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NCT01736683 · OUTCOME

Kaplan-Meier Estimates for Progression-free Survival

Study of Sotatercept for the Treatment of Anemia in low-or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) or Non-proliferative Chronic Myelomonocytic Leukemia (CMML) · Source last updated 2022-10-24

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

Measure
MEDIAN
Unit
weeks
Interval / dispersion
95% Confidence Interval
Time frame
Day 1 to 257.3 weeks

What was measured

Participants who had disease progression were considered to have events. Participants who died without acute myeloid leukemia (AML) were also considered to have events with the event date as the date of death. Those who did not have disease progression and who were lost to follow-up were censored at the last known disease progression assessment date. Participants without disease progression at the last follow-up contact were censored at the date of the last follow-up contact date. Disease Progression to AML used criteria by the International Working Group (IWG) Response Criteria in Myelodysplasia (Cheson, 2006). Progression is considered if any of the following are met: - \>=50% increase in blasts - \>=50% decrement from maximum remission/response levels in granulocytes or platelets - Reduction in hemoglobin (Hgb) concentration by \>=2 g/dL - Transfusion dependence

Analysis population: Efficacy Evaluable Population

Groups in this outcome

Sotatercept 0.1 mg/kg

Sotatercept 0.1 mg/kg administered via subcutaneous injection every third week (Q3W). Participants were randomized to either the 0.1 mg/kg arm or the 0.3 mg/kg arm. Efficacy and safety data were assessed by a Steering Committee. Based upon the occurrence of dose-limiting toxicity (DLT) in ≤ 1/5 subjects in Cycle 1 in the 0.1 mg/kg and 0.3 mg/kg treatment groups, the 0.5 mg/kg treatment group began inclusion in the randomization scheme.

Sotatercept 0.3 mg/kg

Sotatercept 0.3 mg/kg administered via subcutaneous injection every third week (Q3W). Participants were randomized to either the 0.1 mg/kg arm or the 0.3 mg/kg arm. Efficacy and safety data were assessed by a Steering Committee. Based upon the occurrence of dose-limiting toxicity (DLT) in ≤ 1/5 subjects in Cycle 1 in the 0.1 mg/kg and 0.3 mg/kg treatment groups, the 0.5 mg/kg treatment group began inclusion in the randomization scheme.

Sotatercept 0.5 mg/kg

Sotatercept 0.5 mg/kg administered via subcutaneous injection every third week (Q3W). Participants were randomized to one of the active treatment arms. Efficacy and safety data were assessed by a Steering Committee. Based upon the occurrence of dose-limiting toxicity (DLT) in ≤ 1/6 subjects in Cycle 1 in the 0.5 mg/kg treatment group, the 1.0 mg/kg treatment group began inclusion in the randomization scheme.

Sotatercept 1.0 mg/kg

Sotatercept 1.0 mg/kg administered via subcutaneous injection every third week (Q3W). Participants were randomized to one of the active treatment arms. Efficacy and safety data were assessed by a Steering Committee. Based upon the occurrence of dose-limiting toxicity (DLT) in ≤ 1/6 subjects in Cycle 1 in the 1.0 mg/kg treatment group, the 2.0 mg/kg treatment group began inclusion in the randomization scheme. Following evaluation of all treatment group data by the Steering Committee, enrollment continued only in the 1.0 mg/kg arm because the arm had the greatest frequency of erythroid hematological improvement (HI-E).

Sotatercept 2.0 mg/kg

Sotatercept 2.0 mg/kg administered via subcutaneous injection every third week (Q3W). Participants were randomized to one of the active treatment arms. Efficacy and safety data were assessed by a Steering Committee. The 2.0 mg/kg sotatercept dose was reduced to 1.5 mg/kg for ongoing and newly enrolled participants by protocol amendment.

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

Analysis denominator · Participants

GroupSource count
Sotatercept 0.1 mg/kg7
Sotatercept 0.3 mg/kg6
Sotatercept 0.5 mg/kg21
Sotatercept 1.0 mg/kg35
Sotatercept 2.0 mg/kg5

Reported measurements

Source class 1
Values in weeks · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Sotatercept 0.1 mg/kg82.7Not reported15.182.7Not reported
Sotatercept 0.3 mg/kgNANot reported91.1NAnot enough events to allow for calculation
Sotatercept 0.5 mg/kgNANot reported58.6NAnot enough events to allow for calculation
Sotatercept 1.0 mg/kgNANot reportedNANAnot enough events to allow for calculation
Sotatercept 2.0 mg/kgNANot reported79.9NAnot enough events to allow for calculation
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        15.1
        upper Limit
        82.7
        value
        82.7
      2. comment
        not enough events to allow for calculation
        group Id
        OG001
        lower Limit
        91.1
        upper Limit
        NA
        value
        NA
      3. comment
        not enough events to allow for calculation
        group Id
        OG002
        lower Limit
        58.6
        upper Limit
        NA
        value
        NA
      4. comment
        not enough events to allow for calculation
        group Id
        OG003
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      5. comment
        not enough events to allow for calculation
        group Id
        OG004
        lower Limit
        79.9
        upper Limit
        NA
        value
        NA
denoms
  1. counts
    1. group Id
      OG000
      value
      7
    2. group Id
      OG001
      value
      6
    3. group Id
      OG002
      value
      21
    4. group Id
      OG003
      value
      35
    5. group Id
      OG004
      value
      5
    units
    Participants
description
Participants who had disease progression were considered to have events. Participants who died without acute myeloid leukemia (AML) were also considered to have events with the event date as the date of death. Those who did not have disease progression and who were lost to follow-up were censored at the last known disease progression assessment date. Participants without disease progression at the last follow-up contact were censored at the date of the last follow-up contact date. Disease Progression to AML used criteria by the International Working Group (IWG) Response Criteria in Myelodysplasia (Cheson, 2006). Progression is considered if any of the following are met: - \>=50% increase in blasts - \>=50% decrement from maximum remission/response levels in granulocytes or platelets - Reduction in hemoglobin (Hgb) concentration by \>=2 g/dL - Transfusion dependence
dispersion Type
95% Confidence Interval
groups
  1. description
    Sotatercept 0.1 mg/kg administered via subcutaneous injection every third week (Q3W). Participants were randomized to either the 0.1 mg/kg arm or the 0.3 mg/kg arm. Efficacy and safety data were assessed by a Steering Committee. Based upon the occurrence of dose-limiting toxicity (DLT) in ≤ 1/5 subjects in Cycle 1 in the 0.1 mg/kg and 0.3 mg/kg treatment groups, the 0.5 mg/kg treatment group began inclusion in the randomization scheme.
    id
    OG000
    title
    Sotatercept 0.1 mg/kg
  2. description
    Sotatercept 0.3 mg/kg administered via subcutaneous injection every third week (Q3W). Participants were randomized to either the 0.1 mg/kg arm or the 0.3 mg/kg arm. Efficacy and safety data were assessed by a Steering Committee. Based upon the occurrence of dose-limiting toxicity (DLT) in ≤ 1/5 subjects in Cycle 1 in the 0.1 mg/kg and 0.3 mg/kg treatment groups, the 0.5 mg/kg treatment group began inclusion in the randomization scheme.
    id
    OG001
    title
    Sotatercept 0.3 mg/kg
  3. description
    Sotatercept 0.5 mg/kg administered via subcutaneous injection every third week (Q3W). Participants were randomized to one of the active treatment arms. Efficacy and safety data were assessed by a Steering Committee. Based upon the occurrence of dose-limiting toxicity (DLT) in ≤ 1/6 subjects in Cycle 1 in the 0.5 mg/kg treatment group, the 1.0 mg/kg treatment group began inclusion in the randomization scheme.
    id
    OG002
    title
    Sotatercept 0.5 mg/kg
  4. description
    Sotatercept 1.0 mg/kg administered via subcutaneous injection every third week (Q3W). Participants were randomized to one of the active treatment arms. Efficacy and safety data were assessed by a Steering Committee. Based upon the occurrence of dose-limiting toxicity (DLT) in ≤ 1/6 subjects in Cycle 1 in the 1.0 mg/kg treatment group, the 2.0 mg/kg treatment group began inclusion in the randomization scheme. Following evaluation of all treatment group data by the Steering Committee, enrollment continued only in the 1.0 mg/kg arm because the arm had the greatest frequency of erythroid hematological improvement (HI-E).
    id
    OG003
    title
    Sotatercept 1.0 mg/kg
  5. description
    Sotatercept 2.0 mg/kg administered via subcutaneous injection every third week (Q3W). Participants were randomized to one of the active treatment arms. Efficacy and safety data were assessed by a Steering Committee. The 2.0 mg/kg sotatercept dose was reduced to 1.5 mg/kg for ongoing and newly enrolled participants by protocol amendment.
    id
    OG004
    title
    Sotatercept 2.0 mg/kg
param Type
MEDIAN
population Description
Efficacy Evaluable Population
reporting Status
POSTED
time Frame
Day 1 to 257.3 weeks
title
Kaplan-Meier Estimates for Progression-free Survival
type
SECONDARY
unit Of Measure
weeks

Download exact source JSON → · Snapshot ctgov-results-25a395029948b04d52b1253f

Preserved source evidence · Not medical advice