Ruxolitinib efficacy and safety in patients with HU resistant or intolerant polycythemia vera vs best available therapy

Randomized, open label, multicenter phase IIIb study evaluating the efficacy and safety of ruxolitinib versus best available therapy in patients with polycythemia vera who are hydroxyurea resistant or intolerant (Response 2) - Response 2

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-003583-31-DE

Enrollment

130

Registered

2013-12-02

Start date

2014-02-17

Completion date

Unknown

Last updated

2021-04-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Polycythemia vera resistant or intolerant to hydroxyurea MedDRA version: 20.0 Level: LLT Classification code 10036061 Term: Polycythemia vera System Organ Class: 100000004864

Interventions

Trade Name: Jakavi 5 mg Tabletten Product Name: Ruxolitinib Product Code: INC424 Pharmaceutical Form: Tablet INN or Proposed INN: Ruxolitinib CAS Number: 1092939-17-7 Current Sponsor code: INC424 Othe

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Confirmed diagnosis of PV according to the 2008 World Health Organization criteria 2. Non-palpable spleen at Screening and Baseline 3. Phlebotomy dependent, Resistant to or intolerant of hydroxyurea 4. ECOG performance status of 0, 1 or 2. Other inclusion criteria as defined by protocol may apply Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 105 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 25

Exclusion criteria

Exclusion criteria: - Inadequate liver or renal function - Significant bacterial, fungal, parasitic, or viral infection requiring treatment - Active malignancy within the past 5 years, excluding specific skin cancers - Previously received treatment with a JAK inhibitor - Being treated with any investigational agent - Women who are pregnant or nursing. Other exclusion criteria as defined by protocol may apply.

Design outcomes

Primary

Measure	Time frame
Main Objective: efficacy of ruxolitinib to BAT as assessed by Hct control at Week 28;Secondary Objective: Key 1. efficacy of RUX to BAT assessed by complete hematological remission at W28 Other 2. efficacy of RUX to BAT assessed by durable Hct control at W52 and W80 3. efficacy of RUX to BAT assessed by durable complete hematological remission at W52 & W80 4. assess phlebotomies over time 5. change in Hct 6. asses spleen length 7. ECOG status change 8. efficacy of RUX to BAT assessed by partial remission based on ELN+IWG-MRT criteria at W28 9. efficacy of RUX to BAT assessed by durable partial remission based on ELN+IWG-MRT criteria at W52 & W80 10. efficacy of BAT pts after crossover 11. efficacy of RUX pts measured by durable Hct control at W104, 156, 208, 260 12. efficacy of RUX pts measured by durable complete hematological remission at W104, 156, 208, 260 13. efficacy of RUX pts by partial remission based on ELN+IWG-MRT criteria at W104, 156, 208, 260 14. transformation-free survival 15. OS 16. changes in PRO;Primary end point(s): Proportion of patients achieving Hct control at Week 28 as 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 will be 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 will occur 2 to 14 days subsequent to the initial observation.;Timepoint(s) of evaluation of this end point: week 28	—

Measure

Time frame

Main Objective: efficacy of ruxolitinib to BAT as assessed by Hct control at Week 28;Secondary Objective: Key 1. efficacy of RUX to BAT assessed by complete hematological remission at W28 Other 2. efficacy of RUX to BAT assessed by durable Hct control at W52 and W80 3. efficacy of RUX to BAT assessed by durable complete hematological remission at W52 & W80 4. assess phlebotomies over time 5. change in Hct 6. asses spleen length 7. ECOG status change 8. efficacy of RUX to BAT assessed by partial remission based on ELN+IWG-MRT criteria at W28 9. efficacy of RUX to BAT assessed by durable partial remission based on ELN+IWG-MRT criteria at W52 & W80 10. efficacy of BAT pts after crossover 11. efficacy of RUX pts measured by durable Hct control at W104, 156, 208, 260 12. efficacy of RUX pts measured by durable complete hematological remission at W104, 156, 208, 260 13. efficacy of RUX pts by partial remission based on ELN+IWG-MRT criteria at W104, 156, 208, 260 14. transformation-free survival 15. OS 16. changes in PRO;Primary end point(s): Proportion of patients achieving Hct control at Week 28 as 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 will be 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 will occur 2 to 14 days subsequent to the initial observation.;Timepoint(s) of evaluation of this end point: week 28

—

Secondary

Measure	Time frame
Timepoint(s) of evaluation of this end point: For key secondary, 1. week 28 For other secondary: 1. week 52 2. week 52 3. week 4, 8, 12, 16, 20, 24, 28, 40, 52/End of Treatment 4. week 4, 8, 12, 16, 20, 24, 28, 40, 52/End of Treatment 5. week 4, 8, 12, 16, 20, 24, 28, 40, 52/End of Treatment 6. week 28 7. week 28 8. week 52 9. +4, +8, +12, +16, +20, +24 weeks after crossover 10. week 104 11. week 104 12. week 104;Secondary end point(s): Key secondary: Proportion of patients achieving a complete hematological remission at Week 28 as defined by: • Hct control at Week 28 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, and • WBC < 10 x109/L at Week 28, and • Platelets = 400 x 109/L at Week 28 Other secondary: 1. Proportion of patients achieving a Hct control at Week 52 as defined by: • the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 52, with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8 In the BAT arm, no change in the treatment regimen or crossover to the ruxolitinib arm. Endpoint for Week 80 is defined, similarly. 2. Proportion of patients achieving a complete hematological remission at Week 52 as defined by: • Hct control at Week 52 as defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 52, with no more than one phlebotomy eligibility occurring post randomization and prior to week 8, and • WBC < 10 x109/L at Week 52, and • Platelets = 400 x 109/L at Week 52, and In the BAT arm, no change in the treatment regimen or crossover to the ruxolitinib arm. Endpoint for Week 80 is defined, similarly. 3. Number of phlebotomies from Baseline up to Week 28 4. Change from Baseline in Hct at each visit 5. Summary of spleen length by visit 6. Change in ECOG status from baseline to W	—

Measure

Time frame

Timepoint(s) of evaluation of this end point: For key secondary, 1. week 28 For other secondary: 1. week 52 2. week 52 3. week 4, 8, 12, 16, 20, 24, 28, 40, 52/End of Treatment 4. week 4, 8, 12, 16, 20, 24, 28, 40, 52/End of Treatment 5. week 4, 8, 12, 16, 20, 24, 28, 40, 52/End of Treatment 6. week 28 7. week 28 8. week 52 9. +4, +8, +12, +16, +20, +24 weeks after crossover 10. week 104 11. week 104 12. week 104;Secondary end point(s): Key secondary: Proportion of patients achieving a complete hematological remission at Week 28 as defined by: • Hct control at Week 28 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, and • WBC < 10 x109/L at Week 28, and • Platelets = 400 x 109/L at Week 28 Other secondary: 1. Proportion of patients achieving a Hct control at Week 52 as defined by: • the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 52, with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8 In the BAT arm, no change in the treatment regimen or crossover to the ruxolitinib arm. Endpoint for Week 80 is defined, similarly. 2. Proportion of patients achieving a complete hematological remission at Week 52 as defined by: • Hct control at Week 52 as defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 52, with no more than one phlebotomy eligibility occurring post randomization and prior to week 8, and • WBC < 10 x109/L at Week 52, and • Platelets = 400 x 109/L at Week 52, and In the BAT arm, no change in the treatment regimen or crossover to the ruxolitinib arm. Endpoint for Week 80 is defined, similarly. 3. Number of phlebotomies from Baseline up to Week 28 4. Change from Baseline in Hct at each visit 5. Summary of spleen length by visit 6. Change in ECOG status from baseline to W

—

Countries

Australia, Belgium, Canada, France, Germany, Hungary, India, Israel, Italy, Korea, Republic of, Spain, Switzerland, Turkey

Contacts

Public ContactMedizinischer Infoservice (MCC)

Novartis Pharma GmbH

infoservice.novartis@novartis.com+491802 232300

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026