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NCT03580655 · OUTCOME

Clinical Benefit Rate

(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis · Source last updated 2026-04-24

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
Baseline through Month 65

What was measured

The clinical benefit rate was defined as the percentage of participants with a confirmed best response of CR, CRh, PR, CI, and SD by mIWG-MRT-ECNM criteria.

Analysis population: Response-evaluable Population: all participants who received ≥1 dose of avapritinib, were deemed evaluable per mIWG-MRT-ECNM criteria at baseline as assessed by study steering committee review and had 1 of the following conditions: ≥2 complete postbaseline bone marrow assessments and had been on study for ≥6 cycles; had an end of study visit.

Groups in this outcome

Avapritinib

Avapritinib was administered once daily as an immediate-release tablet, orally, in 28-day 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
Avapritinib83

Reported measurements

Source class 1
Values in Percentage of Participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Avapritinib89.2Not reported80.494.9Not reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        80.4
        upper Limit
        94.9
        value
        89.2
denoms
  1. counts
    1. group Id
      OG000
      value
      83
    units
    Participants
description
The clinical benefit rate was defined as the percentage of participants with a confirmed best response of CR, CRh, PR, CI, and SD by mIWG-MRT-ECNM criteria.
dispersion Type
95% Confidence Interval
groups
  1. description
    Avapritinib was administered once daily as an immediate-release tablet, orally, in 28-day cycles.
    id
    OG000
    title
    Avapritinib
param Type
NUMBER
population Description
Response-evaluable Population: all participants who received ≥1 dose of avapritinib, were deemed evaluable per mIWG-MRT-ECNM criteria at baseline as assessed by study steering committee review and had 1 of the following conditions: ≥2 complete postbaseline bone marrow assessments and had been on study for ≥6 cycles; had an end of study visit.
reporting Status
POSTED
time Frame
Baseline through Month 65
title
Clinical Benefit Rate
type
SECONDARY
unit Of Measure
Percentage of Participants

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

Preserved source evidence · Not medical advice