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NCT01243944 · STUDY IN CONTEXT

Study of Efficacy and Safety in Polycythemia Vera Subjects Who Are Resistant to or Intolerant of Hydroxyurea: JAK Inhibitor INC424 (INCB018424) Tablets Versus Best Available Care:

Draft scope assessment: narrower. Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded. The condition was transformed or resolved through the terminology hierarchy to reach this cancer type, so the link itself is a candidate a reviewer must confirm. Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.

Read the full study record and all posted results →

Linked outcomes

Measures are not ranked. Open each original source section for treatment groups, denominators, units and results.

primary · POSTED

Primary response was defined as having achieved hematocrit control (the absence of phlebotomy eligibility beginning at the Week 8 visit and continuing through Week 32) and Spleen Volume Reduction (a greater than or equal to 35% reduction from baseline in spleen volume at Week 32).

Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.

→

secondary · POSTED

Durable Primary Response was defined as any participant who achieved the primary outcome measure and who maintained their response up to 48 weeks after randomization.

Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.

→

secondary · POSTED

Complete Hematological Remission at Week 32 was defined as any participant who achieved hematocrit control with a platelet count less than or equal to 400 X 10\^9/L and a white blood cell count less than or equal to 10 X 10\^9/L.

Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.

→

secondary · POSTED

Durable Complete Hematological Remission was defined as any participant who achieved Complete Hematological Remission at Week 32 and maintained their response up to 48 weeks after randomization.

Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.

→

secondary · POSTED

Durable Hematocrit Control was defined as any participant who achieved phlebotomy eligibility independence from Week 8 to Week 32 and maintained hematocrit control up to 48 weeks after randomization.

Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.

→

secondary · POSTED

Durable Spleen Volume Reduction was defined as a participant who achieved at least 35% reduction from baseline in spleen volume at Week 32 and maintained that response 48 weeks after randomization.

Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.

→

Reported safety

Serious events are not the same as severe events. Compare the original safety groups and their at-risk populations.

Open the study safety table →

Quality of life and patient-reported measures

No linked quality-of-life measure is recorded for this setting and study.

Full draft applicability and source-link record
trial
release id
clinical-indications-draft-1
disease state id
ds-myeloid-neoplasm
nct id
NCT01243944
trial label
Study of Efficacy and Safety in Polycythemia Vera Subjects Who Are Resistant to or Intolerant of Hydroxyurea: JAK Inhibitor INC424 (INCB018424) Tablets Versus Best Available Care:
applicability
narrower
applicability note
Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded. The condition was transformed or resolved through the terminology hierarchy to reach this cancer type, so the link itself is a candidate a reviewer must confirm.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
provenance class
regulator-cited
discovery note
Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.
outcome Count
6
harms Count
3
qol Count
0
outcomes
  1. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0000
    source section id
    NCT01243944-om-0000
    source outcome index
    0
    endpoint
    The Percentage of Participants Achieving a Primary Response at Week 32
    endpoint definition
    Primary response was defined as having achieved hematocrit control (the absence of phlebotomy eligibility beginning at the Week 8 visit and continuing through Week 32) and Spleen Volume Reduction (a greater than or equal to 35% reduction from baseline in spleen volume at Week 32).
    time origin
    32 Weeks
    analysis role
    primary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  2. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0001
    source section id
    NCT01243944-om-0001
    source outcome index
    1
    endpoint
    The Percentage of Participants Achieving a Durable Primary Response at Week 48
    endpoint definition
    Durable Primary Response was defined as any participant who achieved the primary outcome measure and who maintained their response up to 48 weeks after randomization.
    time origin
    48 Weeks
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  3. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0002
    source section id
    NCT01243944-om-0002
    source outcome index
    2
    endpoint
    The Percentage of Participants Achieving Complete Hematological Remission at Week 32
    endpoint definition
    Complete Hematological Remission at Week 32 was defined as any participant who achieved hematocrit control with a platelet count less than or equal to 400 X 10\^9/L and a white blood cell count less than or equal to 10 X 10\^9/L.
    time origin
    32 Weeks
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  4. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0003
    source section id
    NCT01243944-om-0003
    source outcome index
    3
    endpoint
    The Percentage of Participants Who Achieved a Durable Complete Hematological Remission at Week 48
    endpoint definition
    Durable Complete Hematological Remission was defined as any participant who achieved Complete Hematological Remission at Week 32 and maintained their response up to 48 weeks after randomization.
    time origin
    48 Weeks
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  5. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0004
    source section id
    NCT01243944-om-0004
    source outcome index
    4
    endpoint
    The Percentage of Participants Who Achieved a Durable Hematocrit Control at Week 48
    endpoint definition
    Durable Hematocrit Control was defined as any participant who achieved phlebotomy eligibility independence from Week 8 to Week 32 and maintained hematocrit control up to 48 weeks after randomization.
    time origin
    48 Weeks
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  6. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0005
    source section id
    NCT01243944-om-0005
    source outcome index
    5
    endpoint
    The Percentage of Participants Who Achieved Durable Spleen Volume Reduction at Week 48
    endpoint definition
    Durable Spleen Volume Reduction was defined as a participant who achieved at least 35% reduction from baseline in spleen volume at Week 32 and maintained that response 48 weeks after randomization.
    time origin
    48 Weeks
    analysis role
    secondary
    reporting status
    POSTED
    data cutoff
    Source null
    population note
    Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
arms
  1. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0000
    arm key
    OG000
    arm label
    Ruxolitinib
    regimen
    Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
    analyzed count
    110
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT01243944-om-0000#groups/OG000
  2. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0000
    arm key
    OG001
    arm label
    Best Available Therapy
    regimen
    Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
    analyzed count
    112
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT01243944-om-0000#groups/OG001
  3. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0001
    arm key
    OG000
    arm label
    Ruxolitinib
    regimen
    Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
    analyzed count
    110
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT01243944-om-0001#groups/OG000
  4. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0001
    arm key
    OG001
    arm label
    Best Available Therapy
    regimen
    Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
    analyzed count
    112
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT01243944-om-0001#groups/OG001
  5. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0002
    arm key
    OG000
    arm label
    Ruxolitinib
    regimen
    Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
    analyzed count
    110
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT01243944-om-0002#groups/OG000
  6. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0002
    arm key
    OG001
    arm label
    Best Available Therapy
    regimen
    Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
    analyzed count
    112
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT01243944-om-0002#groups/OG001
  7. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0003
    arm key
    OG000
    arm label
    Ruxolitinib
    regimen
    Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
    analyzed count
    110
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT01243944-om-0003#groups/OG000
  8. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0003
    arm key
    OG001
    arm label
    Best Available Therapy
    regimen
    Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
    analyzed count
    112
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT01243944-om-0003#groups/OG001
  9. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0004
    arm key
    OG000
    arm label
    Ruxolitinib
    regimen
    Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
    analyzed count
    110
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT01243944-om-0004#groups/OG000
  10. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0004
    arm key
    OG001
    arm label
    Best Available Therapy
    regimen
    Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
    analyzed count
    112
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT01243944-om-0004#groups/OG001
  11. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0005
    arm key
    OG000
    arm label
    Ruxolitinib
    regimen
    Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
    analyzed count
    110
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT01243944-om-0005#groups/OG000
  12. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    outcome key
    om-0005
    arm key
    OG001
    arm label
    Best Available Therapy
    regimen
    Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
    analyzed count
    112
    analyzed unit
    Participants
    is comparator
    Source null
    source pointer
    NCT01243944-om-0005#groups/OG001
harms
  1. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    harm key
    EG000
    source section id
    NCT01243944-ae
    event group key
    EG000
    event group label
    Ruxolitinib - Through Week 32
    serious affected
    15
    serious at risk
    110
    other affected
    105
    other at risk
    110
    frequency threshold
    5
    collection period
    The initial data reported was for the study up to Week 32. This time frame defines the comparative phase of the study where the majority of participants remained on their original randomized assignment and exposure between ruxolitinib and best available therapy was similar. Data inclusive of end of study was reported up to week 256 for Ruxolitinib group. Long term Follow Up for BAT was not reported as all patients have completed the study or crossed over to Ruxolitinib group by week 80.
    event definition
    Safety population: All participants who were enrolled and took at least 1 dose of study medication. The initial data reported at Week 32 was based on MedDRA 15.1; the remaining data reported for end of study was based on MedDRA 20.1.
    undenominated note
    Source null
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  2. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    harm key
    EG001
    source section id
    NCT01243944-ae
    event group key
    EG001
    event group label
    Best Available Therapy - Through Week 32
    serious affected
    10
    serious at risk
    111
    other affected
    104
    other at risk
    111
    frequency threshold
    5
    collection period
    The initial data reported was for the study up to Week 32. This time frame defines the comparative phase of the study where the majority of participants remained on their original randomized assignment and exposure between ruxolitinib and best available therapy was similar. Data inclusive of end of study was reported up to week 256 for Ruxolitinib group. Long term Follow Up for BAT was not reported as all patients have completed the study or crossed over to Ruxolitinib group by week 80.
    event definition
    Safety population: All participants who were enrolled and took at least 1 dose of study medication. The initial data reported at Week 32 was based on MedDRA 15.1; the remaining data reported for end of study was based on MedDRA 20.1.
    undenominated note
    Source null
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
  3. release id
    clinical-indications-draft-1
    disease state id
    ds-myeloid-neoplasm
    nct id
    NCT01243944
    harm key
    EG002
    source section id
    NCT01243944-ae
    event group key
    EG002
    event group label
    Ruxolitinib - Week 256 Close Out
    serious affected
    44
    serious at risk
    110
    other affected
    107
    other at risk
    110
    frequency threshold
    5
    collection period
    The initial data reported was for the study up to Week 32. This time frame defines the comparative phase of the study where the majority of participants remained on their original randomized assignment and exposure between ruxolitinib and best available therapy was similar. Data inclusive of end of study was reported up to week 256 for Ruxolitinib group. Long term Follow Up for BAT was not reported as all patients have completed the study or crossed over to Ruxolitinib group by week 80.
    event definition
    Safety population: All participants who were enrolled and took at least 1 dose of study medication. The initial data reported at Week 32 was based on MedDRA 15.1; the remaining data reported for end of study was based on MedDRA 20.1.
    undenominated note
    Source null
    curation status
    draft
    reviewed by
    Source null
    reviewed at
    Source null
    created at
    2026-09-10T00:00:00Z
quality of life
    setting source links

      Preserved source evidence · Not medical advice