Skip to content
Triangle logo TriangleGet started
← Full study record

NCT03580655 · OUTCOME

Overall Response Rate (ORR) Based on Modified International Working Group-Myeloproliferative Neoplasms Research and Treatment and European Competence Network on Mastocytosis (mIWG-MRT-ECNM) Response Criteria

(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

ORR was defined as the percentage of participants with a confirmed best response of complete remission (CR), complete remission with partial recovery of peripheral blood counts (CRh), partial remission (PR), or clinical improvement (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 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
Avapritinib73.5Not reported62.782.6Not reported

Source statistical analyses

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

  1. group Description
    Statistical test on binomial proportion of ORR against a null hypothesis of 28%, one sided alpha=0.025.
    group Ids
    1. OG000
    non Inferiority Type
    SUPERIORITY
    p Value
    < 0.0001
    statistical Method
    Wald Test
Complete source fields
analyses
  1. group Description
    Statistical test on binomial proportion of ORR against a null hypothesis of 28%, one sided alpha=0.025.
    group Ids
    1. OG000
    non Inferiority Type
    SUPERIORITY
    p Value
    < 0.0001
    statistical Method
    Wald Test
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        62.7
        upper Limit
        82.6
        value
        73.5
denoms
  1. counts
    1. group Id
      OG000
      value
      83
    units
    Participants
description
ORR was defined as the percentage of participants with a confirmed best response of complete remission (CR), complete remission with partial recovery of peripheral blood counts (CRh), partial remission (PR), or clinical improvement (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 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
Overall Response Rate (ORR) Based on Modified International Working Group-Myeloproliferative Neoplasms Research and Treatment and European Competence Network on Mastocytosis (mIWG-MRT-ECNM) Response Criteria
type
PRIMARY
unit Of Measure
Percentage of Participants

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

Preserved source evidence · Not medical advice