for oral use.
Best Available Therapy (BAT)DRUGBAT was based on the investigator's best judgment, taking into account the manufacturer's instructions, labeling, patient's medical condition, and institutional guidelines for any dose adjustment. The BAT in this study was identified by the investigator prior to patient randomization among the following treatments currently used in this setting: anti-thymocyte globulin (ATG), extracorporeal photopheresis (ECP), mesenchymal stromal cells (MSC), low-dose methotrexate (MTX), mycophenolate mofetil (MMF), mTOR inhibitors (everolimus or sirolimus), etanercept, or infliximab. No other types or combinations of BAT were permitted.
Best Available TherapyDRUGBest Available Therapy as selected by the investigator from: Hydroxyurea, Pegylated-Interferon (IFN/PEG-IFN), pipobroman, anagrelide, IMIDs, or observation. Participants randomized to BAT who did not respond by Week 28 were eligible to crossover and start treatment with ruxolitinib.
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Eligibility Criteria
Age Range12 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites61

Inclusion Criteria: Patient or guardian/health proxy must provide informed consent (and assent if applicable) before any study assessment is performed. Male and female patients aged ≥ 12 years (or ≥ the lower age limit allowed by Health Authority and/or Ethics Committee/Institutional Review Board ...

Countries:United StatesArgentinaBrazilColombiaFranceGermanyMexicoPeruRussiaSpainTurkey (Türkiye)United KingdomAustraliaAustriaBulgariaCanadaCzechiaDenmarkGreeceHong KongIsraelItalyJapanNetherlandsNorwaySaudi ArabiaSouth KoreaTaiwanBelgiumChilePortugalSwedenThailandHungaryIndiaLebanonChina
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Competitive Landscape -Polycythemia Vera 11 trials