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NCT01524978 · OUTCOME

Overall Response Rate (ORR)

A Study of Vemurafenib in Participants With BRAF V600 Mutation-Positive Cancers · Source last updated 2017-11-20

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure
NUMBER
Unit
percentage of participants
Interval / dispersion
95% Confidence Interval
Time frame
Up to approximately 3 years

What was measured

ORR: percentage of participants with an objective response (OR) (CR, PR, sCR or VGPR) on 2 occasions \>/= 4 weeks apart as assessed by the Investigator using RECIST v1.1. or IMWG criteria. RECIST v1.1: CR: disappearance of all target lesions; PR: \>/= 30% decrease in the sum of diameters of target lesions. IMWG: CR: negative immunofixation on serum and urine, disappearance of any soft tissue plasmacytomas and \</= 5% plasma cells in bone marrow; PR: \>/= 50% reduction of serum M-protein and reduction in 24-hour urinary M-protein by \>/= 90% or to \<200 mg per 24 hours. VGPR: serum and urine M-protein detectable by immunofixation but not on electrophoresis or \>/= 90% reduction in serum M-protein plus urine M-protein level \< 100 mg per 24 hour; sCR: CR plus normal FLC ratio and no clonal cells in bone marrow.

Analysis population: ITT population included all participants enrolled in the study irrespective of whether they had received study medication or not.

Groups in this outcome

Cohort 1: Non-Small Cell Lung Cancer (NSCLC) - Vemurafenib

Participants with NSCLC were treated with vemurafenib monotherapy.

Cohort 2: Ovarian Cancer - Vemurafenib

Participants with ovarian cancer were treated with vemurafenib monotherapy.

Cohort 3a: Colorectal Cancer - Vemurafenib

Participants with colorectal cancer were treated with vemurafenib monotherapy.

Cohort 3b: Colorectal Cancer - Vemurafenib + Cetuximab

Participants with colorectal cancer were treated with vemurafenib and cetuximab combination therapy.

Cohort 4: Cholangiocarcinoma - Vemurafenib

Participants with cholangiocarcinoma were treated with vemurafenib monotherapy.

Cohort 6: Multiple Myeloma - Vemurafenib

Participants with multiple myeloma were treated with vemurafenib monotherapy.

Cohort 7a: ECD/LCH - Vemurafenib

Participants with Erdheim-Chester disease (ECD) or Langerhans cell histiocytosis (LCH) were treated with vemurafenib monotherapy.

Cohort 7b: Anaplastic Thyroid Cancer - Vemurafenib

Participants with anaplastic thyroid cancer were treated with vemurafenib monotherapy.

Cohort 7c: Advanced Stage Astrocytoma - Vemurafenib

Participants with advanced stage astrocytoma were treated with vemurafenib monotherapy.

Cohort 7d: Early Stage Astrocytoma - Vemurafenib

Participants with early stage astrocytoma were treated with vemurafenib monotherapy.

Cohort 7: Other BRAF V600-positive Tumors - Vemurafenib

Participants with other BRAF V600-positive tumors were treated with vemurafenib monotherapy.

Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.

Analysis denominator · Participants

GroupSource count
Cohort 1: Non-Small Cell Lung Cancer (NSCLC) - Vemurafenib62
Cohort 2: Ovarian Cancer - Vemurafenib4
Cohort 3a: Colorectal Cancer - Vemurafenib10
Cohort 3b: Colorectal Cancer - Vemurafenib + Cetuximab27
Cohort 4: Cholangiocarcinoma - Vemurafenib9
Cohort 6: Multiple Myeloma - Vemurafenib9
Cohort 7a: ECD/LCH - Vemurafenib26
Cohort 7b: Anaplastic Thyroid Cancer - Vemurafenib12
Cohort 7c: Advanced Stage Astrocytoma - Vemurafenib12
Cohort 7d: Early Stage Astrocytoma - Vemurafenib9
Cohort 7: Other BRAF V600-positive Tumors - Vemurafenib28

Reported measurements

CR
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Cohort 1: Non-Small Cell Lung Cancer (NSCLC) - Vemurafenib0Not reported0.005.78Not reported
Cohort 2: Ovarian Cancer - Vemurafenib0Not reported0.0060.24Not reported
Cohort 3a: Colorectal Cancer - Vemurafenib0Not reported0.0030.85Not reported
Cohort 3b: Colorectal Cancer - Vemurafenib + Cetuximab0Not reported0.0012.77Not reported
Cohort 4: Cholangiocarcinoma - Vemurafenib0Not reported0.0033.63Not reported
Cohort 6: Multiple Myeloma - Vemurafenib0Not reported0.0033.63Not reported
Cohort 7a: ECD/LCH - Vemurafenib7.7Not reported0.9525.13Not reported
Cohort 7b: Anaplastic Thyroid Cancer - Vemurafenib8.3Not reported0.2138.48Not reported
Cohort 7c: Advanced Stage Astrocytoma - Vemurafenib8.3Not reported0.2138.48Not reported
Cohort 7d: Early Stage Astrocytoma - Vemurafenib0Not reported0.0033.63Not reported
Cohort 7: Other BRAF V600-positive Tumors - Vemurafenib3.6Not reported0.0918.35Not reported
PR
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Cohort 1: Non-Small Cell Lung Cancer (NSCLC) - Vemurafenib37.1Not reported25.1650.31Not reported
Cohort 2: Ovarian Cancer - Vemurafenib50.0Not reported6.7693.24Not reported
Cohort 3a: Colorectal Cancer - Vemurafenib0Not reported0.0030.85Not reported
Cohort 3b: Colorectal Cancer - Vemurafenib + Cetuximab7.4Not reported0.9124.29Not reported
Cohort 4: Cholangiocarcinoma - Vemurafenib22.2Not reported2.8160.01Not reported
Cohort 6: Multiple Myeloma - Vemurafenib11.1Not reported0.2848.25Not reported
Cohort 7a: ECD/LCH - Vemurafenib53.8Not reported33.3773.41Not reported
Cohort 7b: Anaplastic Thyroid Cancer - Vemurafenib16.7Not reported2.0948.41Not reported
Cohort 7c: Advanced Stage Astrocytoma - Vemurafenib8.3Not reported0.2138.48Not reported
Cohort 7d: Early Stage Astrocytoma - Vemurafenib33.3Not reported7.4970.07Not reported
Cohort 7: Other BRAF V600-positive Tumors - Vemurafenib14.3Not reported4.0332.67Not reported
VGPR
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Cohort 1: Non-Small Cell Lung Cancer (NSCLC) - VemurafenibNANot reportedNANAN/A= VGPR data only collected in multiple myeloma cohort
Cohort 2: Ovarian Cancer - VemurafenibNANot reportedNANAN/A= VGPR data only collected in multiple myeloma cohort
Cohort 3a: Colorectal Cancer - VemurafenibNANot reportedNANAN/A= VGPR data only collected in multiple myeloma cohort
Cohort 3b: Colorectal Cancer - Vemurafenib + CetuximabNANot reportedNANAN/A= VGPR data only collected in multiple myeloma cohort
Cohort 4: Cholangiocarcinoma - VemurafenibNANot reportedNANAN/A= VGPR data only collected in multiple myeloma cohort
Cohort 6: Multiple Myeloma - Vemurafenib11.1Not reported0.2848.25Not reported
Cohort 7a: ECD/LCH - VemurafenibNANot reportedNANAN/A= VGPR data only collected in multiple myeloma cohort
Cohort 7b: Anaplastic Thyroid Cancer - VemurafenibNANot reportedNANAN/A= VGPR data only collected in multiple myeloma cohort
Cohort 7c: Advanced Stage Astrocytoma - VemurafenibNANot reportedNANAN/A= VGPR data only collected in multiple myeloma cohort
Cohort 7d: Early Stage Astrocytoma - VemurafenibNANot reportedNANAN/A= VGPR data only collected in multiple myeloma cohort
Cohort 7: Other BRAF V600-positive Tumors - VemurafenibNANot reportedNANAN/A= VGPR data only collected in multiple myeloma cohort
sCR
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Cohort 1: Non-Small Cell Lung Cancer (NSCLC) - VemurafenibNANot reportedNANAN/A= sCR data only collected in multiple myeloma cohort
Cohort 2: Ovarian Cancer - VemurafenibNANot reportedNANAN/A= sCR data only collected in multiple myeloma cohort
Cohort 3a: Colorectal Cancer - VemurafenibNANot reportedNANAN/A= sCR data only collected in multiple myeloma cohort
Cohort 3b: Colorectal Cancer - Vemurafenib + CetuximabNANot reportedNANAN/A= sCR data only collected in multiple myeloma cohort
Cohort 4: Cholangiocarcinoma - VemurafenibNANot reportedNANAN/A= sCR data only collected in multiple myeloma cohort
Cohort 6: Multiple Myeloma - Vemurafenib0Not reported0.0033.63Not reported
Cohort 7a: ECD/LCH - VemurafenibNANot reportedNANAN/A= sCR data only collected in multiple myeloma cohort
Cohort 7b: Anaplastic Thyroid Cancer - VemurafenibNANot reportedNANAN/A= sCR data only collected in multiple myeloma cohort
Cohort 7c: Advanced Stage Astrocytoma - VemurafenibNANot reportedNANAN/A= sCR data only collected in multiple myeloma cohort
Cohort 7d: Early Stage Astrocytoma - VemurafenibNANot reportedNANAN/A= sCR data only collected in multiple myeloma cohort
Cohort 7: Other BRAF V600-positive Tumors - VemurafenibNANot reportedNANAN/A= sCR data only collected in multiple myeloma cohort
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        0.00
        upper Limit
        5.78
        value
        0
      2. group Id
        OG001
        lower Limit
        0.00
        upper Limit
        60.24
        value
        0
      3. group Id
        OG002
        lower Limit
        0.00
        upper Limit
        30.85
        value
        0
      4. group Id
        OG003
        lower Limit
        0.00
        upper Limit
        12.77
        value
        0
      5. group Id
        OG004
        lower Limit
        0.00
        upper Limit
        33.63
        value
        0
      6. group Id
        OG005
        lower Limit
        0.00
        upper Limit
        33.63
        value
        0
      7. group Id
        OG006
        lower Limit
        0.95
        upper Limit
        25.13
        value
        7.7
      8. group Id
        OG007
        lower Limit
        0.21
        upper Limit
        38.48
        value
        8.3
      9. group Id
        OG008
        lower Limit
        0.21
        upper Limit
        38.48
        value
        8.3
      10. group Id
        OG009
        lower Limit
        0.00
        upper Limit
        33.63
        value
        0
      11. group Id
        OG010
        lower Limit
        0.09
        upper Limit
        18.35
        value
        3.6
    title
    CR
  2. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        25.16
        upper Limit
        50.31
        value
        37.1
      2. group Id
        OG001
        lower Limit
        6.76
        upper Limit
        93.24
        value
        50.0
      3. group Id
        OG002
        lower Limit
        0.00
        upper Limit
        30.85
        value
        0
      4. group Id
        OG003
        lower Limit
        0.91
        upper Limit
        24.29
        value
        7.4
      5. group Id
        OG004
        lower Limit
        2.81
        upper Limit
        60.01
        value
        22.2
      6. group Id
        OG005
        lower Limit
        0.28
        upper Limit
        48.25
        value
        11.1
      7. group Id
        OG006
        lower Limit
        33.37
        upper Limit
        73.41
        value
        53.8
      8. group Id
        OG007
        lower Limit
        2.09
        upper Limit
        48.41
        value
        16.7
      9. group Id
        OG008
        lower Limit
        0.21
        upper Limit
        38.48
        value
        8.3
      10. group Id
        OG009
        lower Limit
        7.49
        upper Limit
        70.07
        value
        33.3
      11. group Id
        OG010
        lower Limit
        4.03
        upper Limit
        32.67
        value
        14.3
    title
    PR
  3. categories
    1. measurements
      1. comment
        N/A= VGPR data only collected in multiple myeloma cohort
        group Id
        OG000
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      2. comment
        N/A= VGPR data only collected in multiple myeloma cohort
        group Id
        OG001
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      3. comment
        N/A= VGPR data only collected in multiple myeloma cohort
        group Id
        OG002
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      4. comment
        N/A= VGPR data only collected in multiple myeloma cohort
        group Id
        OG003
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      5. comment
        N/A= VGPR data only collected in multiple myeloma cohort
        group Id
        OG004
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      6. group Id
        OG005
        lower Limit
        0.28
        upper Limit
        48.25
        value
        11.1
      7. comment
        N/A= VGPR data only collected in multiple myeloma cohort
        group Id
        OG006
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      8. comment
        N/A= VGPR data only collected in multiple myeloma cohort
        group Id
        OG007
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      9. comment
        N/A= VGPR data only collected in multiple myeloma cohort
        group Id
        OG008
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      10. comment
        N/A= VGPR data only collected in multiple myeloma cohort
        group Id
        OG009
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      11. comment
        N/A= VGPR data only collected in multiple myeloma cohort
        group Id
        OG010
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
    title
    VGPR
  4. categories
    1. measurements
      1. comment
        N/A= sCR data only collected in multiple myeloma cohort
        group Id
        OG000
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      2. comment
        N/A= sCR data only collected in multiple myeloma cohort
        group Id
        OG001
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      3. comment
        N/A= sCR data only collected in multiple myeloma cohort
        group Id
        OG002
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      4. comment
        N/A= sCR data only collected in multiple myeloma cohort
        group Id
        OG003
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      5. comment
        N/A= sCR data only collected in multiple myeloma cohort
        group Id
        OG004
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      6. group Id
        OG005
        lower Limit
        0.00
        upper Limit
        33.63
        value
        0
      7. comment
        N/A= sCR data only collected in multiple myeloma cohort
        group Id
        OG006
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      8. comment
        N/A= sCR data only collected in multiple myeloma cohort
        group Id
        OG007
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      9. comment
        N/A= sCR data only collected in multiple myeloma cohort
        group Id
        OG008
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      10. comment
        N/A= sCR data only collected in multiple myeloma cohort
        group Id
        OG009
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      11. comment
        N/A= sCR data only collected in multiple myeloma cohort
        group Id
        OG010
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
    title
    sCR
denoms
  1. counts
    1. group Id
      OG000
      value
      62
    2. group Id
      OG001
      value
      4
    3. group Id
      OG002
      value
      10
    4. group Id
      OG003
      value
      27
    5. group Id
      OG004
      value
      9
    6. group Id
      OG005
      value
      9
    7. group Id
      OG006
      value
      26
    8. group Id
      OG007
      value
      12
    9. group Id
      OG008
      value
      12
    10. group Id
      OG009
      value
      9
    11. group Id
      OG010
      value
      28
    units
    Participants
description
ORR: percentage of participants with an objective response (OR) (CR, PR, sCR or VGPR) on 2 occasions \>/= 4 weeks apart as assessed by the Investigator using RECIST v1.1. or IMWG criteria. RECIST v1.1: CR: disappearance of all target lesions; PR: \>/= 30% decrease in the sum of diameters of target lesions. IMWG: CR: negative immunofixation on serum and urine, disappearance of any soft tissue plasmacytomas and \</= 5% plasma cells in bone marrow; PR: \>/= 50% reduction of serum M-protein and reduction in 24-hour urinary M-protein by \>/= 90% or to \<200 mg per 24 hours. VGPR: serum and urine M-protein detectable by immunofixation but not on electrophoresis or \>/= 90% reduction in serum M-protein plus urine M-protein level \< 100 mg per 24 hour; sCR: CR plus normal FLC ratio and no clonal cells in bone marrow.
dispersion Type
95% Confidence Interval
groups
  1. description
    Participants with NSCLC were treated with vemurafenib monotherapy.
    id
    OG000
    title
    Cohort 1: Non-Small Cell Lung Cancer (NSCLC) - Vemurafenib
  2. description
    Participants with ovarian cancer were treated with vemurafenib monotherapy.
    id
    OG001
    title
    Cohort 2: Ovarian Cancer - Vemurafenib
  3. description
    Participants with colorectal cancer were treated with vemurafenib monotherapy.
    id
    OG002
    title
    Cohort 3a: Colorectal Cancer - Vemurafenib
  4. description
    Participants with colorectal cancer were treated with vemurafenib and cetuximab combination therapy.
    id
    OG003
    title
    Cohort 3b: Colorectal Cancer - Vemurafenib + Cetuximab
  5. description
    Participants with cholangiocarcinoma were treated with vemurafenib monotherapy.
    id
    OG004
    title
    Cohort 4: Cholangiocarcinoma - Vemurafenib
  6. description
    Participants with multiple myeloma were treated with vemurafenib monotherapy.
    id
    OG005
    title
    Cohort 6: Multiple Myeloma - Vemurafenib
  7. description
    Participants with Erdheim-Chester disease (ECD) or Langerhans cell histiocytosis (LCH) were treated with vemurafenib monotherapy.
    id
    OG006
    title
    Cohort 7a: ECD/LCH - Vemurafenib
  8. description
    Participants with anaplastic thyroid cancer were treated with vemurafenib monotherapy.
    id
    OG007
    title
    Cohort 7b: Anaplastic Thyroid Cancer - Vemurafenib
  9. description
    Participants with advanced stage astrocytoma were treated with vemurafenib monotherapy.
    id
    OG008
    title
    Cohort 7c: Advanced Stage Astrocytoma - Vemurafenib
  10. description
    Participants with early stage astrocytoma were treated with vemurafenib monotherapy.
    id
    OG009
    title
    Cohort 7d: Early Stage Astrocytoma - Vemurafenib
  11. description
    Participants with other BRAF V600-positive tumors were treated with vemurafenib monotherapy.
    id
    OG010
    title
    Cohort 7: Other BRAF V600-positive Tumors - Vemurafenib
param Type
NUMBER
population Description
ITT population included all participants enrolled in the study irrespective of whether they had received study medication or not.
reporting Status
POSTED
time Frame
Up to approximately 3 years
title
Overall Response Rate (ORR)
type
SECONDARY
unit Of Measure
percentage of participants

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Preserved source evidence · Not medical advice