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NCT03580655 · OUTCOME

ORR Based on mIWG-MRT-ECNM Response Criteria as Assessed by Local Investigator

(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 19

What was measured

ORR was defined as the percentage of participants with a confirmed best response of CR, CRh, PR, or CI by mIWG-MRT-ECNM criteria. The mIWG-MRT-ECNM criteria were based on findings from bone marrow biopsy and aspirate, and peripheral blood smear; bone marrow cytogenetics; other extracutaneous tissue biopsies (when available); liver and spleen imaging; and other clinical and laboratory parameters.

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 local investigator 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. Here, 'Overall Number of Participants Analyzed' signifies those participants evaluable for this outcome measure.

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
Avapritinib32

Reported measurements

Source class 1
Values in Percentage of Participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Avapritinib53.1Not reported34.770.9Not reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        34.7
        upper Limit
        70.9
        value
        53.1
denoms
  1. counts
    1. group Id
      OG000
      value
      32
    units
    Participants
description
ORR was defined as the percentage of participants with a confirmed best response of CR, CRh, PR, or CI by mIWG-MRT-ECNM criteria. The mIWG-MRT-ECNM criteria were based on findings from bone marrow biopsy and aspirate, and peripheral blood smear; bone marrow cytogenetics; other extracutaneous tissue biopsies (when available); liver and spleen imaging; and other clinical and laboratory parameters.
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 local investigator 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. Here, 'Overall Number of Participants Analyzed' signifies those participants evaluable for this outcome measure.
reporting Status
POSTED
time Frame
Baseline through Month 19
title
ORR Based on mIWG-MRT-ECNM Response Criteria as Assessed by Local Investigator
type
SECONDARY
unit Of Measure
Percentage of Participants

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

Preserved source evidence · Not medical advice