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NCT00434161 · OUTCOME

Maximum Severity of Oral Mucositis (World Health Organization (WHO) Grades 0/1, 2, 3, or 4)

A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Multiple Myeloma · Source last updated 2015-03-30

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

Measure
NUMBER
Unit
Participants
Interval / dispersion
Not reported
Time frame
at Day 32

What was measured

For the primary efficacy endpoint maximum severity of Oral Mucositis (OM) was assessed, the number of participants who had the different severity. To assess severity of OM, a 5-grade WHO scale (0, 1, 2, 3, or 4) was used. 0 = no findings or erythema only, 1= soreness present with or without erythema, 2=ulcers present but able to take solid food, 3 =ulcers present and only able to take liquids, 4 =ulcers present/not able to take anything orally.

Analysis population: Full analysis set that includes all randomized subjects, and was used to compare treatment effects for all efficacy endpoints. This set of subjects was analyzed according to their randomized treatment assignment.

Groups in this outcome

Palifermin Before Only

Subjects to receive palifermin before-high dose chemotherapy and matched placebo after-high dose chemotherapy

Placebo

Subjects to receive matched placebo before- and after-high dose chemotherapy

Palifermin Before and After

Subjects to receive palifermin before- and after-high dose chemotherapy

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

Analysis denominator · Participants

GroupSource count
Palifermin Before Only109
Placebo57
Palifermin Before and After115

Reported measurements

WHO grade 0/1 or 2
Values in Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Palifermin Before Only79Not reportedNot reportedNot reportedNot reported
Placebo36Not reportedNot reportedNot reportedNot reported
Palifermin Before and After68Not reportedNot reportedNot reportedNot reported
WHO grade 3 or 4
Values in Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Palifermin Before Only26Not reportedNot reportedNot reportedNot reported
Placebo21Not reportedNot reportedNot reportedNot reported
Palifermin Before and After44Not reportedNot reportedNot reportedNot reported
Unknown
Values in Participants · Interval/dispersion: Not reported
GroupValueSpreadLowerUpperComment
Palifermin Before Only4Not reportedNot reportedNot reportedNot reported
Placebo0Not reportedNot reportedNot reportedNot reported
Palifermin Before and After3Not reportedNot reportedNot reportedNot reported

Source statistical analyses

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

  1. ci Lower Limit
    0.351
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    97.5
    ci Upper Limit
    1.313
    estimate Comment
    The odds ratio was defined to be the odds of a subject receiving placebo experiencing OM divided by the odds of a subject receiving palifermin developing OM.
    group Description
    The null hypothesis was that the severity distribution for OM was identical for the placebo and each of the two palifermin groups, and the alternative hypothesis was that palifermin resulted in a shift in the distribution to less severe mucositis than placebo. A total of 275 subjects would give at least 95% power, with a 2.5% type I error rate for each comparison to detect an odds ratio of at least 3.5 between the placebo group and each of the two palifermin groups.
    group Ids
    1. OG001
    2. OG002
    non Inferiority Type
    SUPERIORITY_OR_OTHER
    p Value
    0.188
    p Value Comment
    A 2.5% type I error rate for each comparison gives an overall type I error rate of 5%.
    param Type
    Odds Ratio (OR)
    param Value
    0.679
    statistical Comment
    The proportional odds model including the randomization factors as covariates was used for the primary endpoint, maximum severity of OM.
    statistical Method
    Proportional odds model
    tested Non Inferiority
    false
  2. ci Lower Limit
    0.635
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    97.5
    ci Upper Limit
    2.431
    estimate Comment
    The odds ratio was defined to be the odds of a subject receiving placebo experiencing OM divided by the odds of a subject receiving palifermin developing OM.
    group Description
    The null hypothesis was that the severity distribution for OM was identical for the placebo and each of the two palifermin groups, and the alternative hypothesis was that palifermin resulted in a shift in the distribution to less severe mucositis than placebo. A total of 275 subjects would give at least 95% power, with a 2.5% type I error rate for each comparison to detect an odds ratio of at least 3.5 between the placebo group and each of the two palifermin groups.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY_OR_OTHER
    p Value
    0.468
    p Value Comment
    A 2.5% type I error rate for each comparison gives an overall type I error rate of 5%.
    param Type
    Odds Ratio (OR)
    param Value
    1.242
    statistical Comment
    The proportional odds model including the randomization factors as covariates was used for the primary endpoint, maximum severity of OM.
    statistical Method
    Proportional odds model
    tested Non Inferiority
    false
Complete source fields
analyses
  1. ci Lower Limit
    0.351
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    97.5
    ci Upper Limit
    1.313
    estimate Comment
    The odds ratio was defined to be the odds of a subject receiving placebo experiencing OM divided by the odds of a subject receiving palifermin developing OM.
    group Description
    The null hypothesis was that the severity distribution for OM was identical for the placebo and each of the two palifermin groups, and the alternative hypothesis was that palifermin resulted in a shift in the distribution to less severe mucositis than placebo. A total of 275 subjects would give at least 95% power, with a 2.5% type I error rate for each comparison to detect an odds ratio of at least 3.5 between the placebo group and each of the two palifermin groups.
    group Ids
    1. OG001
    2. OG002
    non Inferiority Type
    SUPERIORITY_OR_OTHER
    p Value
    0.188
    p Value Comment
    A 2.5% type I error rate for each comparison gives an overall type I error rate of 5%.
    param Type
    Odds Ratio (OR)
    param Value
    0.679
    statistical Comment
    The proportional odds model including the randomization factors as covariates was used for the primary endpoint, maximum severity of OM.
    statistical Method
    Proportional odds model
    tested Non Inferiority
    false
  2. ci Lower Limit
    0.635
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    97.5
    ci Upper Limit
    2.431
    estimate Comment
    The odds ratio was defined to be the odds of a subject receiving placebo experiencing OM divided by the odds of a subject receiving palifermin developing OM.
    group Description
    The null hypothesis was that the severity distribution for OM was identical for the placebo and each of the two palifermin groups, and the alternative hypothesis was that palifermin resulted in a shift in the distribution to less severe mucositis than placebo. A total of 275 subjects would give at least 95% power, with a 2.5% type I error rate for each comparison to detect an odds ratio of at least 3.5 between the placebo group and each of the two palifermin groups.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY_OR_OTHER
    p Value
    0.468
    p Value Comment
    A 2.5% type I error rate for each comparison gives an overall type I error rate of 5%.
    param Type
    Odds Ratio (OR)
    param Value
    1.242
    statistical Comment
    The proportional odds model including the randomization factors as covariates was used for the primary endpoint, maximum severity of OM.
    statistical Method
    Proportional odds model
    tested Non Inferiority
    false
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        value
        79
      2. group Id
        OG001
        value
        36
      3. group Id
        OG002
        value
        68
    title
    WHO grade 0/1 or 2
  2. categories
    1. measurements
      1. group Id
        OG000
        value
        26
      2. group Id
        OG001
        value
        21
      3. group Id
        OG002
        value
        44
    title
    WHO grade 3 or 4
  3. categories
    1. measurements
      1. group Id
        OG000
        value
        4
      2. group Id
        OG001
        value
        0
      3. group Id
        OG002
        value
        3
    title
    Unknown
denoms
  1. counts
    1. group Id
      OG000
      value
      109
    2. group Id
      OG001
      value
      57
    3. group Id
      OG002
      value
      115
    units
    Participants
description
For the primary efficacy endpoint maximum severity of Oral Mucositis (OM) was assessed, the number of participants who had the different severity. To assess severity of OM, a 5-grade WHO scale (0, 1, 2, 3, or 4) was used. 0 = no findings or erythema only, 1= soreness present with or without erythema, 2=ulcers present but able to take solid food, 3 =ulcers present and only able to take liquids, 4 =ulcers present/not able to take anything orally.
groups
  1. description
    Subjects to receive palifermin before-high dose chemotherapy and matched placebo after-high dose chemotherapy
    id
    OG000
    title
    Palifermin Before Only
  2. description
    Subjects to receive matched placebo before- and after-high dose chemotherapy
    id
    OG001
    title
    Placebo
  3. description
    Subjects to receive palifermin before- and after-high dose chemotherapy
    id
    OG002
    title
    Palifermin Before and After
param Type
NUMBER
population Description
Full analysis set that includes all randomized subjects, and was used to compare treatment effects for all efficacy endpoints. This set of subjects was analyzed according to their randomized treatment assignment.
reporting Status
POSTED
time Frame
at Day 32
title
Maximum Severity of Oral Mucositis (World Health Organization (WHO) Grades 0/1, 2, 3, or 4)
type
PRIMARY
unit Of Measure
Participants

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Preserved source evidence · Not medical advice