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NCT02601950 · OUTCOME

Cohorts 1, 3, 4, 5, 6 and 7: Objective Response Rate (ORR)

A Study of Tazemetostat in Adult Participants With Soft Tissue Sarcoma · Source last updated 2025-06-22

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
Tumor assessment performed at screening and every 8 weeks beginning at Cycle 3 Day 1, until disease progression or the start of subsequent anti-cancer therapy, whichever occurred first, up to 5.75 years (cycle duration: 28 days)

What was measured

ORR was defined as percentage of participants who achieved a confirmed complete response (CR) or partial response (PR) based on the investigator review from start of treatment until progressive disease (PD) or start of subsequent anti-cancer therapy or cancer-related surgery, whichever was earlier, as per response assessment in neuro-oncology (RANO) criteria for primary brain tumors or response evaluation criteria in solid tumors (RECIST) version (v) 1.1 criteria for all other solid tumors. CR was defined as disappearance of all target and non-target lesions. Any pathological lymph nodes must be \<10 millimeter (mm) in short axis. PR was defined as at least 30% decrease in sum of diameters of target lesions, taking as a reference, baseline sum of diameters. PD was defined as at least a 20% increase in sum of diameters of target lesions, taking as a reference, smallest sum of diameters recorded since treatment started. In addition, sum must have an absolute increase from nadir of 5 mm.

Analysis population: ITT population included all participants who received at least 1 dose of tazemetostat.

Groups in this outcome

Rhabdoid Tumors: Cohort 1

Participants with rhabdoid tumors (MRT, RTK, ATRT, and selected tumors with rhabdoid features, including SCCOHT, also known as MRTO) received tazemetostat 800 mg BID orally in continuous 28-day cycles until disease progression, development of an unacceptable toxicity, withdrawal of consent, or termination of the study.

Other INI1-negative: Cohort 3

Participants with other INI-1-negative tumors or any solid tumor with EZH2 GOF mutation, including EMPNST, EMC, myoepithelial carcinoma, other INI-1-negative malignant tumors with sponsor approval, any solid tumor with EZH2 GOF mutation including but not limited to Ewing's sarcoma and melanoma received tazemetostat 800 mg BID orally in continuous 28-day cycles until disease progression, development of an unacceptable toxicity, withdrawal of consent, or termination of the study.

RMC: Cohort 4

Participants with RMC received tazemetostat 800 mg BID orally in continuous 28-day cycles until disease progression, development of an unacceptable toxicity, withdrawal of consent, or termination of the study.

ES: Cohort 5

Participants with ES received tazemetostat 800 mg BID orally in continuous 28-day cycles until disease progression, development of an unacceptable toxicity, withdrawal of consent, or termination of the study.

ES Paired Biopsy: Cohort 6

Participants with ES undergoing optional tumour biopsy received tazemetostat 800 mg BID orally in continuous 28-day cycles until disease progression, development of an unacceptable toxicity, withdrawal of consent, or termination of the study.

Chordoma: Cohort 7

Participants with poorly differentiated chordoma (or other chordoma with sponsor approval) received tazemetostat 800 mg BID orally in continuous 28-day cycles until disease progression, development of an unacceptable toxicity, withdrawal of consent, or termination of the study.

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

Analysis denominator · Participants

GroupSource count
Rhabdoid Tumors: Cohort 132
Other INI1-negative: Cohort 332
RMC: Cohort 414
ES: Cohort 562
ES Paired Biopsy: Cohort 669
Chordoma: Cohort 718

Reported measurements

Source class 1
Values in Percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Rhabdoid Tumors: Cohort 19.4Not reported2.025.0Not reported
Other INI1-negative: Cohort 39.4Not reported2.025.0Not reported
RMC: Cohort 40.0Not reported0.023.2Not reported
ES: Cohort 516.1Not reported8.027.7Not reported
ES Paired Biopsy: Cohort 615.9Not reported8.226.7Not reported
Chordoma: Cohort 75.6Not reported0.127.3Not reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        2.0
        upper Limit
        25.0
        value
        9.4
      2. group Id
        OG001
        lower Limit
        2.0
        upper Limit
        25.0
        value
        9.4
      3. group Id
        OG002
        lower Limit
        0.0
        upper Limit
        23.2
        value
        0.0
      4. group Id
        OG003
        lower Limit
        8.0
        upper Limit
        27.7
        value
        16.1
      5. group Id
        OG004
        lower Limit
        8.2
        upper Limit
        26.7
        value
        15.9
      6. group Id
        OG005
        lower Limit
        0.1
        upper Limit
        27.3
        value
        5.6
denoms
  1. counts
    1. group Id
      OG000
      value
      32
    2. group Id
      OG001
      value
      32
    3. group Id
      OG002
      value
      14
    4. group Id
      OG003
      value
      62
    5. group Id
      OG004
      value
      69
    6. group Id
      OG005
      value
      18
    units
    Participants
description
ORR was defined as percentage of participants who achieved a confirmed complete response (CR) or partial response (PR) based on the investigator review from start of treatment until progressive disease (PD) or start of subsequent anti-cancer therapy or cancer-related surgery, whichever was earlier, as per response assessment in neuro-oncology (RANO) criteria for primary brain tumors or response evaluation criteria in solid tumors (RECIST) version (v) 1.1 criteria for all other solid tumors. CR was defined as disappearance of all target and non-target lesions. Any pathological lymph nodes must be \<10 millimeter (mm) in short axis. PR was defined as at least 30% decrease in sum of diameters of target lesions, taking as a reference, baseline sum of diameters. PD was defined as at least a 20% increase in sum of diameters of target lesions, taking as a reference, smallest sum of diameters recorded since treatment started. In addition, sum must have an absolute increase from nadir of 5 mm.
dispersion Type
95% Confidence Interval
groups
  1. description
    Participants with rhabdoid tumors (MRT, RTK, ATRT, and selected tumors with rhabdoid features, including SCCOHT, also known as MRTO) received tazemetostat 800 mg BID orally in continuous 28-day cycles until disease progression, development of an unacceptable toxicity, withdrawal of consent, or termination of the study.
    id
    OG000
    title
    Rhabdoid Tumors: Cohort 1
  2. description
    Participants with other INI-1-negative tumors or any solid tumor with EZH2 GOF mutation, including EMPNST, EMC, myoepithelial carcinoma, other INI-1-negative malignant tumors with sponsor approval, any solid tumor with EZH2 GOF mutation including but not limited to Ewing's sarcoma and melanoma received tazemetostat 800 mg BID orally in continuous 28-day cycles until disease progression, development of an unacceptable toxicity, withdrawal of consent, or termination of the study.
    id
    OG001
    title
    Other INI1-negative: Cohort 3
  3. description
    Participants with RMC received tazemetostat 800 mg BID orally in continuous 28-day cycles until disease progression, development of an unacceptable toxicity, withdrawal of consent, or termination of the study.
    id
    OG002
    title
    RMC: Cohort 4
  4. description
    Participants with ES received tazemetostat 800 mg BID orally in continuous 28-day cycles until disease progression, development of an unacceptable toxicity, withdrawal of consent, or termination of the study.
    id
    OG003
    title
    ES: Cohort 5
  5. description
    Participants with ES undergoing optional tumour biopsy received tazemetostat 800 mg BID orally in continuous 28-day cycles until disease progression, development of an unacceptable toxicity, withdrawal of consent, or termination of the study.
    id
    OG004
    title
    ES Paired Biopsy: Cohort 6
  6. description
    Participants with poorly differentiated chordoma (or other chordoma with sponsor approval) received tazemetostat 800 mg BID orally in continuous 28-day cycles until disease progression, development of an unacceptable toxicity, withdrawal of consent, or termination of the study.
    id
    OG005
    title
    Chordoma: Cohort 7
param Type
NUMBER
population Description
ITT population included all participants who received at least 1 dose of tazemetostat.
reporting Status
POSTED
time Frame
Tumor assessment performed at screening and every 8 weeks beginning at Cycle 3 Day 1, until disease progression or the start of subsequent anti-cancer therapy, whichever occurred first, up to 5.75 years (cycle duration: 28 days)
title
Cohorts 1, 3, 4, 5, 6 and 7: Objective Response Rate (ORR)
type
PRIMARY
unit Of Measure
Percentage of participants

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Preserved source evidence · Not medical advice