TriangleNCT02038036 · CITED IN SOURCE DOCUMENTS
Ruxolitinib Efficacy and Safety in Patients With HU Resistant or Intolerant Polycythemia Vera vs Best Available Therapy.
An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.
- Phase
- PHASE3
- Status at capture
- COMPLETED
- Registry last update
- 2021-07-20
This study compared the efficacy and safety of ruxolitinib to Best Available Therapy (BAT) in patients with polycythemia vera (PV) who were hydroxyurea (HU) resistant or intolerant and did not have a palpable spleen.
Open the original ClinicalTrials.gov record → · Download preserved record
What did the study report?
Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.
Participant flow
Baseline characteristics
Number of Participants Achieving Hematocrit (Hct) Control at Week 28
Number of Participants Achieving a Complete Hematological Remission at Week 28
Number of Participants Achieving a Hematocrit (Hct) Control at Week 52 and Week 80
Number of Participants Achieving a Complete Hematological Remission at Week 52 and Week 80
Number of Participants With Phlebotomies Over Time
Change From Baseline in Hematocrit (Hct) at Each Visit
Change From Baseline in Hematocrit (Hct) at Each Scheduled Visit After Crossover in Participants Randomized to BAT Who Cross Over to Ruxolitinib
Spleen Length by Visit
Change From Baseline in Eastern Cooperative Oncology Group (ECOG) Performance Status to Week 28
Number of Participants Achieving a Partial Remission Based on the European Leukemia Net (ELN) and International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) Criteria at Week 28
Number of Participants Who Achieved Partial Remission Based on the European Leukemia Net (ELN) and International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) Criteria at Week 52 and Week 80
Number of Participants Achieving a Hematocrit (Hct) Control at Week 104, Week 156, Week 208 and Week 260.
Number of Participants Achieving a Complete Hematological Remission at Week 104, Week 156, Week 208 and Week 260
Number of Participants Who Achieved Partial Remission Based on the European Leukemia Net (ELN) and International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) Criteria at Week 104, Week 156, Week 208 and Week 260.
Number of Participants With Transformation Free Survival Events
Number of Participants With Overall Survival (OS) Events
Change From Baseline in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)
Change From Baseline in Total Scores of MPN-SAF by Visit in Patients From BAT Group Who Cross Over to Ruxolitinib After Crossover
Change From Baseline in Score as Per European Quality of Life 5-Dimension 5-level (EQ-5D-5L) Questionnaire
Change From Baseline in EQ-5D-5L VAS, by Visit in Patients From BAT Group Who Cross Over to Ruxolitinib After Crossover
Change From Baseline in Work Productivity and Activity Impairment (WPAI) Questionnaire
Change From Baseline in Work Productivity and Activity Impairment Questionnaire (WPAI), by Visit in Patients From BAT Group Who Cross Over to Ruxolitinib After Crossover
Patient Global Impression of Change (PGIC)
Summary of Patient Global Impression of Change (PGIC), by Visit in Patients From BAT Group Who Cross Over to Ruxolitinib After Crossover
Number of Participants Developing Thrombosis
Total Number of Deaths
Adverse events
Limitations, agreements, and source contact
Who could take part
- eligibility Criteria
- Inclusion Criteria: Confirmed diagnosis of PV according to the 2008 World Health Organization criteria, Non-palpable spleen, Phlebotomy dependent, Resistant to or intolerant of hydroxyurea, ECOG performance status of 0, 1 or 2. 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 inclusion/exclusion criteria apply.
- healthy Volunteers
- false
- minimum Age
- 18 Years
- sex
- ALL
- std Ages
- ADULT
- OLDER_ADULT
Treatment arms and interventions
- arm Groups
- description
- Ruxolitinib was administered at a starting dose of 10 mg twice a day (bid). Dose was adjusted based on efficacy and safety parameters up to a maximum dose of 25 mg bid.
- intervention Names
- Drug: Ruxolitinib
- label
- Ruxolitinib
- type
- EXPERIMENTAL
- description
- Best 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.
- intervention Names
- Drug: Best Available Therapy
- label
- Best Available Therapy (BAT)
- type
- ACTIVE_COMPARATOR
- interventions
- arm Group Labels
- Best Available Therapy (BAT)
- description
- Best 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.
- name
- Best Available Therapy
- other Names
- BAT
- type
- DRUG
- arm Group Labels
- Ruxolitinib
- description
- Ruxolitinib was administered at a starting dose of 10 mg twice a day (bid). Dose was adjusted based on efficacy and safety parameters up to a maximum dose of 25 mg bid.
- name
- Ruxolitinib
- other Names
- INC424
- type
- DRUG
Study design
- allocation
- RANDOMIZED
- intervention Model
- PARALLEL
- masking Info
- masking
- NONE
- primary Purpose
- TREATMENT
Enrollment
- count
- 149
- type
- ACTUAL
Registered outcome plans (not posted results)
- primary Outcomes
- description
- 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.
- measure
- Number of Participants Achieving Hematocrit (Hct) Control at Week 28
- time Frame
- Week 28
- secondary Outcomes
- description
- Proportion of patients achieving a complete hematological remission at Week 28 was 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
- measure
- Number of Participants Achieving a Complete Hematological Remission at Week 28
- time Frame
- Week 28
- description
- Proportion of patients achieving a Hct control at Week 52 was defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 52, and no more than one phlebotomy eligibility occurring post randomization and prior to Week 8 \- Endpoint for Week 80 was defined, similarly.
- measure
- Number of Participants Achieving a Hematocrit (Hct) Control at Week 52 and Week 80
- time Frame
- Week 52 and 80
- description
- Proportion of patients achieving a complete hematological remission at Week 52, was 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 * White Blood Count (WBC) \< 10 x10\^9/L at Week 52, and * Platelets ≤ 400 x 10\^9/L at Week 52 * Endpoint for Week 80 was defined, similarly.
- measure
- Number of Participants Achieving a Complete Hematological Remission at Week 52 and Week 80
- time Frame
- Week 52 and 80
- description
- 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.
- measure
- Number of Participants With Phlebotomies Over Time
- time Frame
- Baseline to Week 260
- description
- Hematocrit is the volume percentage of red blood cells (RBC) in the blood.
- measure
- Change From Baseline in Hematocrit (Hct) at Each Visit
- time Frame
- Baseline, Week 4, 8, 12, 16, 20, 24, 28, 40, 52, 66, 80, 92, 104, 117, 130, 143, 156, 169, 182, 195, 208, 221, 234, 247 and 260
- description
- Hematocrit is the percentage of red blood cells (RBC) in the blood.
- measure
- Change From Baseline in Hematocrit (Hct) at Each Scheduled Visit After Crossover in Participants Randomized to BAT Who Cross Over to Ruxolitinib
- time Frame
- Baseline (last assessment before cross over), Week 4, 8, 12, 16, 20, 24, 28, 40, 52, 64, 76, 89, 102, 115, 128, 141, 154, 167, 180, 193, 206, 219 and 232 after cross-over
- description
- Spleen length was assessed by manual palpation at every study visit.
- measure
- Spleen Length by Visit
- time Frame
- Week 4, 8, 12, 16, 20, 24, 28, 40, 52, 66, 80, 92, 104, 117, 130, 143, 156, 169, 182, 195, 208, 221, 234, 247 and 260
- description
- The ECOG scale of performance status described the level of functioning of participants in terms of their ability to care for themselves, daily activity, and physical ability. The ECOG performance was recorded as per ECOG performance status grades ranging from 0 (fully active, able to carry on all pre-disease performance without restriction) to 5 (dead).
- measure
- Change From Baseline in Eastern Cooperative Oncology Group (ECOG) Performance Status to Week 28
- time Frame
- Baseline and Week 28
- description
- Proportion of patients achieving a partial remission at Week 28, based on the ELN and IWG-MRT criteria, as defined by: * Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) score reduction of greater than or equal to 10 points from baseline to Week 28, and * Hct control 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 x10\^9/L at Week 28, and * Platelets ≤ 400 x 10\^9/L at Week 28, and * No palpable spleen at Week 28, and * No hemorrhagic or thrombotic events, and * No transformation into post-PV myelofibrosis, myelodysplastic syndrome (IWG-MRT criteria) or acute leukemia (WHO criteria).
- measure
- Number of Participants Achieving a Partial Remission Based on the European Leukemia Net (ELN) and International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) Criteria at Week 28
- time Frame
- Week 28
- description
- Proportion of patients who achieved partial remission at Week 52 based on the ELN and IWG-MRT criteria, as defined by: * MPN-SAF TSS score reduction of greater than or equal to 10 points from baseline to Week 52 and * Hct control 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 * No palpable spleen at Week 52 and * No hemorrhagic or thrombotic events, and * No transformation into post-PV myelofibrosis, myelodysplastic syndrome (IWG-MRT criteria) or acute leukemia (WHO criteria). * Endpoint for Week 80 was defined, similarly.
- measure
- Number of Participants Who Achieved Partial Remission Based on the European Leukemia Net (ELN) and International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) Criteria at Week 52 and Week 80
- time Frame
- Week 52 and 80
- description
- Proportion of patients achieving a Hct control at Week 104 as defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 104 and with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8 Endpoint for Week 156, Week 208 and Week 260 were defined, similarly.
- measure
- Number of Participants Achieving a Hematocrit (Hct) Control at Week 104, Week 156, Week 208 and Week 260.
- time Frame
- From Week 8 to Week 104, 156, 208 and 260
- description
- Proportion of patients achieving a complete hematological remission at Week 104 as defined by Hct control defined by the absence of phlebotomy eligibility starting at Week 8 and continuing through Week 104, with no more than one phlebotomy eligibility occurring post randomization and prior to Week 8, and * WBC \< 10 x10\^9/L at Week 104, and * Platelets ≤ 400 x 10\^9/L at Week 104 Endpoint for Week 156, Week 208 and Week 260 were defined, similarly.
- measure
- Number of Participants Achieving a Complete Hematological Remission at Week 104, Week 156, Week 208 and Week 260
- time Frame
- From Week 8 to Week 104, 156, 208 and 260
Full study description
- brief Summary
- This study compared the efficacy and safety of ruxolitinib to Best Available Therapy (BAT) in patients with polycythemia vera (PV) who were hydroxyurea (HU) resistant or intolerant and did not have a palpable spleen.
- detailed Description
- This was a prospective, multi-center, open-label, randomized, Phase IIIb study evaluating efficacy and safety of ruxolitinib versus BAT as selected by the Investigator in patients with PV who are resistant to, or intolerant of HU. The study comprised of the following periods: Screening Period (up to 5 weeks: Day -35 to Day -1): Screening evaluations were performed at one or more clinic visits, and reviewed to determine eligibility before the patient was randomized in the study. Core Treatment Period (Day 1 to Week 80): Patients were randomized in 1:1 ratio to either treatment group (ruxolitinib or BAT) and were to be treated with their randomized treatment. Crossover Treatment Period (Week 28 or after) for BAT patients only: Patients randomized to BAT who did not respond by Week 28 were eligible to crossover and start treatment with ruxolitinib. Patients crossing over on or after Week 28 had to complete all assessments for the End of Treatment (EoT) visit of the Core Treatment Period followed by the assessments in Cross-over visit evaluation schedule. Extended Treatment Period (Week 80 to Week 260): Patients receiving ruxolitinib at Week 80 (including patients who have crossed over from BAT) were eligible to continue up to Week 260 in the Extended Treatment Period. Patients continued the ruxolitinib dose that they received at Week 80. Patients who received BAT at Week 80 were not eligible to enter the Extended Treatment Period and had to have the EoT visit at Week 80 and an End of study (EoS) visit 30 days after the EoT visit. Follow-up Period: Patients were followed for safety during 30 days after the last dose of study drug and EoS visit assessments were performed post 30 days after the last dose of study drug. Patients who completed EoT (Week 80 for patients who received BAT, Week 260 for patients who received ruxolitinib or from the time of premature discontinuation) were to be followed-up for every 3 months for survival till the end of the study.
Source references
- references
- citation
- Harrison CN, Kiladjian JJ, Hamer-Maansson JE, Naim A, Palandri F, Passamonti F. Ruxolitinib treatment in patients with polycythemia vera reduces JAK2 variant allele frequency and improves symptom burden and hematocrit control. Ther Adv Hematol. 2026 Mar 24;17:20406207261425083. doi: 10.1177/20406207261425083. eCollection 2026.
- pmid
- 41890273
- type
- DERIVED
- citation
- Passamonti F, Palandri F, Saydam G, Callum J, Devos T, Guglielmelli P, Vannucchi AM, Zor E, Zuurman M, Gilotti G, Zhang Y, Griesshammer M. Ruxolitinib versus best available therapy in inadequately controlled polycythaemia vera without splenomegaly (RESPONSE-2): 5-year follow up of a randomised, phase 3b study. Lancet Haematol. 2022 Jul;9(7):e480-e492. doi: 10.1016/S2352-3026(22)00102-8. Epub 2022 May 18.
- pmid
- 35597252
- type
- DERIVED
- citation
- Kiladjian JJ, Guglielmelli P, Griesshammer M, Saydam G, Masszi T, Durrant S, Passamonti F, Jones M, Zhen H, Li J, Gadbaw B, Perez Ronco J, Khan M, Verstovsek S. Efficacy and safety of ruxolitinib after and versus interferon use in the RESPONSE studies. Ann Hematol. 2018 Apr;97(4):617-627. doi: 10.1007/s00277-017-3225-1. Epub 2018 Feb 2.
- pmid
- 29396713
- type
- DERIVED
- citation
- Passamonti F, Griesshammer M, Palandri F, Egyed M, Benevolo G, Devos T, Callum J, Vannucchi AM, Sivgin S, Bensasson C, Khan M, Mounedji N, Saydam G. Ruxolitinib for the treatment of inadequately controlled polycythaemia vera without splenomegaly (RESPONSE-2): a randomised, open-label, phase 3b study. Lancet Oncol. 2017 Jan;18(1):88-99. doi: 10.1016/S1470-2045(16)30558-7. Epub 2016 Dec 2.
- pmid
- 27916398
- type
- DERIVED
Source notices and limitations
Discovery and provenance
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- September 27, 2024
Preserved source evidence · Not medical advice