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NCT04512235 · OUTCOME

A Hierarchical Combination of Time to All-cause Mortality and Frequency of Cardiovascular Hospitalizations (CVHs) Analyzed by Win Ratio

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES) · Source last updated 2026-06-17

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

Measure
NUMBER
Unit
win ratio
Interval / dispersion
95% Confidence Interval
Time frame
Day 1 through end of the participant's PETP (Participants were enrolled on a rolling basis. Therefore, the primary analysis for the study was conducted 18-49 months after the study randomization.)

What was measured

Time to all-cause mortality was defined as the number of weeks from the date of randomization to the date of death if it is on or before the end of PETP. Participants alive at the end of PETP (LPI+18 months) were censored at their last known alive date recorded within the PETP. CVHs were events adjudicated and confirmed as such by the Clinical Event Adjudication Committee (CEAC). Hypothesis testing with the Finkelstein-Schoenfeld test and treatment effect estimation with the win-ratio method based on pairwise comparisons of participant outcomes with comparisons performed in a hierarchical manner. To calculate win ratio, participant outcomes based on time to all-cause mortality were first compared and if there was no 'winner' due to censoring then a comparison based on frequency of cardiovascular hospitalization was made. A win ratio \>1 represents a more favorable outcome for CAEL-101 over placebo.

Analysis population: Intent-to-Treat Set: all participants who were randomized. As prespecified, data collected and reported as 'Overall (All Randomized Participants)' and per subgroups 'Kappa FLC Subtype' and 'Lambda FLC Subtype'. For the win-ratio analysis, comparisons were made between participants in the 'CAEL-101 + Plasma Cell Dyscrasia Treatment' arm and the 'Placebo + Plasma Cell Dyscrasia Treatment' arm of each reporting group.

Groups in this outcome

Combined CAEL-101 + Plasma Cell Dyscrasia Treatment and Placebo + Plasma Cell Dyscrasia Treatment

In addition to SoC plasma cell dyscrasia treatment, participants were administered either CAEL-101 or placebo as an IV infusion over approximately 2 hours until LPI+18 months.

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

Analysis denominator · Participants

GroupSource count
Combined CAEL-101 + Plasma Cell Dyscrasia Treatment and Placebo + Plasma Cell Dyscrasia Treatment281

Reported measurements

Overall (All Randomized Participants)

Analysis denominator · Participants

GroupSource count
Combined CAEL-101 + Plasma Cell Dyscrasia Treatment and Placebo + Plasma Cell Dyscrasia Treatment281
Values in win ratio · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Combined CAEL-101 + Plasma Cell Dyscrasia Treatment and Placebo + Plasma Cell Dyscrasia Treatment1.23Not reported0.831.84Not reported
FLC Subtype Kappa

Analysis denominator · Participants

GroupSource count
Combined CAEL-101 + Plasma Cell Dyscrasia Treatment and Placebo + Plasma Cell Dyscrasia Treatment41
Values in win ratio · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Combined CAEL-101 + Plasma Cell Dyscrasia Treatment and Placebo + Plasma Cell Dyscrasia Treatment2.68Not reported1.036.99Not reported
FLC Subtype Lambda

Analysis denominator · Participants

GroupSource count
Combined CAEL-101 + Plasma Cell Dyscrasia Treatment and Placebo + Plasma Cell Dyscrasia Treatment237
Values in win ratio · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Combined CAEL-101 + Plasma Cell Dyscrasia Treatment and Placebo + Plasma Cell Dyscrasia Treatment1.04Not reported0.661.63Not reported

Source statistical analyses

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

  1. group Description
    Overall
    group Ids
    1. OG000
    non Inferiority Comment
    Treatment Effect Estimation
    non Inferiority Type
    OTHER
    p Value
    0.259
    statistical Method
    Finkelstein-Schoenfeld Test
  2. group Description
    FLC Subtype Kappa
    group Ids
    1. OG000
    non Inferiority Comment
    Treatment Effect Estimation
    non Inferiority Type
    OTHER
    p Value
    0.070
    statistical Method
    Finkelstein-Schoenfeld Test
  3. group Description
    FLC Subtype Lambda
    group Ids
    1. OG000
    non Inferiority Comment
    Treatment Effect Estimation
    non Inferiority Type
    OTHER
    p Value
    0.633
    statistical Method
    Finkelstein-Schoenfeld Test
Complete source fields
analyses
  1. group Description
    Overall
    group Ids
    1. OG000
    non Inferiority Comment
    Treatment Effect Estimation
    non Inferiority Type
    OTHER
    p Value
    0.259
    statistical Method
    Finkelstein-Schoenfeld Test
  2. group Description
    FLC Subtype Kappa
    group Ids
    1. OG000
    non Inferiority Comment
    Treatment Effect Estimation
    non Inferiority Type
    OTHER
    p Value
    0.070
    statistical Method
    Finkelstein-Schoenfeld Test
  3. group Description
    FLC Subtype Lambda
    group Ids
    1. OG000
    non Inferiority Comment
    Treatment Effect Estimation
    non Inferiority Type
    OTHER
    p Value
    0.633
    statistical Method
    Finkelstein-Schoenfeld Test
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        0.83
        upper Limit
        1.84
        value
        1.23
    denoms
    1. counts
      1. group Id
        OG000
        value
        281
      units
      Participants
    title
    Overall (All Randomized Participants)
  2. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        1.03
        upper Limit
        6.99
        value
        2.68
    denoms
    1. counts
      1. group Id
        OG000
        value
        41
      units
      Participants
    title
    FLC Subtype Kappa
  3. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        0.66
        upper Limit
        1.63
        value
        1.04
    denoms
    1. counts
      1. group Id
        OG000
        value
        237
      units
      Participants
    title
    FLC Subtype Lambda
denoms
  1. counts
    1. group Id
      OG000
      value
      281
    units
    Participants
description
Time to all-cause mortality was defined as the number of weeks from the date of randomization to the date of death if it is on or before the end of PETP. Participants alive at the end of PETP (LPI+18 months) were censored at their last known alive date recorded within the PETP. CVHs were events adjudicated and confirmed as such by the Clinical Event Adjudication Committee (CEAC). Hypothesis testing with the Finkelstein-Schoenfeld test and treatment effect estimation with the win-ratio method based on pairwise comparisons of participant outcomes with comparisons performed in a hierarchical manner. To calculate win ratio, participant outcomes based on time to all-cause mortality were first compared and if there was no 'winner' due to censoring then a comparison based on frequency of cardiovascular hospitalization was made. A win ratio \>1 represents a more favorable outcome for CAEL-101 over placebo.
dispersion Type
95% Confidence Interval
groups
  1. description
    In addition to SoC plasma cell dyscrasia treatment, participants were administered either CAEL-101 or placebo as an IV infusion over approximately 2 hours until LPI+18 months.
    id
    OG000
    title
    Combined CAEL-101 + Plasma Cell Dyscrasia Treatment and Placebo + Plasma Cell Dyscrasia Treatment
param Type
NUMBER
population Description
Intent-to-Treat Set: all participants who were randomized. As prespecified, data collected and reported as 'Overall (All Randomized Participants)' and per subgroups 'Kappa FLC Subtype' and 'Lambda FLC Subtype'. For the win-ratio analysis, comparisons were made between participants in the 'CAEL-101 + Plasma Cell Dyscrasia Treatment' arm and the 'Placebo + Plasma Cell Dyscrasia Treatment' arm of each reporting group.
reporting Status
POSTED
time Frame
Day 1 through end of the participant's PETP (Participants were enrolled on a rolling basis. Therefore, the primary analysis for the study was conducted 18-49 months after the study randomization.)
title
A Hierarchical Combination of Time to All-cause Mortality and Frequency of Cardiovascular Hospitalizations (CVHs) Analyzed by Win Ratio
type
PRIMARY
unit Of Measure
win ratio

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

Preserved source evidence · Not medical advice