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NCT02065557 · OUTCOME

Ranked Secondary Endpoint 3: Percentage of Participants With Clinical Remission Per FMS at Week 52 in Week 8 Remitters Per PMS - Maintenance Period

Efficacy and Safety of Adalimumab in Pediatric Subjects With Moderate to Severe Ulcerative Colitis · Source last updated 2020-10-05

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
Week 52

What was measured

The FMS ranges from 0 (normal or inactive disease) to 12 (severe disease) and is calculated as the sum of 4 subscores (stool frequency, rectal bleeding, endoscopy, and physician's global assessment), each of which ranges from 0 (normal) to 3 (severe disease). The PMS (Mayo score without endoscopy) ranges from 0 (normal or inactive disease) to 9 (severe disease) and is calculated as the sum of 3 subscores (stool frequency, rectal bleeding and physician's global assessment). Negative changes indicate improvement. PMS remitters are defined as those participants with a PMS ≤ 2 and no individual subscore \> 1. Clinical remission per FMS is defined as Mayo Score ≤ 2 and no individual subscore \> 1.

Analysis population: Modified intent-to-treat (mITT): all Week 8 PMS responders who were re-randomized at Week 8 and received at least 1 dose of study drug during the Maintenance period; analyzed as randomized at the beginning of the Maintenance phase. Participants who were also Week 8 remitters. NRI: missing data is imputed as not having met the endpoint.

Groups in this outcome

Main Study: M-PL

(Prior to Amendment 4) participants demonstrating a clinical response per PMS (defined as a decrease in PMS ≥ 2 points and ≥ 30% from Baseline) at Week 8 randomized to maintenance placebo. Participants were to continue their blinded treatment during the maintenance period until Week 52 unless they had ≥ 2 flares and got open label adalimumab rescue therapy after the second flare.

Main Study: M-SD

Participants demonstrating a clinical response per PMS (defined as a decrease in PMS ≥ 2 points and ≥ 30% from Baseline) at Week 8 randomized to adalimumab maintenance standard dose (0.6 mg/kg \[maximum dose of 40 mg\] eow). Participants were to continue their blinded treatment during the maintenance period until Week 52 unless they had ≥ 2 flares and got open label rescue therapy after the second flare.

Main Study: M-HD

Participants demonstrating a clinical response per PMS (defined as a decrease in PMS ≥ 2 points and ≥ 30% from Baseline) at Week 8 randomized to adalimumab maintenance high dose (0.6 mg/kg \[maximum dose of 40 mg\] ew). Participants were to continue their blinded treatment during the maintenance period until Week 52 unless they had ≥ 2 flares and got open label rescue therapy after the second flare.

Main Study: M-SD + M-HD

Combined M-SD + M-HD arms (see above).

Integrated Study (Main + Japan Sub-Study): M-PL

(Prior to Amendment 4) participants demonstrating a clinical response per PMS (defined as a decrease in PMS ≥ 2 points and ≥ 30% from Baseline) at Week 8 randomized to maintenance placebo. Participants were to continue their blinded treatment during the maintenance period until Week 52 unless they had ≥ 2 flares and got open label adalimumab rescue therapy after the second flare.

Integrated Study (Main + Japan Sub-Study): M-SD

Participants demonstrating a clinical response per PMS (defined as a decrease in PMS ≥ 2 points and ≥ 30% from Baseline) at Week 8 randomized to adalimumab maintenance standard dose (0.6 mg/kg \[maximum dose of 40 mg\] eow). Participants were to continue their blinded treatment during the maintenance period until Week 52 unless they had ≥ 2 flares and got open label rescue therapy after the second flare.

Integrated Study (Main + Japan Sub-Study): M-HD

Participants demonstrating a clinical response per PMS (defined as a decrease in PMS ≥ 2 points and ≥ 30% from Baseline) at Week 8 randomized to adalimumab maintenance high dose (0.6 mg/kg \[maximum dose of 40 mg\] ew). Participants were to continue their blinded treatment during the maintenance period until Week 52 unless they had ≥ 2 flares and got open label rescue therapy after the second flare.

Integrated Study (Main + Japan Sub-Study): M-SD + M-HD

Combined M-SD + M-HD arms (see above).

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

Analysis denominator · Participants

GroupSource count
Main Study: M-PL8
Main Study: M-SD21
Main Study: M-HD22
Main Study: M-SD + M-HD43
Integrated Study (Main + Japan Sub-Study): M-PL8
Integrated Study (Main + Japan Sub-Study): M-SD21
Integrated Study (Main + Japan Sub-Study): M-HD25
Integrated Study (Main + Japan Sub-Study): M-SD + M-HD46

Reported measurements

Source class 1
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Main Study: M-PL37.5Not reported8.5275.51Not reported
Main Study: M-SD42.9Not reported21.8265.98Not reported
Main Study: M-HD45.5Not reported24.3967.79Not reported
Main Study: M-SD + M-HD44.2Not reported29.0860.12Not reported
Integrated Study (Main + Japan Sub-Study): M-PL37.5Not reported8.5275.51Not reported
Integrated Study (Main + Japan Sub-Study): M-SD42.9Not reported21.8265.98Not reported
Integrated Study (Main + Japan Sub-Study): M-HD44.0Not reported24.4065.07Not reported
Integrated Study (Main + Japan Sub-Study): M-SD + M-HD43.5Not reported28.9358.89Not reported

Source statistical analyses

Group order, estimate direction, methods and comments are retained. No treatment ranking is inferred.

  1. group Description
    Efficacy analysis was based on the modified Intent-To-Treat-Efficacy (mITT-E) population for the maintenance period. The mITT-E population was a subpopulation of the mITT population, where participants in M-PL arm were excluded.
    group Ids
    1. OG003
    non Inferiority Comment
    one-sample two-sided Chi-square test
    non Inferiority Type
    SUPERIORITY
    p Value
    < 0.001
    p Value Comment
    Adjusted p-value from a sequentially rejective multiple test procedure, testing co-primary and ranked secondary endpoints 1st for M-SD+M-HD, then for individual adalimumab dose groups (M-HD, M-SD) against the respective external placebo rate (14.79%)
    statistical Comment
    controlling familywise Type I error of 5% in a strong sense
    statistical Method
    rejective multiple test procedure
  2. group Description
    Efficacy analysis was based on the modified Intent-To-Treat-Efficacy (mITT-E) population for the maintenance period. The mITT-E population was a subpopulation of the mITT population, where participants in M-PL arm were excluded.
    group Ids
    1. OG002
    non Inferiority Comment
    one-sample two-sided Chi-square test
    non Inferiority Type
    SUPERIORITY
    p Value
    < 0.001
    p Value Comment
    Adjusted p-value from a sequentially rejective multiple test procedure, testing co-primary and ranked secondary endpoints 1st for M-SD+M-HD, then for individual adalimumab dose groups (M-HD, M-SD) against the respective external placebo rate (14.79%)
    statistical Comment
    controlling familywise Type I error of 5% in a strong sense
    statistical Method
    rejective multiple test procedure
  3. group Description
    Efficacy analysis was based on the modified Intent-To-Treat-Efficacy (mITT-E) population for the maintenance period. The mITT-E population was a subpopulation of the mITT population, where participants in M-PL arm were excluded.
    group Ids
    1. OG001
    non Inferiority Comment
    one-sample two-sided Chi-square test
    non Inferiority Type
    SUPERIORITY
    p Value
    0.292
    p Value Comment
    Adjusted p-value from a sequentially rejective multiple test procedure, testing co-primary and ranked secondary endpoints 1st for M-SD+M-HD, then for individual adalimumab dose groups (M-HD, M-SD) against the respective external placebo rate (14.79%)
    statistical Comment
    controlling familywise Type I error of 5% in a strong sense
    statistical Method
    rejective multiple test procedure
  4. group Description
    Efficacy analysis was based on the modified Intent-To-Treat-Efficacy (mITT-E) population for the maintenance period. The mITT-E population was a subpopulation of the mITT population, where participants in M-PL arm were excluded.
    group Ids
    1. OG007
    non Inferiority Comment
    one-sample two-sided Chi-square test
    non Inferiority Type
    SUPERIORITY
    p Value
    < 0.001
    p Value Comment
    Adjusted p-value from a sequentially rejective multiple test procedure, testing co-primary and ranked secondary endpoints 1st for M-SD+M-HD, then for individual adalimumab dose groups (M-HD, M-SD) against the respective external placebo rate (14.79%)
    statistical Comment
    controlling familywise Type I error of 5% in a strong sense
    statistical Method
    rejective multiple test procedure
  5. group Description
    Efficacy analysis was based on the modified Intent-To-Treat-Efficacy (mITT-E) population for the maintenance period. The mITT-E population was a subpopulation of the mITT population, where participants in M-PL arm were excluded.
    group Ids
    1. OG006
    non Inferiority Comment
    one-sample two-sided Chi-square test
    non Inferiority Type
    SUPERIORITY
    p Value
    < 0.001
    p Value Comment
    Adjusted p-value from a sequentially rejective multiple test procedure, testing co-primary and ranked secondary endpoints 1st for M-SD+M-HD, then for individual adalimumab dose groups (M-HD, M-SD) against the respective external placebo rate (14.79%)
    statistical Comment
    controlling familywise Type I error of 5% in a strong sense
    statistical Method
    rejective multiple test procedure
  6. group Description
    Efficacy analysis was based on the modified Intent-To-Treat-Efficacy (mITT-E) population for the maintenance period. The mITT-E population was a subpopulation of the mITT population, where participants in M-PL arm were excluded.
    group Ids
    1. OG005
    non Inferiority Comment
    one-sample two-sided Chi-square test
    non Inferiority Type
    SUPERIORITY
    p Value
    0.292
    p Value Comment
    Adjusted p-value from a sequentially rejective multiple test procedure, testing co-primary and ranked secondary endpoints 1st for M-SD+M-HD, then for individual adalimumab dose groups (M-HD, M-SD) against the respective external placebo rate (14.79%)
    statistical Comment
    controlling familywise Type I error of 5% in a strong sense
    statistical Method
    rejective multiple test procedure
Complete source fields
analyses
  1. group Description
    Efficacy analysis was based on the modified Intent-To-Treat-Efficacy (mITT-E) population for the maintenance period. The mITT-E population was a subpopulation of the mITT population, where participants in M-PL arm were excluded.
    group Ids
    1. OG003
    non Inferiority Comment
    one-sample two-sided Chi-square test
    non Inferiority Type
    SUPERIORITY
    p Value
    < 0.001
    p Value Comment
    Adjusted p-value from a sequentially rejective multiple test procedure, testing co-primary and ranked secondary endpoints 1st for M-SD+M-HD, then for individual adalimumab dose groups (M-HD, M-SD) against the respective external placebo rate (14.79%)
    statistical Comment
    controlling familywise Type I error of 5% in a strong sense
    statistical Method
    rejective multiple test procedure
  2. group Description
    Efficacy analysis was based on the modified Intent-To-Treat-Efficacy (mITT-E) population for the maintenance period. The mITT-E population was a subpopulation of the mITT population, where participants in M-PL arm were excluded.
    group Ids
    1. OG002
    non Inferiority Comment
    one-sample two-sided Chi-square test
    non Inferiority Type
    SUPERIORITY
    p Value
    < 0.001
    p Value Comment
    Adjusted p-value from a sequentially rejective multiple test procedure, testing co-primary and ranked secondary endpoints 1st for M-SD+M-HD, then for individual adalimumab dose groups (M-HD, M-SD) against the respective external placebo rate (14.79%)
    statistical Comment
    controlling familywise Type I error of 5% in a strong sense
    statistical Method
    rejective multiple test procedure
  3. group Description
    Efficacy analysis was based on the modified Intent-To-Treat-Efficacy (mITT-E) population for the maintenance period. The mITT-E population was a subpopulation of the mITT population, where participants in M-PL arm were excluded.
    group Ids
    1. OG001
    non Inferiority Comment
    one-sample two-sided Chi-square test
    non Inferiority Type
    SUPERIORITY
    p Value
    0.292
    p Value Comment
    Adjusted p-value from a sequentially rejective multiple test procedure, testing co-primary and ranked secondary endpoints 1st for M-SD+M-HD, then for individual adalimumab dose groups (M-HD, M-SD) against the respective external placebo rate (14.79%)
    statistical Comment
    controlling familywise Type I error of 5% in a strong sense
    statistical Method
    rejective multiple test procedure
  4. group Description
    Efficacy analysis was based on the modified Intent-To-Treat-Efficacy (mITT-E) population for the maintenance period. The mITT-E population was a subpopulation of the mITT population, where participants in M-PL arm were excluded.
    group Ids
    1. OG007
    non Inferiority Comment
    one-sample two-sided Chi-square test
    non Inferiority Type
    SUPERIORITY
    p Value
    < 0.001
    p Value Comment
    Adjusted p-value from a sequentially rejective multiple test procedure, testing co-primary and ranked secondary endpoints 1st for M-SD+M-HD, then for individual adalimumab dose groups (M-HD, M-SD) against the respective external placebo rate (14.79%)
    statistical Comment
    controlling familywise Type I error of 5% in a strong sense
    statistical Method
    rejective multiple test procedure
  5. group Description
    Efficacy analysis was based on the modified Intent-To-Treat-Efficacy (mITT-E) population for the maintenance period. The mITT-E population was a subpopulation of the mITT population, where participants in M-PL arm were excluded.
    group Ids
    1. OG006
    non Inferiority Comment
    one-sample two-sided Chi-square test
    non Inferiority Type
    SUPERIORITY
    p Value
    < 0.001
    p Value Comment
    Adjusted p-value from a sequentially rejective multiple test procedure, testing co-primary and ranked secondary endpoints 1st for M-SD+M-HD, then for individual adalimumab dose groups (M-HD, M-SD) against the respective external placebo rate (14.79%)
    statistical Comment
    controlling familywise Type I error of 5% in a strong sense
    statistical Method
    rejective multiple test procedure
  6. group Description
    Efficacy analysis was based on the modified Intent-To-Treat-Efficacy (mITT-E) population for the maintenance period. The mITT-E population was a subpopulation of the mITT population, where participants in M-PL arm were excluded.
    group Ids
    1. OG005
    non Inferiority Comment
    one-sample two-sided Chi-square test
    non Inferiority Type
    SUPERIORITY
    p Value
    0.292
    p Value Comment
    Adjusted p-value from a sequentially rejective multiple test procedure, testing co-primary and ranked secondary endpoints 1st for M-SD+M-HD, then for individual adalimumab dose groups (M-HD, M-SD) against the respective external placebo rate (14.79%)
    statistical Comment
    controlling familywise Type I error of 5% in a strong sense
    statistical Method
    rejective multiple test procedure
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        8.52
        upper Limit
        75.51
        value
        37.5
      2. group Id
        OG001
        lower Limit
        21.82
        upper Limit
        65.98
        value
        42.9
      3. group Id
        OG002
        lower Limit
        24.39
        upper Limit
        67.79
        value
        45.5
      4. group Id
        OG003
        lower Limit
        29.08
        upper Limit
        60.12
        value
        44.2
      5. group Id
        OG004
        lower Limit
        8.52
        upper Limit
        75.51
        value
        37.5
      6. group Id
        OG005
        lower Limit
        21.82
        upper Limit
        65.98
        value
        42.9
      7. group Id
        OG006
        lower Limit
        24.40
        upper Limit
        65.07
        value
        44.0
      8. group Id
        OG007
        lower Limit
        28.93
        upper Limit
        58.89
        value
        43.5
denoms
  1. counts
    1. group Id
      OG000
      value
      8
    2. group Id
      OG001
      value
      21
    3. group Id
      OG002
      value
      22
    4. group Id
      OG003
      value
      43
    5. group Id
      OG004
      value
      8
    6. group Id
      OG005
      value
      21
    7. group Id
      OG006
      value
      25
    8. group Id
      OG007
      value
      46
    units
    Participants
description
The FMS ranges from 0 (normal or inactive disease) to 12 (severe disease) and is calculated as the sum of 4 subscores (stool frequency, rectal bleeding, endoscopy, and physician's global assessment), each of which ranges from 0 (normal) to 3 (severe disease). The PMS (Mayo score without endoscopy) ranges from 0 (normal or inactive disease) to 9 (severe disease) and is calculated as the sum of 3 subscores (stool frequency, rectal bleeding and physician's global assessment). Negative changes indicate improvement. PMS remitters are defined as those participants with a PMS ≤ 2 and no individual subscore \> 1. Clinical remission per FMS is defined as Mayo Score ≤ 2 and no individual subscore \> 1.
dispersion Type
95% Confidence Interval
groups
  1. description
    (Prior to Amendment 4) participants demonstrating a clinical response per PMS (defined as a decrease in PMS ≥ 2 points and ≥ 30% from Baseline) at Week 8 randomized to maintenance placebo. Participants were to continue their blinded treatment during the maintenance period until Week 52 unless they had ≥ 2 flares and got open label adalimumab rescue therapy after the second flare.
    id
    OG000
    title
    Main Study: M-PL
  2. description
    Participants demonstrating a clinical response per PMS (defined as a decrease in PMS ≥ 2 points and ≥ 30% from Baseline) at Week 8 randomized to adalimumab maintenance standard dose (0.6 mg/kg \[maximum dose of 40 mg\] eow). Participants were to continue their blinded treatment during the maintenance period until Week 52 unless they had ≥ 2 flares and got open label rescue therapy after the second flare.
    id
    OG001
    title
    Main Study: M-SD
  3. description
    Participants demonstrating a clinical response per PMS (defined as a decrease in PMS ≥ 2 points and ≥ 30% from Baseline) at Week 8 randomized to adalimumab maintenance high dose (0.6 mg/kg \[maximum dose of 40 mg\] ew). Participants were to continue their blinded treatment during the maintenance period until Week 52 unless they had ≥ 2 flares and got open label rescue therapy after the second flare.
    id
    OG002
    title
    Main Study: M-HD
  4. description
    Combined M-SD + M-HD arms (see above).
    id
    OG003
    title
    Main Study: M-SD + M-HD
  5. description
    (Prior to Amendment 4) participants demonstrating a clinical response per PMS (defined as a decrease in PMS ≥ 2 points and ≥ 30% from Baseline) at Week 8 randomized to maintenance placebo. Participants were to continue their blinded treatment during the maintenance period until Week 52 unless they had ≥ 2 flares and got open label adalimumab rescue therapy after the second flare.
    id
    OG004
    title
    Integrated Study (Main + Japan Sub-Study): M-PL
  6. description
    Participants demonstrating a clinical response per PMS (defined as a decrease in PMS ≥ 2 points and ≥ 30% from Baseline) at Week 8 randomized to adalimumab maintenance standard dose (0.6 mg/kg \[maximum dose of 40 mg\] eow). Participants were to continue their blinded treatment during the maintenance period until Week 52 unless they had ≥ 2 flares and got open label rescue therapy after the second flare.
    id
    OG005
    title
    Integrated Study (Main + Japan Sub-Study): M-SD
  7. description
    Participants demonstrating a clinical response per PMS (defined as a decrease in PMS ≥ 2 points and ≥ 30% from Baseline) at Week 8 randomized to adalimumab maintenance high dose (0.6 mg/kg \[maximum dose of 40 mg\] ew). Participants were to continue their blinded treatment during the maintenance period until Week 52 unless they had ≥ 2 flares and got open label rescue therapy after the second flare.
    id
    OG006
    title
    Integrated Study (Main + Japan Sub-Study): M-HD
  8. description
    Combined M-SD + M-HD arms (see above).
    id
    OG007
    title
    Integrated Study (Main + Japan Sub-Study): M-SD + M-HD
param Type
NUMBER
population Description
Modified intent-to-treat (mITT): all Week 8 PMS responders who were re-randomized at Week 8 and received at least 1 dose of study drug during the Maintenance period; analyzed as randomized at the beginning of the Maintenance phase. Participants who were also Week 8 remitters. NRI: missing data is imputed as not having met the endpoint.
reporting Status
POSTED
time Frame
Week 52
title
Ranked Secondary Endpoint 3: Percentage of Participants With Clinical Remission Per FMS at Week 52 in Week 8 Remitters Per PMS - Maintenance Period
type
SECONDARY
unit Of Measure
percentage of participants

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Preserved source evidence · Not medical advice