Skip to content
Triangle logo TriangleGet started
← Full study record

NCT03493854 · OUTCOME

Percentage of Participants With Total Pathological Complete Response (tpCR), According to Local Pathologist Assessment

A Study to Evaluate the Pharmacokinetics, Efficacy, and Safety of Subcutaneous Administration of the Fixed-Dose Combination of Pertuzumab and Trastuzumab in Combination With Chemotherapy in Participants With HER2-Positive Early Breast Cancer · Source last updated 2024-06-26

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
Following completion of surgery (up to 33 weeks)

What was measured

Total pCR (tpCR) was defined as eradication of invasive disease in the breast and axilla; that is, ypT0/is ypN0, according to the local pathologists' assessment. Pathologic response to therapy was determined at the time of surgery. The tpCR rate is the percentage of participants in the ITT population who achieved a tpCR. Participants with missing data for tpCR (i.e., do not undergo surgery or have an invalid pCR assessment) were included in the analysis and classified as non-responders. Rates of tpCR were calculated in each treatment arm and were assessed using the difference between the Arm B: Pertuzumab and Trastuzumab FDC SC and the Arm A: Pertuzumab IV and Trastuzumab IV tpCR rates and corresponding 95% Clopper-Pearson confidence intervals (CIs). The difference between the tpCR rates along with corresponding 95% Hauck-Anderson CIs were calculated. The lower bound of the CI will reliably reflect the largest tpCR difference that can be considered unlikely.

Analysis population: ITT Population: includes all enrolled participants.

Groups in this outcome

Arm A: Pertuzumab IV + Trastuzumab IV + Chemotherapy

Participants received 8 cycles of investigator's choice of neoadjuvant chemotherapy. This included either: 1) 4 cycles of dose-dense doxorubicin plus cyclophosphamide (ddAC) once every 2 weeks (Q2W) (given with granulocyte colony-stimulating factor \[G-CSF\] support as needed according to local guidelines) followed by paclitaxel QW for 12 weeks; or 2) 4 cycles of doxorubicin plus cyclophosphamide (AC) once every 3 weeks (Q3W) followed by docetaxel Q3W for 4 cycles. Pertuzumab and trastuzumab were given intravenously (IV) for 4 cycles Q3W concurrently with the taxane component of chemotherapy. After completing their neoadjuvant therapy, participants underwent surgery. Thereafter, participants received an additional 14 cycles of pertuzumab IV and trastuzumab IV for a total of 18 cycles.

Arm B: Pertuzumab and Trastuzumab FDC SC + Chemotherapy

Participants received 8 cycles of investigator's choice of neoadjuvant chemotherapy. This included either: 1) 4 cycles of ddAC Q2W (given with G-CSF support as needed according to local guidelines) followed by paclitaxel once every week (QW) for 12 weeks; or 2) 4 cycles of AC Q3W followed by docetaxel Q3W for 4 cycles. The fixed-dose combination (FDC) of pertuzumab and trastuzumab was given subcutaneously (SC) for 4 cycles (Q3W) concurrently with the taxane component of chemotherapy. After completing their neoadjuvant therapy, participants underwent surgery. Thereafter, participants received an additional 14 cycles of the FDC of pertuzumab and trastuzumab SC for a total of 18 cycles.

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

Analysis denominator · Participants

GroupSource count
Arm A: Pertuzumab IV + Trastuzumab IV + Chemotherapy252
Arm B: Pertuzumab and Trastuzumab FDC SC + Chemotherapy248

Reported measurements

Source class 1
Values in Percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Arm A: Pertuzumab IV + Trastuzumab IV + Chemotherapy59.5Not reported53.265.6Not reported
Arm B: Pertuzumab and Trastuzumab FDC SC + Chemotherapy59.7Not reported53.365.8Not reported

Source statistical analyses

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

  1. ci Lower Limit
    -8.67
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    8.97
    estimate Comment
    Difference in tpCR rate was calculated as Arm B: PH FDC SC minus Arm A: P+H IV.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Comment
    Descriptive analysis only. tpCR was analyzed outside of a hypothesis-testing framework and according to the methodology outlined in the outcome measure description.
    non Inferiority Type
    OTHER
    param Type
    Difference in tpCR Rate
    param Value
    0.15
Complete source fields
analyses
  1. ci Lower Limit
    -8.67
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    8.97
    estimate Comment
    Difference in tpCR rate was calculated as Arm B: PH FDC SC minus Arm A: P+H IV.
    group Ids
    1. OG000
    2. OG001
    non Inferiority Comment
    Descriptive analysis only. tpCR was analyzed outside of a hypothesis-testing framework and according to the methodology outlined in the outcome measure description.
    non Inferiority Type
    OTHER
    param Type
    Difference in tpCR Rate
    param Value
    0.15
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        53.2
        upper Limit
        65.6
        value
        59.5
      2. group Id
        OG001
        lower Limit
        53.3
        upper Limit
        65.8
        value
        59.7
denoms
  1. counts
    1. group Id
      OG000
      value
      252
    2. group Id
      OG001
      value
      248
    units
    Participants
description
Total pCR (tpCR) was defined as eradication of invasive disease in the breast and axilla; that is, ypT0/is ypN0, according to the local pathologists' assessment. Pathologic response to therapy was determined at the time of surgery. The tpCR rate is the percentage of participants in the ITT population who achieved a tpCR. Participants with missing data for tpCR (i.e., do not undergo surgery or have an invalid pCR assessment) were included in the analysis and classified as non-responders. Rates of tpCR were calculated in each treatment arm and were assessed using the difference between the Arm B: Pertuzumab and Trastuzumab FDC SC and the Arm A: Pertuzumab IV and Trastuzumab IV tpCR rates and corresponding 95% Clopper-Pearson confidence intervals (CIs). The difference between the tpCR rates along with corresponding 95% Hauck-Anderson CIs were calculated. The lower bound of the CI will reliably reflect the largest tpCR difference that can be considered unlikely.
dispersion Type
95% Confidence Interval
groups
  1. description
    Participants received 8 cycles of investigator's choice of neoadjuvant chemotherapy. This included either: 1) 4 cycles of dose-dense doxorubicin plus cyclophosphamide (ddAC) once every 2 weeks (Q2W) (given with granulocyte colony-stimulating factor \[G-CSF\] support as needed according to local guidelines) followed by paclitaxel QW for 12 weeks; or 2) 4 cycles of doxorubicin plus cyclophosphamide (AC) once every 3 weeks (Q3W) followed by docetaxel Q3W for 4 cycles. Pertuzumab and trastuzumab were given intravenously (IV) for 4 cycles Q3W concurrently with the taxane component of chemotherapy. After completing their neoadjuvant therapy, participants underwent surgery. Thereafter, participants received an additional 14 cycles of pertuzumab IV and trastuzumab IV for a total of 18 cycles.
    id
    OG000
    title
    Arm A: Pertuzumab IV + Trastuzumab IV + Chemotherapy
  2. description
    Participants received 8 cycles of investigator's choice of neoadjuvant chemotherapy. This included either: 1) 4 cycles of ddAC Q2W (given with G-CSF support as needed according to local guidelines) followed by paclitaxel once every week (QW) for 12 weeks; or 2) 4 cycles of AC Q3W followed by docetaxel Q3W for 4 cycles. The fixed-dose combination (FDC) of pertuzumab and trastuzumab was given subcutaneously (SC) for 4 cycles (Q3W) concurrently with the taxane component of chemotherapy. After completing their neoadjuvant therapy, participants underwent surgery. Thereafter, participants received an additional 14 cycles of the FDC of pertuzumab and trastuzumab SC for a total of 18 cycles.
    id
    OG001
    title
    Arm B: Pertuzumab and Trastuzumab FDC SC + Chemotherapy
param Type
NUMBER
population Description
ITT Population: includes all enrolled participants.
reporting Status
POSTED
time Frame
Following completion of surgery (up to 33 weeks)
title
Percentage of Participants With Total Pathological Complete Response (tpCR), According to Local Pathologist Assessment
type
SECONDARY
unit Of Measure
Percentage of participants

Download exact source JSON → · Snapshot ctgov-results-191f4516d4760045304b24a0

Preserved source evidence · Not medical advice