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NCT03037385 · OUTCOME

Phase 1 and Phase 2: DOR in RET-fusion Positive TC Participants With Specific RET Gene Status

Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Participants With Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors · Source last updated 2025-05-31

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

Measure
MEDIAN
Unit
months
Interval / dispersion
95% Confidence Interval
Time frame
Up to approximately 79.8 months

What was measured

Oncogenic RET activation has been implicated as a driver in MTC. These rearrangements typically produce chimeric transcripts that encode a fusion protein consisting of the RET kinase domain coupled to a protein with a dimerization domain (e.g., KIF5B, CCDC6, NCOA4). RET genotypes were determined by local testing and/or central analysis of ctDNA. DOR was assessed in participants having specific RET rearrangements. DOR=time from first documented CR/PR to date of first documented PD or death due to any cause, whichever occurs first. Per RECIST, CR= disappearance of all target lesions or any pathological lymph nodes (target or non-target) having a reduction in the short axis to \<10 mm. PR=at least a 30% decrease in the SOD of all target lesions, taking as reference the baseline SOD, in absence of CR. PD=as at least a 20% increase in SOD of target lesions, taking as reference smallest SOD on study (including baseline). DOR was analyzed using the KM method.

Analysis population: RET-altered measurable disease population included participants in the efficacy population who have measurable (target) disease per RECIST v1.1, at baseline according to BICR \& sufficient evidence of a RET alteration. As prespecified in SAP, Phase 1 participants treated at 400 mg QD are pooled with Phase 2 participants for efficacy analysis. Participants who had a response of CR/PR were analyzed in this outcome measure. Number analyzed is the number of participants with the specified mutation.

Groups in this outcome

RET-fusion Positive TC

Participants who had RET-fusion positive TC received pralsetinib 400 mg, orally, QD until discontinuation due to toxicity, disease progression, or other reasons.

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

Analysis denominator · Participants

GroupSource count
RET-fusion Positive TC23

Reported measurements

CCDC6

Analysis denominator · Participants

GroupSource count
RET-fusion Positive TC14
Values in months · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
RET-fusion Positive TCNANot reported11.2NAThe median and upper limit of 95% CI was not estimable due to insufficient number of participants with events.
NCOA4

Analysis denominator · Participants

GroupSource count
RET-fusion Positive TC6
Values in months · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
RET-fusion Positive TC15.2Not reported6.9NAThe upper limit of 95% CI was not estimable due to insufficient number of participants with events.
Other

Analysis denominator · Participants

GroupSource count
RET-fusion Positive TC3
Values in months · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
RET-fusion Positive TCNANot reported23.6NAThe median and upper limit of 95% CI was not estimable due to insufficient number of participants with events.
Complete source fields
classes
  1. categories
    1. measurements
      1. comment
        The median and upper limit of 95% CI was not estimable due to insufficient number of participants with events.
        group Id
        OG000
        lower Limit
        11.2
        upper Limit
        NA
        value
        NA
    denoms
    1. counts
      1. group Id
        OG000
        value
        14
      units
      Participants
    title
    CCDC6
  2. categories
    1. measurements
      1. comment
        The upper limit of 95% CI was not estimable due to insufficient number of participants with events.
        group Id
        OG000
        lower Limit
        6.9
        upper Limit
        NA
        value
        15.2
    denoms
    1. counts
      1. group Id
        OG000
        value
        6
      units
      Participants
    title
    NCOA4
  3. categories
    1. measurements
      1. comment
        The median and upper limit of 95% CI was not estimable due to insufficient number of participants with events.
        group Id
        OG000
        lower Limit
        23.6
        upper Limit
        NA
        value
        NA
    denoms
    1. counts
      1. group Id
        OG000
        value
        3
      units
      Participants
    title
    Other
denoms
  1. counts
    1. group Id
      OG000
      value
      23
    units
    Participants
description
Oncogenic RET activation has been implicated as a driver in MTC. These rearrangements typically produce chimeric transcripts that encode a fusion protein consisting of the RET kinase domain coupled to a protein with a dimerization domain (e.g., KIF5B, CCDC6, NCOA4). RET genotypes were determined by local testing and/or central analysis of ctDNA. DOR was assessed in participants having specific RET rearrangements. DOR=time from first documented CR/PR to date of first documented PD or death due to any cause, whichever occurs first. Per RECIST, CR= disappearance of all target lesions or any pathological lymph nodes (target or non-target) having a reduction in the short axis to \<10 mm. PR=at least a 30% decrease in the SOD of all target lesions, taking as reference the baseline SOD, in absence of CR. PD=as at least a 20% increase in SOD of target lesions, taking as reference smallest SOD on study (including baseline). DOR was analyzed using the KM method.
dispersion Type
95% Confidence Interval
groups
  1. description
    Participants who had RET-fusion positive TC received pralsetinib 400 mg, orally, QD until discontinuation due to toxicity, disease progression, or other reasons.
    id
    OG000
    title
    RET-fusion Positive TC
param Type
MEDIAN
population Description
RET-altered measurable disease population included participants in the efficacy population who have measurable (target) disease per RECIST v1.1, at baseline according to BICR \& sufficient evidence of a RET alteration. As prespecified in SAP, Phase 1 participants treated at 400 mg QD are pooled with Phase 2 participants for efficacy analysis. Participants who had a response of CR/PR were analyzed in this outcome measure. Number analyzed is the number of participants with the specified mutation.
reporting Status
POSTED
time Frame
Up to approximately 79.8 months
title
Phase 1 and Phase 2: DOR in RET-fusion Positive TC Participants With Specific RET Gene Status
type
SECONDARY
unit Of Measure
months

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Preserved source evidence · Not medical advice